The Comparative-Effectiveness Controversy

Government funding is slated to boost comparative studies of prescription drugs.
May 02, 2009
Volume 33, Issue 5

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

Comparative effectiveness (CE) research enthusiasts insist that more and better information about which medical products and procedures are most effective for treating certain conditions can improve care and cut unnecessary costs. A white paper last year from the Congressional Budget Office (CBO) looked to CE research to "provide a basis for applying costly new technologies only when they are likely to confer added benefits." And the Obama administration has stated frequently that reducing healthcare spending is critical to economic recovery and to maintaining a viable Medicare program.

However, the initial CE research legislation set off a firestorm by stating that by "knowing what works best and presenting this information more broadly to patients and health professionals," procedures and interventions "found to be less effective and, in some cases, more expensive, will no longer be prescribed." Conservative columnists raised the specter of government rationing and "cookbook medicine" that offers the same treatment for everyone. Pharmaceutical companies predicted that comparative cost assessments would lead to price controls if payers refused to reimburse companies for drugs that fall below a cost-effectiveness threshold.

The final ARRA legislative report defused the outcry 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, though, that once CE research results are available to the public, health-plan managers and payers will be free to use the data as they see fit, which could include imposing limits on coverage and reimbursement.

More new research

In any case, the new government-funded CE research program opens the door to a much broader role for the federal government in selecting treatments and topics to be analyzed and in shaping methods for conducting comparative studies. Even though ARRA does not establish a new, multibillion-dollar independent entity to carry out CE research, as envisioned by leading health authority Gail Wilensky, senior fellow at Project HOPE, in her seminal November 2006 article in Health Affairs, it promises to expand the scope of CE research considerably. Whether or not an independent CE agency is established is not the critical issue, Wilensky says. The important questions are whether CE data have credibility, whether research practices are open and transparent, and whether studies are objective and not politically motivated.

As an initial step, the legislation divides the $1.1 billion in funds among three arms of the US 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 support CE research conducted by its various Institutes, in addition to the $10 billion in ARRA to fund general research and medical-facility improvements. The remaining $400 million goes to the HHS secretary for activities such as standards development and support for registries and electronic data systems to generate outcomes data.

In Washington This Month
How these agencies dole out the money will be shaped by a report from the Institute of Medicine (IOM) recommending priorities for CE research. The legislation provides $1.5 million for an IOM panel chaired by Harold Cox, editor of the Annals of Internal Medicine, to issue a priorities assessment by June 30, 2009. The importance of the committee's deliberations was seen in the dozens of interested parties who presented their opinions at a March 2009 public meeting about 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, and the disabled, and will look for research that includes women and minorities.

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