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Last week the U.S. Government Accountability Office (GAO) appointed several new members to the board of the Patient-Centered Outcomes Research Institute (PCORI). This group will oversee the application of comparative effectiveness research in health care reform. The panel’s composition was the subject of two posts today by Merrill Goozner, a long-standing observer of health care economics and journalist.

The group may play a key role in forming health care policy in the years ahead. According to the Patient Protection and Affordable Care Act, signed into law in March of this year, the PCORI is slated as a private, non-profit agency. The Institute’s purpose is:

to assist patients, clinicians, purchasers, and policy-makers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis that considers variations in patient subpopulations, and the dissemination of research findings with respect to the relative health outcomes, clinical effectiveness, and appropriateness of the medical treatments, services, …”

This is no small task, to say the least.

What is comparative effectiveness research and why does it matter? The idea, basically, is to inform medical decisions with relevant data derived from well-designed clinical trials. This sort of research will provide the foundation for evidence-based medicine (EBM).

Some casual readers of this blog may not appreciate the extent to which I support these endeavors. I do, 100 percent. I say this for several reasons:

First, as a patient: We need to obtain as much information as possible on the best way to treat common and rare medical conditions that arise in humans. Knowing which treatments work, and don’t, ultimately will promote care that’s effective and diminish use of procedures, devices and medications that are ineffective and/or harmful.

Second, as a citizen: We don’t have a surplus of health-care resources to throw around, so to speak, in vain efforts to treat things that can’t be fixed.

As an oncologist, I was not one to give treatments that I didn’t believe would help a patient to feel better and, ideally, get well. Rather, I think the value of chemotherapy and other treatments needs be demonstrated in clinical trials prior to their routine administration, or that their worth be measured otherwise – such as by careful testing of how a drug might work in a subset of patients or even in a particular patient who has a condition like breast cancer and, upon careful monitoring, it is apparent that nothing helps except Avastin in her case.

Third, as a doctor: I would never want to cause avoidable harm to a patient. If a drug, device or procedure is ineffective or damaging, we shouldn’t often prescribe that, if ever. The best way to help patients get well, and harm them less, is by knowing what works and what doesn’t in most cases and by knowing the limitations of aggregated, statistically-valid data applied to individual patients.

Cost analyses are needed, too, but that’s a somewhat separate issue.

I wish the panel good luck in their important work ahead.

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