OBJECTIVES: The use of patient experience data (PED) is increasingly considered important in healthcare decision-making, including regulatory evaluation. Despite clear interest, it is unclear if and how PED is considered by the European Medicines Agency (EMA) when evaluating marketing authorization applications. This study aimed to investigate if, how, and to what extent PED is currently reported in European public assessment reports (EPARs).

METHODS: A document analysis was performed to evaluate and compare the reporting of PED in EPARs in two disease areas; i) COVID-19 treatments and vaccines and ii) oncology including breast neoplasms, neuroendocrine tumours, neuroblastomas, ovarian neoplasms and endometrial neoplasms (published between 1/1/2015 – 30/9/2022). The EPARs of six COVID-19 vaccines, eight COVID-19 treatments and 47 cancer treatments were systematically screened. Data was analysed descriptively.

RESULTS: Patient input (PI) was observed to be reported in 84% of the analysed EPARs, mostly (74 %) reflected in the section for ‘user consultation for the package leaflet’. PI collection methods, such as the use of electronic diaries, was more reported in COVID-19 EPARs (57%) compared to cancer EPARs (4%). Patient reported outcome measures are reported in 50% of the cancer assessment documents but were almost completely absent (14%) for COVID-19. Patient reported experiences and patient preferences were both only reported in one EPAR. Whilst PED was always reported in the results section of the EPAR, it seldom appeared in the benefit-risk section. Only in a few EPARs in the field of oncology did the EMA ask for more PED.

CONCLUSIONS: Preliminary findings of this study suggest that PI is the most common type of PED captured in EMA regulatory evaluation dossiers of oncology and COVID-19 products. Results underscore a need for more transparency, and clear guidance from regulators on how and where to include PED in regulatory submissions and whether the EMA considers submitted PED.