Although patients and manufacturers support CER generally, they are wary that government-funded studies will steer health coverage decisions towards low-cost remedies and away innovative products with high price tags. Biopharmaceutical companies have worked hard to shift the focus of the federal program take away from drug–drug comparisons and toward the more difficult task of evaluating medical procedures and methods of care. Personalized medicine advocates, moreover, are pressing for CER to consider treatment effects on patient subpopulations, including minorities, children and individuals with uncommon health problems—and not what works best in the average patient. Yet, designing studies that are able to detect such differences is tricky and may increase the scope and cost of research.
LAUNCHING PCORIA range of public and private researchers and payers have been sponsoring CER studies for many years in an effort to identify the most effective medical therapies and practices, as well as those that are inappropriate or even harmful. Comparison of the costs and effectiveness of new drugs and medical technology is the objective of most CER initiatives, including the United Kingdom's National Institute for Health and Clinical Excellence (NICE) and the University of Oregon-based Drug Effectiveness Review Project (DERP), which evaluates drugs for state Medicaid programs and other payers. In establishing the Medicare drug benefit in 2003, Congress provided additional funds (nearly $150 million over the last five years) for the Agency for Healthcare Research and Quality (AHRQ) to solicit systematic reviews and literature syntheses related to treatment of prevalent conditions affecting Medicare beneficiaries.
The federal stimulus legislation enacted in 2009 (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 (HHS) to set priorities for and fund comparative studies. Much of the money went to AHRQ and the National Institutes of Health (NIH) to support CER projects and infrastructure development.
This year's health reform legislation built on ARRA by establishing PCORI as an independent, non-profit organization. The initiative's $500 million annual budget, beginning in 2014, will be funded largely by a 1% tax on health insurance premiums—a strategy designed to insulate the program from the highly political annual Congressional appropriations process and provide more stability and predictability.
The PCORI board members, who were announced by the Government Accountability Office (GAO) in late September, represent payers, providers, patients and industry, with an emphasis on women and minorities. NIH director Francis Collins and AHRQ chief Carolyn Clancy are on the panel, but do not chair it; that honor goes to UCLA Vice Chancellor and Dean Eugene Washington, with Steven Lipstein, president of the non-profit BJC healthcare system, as vice-chair. Biopharmaceutical industry representatives include Pfizer Chief Medical Officer and Senior Vice-President Freda Lewis-Hall, Harlan Weisman, head of medical devices and diagnostics at Johnson & Johnson, and Richard Kuntz, senior vice president of Medtronic. These three board members reflect industry efforts to gain a seat at the table, to keep the program independent of the federal government, and to establish a transparent process for authorizing studies and releasing research findings.
Almost as important as the structure of PCORI is the role of its methodology committee, to be named shortly by GAO. That committee's assignment is to define appropriate CER study designs and methods and submit initial recommendations in a report to Congress in only 18 months. The panel will weigh criteria for internal validity, generalizability, feasibility, and timeliness of CER studies. Selecting appropriate comparators and determining strengths and weaknesses of observational studies versus randomized controlled trials (RCTs) are key tasks, and are sure to ignite debate on the value of "pragmatic" clinical trials and observational studies, the ethics of conducting RCTs on available treatments, and challenges in designing studies that include diverse subgroups. Ideally, the process of developing evidentiary standards and common definitions for multiple CER activities and processes will link the many diverse guidelines and methods set by regulators and various payers.
An important consideration for industry is whether the demand for more information on how drugs and clinical treatments work in real-world settings will stymie innovation by expanding the scope of data needed to bring new drugs to market. The US FDA does not require comparative or cost information to approve a new drug, although some foreign regulatory authorities do so. Many sponsors now include comparative and clinical-use measures in preapproval trials to meet demands of private payers, and decisions on advancing from Phase 2 to Phase 3 studies increasingly weigh the feasibility of gathering evidence of product value during development. Biopharmaceutical companies oppose mandates to provide such information as a condition of FDA approval. Yet adoption of CER research methods and standards by PCORI is likely to shape study processes for all effectiveness and outcomes research, including studies required by the FDA as part of postmarketing oversight.