Transcript Comparative Effectiveness Research and Health Policy

Comparative Effectiveness Research:
Now and in the Future
Children’s Hospital of Philadelphia
July 13, 2009
Patrick Conway, MD, MSCE
Chief Medical Officer, OS/ASPE
Department of Health and Human Services
Disclosure
 I am an employee of federal government
 Views expressed are my own and do not
represent official policy or guidance from the
Department of Health and Human Services
Agenda
 Short Background
 Comparative Effectiveness Research –
What is it and where we are currently
 Potential implications for academic
community of comparative
effectiveness research and broader
health reform
Background
 RWJ Clinical Scholar at Penn (and attending at
CHOP) – thesis was comparative effectiveness study
with Ron Keren as mentor
 Faculty (now on leave) Cincinnati Children’s Hospital
 White House Fellow 2007-08 – split between
Secretary of HHS and Director of AHRQ
 Chief Medical officer in policy division for Office of
Secretary
 Executive Director of Federal Coordinating Council
on Comparative Effectiveness Research
Portfolio at HHS
 Quality measurement and link to
payment
– National Quality Forum
– CHIPRA – quality measures
 Health Information Technology (e.g.
meaningful use)
 Health reform
 Comparative Effectiveness Research
 Whatever is asked
Background on American Recovery and
Reinvestment Act Legislation
 Allocated $1.1 billion for CER: $400 million to Office
of the Secretary, $400 million to NIH and $300 million
to AHRQ
 All money obligated (not spent) by end of FY10
 Established Federal Coordinating Council for CER
composed of senior federal officials and over half
physicians
 Required FCC report to the President and Congress
by June 30 on recommended priorities for OS CER
funds
 Required IOM report by June 30 on CER priorities
 Mandated operational plan from Secretary for
combined OS, AHRQ, and NIH CER funds by July 30
Federal Coordinating Council for
CER Objectives
 Develop a definition, establish prioritization
criteria, create a strategic framework, and
identify priorities that lay the foundation for
CER.
 Foster optimum coordination of comparative
effectiveness research conducted or
supported by relevant Federal departments.
 Formulate recommendations for investing
the $400 million appropriated to the HHS
Office of Secretary as part of this Report to
Congress.
Definition
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.
 To provide this information, comparative effectiveness research must
assess a comprehensive array of health-related outcomes for diverse
patient populations and sub-groups.
 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.
Minimum Threshold Criteria
1. Included within statutory limits of Recovery
Act and FCC definition of CER
2. Potential to inform decision-making by
patients, clinicians, and/or other
stakeholders
3. Responsiveness to expressed needs of
patients, clinicians, and/or other
stakeholders
4. Feasibility of research topic (including time
necessary for research)
Prioritization Criteria
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The criteria for scientifically meritorious research and
investments are:
Potential impact (based on prevalence of condition, burden of
disease, variability in outcomes, costs, potential for
increased patient benefit or decreased harm)
Potential to evaluate comparative effectiveness in diverse
populations and patient sub-groups and engage communities
in research
Uncertainty within the clinical and public health communities
regarding management decisions and variability in practice
Addresses need or gap unlikely to be addressed through
other organizations
Potential for multiplicative effect (e.g. lays foundation for
future CER such as data infrastructure and methods
development and training)
FCC Report: Strategic Framework for CER
Research
Human &
Scientific Capital
Data Infrastructure
Priority Populations
CrossCutting
Priority
Themes
Priority Conditions
Types of Interventions
Investments can be made in a
single category and/or be crosscutting in one of the themes
Dissemination and
Translation
CER Investment and
Activities
Four major categories:
• Research (e.g., comparing patient outcomes between
different treatments or care delivery models for a specific
condition)
• Human and Scientific Capital (e.g., training new researchers
to conduct CER, developing CER methodology)
• CER Data Infrastructure (e.g., developing longitudinal,
linked administrative or Electronic Health Record (EHR)
databases, or patient registries)
• Dissemination and Translation of CER (e.g., improving
methods to disseminate CER findings to clinicians and
patients and translate CER into practice)
Examples of Potential Focus Areas for
Cross-Cutting Theme Investments
Priority Populations
• Children
• Patients with multiple chronic conditions
• Racial and ethnic minorities
• Elderly
• Persons with disabilities
Priority Conditions
• Cancer
• Heart disease
• Diabetes
Types of Interventions
• Medications, devices
• procedures
• Behavioral change
• Delivery system changes
• Prevention
Council’s Recommended OS Investment Strategy
Research
Human &
Scientific Capital
Data Infrastructure
Dissemination and
Translation
Priority Populations
Crosscutting
Priority
Themes
Priority Conditions
Types of Interventions
Legend
Primary investment
Secondary investments
Supporting investment
Coordinated complimentary funding
 AHRQ and NIH will likely devote significant
amounts to research category (as well as
training and cross-cutting themes)
 OS could make short term investments in
infrastructure (both data and translational) to
lay foundation for CER and complement
AHRQ and NIH funding
IOM Report
 Purpose:
- to prepare a list of priorities for research funding
and recommendations to implement a sustained
CER effort
 Similar definition:
“CER is the generation and synthesis of evidence that
compares the benefits and harms of alternative methods to
prevent, diagnose, treat, and monitor a clinical condition or
to improve the delivery of care. The purpose of CER is to
assist consumers, clinicians, purchasers, and policy
makers to make informed decisions that will improve health
care at both the individual and population levels.”
IOM Report
 Recommended 100 priority CER topics and
research questions divided into four
quartiles
 Examples:
 Compare the effectiveness of treatment strategies for
atrial fibrillation
 Compare the effectiveness of comprehensive care
coordination programs, such as the medical home model
 Compare the effectiveness of various screening,
prophylaxis and treatment interventions in eradicating
methicillin resistant staph aureus
IOM General Recommendations
 Establish coordinating advisory body
 Develop and promote robust data and information
systems
 Strengthen the CER workforce
 Promote rapid adoption of CER findings
 Obtain public input and maintain transparent
processes
Interested to Read More
 Full FCC report and IOM report
available online
 Short summaries in NEJM online June
30th1,2
Conway PH, Clancy C. Comparative Effectiveness Research – Implications
of the Federal Coordinating Council’s Report. N Engl J Med 2009 June 30.
2 Iglehart R. Prioritizing Comparative Effectiveness Research – IOM Recommendations.
N Engl J Med 2009 June 30.
1
Where might CER fit in broader health care
reform and research funding in the future
 If we want to improve quality and value
in health care, we need to know:1,2
– What works for which specific patient
subgroups
– The quality outcomes for a given cost
– How to implement across systems of care
and measure results
1 Dougherty
D, Conway PH. The T3 Roadmap to Transform U.S. Health Care:
The How of High Quality Care. JAMA. 2008 May 21; 299(19): 2319-21.
2 Conway, PH, Clancy C. Transformation of Health Care at the Frontline. JAMA.
2009 Feb 18;301(7):763-5.
Health Reform
 All of the major current reform bills include
comparative effectiveness research or similar
research with different name (e.g. patient-centered
outcomes research) and typically with significant
funding
 Different proposals for where CER might primarily
reside (e.g. within a current government agency, new
entity, public-private governance structure)
Thoughts on future
 In terms of growth of research funding, this
may be a major area for future growth
 Data and link to HIT is critical for these
questions
 Will require further methods development and
training of researchers
 Need to strengthen link between questions
that patients and clinicians need answered
and the research enterprise
Immediate timeline
 Spend plans under review and ARRA calls
for release of combined plan July 30th
 Once plans approved (NIH, AHRQ, OS), funds
can be obligated
 All funds obligated by September 2010
 Spend may be able to extend 2-3 years from
date obligated at least for significant portion
of funds
Acknowledgement
 Mentorship of faculty at CHOP such as
Dr. Ron Keren and faculty through RWJ
Clinical Scholars program and CCEB
Questions?