Improving the Evidence

All good physicians want to do the right thing. They want to recommend effective therapies to their patients that will improve outcomes or alleviate symptoms. It is widely accepted that the best way to discover new effective therapies is through the use of clinical trials. Among clinical trials, the reference standard is the randomized, double-blinded, placebo-controlled trial, which is designed to minimize bias in the selection of therapies or the interpretation of results.

I have written before about the limitations of clinical research in advancing medical practice. As I have said, it is literally impossible to study every clinically relevant question, and it is also impossible even in theory to use randomized controlled trials as the methodology for many of the questions that can be studied.

A recent article in the New York Times highlighted another challenge to the paradigm of clinical trials as the engine for improving medical practice. The piece was about a change in policy at the National Institutes of Health, being implemented by Michael Lauer, the “newly appointed deputy director for extramural research.” In the interest of full disclosure, I have known Mike for many years (we were cardiology fellows in the same program at Boston’s Beth Israel Hospital in the late 1980’s) and you would be hard-pressed to find a nicer, smarter or more upstanding guy.

The new policy grew out of a disturbing observation that the results of many studies funded through NIH grants were never published. To paraphrase Lauer, it is as if those unpublished studies were never performed. As a result, the dollars spent to do those studies did not yield insights that could inform medical practice, and the implicit promise to study participants that they would be able to contribute to advancing knowledge was broken.

In response, the NIH is now shifting focus. The stated intent is to fund “fewer, but deeper, studies, to focus resources on efforts with real-world impact and life-or-death implications” and to require that all studies post their findings in a federal database, even if the results were not published elsewhere.

The idea behind these changes is to direct scarce resources to exploring issues that can have a profound effect on medical practice, and to make sure that the results are broadly available. Seems like progress to me.

What do you think?

2 thoughts on “Improving the Evidence

  1. Great article. Raises interesting issues about why results were not published…were the studies completed? Were they not publishable ? Perhaps we need a journal for well done studies that proved the thesis was incorrect? I hope though that this won’t steer money away from research on rare diseases. Can you add a share on Linked in button, if such a thing exists?

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