Practical Clinical Trials and the
AHRQ Experience
Carolyn M. Clancy, MD
Director
Agency for Healthcare Research and Quality
AIFA European Conference on Clinical Research for Decision-Making
Rome – March 30, 2007
AHRQ & PCTs
 AHRQ Overview
 Personalized Health Care
 Practical Clinical Trials
 Q&A
AHRQ’s Mission
Improve the quality, safety,
efficiency and effectiveness of
health care for all Americans
HHS Organizational Focus
NIH
CDC
AHRQ
Biomedical
research to
prevent,
diagnose and
treat diseases
Population health
and the role of
community-based
interventions to
improve health
Long-term and
system-wide
improvement of
health care quality
and effectiveness
Combating the
“Sting of Ignorance”
“The nation faces two yawning medical
information gaps. First, we need more studies
comparing treatments… what about the
insurers, private and governmental, who pay
such a large share of the nation’s $220 billion
annual drug bill? They could support such
studies with the rounding error of their annual
budgets — and then save billions if the findings
were put Into practice. The second problem is
that much of the knowledge we do have is not
communicated…we need an unbiased, efficient
system to get the word out to practitioners on
what works best.”
Jerry Avorn, Professor,
Harvard Medical School
New York Times Op-Ed
September 16, 2006
Comparative Effectiveness:
Effective Health Care Program
 Designed to support the new
Medicare prescription drug
benefit in 2006
 Mandated by Section 1013 of
the Medicare Modernization Act
to improve the quality,
effectiveness and efficiency of
health care delivered through
Medicare, Medicaid and the SCHIP programs
 Builds on years of experience
gained through AHRQ’s
Evidence-Based Practice
Centers
Effective Health Care Program
Translate Evidence into Improvements
(Eisenberg Clinical Decisions &
Communications Science Center)
Review/Compare
Existing Evidence
(Evidence-Based Practice Centers)
Accelerate
Practical Studies
(DEcIDE Network)
Comparative Effectiveness Review:
Management Strategies for Renal
Artery Stenosis
 Major Finding: No clear advantage found to
treating narrowed kidney arteries with
angioplasty, with or without a stent, over drug
therapy
– Narrowed kidney arteries are most common cause
of correctable high blood pressure
 Drug therapy and angioplasty both
improve blood pressure and slow
down the worsening of kidney function
AHRQ Comparative Effectiveness Review No.5, Comparative Effectiveness of
Management of Renal Artery Stenosis, October 2006, Available at
www.effectivehealthcare.ahrq.gov
Comparative Effectiveness
Reviews in Progress
 Comparative Effectiveness of
Drug Therapies for Rheumatoid
or Psoriatic Arthritis
 Comparison of Therapies for Low
Bone Density
 Comparison of Therapies for
Clinically Localized Prostate
Cancer
 Evidence of Off-Label Use of
Atypical Anti-Psychotic
Medications
Sample DEcIDE Projects
Duke
Treatment of in-stent restenosis (JAMA 2007)
Outcomes
Evaluating Patient Registries: A Users’ Guide
Brigham &
Women’s
Antipsychotic treatment in older adults
BWH/RTI
UNC
Hopkins
Vanderbilt
Methodological toolbox for
pharmacoepidemiologic research
Surveillance methods for improving patient
safety in Medicare claims
Effectiveness of new agents for diabetes/bariatric
surgery
DMARDs/Factors modulating treatment
outcomes of rheumatoid arthritis
Eisenberg Clinical Decisions and
Communications Center
 Presents evidence synthesized
by the Evidence-Based Practice
Centers and generated by the
DeCIDE Network in actionable
formats for stakeholders
including patients, providers,
policymakers and the public
Oregon Health &
Sciences University
 The knowledge is used to maximize the benefits of
health care, minimize harm and optimize the use of
health care resources
New Eisenberg Center
Summary Guides
http://effectivehealthcare.ahrq.gov/dsc/products.cfm
2008 Priorities
Patient Safety
 Health IT
 Patient Safety
Ambulatory
Patient Safety
Organizations
 New Patient
 Safety & Quality Measures, Safety Grants
Drug Management and
Patient-Centered Care
 Patient Safety Improvement
Corps
Medical Expenditure
Panel Surveys
Effective Health
Care Program
 Comparative
Effectiveness Reports
 Network of Research
Centers
 Clear Findings for
Multiple Audiences
Other Research &
Dissemination Activities
 Visit-Level Information on  Quality & Cost-Effectiveness, e.g.
Medical Expenditures
 Annual Quality &
Disparities Reports
Prevention and Pharmaceutical
Outcomes
 U.S. Preventive Services
Task Force
Questions are the Answer
Patient Involvement Campaign by
AHRQ and the AD Council
 Series of TV, radio and prints
Public Service Announcements
 A Web site that features a
“Question Builder” for patients to
enhance their medical
appointments
 The message: Get More
Involved With Your Health Care
FY 2008 Request
 FY 2008 Request = $329,564,000
– Increase of $10,872,000, or 3.4%, from the
FY 2007 Continuing Resolution Level
– Department-wide $15,000,000 Initiative
related to Personalized Health Care
FY 2008 Initiative:
Personalized Health Care
 Personalized Health Care
– +$15 million
– The initiative will accelerate the movement
toward personalized health care and help
bring “next generation” effectiveness of
care for individual patients.
– This initiative is essential to our drive for
health care transparency by identifying and
consistently measuring effective, high
quality care.
Partnership
ELECTRONIC
MEDICAL RECORD
CLINICAL
RESEARCH
CLINICAL
RESEARCH
CLINICAL
RESEARCH
CLINICAL
RESEARCH
PUBLIC-PRIVATE PARTNERSHIP
The Promise of Genomics
“We stand on the threshold
of creating a future that will
revolutionize the practice of
medicine by allowing us to
predict disease, develop
more precise therapies and,
ultimately, pre-empt the
development of disease in
the first place.”
Elias Zerhouni, 2006
Disease Diagnosis
Gene Therapy
Drug Design
Specialized Care
Examples of interventions/diagnostics
that can help specific groups of people
Iressa – For
advanced nonsmall cell lung
cancer treatment.
A gene-based test
can help identify
patients who
might be helped
by the drug
Gleevec – For
treatment of
chronic
myelogenous
leukemia
(gene-based)
Challenges
 Using data on outcomes of gene-based testing,
which currently is rarely collected
 Acquiring data on diagnostics, therapeutics and
patient outcomes from many different databases
 Using administrative data for research purposes
 Securely linking data from multiple resources
Solutions
 A secure Web-based system
for sharing gene-based data
among multiple organizations
 Partnerships that encourage
research, development and
increased implementations
 Shared decision-making
Key Questions for Decision-Makers
 Are patient outcomes
improved?
 What are the harms?
 Do the benefits
outweigh the harms?
 What is the incremental
benefit?
 Is the real-world net
benefit the same as in
clinical trials?
 What are the costs?
 Given the net benefit
and costs, is the
intervention justified?
Reliable evidence is essential to
improving health care quality and to
supporting efficient use of limited
resources
Clinical Evaluation
Science: Phase 1
Question
Hypothesis
Study
Publications
Changes in practice
Clinical Evaluation
Science: Phase 2
Publications (multiple)
Evidence synthesis
Guidelines,
Performance Measures
Changes in Practice
Practical Clinical Trials
 Compare clinically relevant
interventions
 Enroll a diverse study population
 Recruit from a variety of practice
settings
 Measure a broad range of relevant
health outcomes
PCTs vs. ECTs
Practical Clinical Trials
Explanatory Clinical Trials
Hypothesis and study design
are formulated based on
information needed to make a
decision
Addresses risks, benefits, and
costs of an intervention as
they would occur in routine
clinical practice
Designed to better understand
how and why an intervention
works
Maximize the chance that
biological effect of a new
treatment will be revealed by
the study
AHRQ GOLD Grant:
Cluster Randomized Trials
 HMO research Network CERT – Harvard
Pilgrim Health Care, Wellesley, MA
– CRTs are rarely used to study the comparative
effectiveness of multiple treatments, such as two
different drug regimens. This study:
 Issues that must be addressed in order to perform CRTs
in a manner that is acceptable to all parties, feasible and
methodologically sound
 Evaluate medical ethics and develop ethical standards
to inform researchers and health plans considering
CRTs.
Duration of Project
September 1, 2000 – September 29, 2007
Istituto di Ricerche
Farmacologiche Mario Negri
 2001 study of 3,567 outpatients with
type 2 diabetes, using AHRQ’s
Diabetes PORT
– Findings involving101 outpatient
diabetic clinics and 103 general
practitioners throughout Italy:
 Self-monitoring of blood glucose can
improve metabolic control as part of a
strategy for promoting patient
autonomy
 For patients not treated with insulin,
self-monitoring is associated with
higher blood glucose levels and
psychological burden
Istituto di Ricerche Farmacologiche Mario Negri
The Guide to Clinical
Preventive Services 2006
 Contains 53 new or
revised clinical preventive
services
recommendations from
the U.S. Preventive
Services Task Force
 AHRQ and United Health
Foundation to distribute
430,000 copies to primary
care physicians
Electronic Preventive
Services Selector (ePSS)
 A quick hands-on tool designed
to help primary care clinicians
identify screening, counseling
and preventive medication
services appropriate for patients
 PDA and Web versions based on
current USPSTF
recommendations, searchable by
specific patient characteristics
 Available at www.ePSS.ahrq.gov
Consumer Version Underway
Why is the Supply of PCTs
Inadequate?
 Inadequate public and private funding
 Costly and often longitudinal studies
 Lack of organized mechanism to identify
high priority questions for decision
makers
Increasing PCTs Supplies
 Development of a mechanism to
establish priorities for these studies
 Significant expansion of an infrastructure
to conduct clinical research within the
health care delivery system
 More reliance on high-quality evidence
by health care decision makers
 Substantial increase in public and
private funding for these studies
Strategies for Improvement
 Systematically identify and prioritize
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
knowledge gaps
Decision makers Insist on high-quality
evidence in making decisions
Create operational infrastructure
Address Methodological and Ethical
Issues
Examine funding options
Your questions?