AHRQ: Comparative Effectiveness Research

November / December 2009


Comparative Effectiveness Research:
Keeping the Patient at the Center

Comparative effectiveness research (CER) — the comparison of existing healthcare interventions to determine which work best for which patients and which pose the greatest benefits and harms—may seem straightforward at first glance. Yet few provisions in the American Recovery and Reinvestment Act of 2009 (the Recovery Act) generated as much interest as the $1.1 billion allotted for CER.

The higher profile of CER has raised many questions: Will CER affect clinicians’ autonomy, and if so, how? How should research findings affect clinical and payment decisions? Which clinical studies represent the best investments for federal research dollars? Should costs be analyzed?

These are interesting, high-level policy questions, and as a physician and a health services researcher, I welcome them. But there is one other question that should be borne in mind as well.

When an elderly woman with heart disease seeks the best way to treat the pain from arthritis, there is only question that matters: “What is the best choice for me?” The same is true for an elderly man seeking treatment for depression. Or an active, middle-aged woman who finds that medication prescribed to treat atrial fibrillation isn’t working, and she is considering surgery. Or the mother of a child with chronic illness who wants to know whether a medical home or other comprehensive care coordination program is right for her family.

Clinicians and patients confront healthcare decisions like these every day, often without adequate information. “What is the best choice for me?” Unfortunately, the answer to this real-world, patient-centered question is often, “We don’t really know.”

Therefore, as we pursue enhanced opportunities for CER, and as we grow to understand the policy implications, I encourage the tough questions, knowing that a direct and honest appraisal of this very important type of research will lead to greater understanding and, ultimately, will enhance healthcare in the United States. But I also hope that the ultimate patient-centered question, “What is the best choice for me?” is not forgotten.  In short, CER is essential to deliver care that is truly patient-centered.

CER, the Recovery Act, and AHRQ’s Effective Health Care Program
Of the $1.1 billion in CER funding under the Recovery Act, $300 million is targeted to the Agency for Healthcare Research and Quality (AHRQ) to expand its existing Effective Health Care (EHC) Program. Of the remaining funds, $400 million each will go to the National Institutes of Health (NIH) and the Office of the Health and Human Services (HHS) Secretary. These funds are to be distributed by the end of fiscal year 2010.

Two federal reports published this summer will guide our immediate work. First, the Institute of Medicine (IOM) has recommended research priorities for the secretary’s funds and gathering input from stakeholders. The IOM has recommended 100 initial priority topics, divided into four quartiles, for CER (Iglehart, 2009; IOM, 2009). Second, the Recovery Act created a new Federal Coordinating Council for CER, which is to offer guidance to the secretary on the use of new CER funding. The Coordinating Council’s Report to the president and Congress (2009, June 30) offered a definition of CER, created a strategic framework, asserted priorities for research, and formulated recommendations for how the HHS portion of the funding should be spent (Conway & Clancy, 2009). Of particular interest, both groups developed similar definitions of CER that explicitly include care delivery as well as clinical interventions.

The Recovery Act raised the profile of CER, but it did not introduce it in the United States. AHRQ has been funding CER since 2003, when Congress created the EHC Program under the Medicare Prescription Drug, Improvement, and Modernization Act. In doing so, Congress mandated that AHRQ develop evidence about the effectiveness and comparative effectiveness of different treatments and clinical approaches to significant health problems and communicate its findings widely to a variety of audiences, including consumers.

The EHC Program was initially funded at $15 million for the first year, an appropriation that increased by 2009 to $50 million. It has successfully grown into a program that is collaborative, transparent, stakeholder-driven, relevant, and timely.  Our goal at all times is to develop evidence-based information that is both rigorous and relevant to clinical decisions and is available when decisions are made. We have learned that to achieve that goal, ongoing dialogue with public and private sector stakeholders is essential.

Since the program’s inception, it has published more than 45 products, including summary guides for clinicians such as a comparison of treatments for osteoarthritis of the knee, treatments for clinically localized prostate cancer, and comparing oral medications for adults with type 2 diabetes. New research has examined the effectiveness and safety of bare metal and drug eluting cardiac stents. And, the program has published tools to assist researchers, such as a user’s guide to registries for evaluating patient outcomes and an assessment of infrastructure to monitor utilization and outcomes of gene-based applications. (More information on AHRQ’s Effective Health Care program can be found at: http://effectivehealthcare.ahrq.gov/.)

Relevance and Transparency
The questions investigated by CER are intended to take medical research from the theoretical to the practical. To be effective, CER must be relevant to its users, including individual consumers.
Patients and clinicians often are faced with clinical circumstances for which more than one treatment course may be a viable option. So, to succeed, CER should provide evidence that informs the choices confronting clinicians and patients and, where possible, should closely align with the sequence of decisions they face. It should also ensure that findings are frequently revisited so that they remain relevant and up-to-date.

And, in order to gain and maintain the trust of all parties, CER must be fully transparent to all. Transparency has been a hallmark of the EHC Program. The transparency begins with an open process for setting research priorities, which the secretary of HHS sets through discussion with and extensive input from stakeholders. Within the boundaries of those priorities, the public and other interested stakeholders have the opportunity to comment on the framing of specific research questions and to critique draft reports. In addition to the open invitation to comment, manufacturers are notified when a comparative effectiveness review begins on one of their products and are invited to submit relevant studies and data. Efforts to encourage outside input ensure that all stakeholders have equal and fair access to the process. (To sign up for updates, visit http://effectivehealthcare.ahrq.gov. To review AHRQ’s standing program and training award announcements, please visit http://www.ahrq.gov/fund/grantix.htm.) 

CER succeeds when it gets the best available evidence at the time of decision-making into the hands of those who need it the most. This is, not coincidentally, one of AHRQ’s highest priorities. We will need to remain vigilant that CER findings are descriptive, not prescriptive, so that CER gives us the potential to create a fairer, more transparent, patient-centered health system. Every patient presents a unique set of clinical circumstances that should be taken into consideration when making treatment decisions. Nothing should compromise the best possible care for each individual.

Carolyn Clancy is director of the Agency for Healthcare Research and Quality, Rockville, Maryland. She is a general internist and holds an academic appointment at George Washington School of Medicine in Washington, D.C. She may be contacted at carolyn.clancy@ahrq.hhs.gov.

Conway, P. H., & Clancy, C. M. (2009). Comparative-effectiveness research — Implications of the federal coordinating council’s report. New England Journal of Medicine, 361, 328-330.

Institute of Medicine. (2009). Initial National Priorities for Comparative Effectiveness Research. Washington, DC: National Academy Press. Available online at http://www.iom.edu/CMS/3809/63608/71025.aspx. Accessed September 2009.