As FDA Commissioner Robert Califf has emphasized, the drug approval process is complex and risky, and the agency takes heat every time an approved medication leads to adverse side effects or even deaths. The Trump administration has signaled it may be open to reworking the system, but it is unclear what changes might look like.
Among the issues that have been raised are the use of surrogate endpoints to speed up approval of drugs, and the tendency for companies to delay post-marketing trials. The latter have been shown to improve the robustness of evidence supporting a drug’s benefits, but the fact that they are often conducted after a product has reached market means that many patients have been exposed to drugs that may not prove as effective as originally thought.
In a recent study, CMMI found that 104 out of 278 drugs that have been granted an Accelerated Approval have at least one confirmatory trial that is past its original scheduled completion date. This raises concerns about the quality of data supporting these drugs, and it suggests that there could be important differences between oncology and non-oncology products.
The accelerated approval pathway has brought a number of innovative therapies to the market, but it also exposes a weakness in the FDA’s system, and some companies have exploited this loophole by charging very high prices for drugs that may not provide clinical benefit. The FDA’s recent decision to withdraw approval of the duchenne muscular dystrophy drug Elevidys reflects this issue.
About the Author
