Comparative effectiveness research studies will alter methods for assessing the value of new therapies and coverage calculations


PCORI now has a chairman and board representing all stakeholders to take on the daunting task of setting the national CER agenda, developing systems for funding research, establishing standards and methods for comparative studies, and devising programs to disseminate results to practitioners and to the public.

Key Points

There is great optimism throughout the healthcare community that comparative effectiveness research (CER) will both enhance the nation's healthcare system and curb unwarranted spending. Although there is concern that CER could limit access to certain treatments and skepticism about just how big the savings will be, a new federal CER program is poised to invest about $500 million a year on research for how to prevent, diagnose, treat, monitor, and manage disease.

The new program will be funded and managed by the new Patient-Centered Outcomes Research Institute (PCORI), authorized by the Affordable Care Act (ACA), enacted last March. PCORI now has a chairman and board representing all stakeholders to take on the daunting task of setting the national CER agenda, developing systems for funding research, establishing standards and methods for comparative studies, and devising programs to disseminate results to practitioners and to the public.

Payers, health plans, pharmaceutical manufacturers, and patient advocates all support CER in general, but are watching closely to see whether the resulting research is based on scientific standards and provides valid information. Personalized medicine advocates, moreover, are pressing for CER to consider treatment effects on patient subpopulations, including minorities, children, and individuals with rare conditions-and not what works best in the average patient. Yet, designing studies able to detect such differences is tricky and may increase the scope and cost of research.


CER, or health technology assessment, has been around for years, funded largely by the National Institutes of Health (NIH) and public and private payers seeking to identify more effective medical products and practices versus those that are inappropriate or even harmful. New drug costs and effectiveness have been a prime target, particularly for the United Kingdom's National Institute for Health and Clinical Excellence (NICE) and the University of Oregon-based Drug Effectiveness Review Project (DERP).

The federal government became more involved in this field when it established the Medicare drug benefit in 2003, which authorized additional funds (nearly $150 million over the last 5 years) for the Agency for Healthcare Research and Quality (AHRQ) to fund systematic reviews of treatments for conditions largely affecting Medicare beneficiaries. The 2009 federal stimulus bill (American Recovery and Reinvestment Act, or ARRA) dramatically advanced federal support for CER by providing $1.1 billion for the Department of Health and Human Services to set priorities for and fund comparative studies, primarily through NIH and AHRQ.

This year's health reform legislation built on ARRA by establishing PCORI as an independent, non-profit organization. The program's annual budget, estimated at $500 million by 2015, will be funded largely by a 1% tax on health insurance premiums-a strategy designed to provide more stability and predictability for the initiative by insulating it from the highly political Congressional appropriations process.

PCORI's 21 board members, who were announced by the Government Accountability Office (GAO) in late September, include payers, providers, patient advocates, and industry. NIH Director Francis Collins, MD, PhD and AHRQ Director Carolyn Clancy, MD, are on the panel, but do not chair it; that honor goes to UCLA Health Sciences Vice Chancellor and Dean A. Eugene Washington, MD, MSc, with Steven Lipstein, MHA, president and CEO of the non-profit healthcare organization BJC Health Care, as vice-chair. Three members represent pharmaceutical and medical device makers, 3 are from consumer groups, and several represent health plans and payers. A PCORI resource guide prepared by the National Pharmaceutical Council provides full biographies of all the members (

The Board is expected to award many of its grants through NIH and AHRQ to take advantage of their peer-review and research infrastructure. But before it can start funding projects, the panel has to hire a staff, establish offices, and craft a charter for operations.

Almost as important as the structure of PCORI is the role of its methodology committee, which GAO is slated to name shortly. That committee's assignment is to define appropriate CER study designs and methods and submit a report with recommendations in 18 months. The panel will weigh criteria for internal study validity, generalizability, feasibility, and selection of appropriate comparators. The task of determining strengths and weaknesses of observational studies versus randomized controlled trials (RCTs) is sure to ignite debate on the value of "pragmatic" clinical trials, the ethics of conducting RCTs on marketed therapies, and design of studies with multiple subgroups. Ideally, the process of establishing common definitions and evidentiary standards will link the many diverse guidelines and methods already adopted by various agencies and payers.

An important consideration for pharmaceutical companies and drug plans is whether the demand for more information on how drugs work in real-world settings expands the scope of data needed to bring new products to market. FDA does not require comparative or cost information to approve a new drug, although some foreign regulatory authorities do so, and patient advocates would like FDA to follow suit. New drug sponsors now regularly include comparative and clinical-use measures in preapproval trials to meet demands of private payers, and decisions on advancing from phase 2 to 3 studies increasingly weigh the feasibility of gathering evidence of product value during development. Adoption of CER methods and standards by PCORI thus is likely to have a broad impact on the design of all effectiveness and outcomes research, from preapproval trials to studies required by FDA as part of postmarketing oversight.


Whether more comparative research will actually limit healthcare spending remains to be seen. Analysts project that the CER initiative will reduce federal outlays for healthcare by about $3 billion over 10 years-just about what the government will spend on PCORI. That calculation assumes that more comparative information will lead to changes in physician practice and patient choice.

Potential savings from CER are curbed by Congress' stipulation that Medicare cannot use study results to establish cost-effectiveness thresholds, set practice guidelines, or make coverage or payment recommendations. However, private insurers and payers are free to tap CER evidence in their coverage decisions, as they have done for years. And more outcomes studies will support efforts by payers to negotiate lower rates and to steer consumers to more high-value therapies and care options.

Consequently, some analysts believe that comparative studies should consider cost and pricing issues. At a CER briefing last month sponsored by Health Affairs, Harold Sox, MD, MACP, professor of medicine at Dartmouth Medical School, recommended that CER studies include data on utilization and costs so that such information is available to health plans and payers and can inform the public about what they're paying for. Harvard researcher Steven Pearson, MD, MSc, FRCP, made the radical proposal that Medicare reward innovation by paying higher prices for products that can document superiority, but only a comparable or "reference" price for those demonstrating comparable clinical effectiveness; new products that fall in the middle would have 3 years after FDA approval to collect data supporting a premium price.

For CER results to have any positive impact on the medical community, Harvard Medical School Professor of Medicine Jerry Avorn, MD, proposed that PCORI support "academic detailing" and "social marketing" of comparative assessments, largely to offset "pharma's hype of newer, more costly products," he commented. Dr Avorn also wants FDA to require "relevant comparisons with alternative treatments" in pharmaceutical promotional materials, and to crack down on unsubstantiated superiority claims.

Public support for CER is fairly strong, according to recent public opinion surveys, but physician backing for the research is necessary to offset Americans' fears of mandatory guidelines that could lead to "one-size-fits-all" medicine, policy experts Alan Gerber and Eric Patashnik reported.

An "early win" for the program could come from some of the studies funded under ARRA that soon should begin publishing results.

"But CER is not a panacea or a silver bullet," observed Kavita Patel, MD, MS, director of the Health Policy Program at the New American Foundation, Washington, D.C. She and others advise PCORI's board to look for some "low-hanging fruit" on the CER priority list that can demonstrate how comparative research can accelerate patient access to treatment. ECRI Institute President and CEO Jeffrey Lerner, PhD, observed that it "will take a great deal of work convincing people that CER is in their favor-that it's something they should want."

Ms Wechsler is a Washington-based reporter specializing in federal and state healthcare issues.

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