A Review of Labeling Based on Patient-Reported Outcome Endpoints for New Oncology Drugs Approved by the European Medicines Agency (2017-2021).

A review of new oncology indications approved by the European Medicines Agency (EMA) for 2012-2016 showed that 33% of new drugs had labeling based on patient-reported outcomes (PROs). We reviewed labeling text based on PRO endpoints for new oncology indications approved during 2017-2021. New oncology drugs approved by EMA to treat indications of cancers during 2017-2021 were identified from the EMA website. PRO-related language reported in EMA summaries of product characteristics (SmPCs) were summarized and compared with similar findings reported for oncology indications approved during 2012-2016. Review documents by the EMA during 2017-2021 were available for 49 new oncology drugs for 70 cancer indications. Submissions for 52 (74.3%) of the 70 indications included PRO data for EMA review. Of all submissions, 14 (20.0%) approvals contained PRO-related language in the SmPC. Broad concepts such as health-related quality of life were most common and found in 8 of 14 (57.1%) PRO-related labels. PRO-related language appeared in SmPCs for 20% of all indications of new oncology drugs approved by EMA during 2017-2021 compared with approximately 33% of EMA approvals during 2012-2016. PRO-related labeling during the same periods showed a greater decline (from 47% to 27%) for indications of new oncology drugs that also included PRO data. One possible reason for this decline may be the increase in open-label studies from 62% between 2012 and 2016 to approximately 79% between 2017 and 2021. • A previous review of new oncology indications approved by the European Medicines Agency for 2012 to 2016 showed that 33% of indications had labeling based on patient-reported outcomes (PROs). Of these, the most commonly included concepts were related to symptoms. • The current review, undertaken for 2017 to 2021, shows that PRO-related labeling was included in only 20% of new oncology indications approved by the European Medicines Agency—a decline of approximately 13%. In this period, broad concepts such as health-related quality of life represented the concepts most commonly included in labeling with PRO language. • One possible reason for the discrepancy observed during these 2 periods may be related to the more frequent use of open-label studies seen in the current review. [ABSTRACT FROM AUTHOR]