AHRQ: The New Effective Health Care Program


January / February 2006


The New Effective Health Care Program

When consumers shop for a new car, the latest electronic gadget, and many other types of products or services, a wealth of comparative information awaits them on newsstands and online. But when it comes to choosing the most effective drugs and medical treatments, getting useful information can be as challenging as finding a parking space at the mall on the day before Christmas.

Information about the effectiveness of many types of medications and therapies is available, but pulling it together and then using it to make healthcare decisions is too complicated for most consumers and many clinicians. Research findings are most often presented in discrete chunks — finding a good overview is far more difficult. As the cost of healthcare continues to grow, consumers are paying ever-higher out-of-pocket costs. They want to know what they are paying for and where their healthcare dollars can buy the best value. And so does America’s biggest purchaser of healthcare services — the federal government.

When the Medicare Modernization Act (MMA) was enacted in 2003, Congress asked the U.S. Agency for Healthcare Research and Quality (AHRQ) to meet the growing need for reliable and understandable information about the relative effectiveness of widely prescribed drugs and medical treatments. AHRQ was the natural choice among Department of Health and Human Services agencies to take on this new role because of its leadership role in health services research and its experience in sponsoring, analyzing, and translating research into practice.

In September 2005, AHRQ launched the Effective Health Care Program, a comprehensive effort to synthesize treatment information, compare treatment outcomes, and translate findings into language that consumers, doctors, and employers can use. Because the Effective Health Care Program was started through the Medicare Modernization Act of 2003, it will initially emphasize issues related to the Medicare program. However, in the future, the focus will expand to include Medicaid and the State Children’s Health Insurance Program (SCHIP), addressing clinical issues affecting Americans of all ages.

The Effective Health Care Program will focus on common clinical problems where significant questions exist about the relative effectiveness of alternative clinical interventions. The new program will enable clinicians and consumers to use the best available information when selecting the alternatives that best meet individual needs.

While AHRQ and others in the health services research community have been involved in effectiveness research for years, the new program will take a more strategic approach (choosing particular topics where there are urgent questions), employing a systematic method of generating evidence and placing increased emphasis on putting evidence into use (including translating that evidence into usable terms).

Naturally, as is true of all effectiveness research, the new Effective Health Care Program will exist in a very dynamic environment, with continued change in the evidence base. Across the program’s various components, continuing emphasis will be on examining new evidence as it becomes available and incorporating it into the knowledge base made available to decision-makers.

It is also important to understand what the new program will not do. The Effective Health Care Program will focus tightly on the relative benefits and risks of alternative therapies. The program will not make recommendations among different types of therapies. It will notinclude cost considerations or other issues outside of questions that have a direct impact on medical effectiveness.

Background: The Current Dilemma
Despite historic attempts to prioritize effectiveness research and to identify bases of knowledge systematically, prior to the Effective Health Care Program, there was no comprehensive effort to synthesize and inventory available research; identify and resolve major gaps in this research; and address the problem of communicating with relevant audiences, including both clinicians and consumers.

In many ways, physicians and other medical decision-makers confront a growing burden in trying to understand the relative benefits and risks of available treatments. The current generation of medical evidence is so multi-faceted that it is increasingly difficult for providers to stay current even within their particular specialties. They need a systematized knowledge base that will bring order to a world of chaos.

Disturbing evidence of inattention to medical evidence has become apparent in recent decades. For example, in the 1970s and 1980s, AHRQ-sponsored research documented dramatic inconsistencies across the country in the treatment of identical conditions. These geographic variations in medical practice suggested the lack of a reliable knowledge base to inform medical decision-makers about practices shown to be most effective. By the 1990s and 2000s, with the growth in interest in medical errors and patient safety and the publication of reports such as the Institute of Medicine’s To Err Is Human(2000), there was increasing awareness that American healthcare was not as advanced and reliable as popular perceptions indicated.

The MMA-Congress Intervenes to Change the Medical Landscape
By enacting the MMA in 2003 and establishing the Effective Health Care Program in Section 1013 of the new law, Congress underscored the impact that effectiveness research can have on the quality of care. The fundamental features of AHRQ’s new mandate are a more systematic approach to identifying the drugs and treatments that work best for specific healthcare conditions and an emphasis on helping to put those findings into practice.

Section 1013 of the MMA reflected growing awareness in Congress and among a variety of medical decision-makers of the importance of a strong medical evidence base. The MMA charges AHRQ to take a systematic approach whereby:


  • The most urgent needs for effectiveness information are identified through an inclusive and comprehensive process.
  • Existing data are reviewed and synthesized based on sound, consistent, and transparent methodologies.
  • Significant gaps in data are targeted to help effectuate productive research as quickly as possible.
  • Findings are disseminated widely and understandably to a variety of users, including payers, policy-makers, clinicians, and consumers.


Stakeholder and Public Input
The new Effective Health Care Program provides opportunities for public input, which will help ensure that the new program responds to the most pressing issues, that it continues to produce new knowledge, and that its products are useful for healthcare decision-makers. A Web site has been established (www.effectivehealthcare.ahrq.gov) to support a public comment process as well as the vital goal of disseminating results.

In December 2004, following public input, AHRQ identified 10 priority conditions — all of special significance to the Medicare program — to be addressed by the Effective Health Care Program:


  • Arthritis and nontraumatic joint disorders
  • Cancer
  • Chronic obstructive pulmonary disease/asthma
  • Dementia, including Alzheimer’s disease
  • Depression and other mood disorders
  • Diabetes mellitus
  • Ischemic heart disease
  • Peptic ulcer/dyspepsia
  • Pneumonia
  • Stroke, including control of hypertension


Future lists will also include priority conditions relevant to Medicaid and SCHIP beneficiaries.

By June 2005, following additional input from stakeholders and the public, AHRQ announced 10 specific topics within these priority areas for the program’s first reports. In December 2005, AHRQ reached an important milestone with the issuance of its first Comparative Effectiveness Review on management strategies for gastroesophageal reflux disease (GERD), one of the most common health conditions affecting older Americans, resulting in $10 billion in direct annual healthcare costs. This report, prepared by the Tufts-New England Medical Center Evidence-based Practice Center, compares the two major types of medications available for GERD as well as surgical interventions. Nine more comparative effectiveness studies are underway, including one on atypical antipsychotic medications, which was recently posted for public comment.

The Three-Part Structure of the New Program
The new Effective Health Care Program is administered through a three-part structure.

First, the program will capitalize on the existing network of AHRQ’s Evidence-Based Practice Centers (EPCs), which were created in 1977 and are located across the United States and in Canada. The EPCs have issued more than 125 reports to date and will produce as many as 20 Comparative Effectiveness Reviews annually under the new effectiveness mission. The EPCs will focus on treatments for the priority conditions listed above and synthesize currently available scientific evidence, including both published and unpublished studies. They will compare treatments, including drugs, to determine relative benefits and risks, and wherever possible, to measure these outcomes for subpopulation groups. Equally importantly, the EPCs will identify major gaps in the existing knowledge/information base.

Second, AHRQ has established the new DEcIDE network (Developing Evidence to Inform Decisions about Effectiveness), a group of 13 research centers whose purpose is to address research gaps, including those identified by the EPCs, as well as developing improved methodological approaches. The DEcIDE network will perform prospective observational studies by evaluating patient-level data stripped of identifying information. The emphasis will be on generating findings expeditiously to address specific issues that do not necessitate larger, more time-consuming randomized clinical trials. The network will have access to databases that contain clinical information for more than 50 million patients but do not identify them individually.

Finally, the new Clinical Decisions and Communications Science Center,based at the Oregon Health and Science University’s Department of Medicine, has been created to ensure that the findings of the Effective Health Care Program are usable to those who need them. The establishment of the new center — named the John M. Eisenberg Center after AHRQ’s esteemed late director — reflects Congressional interest in seeing that effectiveness research leads to real-world quality improvements. The Center will help assure that reports are presented in formats that make them useful and actionable to a range of audiences and will develop tools to facilitate consumer decision-making.

Health Information Technology and Effective Health Care
Health information technology (IT) has great potential to expand the impact of effectiveness research. With the Effective Health Care Program, the federal government has created a systematic approach to synthesizing, generating, and translating medical knowledge. But the real quantum leap will come when evidence-based medicine is combined with health IT to deliver medical decision-making information in real time.

Health IT will make new data linkages possible and will facilitate rapid “real-world” data analyses. Health IT also represents an important conduit for getting information to decision-makers through electronic medical records, personal health records, and decision support for clinicians.

Perhaps now more than ever, the environment in which clinical decisions are made is characterized by a mixed and uncoordinated body of data that is in a state of constant change. An important task, which previously was addressed incompletely, in a rather piecemeal way, is systematizing these data and comparing outcomes to help consumers and providers make treatment choices. That is where the new Effective Health Care Program faces its greatest challenge and its greatest potential for promoting quality healthcare and patient safety. We owe it to both patients and clinicians to share the best we know now, even as we develop systems for learning more and faster in the future.

Carolyn Clancy (cclancy@ahrq.gov) is director of the Agency for Healthcare Research and Quality.


Institute of Medicine. (2000). To err is human: Building a safer health system. L. T. Kohn, J. M. Corrigan, & M. S. Donaldson (Eds.). Washington, DC: National Academy Press.