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News on Comparative Effectiveness Research

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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