A Review of Labeling Based on Patient-Reported Outcome Endpoints for New Oncology Drugs Approved by the European Medicines Agency (2017-2021) [Editor's Choice]

Jun 1, 2023, 00:00 AM
10.1016/j.jval.2023.01.017
https://www.valueinhealthjournal.com/article/S1098-3015(23)00042-6/fulltext
Section Title : PATIENT-REPORTED OUTCOMES
Section Order : 893
First Page : 893

Objective

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.

Methods

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.

Results

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.

Conclusion

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.

https://www.valueinhealthjournal.com/action/showCitFormats?pii=S1098-3015(23)00042-6&doi=10.1016/j.jval.2023.01.017
HEOR Topics :
  • Literature Review & Synthesis
  • Oncology
  • Patient-Centered Research
  • Patient-reported Outcomes & Quality of Life Outcomes
  • Specific Diseases & Conditions
  • Study Approaches
Tags :
  • labeling
  • patient-reported outcomes
  • regulatory review (European Medicines Agency)
  • review
Regions :
  • Global