Dear Editor,
In “Patients deserve better information on new drugs”, the authors make an extrodinary claim: “For example, over two thirds of new cancer medicines, which now comprise the largest category of new treatments, are approved based on single arm trials that have no control groups and only short follow-ups.” In the print edition, this sentence is repeated in bold near the title.
The citation for this claim is study by Salcher-Konrad, Naci, and Davis. However, the citation does not support the claim. The cited study is limited to a sample of 21 cancer drug-indication pairs that (a) received special approval from FDA and/or EMA, and -critically- (b) for “which data on efficacy and safety was less complete than that required for regular approval at time of market entry.” The study design of the citation cannot support the claim that over two thirds of new cancer medications are approves based on single arm trials without control groups. Moreover, in the cited study, the 21 cases are a small subset of the 447 approved by either EMA (between 2006-2016) or FDA (1992-2017), the vast majority of which received regular approval and which are not the subject of the cited study.
I appreciate that Davis and Naci are authors of both articles, and hope they can provide clarity.
Re: Patients deserve better information on new drugs