Representation of core outcomes in regulatory guidance from the FDA and EMA

Ian Saldanha, Susanna Dodd, Rebecca Fish, Sarah Gorst, Deborah Hall, Pamela Jacobsen, Jamie Kirkham, Dominic Trepel, Paula Williamson

Research output: Contribution to journalArticlepeer-review


We conducted an analysis to compare outcomes included within published core outcome sets (COS) against the outcomes recommended in corresponding European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) guidance documents, matched by health condition.

Study Design
We included a sample of COS related to drugs, devices, and gene therapy that involved patients in the consensus process and were published between January 1, 2015, and December 31, 2019.

We assessed the extent of matches between outcomes included within COS and those recommended in corresponding EMA and FDA guidance documents. When outcomes were matched, we considered matches to be general (i.e., non-specific) or specific (i.e., exact). For general matches, we assessed whether the COS or guidance document outcome was narrower.

We found relevant guidance documents for 38/98 eligible COS (39%). Among outcomes in COS, medians of 70% [interquartile range (IQR) 48% to 86%] and 52% [IQR 33% to 76%] were matches with outcomes in EMA and FDA documents, respectively. Medians of 46% [IQR 27% to 68%] and 26% [IQR 18% to 46%] were specific matches with outcomes in EMA and FDA documents, respectively. When outcomes were generally matched, the COS outcome was more frequently narrower than the regulatory outcome (83% and 75% for EMA and FDA, respectively).

Greater adoption of, and reference to, COS in regulatory guidance documents can encourage clinical trialists, especially those in industry, to measure and report consistent and agreed outcomes and improve the quality of guidance. Given the overlap between outcomes in COS and regulatory guidance, and given that most COS now involve patients in the consensus process, COS could serve as a useful resource for regulators when recommending outcomes for studies evaluating regulated products. We encourage COS developers to appraise recommended outcomes in salient regulatory documents when planning a COS.
Original languageEnglish
JournalBMJ Medicine
Publication statusAcceptance date - 22 Sep 2022


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