This report is one of two linked papers that provide valuable accounts of how the FDA is using faster reviews for what it deems to be important new drugs and using supplemental approvals for existing drugs more widely. The other report highlighted that drugs approved for supplemental indications by the FDA were supported by low rates of clinical trials using active comparators or study endpoints directly related to patients’ function/mortality. This was especially true of supplemental indications that expanded the drugs’ approved patient populations.
An editorial expresses concerns about whether most new drugs are any more effective than existing products or whether their safety has been adequately assessed. The commentators note that although the US Congress and the FDA require “substantial evidence of effectiveness” to approve new drugs, they require no evidence of substantial effectiveness. They add that companies provide substantial evidence of effectiveness through trials that in most cases prove only that the product being tested has a non-zero level of effectiveness. The result is that independent reviews find that 85-90% of new drugs provide few or no advantages for patients. They conclude “the FDA’s flexible criteria and low threshold for approval do not reward more research for breakthroughs but instead reward more research for minor variations that can clear this low threshold.”