Slide 1

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 2

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 3

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 4

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 5

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 6

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 7

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 8

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 9

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 10

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 11

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 12

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 13

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 14

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 15

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 16

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 17

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 18

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 19

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 20

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 21

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]


Slide 22

Comparative Effectiveness Research in the
National Drug Abuse Treatment Clinical Trials
Network (CTN)
CDR Steven Sparenborg, Ph.D., Udi Ghitza, Ph.D., CAPT
Betty Tai, Ph.D.

The Center for the Clinical Trials Network
National Institute on Drug Abuse
Rockville, Maryland

What is CER?
• A rigorous evaluation of the impact of
available treatment options for a given medical
condition and particular set of patients.
• The goal is to determine which specific types
of patients would benefit most from a given
healthcare option.

What is CER?
• relative medical benefits/risks of available
options
• similar treatments may be compared
• two or more competing drugs
• surgery vs. drug therapy
• may include assessments of cost-effectiveness.

Why pay attention to CER?
• CER has the potential to contain future healthcare spending
and improve the quality of care.
• Therefore, many proposals to reform health care call for an
expanded role for CER.
• CER rises in prominence as attention to healthcare
increases.
• Federal Coordinating Council for CER created in 2009.
• Patient Protection and Affordable Care Act signed in 2010.

Study Types Used in CER
•
•
•
•
•
•

randomized controlled trial
cohort study
case control study
cross section study
case series
systematic review

The Clinical Trials Network
• established in 1999 by The National Institute on
Drug Abuse
• nearly 12,000 patients in nearly 30 trials in
dozens of sites located in most states
• currently 16 nodes centered in academic/VA
institutions
• each node works with 5 or more local community
treatment programs
• funded by 5-year grants

The Clinical Trials Network:
Purpose and Goals
• validation and comparison of science-based
addiction treatments
• results guide community-based drug addiction
treatment practitioners
• promote translation of scientifically-derived
findings from research to practice
• conduct and advance comparative effectiveness
research (CER) led by community-based
practitioners and academic researchers

Project 1: Opioid Detoxification
• 13-day detoxification program (Ling et al.,
2005)
• 113 in-patients and 231 out-patients
• 6 sites in each condition (i-p; o-p)
• buprenorphine/naloxone (BUP/NX) vs. clonidine
• randomized 2:1
• subject presence on Day 13 with negative urine
sample was considered Tx success

BUP/NX was 3-5 times more effective than
clonidine

Project 2: Motivational Incentives
• Tx-seeking drug abusers randomized to routine
care with or without motivational incentives
• routine care in community outpatient
psychosocial clinics (Petry et al. 2005)
• routine care in community methadone
maintenance clinics (Peirce et al., 2006)
• low-cost prize-based incentives for drug
abstinence

Abstinence-based motivational incentives led to
improved drug-abstinence outcomes
Outpatient Psychosocial
Tx
Weeks of
Incentive
TAU
Continuous
Group
Group
Abstinence
%
%
(n = 209)
(n = 206)

Methadone
Maintenance
Incentive
TAU
Group
Group
%
%
(n = 198)
(n = 190)

≥4

39.7

21.0

23.7

9.0

≥8

26.3

11.7

16.7

2.1

12

18.7

4.9

5.6

0.5

Cost-Effectiveness Analyses
Compared to usual care in counseling-based
drug-free clinics, the incremental cost of using
motivational incentives to lengthen the longest
duration of continuous stimulant and alcohol
abstinence by 1 week was $258, and to obtain
an additional stimulant-free urine sample was
$146.

Cost-Effectiveness Analyses…cont
Compared to usual care in methadonemaintenance clinics, the incremental cost of
using abstinence-based incentives to lengthen
the longest duration of continuous stimulant
and alcohol abstinence by 1 week was $141,
and to obtain an additional stimulant-free urine
sample was $70.

Possible Future CER Studies in the CTN
Co-morbidity of Opiates and Alcohol Abuse
(in collaboration with NIAAA)
• patients with opiate abuse/dependence and comorbid alcohol abuse/dependence
• randomized to treatment with
1) Suboxone plus an anti-alcohol medication
(topiramate, acamprosate, or naltrexone)
2) Suboxone alone, or
3) The selected anti-alcohol medication alone

• 12 weeks Tx with follow-up 12 weeks after the
cessation of pharmacotherapy

Possible Future CER Studies in the CTN
Smoking Cessation Pharmacotherapy Options
• assess the effectiveness of nicotine replacement
therapy, bupropion, and varenicline as aids to
smoking cessation
• multi-site randomized clinical trial with adaptive
design
• cost-effectiveness analyses
• all participants will receive smoking cessation
counseling
• primary endpoint will be smoking cessation

Conclusions
• The CTN is an ideal organization to conduct CER
studies.
• CTN investigators and staff are informed and
experienced.
• The CTN infrastructure is designed to promote
real-world relevant research that can inform
clinicians and policy makers on the best evidencebased interventions for treating drug addiction.

Federal Coordinating Council on CER
Comparative effectiveness research is the conduct
and synthesis of research comparing the benefits and
harms of different interventions and strategies to
prevent, diagnose, treat and monitor health
conditions in “real world” settings. The purpose of
this research is to improve health outcomes by
developing and disseminating evidence-based
information to patients, clinicians, and other
decision-makers, responding to their expressed
needs, about which interventions are most effective
for which patients under specific circumstances.

Federal Coordinating Council on CER
• To provide this information, comparative
effectiveness
research
must
assess
a
comprehensive array of health-related outcomes
for diverse patient populations and subgroups.
• Defined interventions compared may include
medications, procedures, medical and assistive
devices and technologies, diagnostic testing,
behavioral change, and delivery system strategies.
• This research necessitates the development,
expansion, and use of a variety of data sources
and methods to assess comparative effectiveness
and actively disseminate the results.

In the Future....
• generate some near-term results and
momentum for the future
• facilitate the building of needed infrastructure
• expand access to existing infrastructure
• demonstrate proof of concept for
implementation efforts

Patient Protection and
Affordable Care Act
• creates an independent Patient-Centered
Outcomes Research Institute
• a non government entity
• establishes research priorities
• funds comparative clinical effectiveness research
• communicates results to the public
• requires openness, transparency, and scientific
integrity
• requires patient protections against misuse of
research results by policymakers

[email protected]