Comparative Effectiveness Research to Shape Biotech Studies

Authorities are pushing for CE; manufacturers prefer to focus on value.
May 01, 2009
Volume 22, Issue 5

The economic stimulus package approved by Congress in February provides more than $1 billion to support research on competing medical treatments. Although a fairly minor piece of the larger $789 billion American Recovery and Reinvestment Act of 2009 (ARRA), the provision has generated considerable controversy.

Jill Wechsler
Conservatives raised the specter of "government rationing" and "cookbook medicine." Comparative effectiveness (CE) research enthusiasts countered that more and better information about which medical products and procedures are most effective can improve care and cut unnecessary spending.

The final legislation represents a compromise by stating that Congress does not intend for CE research to be used to "mandate coverage, reimbursement or other policies for any public or private payer." However, more evaluation of clinical outcomes and treatment effectiveness opens the door to a much broader role for the federal government in selecting and shaping comparative assessments and to greater reliance on CE studies for directing medical practice.


Instead of establishing a new, independent entity to carry out CE research, as some advocated, the stimulus package divides the $1.1 billion in funds among three arms of the Department of Health and Human Services (HHS). The Agency for Healthcare Research and Quality (AHRQ) gains $300 million to expand its outcomes and effectiveness research program. The National Institutes of Health (NIH) gets $400 million to support research conducted by its various institutes. The remaining $400 million goes to the HHS secretary to support standards development, a registry, and data systems and other activities.

How these agencies dole out the money will be shaped by a June 30 report from the Institute of Medicine (IOM) recommending priorities for CE research. Dozens of interested parties presented their opinions at a public meeting in March on how CE research should be conducted and which topics should be studied. The IOM panel will consider the needs of populations served by federal programs, with an eye to including women and minorities in research.

To ensure broad input to the priority-setting process, the IOM also established an online system for anyone to submit specific proposals for CE studies. The panel is ranking these submissions according to disease burden and severity, variation in care, cost, public interest, and gaps in available information.

A positive result of CE research would be the development of standards and improved methods for conducting comparative studies. At the IOM meeting, Bryan Luce, senior vice president at United BioSource, urged "true transformational thinking" in designing CE research studies; otherwise, he said, we will "waste vast amounts of money answering the wrong questions, or the right questions too late."

A new Federal Coordinating Council for CE Research will coordinate government-funded initiatives and monitor how well HHS meets the IOM priorities in awarding CE research grants. The 15-member council includes top officials from the HHS, NIH, AHRQ, the US Food and Drug Administration and other federal agencies. In the spirit of transparency, HHS will publish information on grants and contracts awarded, disseminate the research findings that result, and report annually to Congress on the program.


Although ARRA funding will spur CE-studies activity, a good deal of this research has been going on for some time. The Medicare Modernization Act of 2003 provided limited funding to AHRQ for research to determine the clinical effectiveness and appropriateness of various health services, including prescription drugs. In the private sector, the Blue Cross and Blue Shield Association's Technology Evaluation Center has been reviewing clinical evidence since 1985 to determine the effectiveness of certain medical procedures, drugs, and medical devices. The Drug Effectiveness Review Project at the Oregon Health & Science University provides state Medicaid agencies and large insurers with comparative information on the efficacy and safety of new, high-cost medicines, and on drugs frequently used off-label.

A number of foreign CE research programs have spurred interest in the US. Most prominent is the National Institute for Health and Clinical Excellence (NICE) in the United Kingdom, which provides Britain's National Health Service with recommendations on coverage of new drugs and diagnostics. NICE sets a clear cost-effectiveness threshold that has led to controversial no-coverage decisions on several new, but costly, biotech therapies.

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