Do Physicians Lack Understanding of FDA Approval Processes? 
[Posted on: Thursday, 14 April , 2016]

A recent report based on a survey of about 1200 physicians provides a critique on how little prescribers know about the process of drug approval by the FDA. According to the report about 90% of the physicians surveyed could not correctly answer all three questions asked about FDA approval process. The survey asked two sets of questions; one set of 3 questions about general FDA review and approval process, and a second set of 5 questions about the breakthrough therapy designation. Based on the answers provided for each category the surveyors paint a bleak picture of physicians over-prescribing new drugs that may have been approved under a lower threshold by the FDA and hence may not be much better than the older drugs. However, a review of the questions posed to the physicians shows that the truth may be muddied by the surveyors’ own lack of understanding of the FDA review and approval process. For example, it is true that approval of a given drug is primarily based on comparing active to placebo, and may not require comparison of the new drug to standard of care or other drugs available for the same indication, but it does not mean that the new drug is “not” as effective as other drugs approved for the same indication. The same FDA review division is involved in all drugs approved for a given indication and during the review process it does compare if the new treatment offers an advantage of available drugs either based on historical experience or scientific prediction. However, the survey considered only the argument of placebo controlled trials. FDA approval does mean that benefits outweigh the risks, and it is based on statistically significant results for clinically relevant endpoints. In all these cases, the physicians correctly answered 70-85% of the times but the survey considered these as incorrect answers. Similarly, regarding the issue of breakthrough therapies, the survey incorrectly emphasized that breakthrough approvals are based on preliminary clinical evidence. On the contrary the breakthrough “designation” is based on preliminary clinical evidence not its approval. It is true that for the approval of a drug designated a breakthrough, FDA provides a lower threshold for approval but there a couple of points the surveyors did not appreciate. First, breakthrough designation is reserved for diseases where there are few treatment options, if any, leading to an unmet medical need. Second, the breakthrough designation is based on the drug showing a significant clinical superiority over the available options. Third, FDA designation as a breakthrough does mean that in the eyes of the FDA, the drug has high-quality evidence of being better both in safety and effectiveness to other available therapies. All these questions were correctly answered by the physicians but deemed incorrect by the surveyors. The answers were indeed based on perceptions as the physicians agreed that they were not very familiar with the breakthrough therapy designation and they put great weight of FDA’s use of the word “Breakthrough” for a drug but that does not make all their answers incorrect. The devil is in the detail. Surveys could be highly influenced by the biases of the surveyors. More importantly, when FDA approves more drugs, it provides patients with more options. FDA’s incentives are based on this logic. It does seem it is having intended effect on physicians perceptions.