Avoiding the Draft (Guidance)

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  • 01/07/2013

There was no telling what people might find out once they felt free to ask whatever questions they wanted to. -- Joseph Heller, Catch 22

Does no good deed go unpunished?

For years the beef was that the FDA was unyielding when it came to anything that even remotely undermined the large-scale random-controlled clinical trial. “The gold standard” was firmly enshrined in FDA dogma, not to be abased, and any challenge to its sacrosanct nature was regulatory heresy.

But times change and even the most ardent supporters of traditional RCT design (such as the FDA’s Bob Temple), now realize we can recruit on a more genetically molecular level. The path to personalized medicine makes every design (to a certain extent) an orphan disease.

According to Temple, smaller, "enrichment strategies" or "enriched studies" can assist drug developers to "exclude poor potential candidates and select those more likely to show a clinical benefit."  Such study designs are "potentially powerful strategies for the pharmaceutical industry because appropriate use of enrichment could result in smaller studies, shortened drug development times, and lower development costs." Temple said conducting clinical trials within a "patient population that has a larger than average response to treatment can greatly reduce the number of patients needed in the study."

Less, it seems, can indeed be more.

For example, the FDA "approved the cystic fibrosis drug ivacaftor (Kalydeco) which works in just 4% of CF patients with a specific genetic abnormality." But if "all CF patients were included in a trial," Dr. Temple said an "effect would have been impossible to detect."

Good news? Well, where you stand depends on where you sit. Some are worried that narrowing the scope of a given patient population involved in a clinical trial has the potential impact of becoming a disincentive for industry continuing to develop treatments for more heterogeneous diseases. 

A valid concern? Perhaps. Unintended consequences certainly aren’t unknown in regulatory environs. Might the FDA ask sponsors for both traditional clinical studies and smaller population trials? It’s possible. And it certainly calls into question the canard of “non-binding advice.” Might a boon for orphan disease trials be yet another costly encumbrance for innovators?

In other words, is it a legitimate concern that less could lead to more?

Yes – and one worth addressing in thoughtful comments to the draft guidance.

Speak out now – or forever hold your Citizen's Petitions.

Sometimes our actions are questions, not answers. -- John Le Carre


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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