Transcript 19_The_Clinical_Trial_ProcessMini Med_Baquet

Participating in Research
and the Clinical Trials
Process
Claudia R. Baquet, MD, MPH
Professor, Department of Medicine, and
Associate Dean for Policy and Planning
University of Maryland School of Medicine
Assuring Diversity in
Clinical Research Participation
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A national priority
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Minority, uninsured, poor, and rural
communities have lower participation rates
in medical research.
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Underserved communities experience
substantial health disparities.
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Substantial barriers across multiple levels
impede participation in clinical trials.
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Building community-academic partnerships
and community trust is essential.
Fostering Public Trust in Research
and Trial Diversity
• Issues:
• Lack of public trust and need for diversity in research
participation
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“Research Literacy”
• Community: Lack of basic information on why
research is important, how it is designed and
reported; concern for “helicopter research; ethical
protections for research participants
• Community Primary Care Clinicians: Fear of losing
control over patient care; distrust of academia and
“stealing “patients; how to refer/enroll patients in
studies; need for feedback from researchers
Barriers to Research Participation
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Patient
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Health care professional
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Structural or organizational
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Knowledge and awareness in general
public
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Insufficient community infrastructure
to support clinical research and trials
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Historical factors
Cancer Detection and
Prevention
University of Maryland
Barriers to Clinical Trials Research
Goal:
examine the health behavior, clinical trials barriers, health care
access, screening and health status of Maryland residents
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Survey of 5,154 English-speaking, non-institutionalized men and women
aged 18+
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13 jurisdictions in Maryland (December 2001-March 2003), including:
• urban Baltimore City
• rural Western Maryland
• rural Eastern Shore
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Cross-sectional study design using random digit dialing (RDD)
methodology
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Clinical trial barriers and factors are predictive of
participation and attitudes and information channels.
Baquet CR, et al. Recruitment and Participation in Clinical Trials: Socio-Demographic, Rural/Urban, and Health Care Access Predictors.
Cancer Detection and Prevention. 2006; 30.
Maryland Clinical Trial Barriers
Research Results
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80.0% of Blacks and 50.9% of Whites reported not knowing what a clinical
trial is.
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Over 95% reported their physician
never discussed clinical
research/trials.
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11.1% reported previous recruitment
into clinical trials.
• Of those, 59.4% actually
participated in clinical trials.
Baquet CR, et al. Recruitment and Participation in Clinical Trials: Socio-Demographic, Rural/Urban, and Health Care Access Predictors.
Cancer Detection and Prevention. 2006; 30.
Maryland Clinical Trial Barrier Research
“Multivariate Predictors of Recruitment and Participation in Clinical Trials”
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Were significantly more likely to be recruited:
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in poor health (OR=1.83, CI=1.21-2.76),
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had public health insurance coverage (OR=1.98, CI=1.57-2.51), and
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had some college or higher level of education (OR=2.32, CI=1.84-2.92).
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Were significantly more likely to actually participate:
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informed about clinical trials by their health care provider (OR=1.69, CI=1.08-2.65),
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knowledgeable about clinical trials (OR=2.09, CI=1.26-3.46), and
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able to make the time commitment (OR=1.67, CI=1.06-2.63).
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Black respondents were significantly less likely to be recruited (OR=0.61, CI=0.44-0.85)
and less likely to participate (OR=0.38, CI=0.21-0.68) in clinical trials.
Baquet CR, et al. Recruitment and Participation in Clinical Trials: Socio-Demographic, Rural/Urban, and Health Care Access
Predictors. Cancer Detection and Prevention. 2006; 30.
Journal Clinical Oncology
Accrual of Maryland Cancer Patients to
NCI sponsored Treatment Clinical Trials Study
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Examined relationship of socio-demographic factors on accrual of Maryland cancer
patients to NCI sponsored cancer treatment trials
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n=2,240 Maryland cancer patients accrued onto NCI-sponsored treatment trials
(1999-2002)
Purpose: To determine extent to which Maryland cancer patients and patients
residing in lower SES and/or rural areas were accrued to cancer trials and were
representative of all cancer patients in Maryland.
Data sources:
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NCI’s Cancer Therapy and Evaluation Program (CTEP) for Maryland cancer
patients
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Maryland Cancer Registry
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US Census and the Department of Agriculture
Baquet CR, Ellison, G, Mishra, S et al. Analysis of Maryland Cancer Patient Participation in NCI Supported Cancer Treatment Clinical
Trials. Journal of Clinical Oncology. July 2008.
Accrual of Maryland Cancer Patients to
NCI sponsored Treatment Clinical Trials
Baquet CR, Ellison, G, Mishra, S et al. Analysis of Maryland Cancer Patient Participation in NCI Supported Cancer Treatment Clinical
Trials. Journal of Clinical Oncology.July 2008.
Maryland Community Clinical Trial
Program Components
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Research: Barriers and Strategies
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Random digit dial (RDD)
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Qualitative research
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Results used to target
educational/awareness efforts
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Theory based
education/outreach
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Track accrual rates/trends
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CTEP analysis
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Health care professionals
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Trial referral
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Continuing Education
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Grand Rounds
Training for Researchers
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Community engagement
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Faith-based
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AHECs
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Print Media
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CBOs
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Local Health Departments
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FQHCs
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Formal Partnerships
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Policy Research and Advocacy
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Community based infrastructure
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Technical Assistance for elected
officials and staff
The Clinical Trials Process
Learning Objectives
 Define a clinical trial
 Name the different types and phases of
clinical trials
 Describe how participants are assigned to
groups in “randomized clinical trials
 Know what a protocol is and its importance
 Know the benefits and risks of taking part in a
clinical trial
What are Clinical Trials?
 Research studies involving people
 Tests new ways to prevent, diagnose or treat
disease
Why are clinical trials important?
 Most of the best treatments are based on
what we learned from clinical trials
 People with cancer and other diseases are
living longer because of clinical trials
Types of Clinical Trials
 Treatment trials
 Prevention trials
 Early-detection/screening trials
 Diagnostic trials
 Genetics trials
 Quality-of-life /supportive care trials
Treatment Trials
 Evaluate the effectiveness of a new drug or
treatment or a new way of using a standard
treatment
 Test chemotherapies, vaccines, or new
approaches to surgery or radiation treatment
Prevention Trials
 Investigate different ways to reduce the risk of
developing cancer
 Conducted in people who have never had cancer
or cancer patients in remission to prevent a
recurrence
Early Detection/Screening
 Evaluate new ways of finding cancer in
people before they have symptoms
 Conducted in people who do not have any
symptoms of cancer
Diagnostic Trials
 Test procedures for identifying cancer more
accurately and at an earlier stage
 Conducted with people who have signs or
symptoms of cancer
Genetics Trials
 Examine the role that genes play in cancer
risk or response to treatment
 Conducted with specific populations
 Look for genetic mutations by looking at DNA
from blood or tissue samples
Quality-of-Life/Supportive Care
 Investigate different ways to improve the
comfort and quality of life of people who have
cancer
Phases of Clinical Trials
 Phase I: Is it safe?
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15-30 people
To find a safe dosage (how often it should be
given?)
To decide how the agent should be given (pill,
injection or intravenous infusion)
To observe how the agent affects the human
body
Participants are cancer patients who have no
known effective treatment options
Phases of Clinical Trials
 Phase II: Does it work?
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Less than 100 people
To determine how well the new agent works
against a particular cancer
To see how the agent or intervention affects
the human body
The dosage found safe in Phase I is used
Participants have been treated with
chemotherapy, surgery, or radiation, but
treatment has not been effective
Phases of Clinical Trials
 Phase III: How well does it work?
 100 – thousands of people
 To compare the new agent or intervention (or new
use of a treatment ) with the current standard
 To find out if new treatment is better than, the same
as or worse than standard treatment
 Eligible participants have an equal chance to be
assigned to one or two more group (also called
“arms”).
 One group gets standard treatment (control)
 Other group gets new treatment (investigational
or intervention group)
Phases of Clinical Trials
 Phase IV: How well does it work in the
general population?
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Several hundred to several thousand people
To further evaluate the long-term safety and
effectiveness of a new treatment
How Research Participants are
Assigned to Groups
 Process called randomization (chance) using
a computer to assign a participant to the
intervention/investigational group or control
group
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Control group gets standard treatment and
investigational group gets new treatment being
tested
Used to prevent bias in research
Occurs primarily in Phase III research studies
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Double blind – means that neither you nor the
doctors know which people are taking the study
agent or control agent
Randomization
Why is randomization important?
 So all groups are as alike as possible
 Provides the best way to prove the
effectiveness of a new agent or intervention
What is the Clinical Trial Protocol?
 A plan for the research study which acts like a
“recipe” or blueprint for conducting the clinical
trial
 Generated by the person in charge of the
study, called a principal investigator
What is included in a Protocol?
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Purpose of the study
How many people will be in the study
Who is eligible to participate in the study
Any agents participants will take, the dosage,
and how often
 What medical tests participants will have and
how often
 What information will be gathered about the
participants
 The endpoints of the study
Benefits of taking part in a
Clinical Trial
 Possible benefits:
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Patients will receive, at a minimum, the best
standard treatment
If the new approach is proven to work, patients
may be among the first to benefit
Patients have a chance to help others and
improve cancer care
Risks of taking part in a
Clinical Trial
 Possible risks:
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Unknown side effects or other risks of new
drug or treatment
New treatment may not help every participants
Costs (e.g. travel to clinical trial site)
Resources
 Cancertrials.gov
 Cancer.org
 1-800-4-CANCER
 American Cancer Society Clinical Trial
Matching Service
References
 A.J.S. Rayl. A Complete Guide to Clinical Trials. Mamm. Nov/Dec
2005
 Cancer Clinical Trials: The Basic Workbook, Clinical Trials
Education Series. U.S. Department of Health and Human Services.
National Institutes of Health, National Cancer Institute. September
2002
 Taking Part in Cancer Treatment Research Studies. U.S.
Department of Health and Human Services. National Institutes of
Health, National Cancer Institute. July 2007.