Jill Wechsler
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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 set off a firestorm about using comparative study results to limit coverage of more expensive medicines.
Conservatives raised the specter of "government rationing" and "cookbook medicine."
Comparative effectiveness (CE) enthusiasts, including Obama administration officials, countered that more and better information
about which medical products and procedures are most effective can improve care and cut unnecessary spending. But a White
Paper last year from the Congressional Budget Office looked to CE research to "provide a basis for applying costly new technologies
only when they are likely to confer added benefits."
The final ARRA legislation compromised 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." Observers noted, however, that once CE research results
are available to the public, health plans and payers can use the data as they see fit. Expanding studies
In any case, the government-funded CE research program opens the door for the federal government to play a larger role in
selecting and shaping comparative assessments. Instead of establishing a new, independent entity to carry out CE research,
as some advocated, the stimulus package divides $1.1 billion among three arms of the Department of Health and Human Services
(HHS).
The Agency for Healthcare Research and Quality (AHRQ) gains $300 million to bolster its relatively small outcomes and effectiveness
research program. The National Institutes of Health (NIH) gets $400 million to fund CE research conducted by its various Institutes.
The remaining $400 million goes to the HHS secretary to support standards development, establish 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. The importance of the committee's deliberations was seen in the dozens of interested parties that
presented their opinions at a March public meeting on how CE research should be conducted and which topics should be studied.
In recommending study areas, the IOM panel will consider the needs of populations served by federal programs, such as the
elderly, children, the disabled, and subpopulations, including women and minorities.
To ensure broad input, the IOM also established an online system for anyone to submit specific proposals for CE studies. The
questionnaire requests a brief summary of the research question, study design, study population, and likely comparators. The
panel is ranking these proposals according to disease burden, disease severity, variation in care, cost, public interest,
and gaps in current information.
A new Federal Coordinating Council for CE Research will monitor how well HHS meets the IOM priorities in awarding CE research
grants. The 15-member council includes top officials from HHS, NIH, AHRQ, OMB, the FDA, and others.
In the spirit of transparency, HHS will publish information on grants and contracts awarded under this program, will disseminate
the research findings that result, and will report annually to Congress on the program. Grants may go to government agencies
and to private organizations with demonstrated experience in this area.
One benefit of a broader CE research program would be to improve methods for conducting comparative studies. Research experts
see a need, and an opportunity, to use some of the federal funding to develop innovative trial designs for real-world assessment
of medical treatments.
At the IOM meeting, Bryan Luce, senior vice president of UBC, urged "true transformational thinking" in designing CE studies;
otherwise, he said, we will "waste vast amounts of money answering the wrong questions, or the right questions too late."
