Food and Drug Administration novel drug decisions in 2017: transparency and disclosure prior to and 5 years following approval

Abstract

Abstract The Food and Drug Administration (FDA) approved 46 novel drugs in 2017. We reviewed availability of results prior to and during the 5 years following each approval. Using the FDA website and ClinicalTrials.gov, we recorded trials cited as evidence for the approval, total number of studies registered in ClinicalTrials.gov, number started and completed before approval, and the frequency and timing of reporting results. The 46 drugs approved in 2017 were evaluated in 1149 studies. The number of studies used to evaluate the 46 drugs ranged from 2 to 165 (mean: 24.98; SD = 28.95). Among these, an average of 9.22 studies (SD = 9.21) were started and 5.82 studies (SD = 6.89) were completed before the approval. A single trial justified approval for 19 of 46 (41%) of the approved products. Public posting of results prior to the FDA approval was available for an average of only 1.42 studies (SD = 3.12). No results were publicly reported before approval for 9 of the 46 drugs (20%). Health care providers and consumers depend on complete and transparent reporting of information about FDA-approved medications. Only a fraction of evidence from completed studies was available before approval and a substantial portion of research evidence remained undisclosed after 5 years.