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FDA and NIH: Natural Allies

Tension between CMS and FDA is a fact of life at HHS. This is not surprising because they have fundamentally different missions and world views. An analysis of the FDA-CMS relationship leads to an interesting conclusion: FDA should be doing a lot more with NIH because they have complementary missions and similar world views. They are natural allies.

FDA’s mission is the approval of safe and effective therapeutic agents. It believes that actionable knowledge comes from prospective, randomized, double-blind controlled clinical trials. Its view of individualized patient treatment: it should be based on the results of rigorous clinical trial information. Yet, they do not impose this on physicians, who may practice medicine as they see fit. FDA will consider information from observational trials, natural history controls and medical literature, but these generally supplement information derived from clinical trials.

In contrast, CMS’ mission is to pay for the health care of individuals eligible for coverage through statute. They try to assure health care access that maximizes quality and minimizes public cost. To them, actionable knowledge is retrospectively derived: therapies approved by FDA and treatments reviewed favorably in compendia and having support in a broad cross-section of medical literature. Increasingly, CMS is interested in supplementing these sources with more retrospective data: population studies and analysis of claims data and electronic medical records. Ultimately, CMS’ target population is beneficiaries, not patients. Knowledge about the needs of classes of beneficiaries is usually thought sufficient without reference to individualized needs.

While my FDA vs. CMS comparison involves some sweeping generalizations, it also explains a lot of behavior. For example, CMS kept offering its Medicare claims data to FDA and, for a long time, FDA wasn’t interested. FDA has now accepted the Medicare data from CMS. However, the dialogue will always be limited by CMS’ perception that they are giving FDA an extremely valuable tool and FDA’s perception that it is potentially useful, but of limited value. As noted in an earlier post: FDA believes that retrospective “real world” data sets = uncontrolled variables + inconsistent data collection + questionable data accuracy. In short, nothing that FDA would base a safety or efficacy decision on…unless it had no choice.

If you look at what type of knowledge counts to FDA, its natural ally is NIH. They both believe in the virtue of prospective clinical trials as the basis of actionable knowledge. NIH generates more clinical trials—directly or through grants—then any other entity in the world.

FDA and the National Eye Institute provide a model of cooperation that should be fostered FDA-wide and NIH-wide.  NEI had started to work with endpoints based on medical imaging and biological evidence of disease progression. FDA’s standard endpoint was still: how many lines on an eye chart had the patient improved or regressed. From working together, both NIH and FDA have advanced knowledge in the field and moved closer to standards appropriate for an increasingly sophisticated therapeutic area.

NIH-funded trials often have to be re-done because FDA won’t accept the endpoints or some other aspect of the clinical trial design. In many of those instances, NIH should be running trials with different endpoints to advance clinical knowledge or validate those endpoints. Many times though, early coordination would reduce the chances that expensive trials will need to be repeated….and patients might get beneficial therapies quicker.  A high level of coordination should be possible because NIH and FDA share similar values about the importance of knowledge derived prospectively. There are differences between them, but they are ones of degree, not kind.

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