Should FDA Be More Flexible For Approval of Drugs For Severe Diseases?

FDA approval is primarily based on avoiding “false positive” or the Type I error, which means approval of a drug that is ineffective or harmful. FDA review is focused on making sure that all products it approves meet the minimum threshold of being safe and effective. According to a policy paper published by an MIT Professor this week, the FDA criteria for approval is unfair as it does not take into consideration the need for a drug intended to treat more severe diseases. The paper provides an interesting but flawed justification for its rationale. The acceptable level of “false positive” by FDA is 2.5%, basically FDA would approve a drug if there is a 2.5% chance of it being ineffective or harmful. This threshold for approval is constant across all drugs irrespective of the target indication. According to the authors of the policy paper, FDA should be “less conservative” when reviewing drugs which treat diseases with high likelihood of death compared to those for less severe diseases. In the authors’ opinion, FDA should approve drugs for diseases such an pancreatic cancer even if there is 1 in 4 chance of the drug being ineffective and harmful, while for diseases such as diabetes it can take a much tougher stance. The paper presents an elaborate statistical justification. There are many flaws with this analysis not to mention the obvious unfairness and unscientific clinical logic. First the paper totally ignores that FDA has already implemented multiple programs such as breakthrough therapy designation, orphan designation, and fast track process to aid faster development of much needed drugs. Also, the compassionate use provisions allow patients to access experimental drugs if the manufacturer is willing to share. More erroneously, the paper uses data from 2010 and before to makes its case. In regulatory jargon using 5 year-old trends are ancient data. There have been numerous developments in the last 5 years to encourage faster approval. Last two years have been years of record-breaking approval. With the current approval rate at above 96%, argument can be made that FDA is probably a lot less conservative, Any more and we could adding to risk to patients by approval of bad drugs. While we should certainly strive to create more opportunities for approval of much needed drugs, biased approval process is not one of them