OBJECTIVES: FDA makes significant public information accessible through its website and databases. In particular, to learn about the role of clinical outcome assessments (COA) data in regulatory review and approval of marketing applications, Drugs@FDA is a comprehensive source. The limitation is that while all updates to the label are posted, typically only the medical reviews of the original marketing application are accessible. Reviews covering label expansion data from supplemental submissions are not readily available. The aim of this project is to review and compare multiple methods to better understand current FDA perspectives on patient experience data.

METHODS: Cemiplimab-rwlc was selected as a test case because two indications were added to the label in early 2021. Several methods for sourcing information about COAs in the associated label expansion trials and the evaluation of COA data by FDA were implemented. Any information available on the public FDA website was recorded. Additionally, a Freedom of Information Act (FOIA) request was generated for redacted FDA medical reviews. Finally, searches of both peer-reviewed and “gray” literature such as medical congress abstracts were conducted.

RESULTS: An understanding of how the manufacturer prioritized and implemented COAs could be gleaned from public information on the internet, including meeting abstracts and corporate press releases. Insights into how FDA Reviewers appraised the COA instruments used in the trial, the endpoints selected, and the data submitted were not readily available. Lastly, use of the FOIA mechanism proved impractical for the present study due to time and costs.

CONCLUSIONS: FDA aims to be as transparent as possible in the context of the regulatory-commercial nature of their remit and generally makes information available to the public in a way that is straightforward and reasonable. In the case of supplemental indications, skilled researchers may pursue other avenues, but the information will invariably be incomplete.