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Statement Before the U.S. House of Representatives Committee on Ways and
Means, Subcommittee on Health
Carolyn M. Clancy, M.D., Director, Agency
for Healthcare Research and Quality, U.S. Department of Health and
Human Services, June 12, 2007
morning, Mr. Chairman and members of the Subcommittee. I am Dr. Carolyn
Clancy, the Director of the Agency for Healthcare Research and Quality (AHRQ),
an Agency of the U.S. Department of Health and Human Services (HHS). I am very
pleased to testify before you on the exciting issue of comparative
effectiveness. I am thrilled about the growing interest in, and attention to,
enhancing the role of comparative effectiveness research in our health care
system. And I am particularly pleased to be able to tell you about AHRQ's
important efforts in this area of research.
is a very interesting time in the history of health and medicine. Our
investments in biomedical research have resulted in many new diagnostic and
therapeutic options. Clinicians and patients can often now choose among an
expanded array of choices for treating hypertension, heart failure, HIV, mental
illness, and other chronic illnesses, and unprecedented innovations in
diagnosis and prediction bring us closer to a vision of personalized health
care than ever.
also are beginning to reap the benefits from the advances in health information
technology (health IT) that can bring this information immediately to
clinicians, patients, and others when and where they need it. Health IT also
is enhancing our research capacity and our ability to diffuse breakthroughs
quickly and efficiently throughout the health care system. Health IT can make
research a natural by-product of delivering health care.
this brave new world of health care presents wonderful opportunities, it also
creates challenges. Chief among them is how to evaluate these innovations and
determine which represent added value, which offer minimal enhancements to
current choices, which fail to reach their potential, and which work for some
patients and not for others. The need to develop better evidence about the
benefits and risks of alternative choices is imperative.
mission of AHRQ is to improve the quality, safety, efficiency, and
effectiveness of health care for all Americans. Effectiveness sits squarely in
our mission—what is the right treatment for the right patient at the right
effectiveness research is a means to an end. Our mission is fulfilled when
health care decisionmakers—including patients, clinicians, purchasers, and
policymakers—use up-to-date, evidence-based information about their treatment
options to make informed health care decisions. This goal was the inspiration
for the creation of AHRQ by those members of Congress who grasped the power of
information to improve the health care system and the health of Americans.
Health Care Program
was granted authority under Section 1013 of the Medicare Prescription Drug,
Improvement, and Modernization Act of 2003 (MMA) to conduct and support evidence
syntheses and research on topics of highest priority to Medicare, Medicaid, and
the State Children's Health Insurance Program (SCHIP).
would like to thank Congress for its recognition of the programs and
infrastructure that AHRQ has established for providing the health care system
with the scientific evidence that it needs to provide safe, high-quality
effective health care. AHRQ's Evidence-based Practice Centers (EPCs), Centers
for Education and Research on Therapeutics, and other research programs have
become a trusted, unbiased national source of information on health care
diagnostics and treatments.
programs are an integral part of AHRQ's Effective Health Care program,
which was created under the authority of Section 1013. AHRQ was able to
establish the Effective Health Care program and begin work very quickly because
of our solid, existing research enterprise.
Secretary of HHS, Michael Leavitt, has established priorities for research
conducted in the Effective Health Care Program by establishing high priority
conditions that have a major impact for the Medicare, Medicaid, and SCHIP
programs. To be effective, comparative effectiveness
research must be relevant to its users. decisionmakers are often faced with
situations for which multiple different treatments are relevant at different
times. For example, should a 55-year-old woman with a scan showing
greatly decreased bone density take drugs, increase Vitamin D and calcium
intake, focus on weight-bearing exercise, or watchfully wait? We know that
drugs are effective, but there is limited information on their long-term
effects. Some women will develop kidney stones after increased calcium;
current evidence does not allow precise formulation of an effective exercise
prescription; and many women will never experience a fracture.
The key to success for this research is that it provides evidence
that informs the choices confronting clinicians and patients and, where
possible, should closely align with the sequence of decisions they face. As MMA
Section 1013 directs, we also need to ensure that findings are frequently revisited,
so they remain relevant and up-to-date. New evidence, such as a genetic test
that identifies people at increased risk of untoward outcomes, affects comparative
effectiveness and should be incorporated into these reviews at the appropriate
the statute, the Secretary of HHS is required to establish priorities, informed
by a transparent priority-setting process that includes all stakeholders.
Priorities for the Effective Health Care Program therefore are set after
receiving broad public input through Federal Register notices, public
listening sessions, and other means.
was much discussion within HHS about how to approach these priorities. During
our discussion of research on diseases or conditions, for example, we debated specific
questions about treating diabetes and heart disease, and whether our research
should center on particular medications and interventions, such as stents or
proton pump inhibitors.
decided to take a disease- and condition-based approach because, at the end of
the day, that is how health care decisions are made. A patient comes to the
health care system with a condition or disease, and all decisions, including
how best to treat it, follow.
December 2004, based on input from stakeholders, the Secretary of HHS identified
10 priority conditions—all of special significance to the Medicare program—to
be the first addressed by the Effective Health Care Program. These conditions
- Arthritis and nontraumatic joint disorders.
- Chronic obstructive pulmonary disease/asthma.
- Dementia, including Alzheimer's disease.
- Depression and other mood disorders.
- Diabetes mellitus.
- Ischemic heart disease.
- Peptic ulcer/dyspepsia.
- Stroke, including control of hypertension.
AHRQ's Effective Health Care Program comprises three parts. The
first capitalizes on effectiveness research conducted by AHRQ's existing 13
EPCs, which were created in 1997. The EPCs develop comparative effectiveness
reviews which focus on treatments for the priority conditions. These reports
synthesize currently available scientific evidence, including both published
and unpublished studies, comparing treatments, including drugs, to determine
relative benefits and risks, and wherever possible, measure these outcomes for
subpopulation groups. In addition, the EPCs identify major gaps in the
existing knowledge base.
To help fill these gaps, AHRQ established the second part of the
Effective Health Care program, called the DEcIDE (Developing
Evidence to Inform Decisions about Effectiveness) network, which will focus on
conducting rapid-cycle research to address specific issues that do not
necessitate larger, more time-consuming randomized clinical trials. The DEcIDE
network consists of 13 research centers that have access to databases that
contain clinical information for more than 50 million patients but do not
identify them individually.
The third part of the Effective Health Care program is the John M.
Eisenberg Clinical Decisions and Communications Science Center, based at the Oregon
Health & Science University's Department of Medicine. The Eisenberg Center was established to ensure that the findings of our comparative effectiveness
research are translated into formats that are understandable for all potential
users. The Center—named the John M. Eisenberg Center in honor of AHRQ's late
director—assists in ensuring that effectiveness research leads to real-world
quality improvements by translating complex scientific findings into
understandable language for different audiences. The Center will help assure
that reports are presented in formats that make them useful to a wide range of
audiences and also will develop tools that encourage and empower consumers to
make informed health care decisions.
important hallmark of the Effective Health Care program is transparency in all
aspects of the process. The transparency begins with the open process for
setting research priorities, described earlier. The public and all interested
stakeholders also have the opportunity to comment on the framing of specific
research questions, as well as commenting on draft reports. In addition to
the open invitation to comment, manufacturers are notified when a study is begun
on one of their products and are invited to submit relevant studies and data.
research questions and reports are posted on AHRQ's Effective Health Care Program
Web site (http://www.effectivehealthcare.ahrq.gov), and the Web site has a
LISTSERV™ that automatically notifies interested parties when draft questions or
draft reports are posted.
date, AHRQ has released seven comparative effectiveness reviews. These reviews
can be found on the Effective Health Care Program Web site, discussed above.
Gastroesophageal Reflux Disease (GERD)
management of gastroesophageal reflux disease, medications called proton pump
inhibitors can be as effective as surgery in relieving the symptoms and
improving quality of life.
women who receive abnormal mammography findings or physical exams, four
common noninvasive tests (magnetic resonance imaging, ultrasonography, positron
emission tomography scanning, and scintimammography) are not accurate enough to
routinely replace biopsies.
Anemia in Cancer Patients
cancer patients undergoing chemotherapy or radiation, there is no clinically significant
difference between epoetin and darbepoetin in the management of anemia. The
drugs show no clinically significant difference in improving hemoglobin
concentration and reducing the need for transfusion.
anti-inflammatory drugs (NSAIDs) and COX-2 inhibitors present similar increased
risks of heart attacks while offering about the same level of pain relief for
patients with osteoarthritis. The exception is naproxen, which presents a lower
risk of heart attack for some patients than other NSAIDs or COX-2 inhibitors.
numbers of patients with narrowed kidney arteries are undergoing
vessel-widening angioplasty and placement of a tubular stent, but evidence does
not show a clear advantage of that treatment over prescription drug therapy.
Off-Label Use of Atypical
Some newer antipsychotic medications approved to treat
schizophrenia and bipolar disorder are being prescribed for depression,
dementia, and other psychiatric disorders without strong evidence that such
off-label uses are effective. Research is urgently needed for new treatments of
dementia patients with severe agitation.
Today's most commonly prescribed antidepressants are similarly
effective to first-generation antidepressants and provide relief to about 6
in 10 patients, but current evidence is insufficient for clinicians to predict
which medications will work best for individual patients. Six in 10 patients
experience at least one side effect, ranging from nausea to sexual dysfunction.
In January 2007, AHRQ released the first summary guide for
consumers and clinicians derived from a Comparative Effectiveness Review by the
Eisenberg Center. The consumer report, titled Choosing Pain Medicine for
Osteoarthritis, translates the information from the comparative
effectiveness report on osteoarthritis drugs into language that will help
consumers choose among their treatment options. The companion guide, Choosing
Non-Opioid Analgesics for Osteoarthritis, further synthesizes the evidence
into a resource that can help clinicians work with their patients to make
informed decisions about treatments for osteoarthritis.
AHRQ has a series of upcoming reports that deal with critically
important issues facing the health care system. They include:
for type 2 diabetes.
- ACEIs (Angiotensin-converting enzyme Inhibitors) versus ARBs (angiotensin
II receptor antagonists) for high blood pressure.
- Surgery versus stents coronary artery disease.
and other treatments (e.g., diet, exercise) for low bone density.
would like to mention briefly the role of health IT, which will make it easier
for researchers to gather information for their research and for users of
research findings to get information in real time when they need it. The
health care system's growing investments in health IT provide us with an
unprecedented opportunity for redefining the possibilities of observational
studies, accelerating and targeting the uptake of relevant information, and
providing feedback to the biomedical enterprise itself.
IT will make it possible for research to answer the pressing questions facing
the health care system more quickly and efficiently. In the future, health IT will
provide us with the vehicle for transforming our health services research
enterprise so that we can evaluate the effectiveness of interventions and
treatments in real time as a byproduct of providing care.
Fiscal Year 2008 budget request includes $15 million for a personalized health
care initiative that will begin the infrastructure for a federated system of
databases that can help answer critical comparative effectiveness questions. This
system would enable researchers to match treatments and outcomes, and in that
way learn from the Nation's day-to-day medical practice and improve safety and
effectiveness of medical treatments.
IT also will greatly improve the ability to diffuse evidence and information
more quickly throughout the health care system. For example, clinical decision
support tools will make it possible to deliver relevant information to clinicians
and patients, at the point of decisionmaking. Most commonly envisioned as a
pop-up reminder on a screen, clinical decision support should include
information communicated directly to patients and caregivers at home—by phone,
computer, or by other means.
AHRQ has implemented the Effective Health Care program, we have some
setting: It is important to set
clear priorities that meet the needs of all the stakeholders in the health
care system. Therefore, end users and stakeholders must continuously provide
input through an open and transparent process.
the research questions: Research
must track closely with how clinicians and patients make health care decisions
every day. The Secretary's decision to use a disease- and condition-based
approach to priorities embodies this perspective. It is also very important to
recognize the importance of revising findings frequently to incorporate new
evidence that may change the conclusions of what works best and for whom.
benefits and harms: Comparative
effectiveness research must provide information on benefits and harms of a particular
medication or intervention. Evaluating the balance of harms and benefits is a
critical component of informed decisionmaking. Few interventions are risk
free, and for many chronic conditions the therapeutic goal is management of
symptoms and disease state rather than cure. Often times, the decision comes
with some assumption of harm—by both patient and clinician—but with the
understanding that the benefits are worth that risk.
is a means, not an end: The ultimate
goal of our research efforts is the development timely, relevant information
for decisionmaking. This requires us to go beyond the products of traditional
research, namely scholarly articles, and translate findings into language and
formats that are appropriate for different audiences. Creating evidence and
information that is not useful and accessible, or that does not take advantage
of the latest communication technologies and vehicles, is a missed opportunity.
as a process, not a structure: As
has been stated, comparative effectiveness research can be a risky business,
and there are winners and losers. Therefore, it is important that there be a
level playing field among stakeholders. AHRQ's Effective Health Care program
has adopted a policy of transparency and inclusion. Manufacturers are notified
when a study is begun, are invited to submit relevant studies and data, and
have the opportunity—along with any other interested party—to comment on the
framing of the specific research questions as well as draft reports. In
addition, it is clear that the program's success is dependent on effective
collaboration with scientists from industry as well as academia. At the same
time, we ensure that the authors of the Comparative Effectiveness Reviews are
free of conflict to make sure that the results are not perceived as being
biased in any way.
question of trust also extends to the integral role that patients play in
research. Although government and the private sector pay for research, patients
assume the risks and benefits of enrolling in clinical trials and other
studies. A question that is the subject of debate is whether study findings can ethically be kept secret from
other researchers and patients themselves. We all need to learn from the
knowledge gained in research, but it can be a matter of life and death for
patients. We must move to an atmosphere where it is unacceptable to hold back
research findings that may have an impact on the care that patients receive.
conclusion, the U.S. health care system is poised to take advantage of advances
in science and health information and communications technology in ways that
have previously only seemed like something out of science fiction.
need for valid, reliable, and accessible information on the comparative
benefits and potential harms of treatment options has gained an urgency due to
recent policies to promote the adoption of interoperable health IT, continued
expansion of diagnostic and treatment options, increased consumer interest in
health and health care decisions, and broad interest in improving value.
Effective Health Care program is a model for how this vision can be achieved:
A transparent, participatory approach that is driven by the needs of users and
encourages broad engagement of stakeholders to mitigate any expected
controversies and to expand opportunities for diffusion of findings of
comparative effectiveness research. The Effective Health Care Program
represents a foundation in which a larger investment in comparative effectiveness
can be built.
you very much, and I would be pleased to answer any questions.
Current as of June 2007