(11) A Review of Patient-Reported Outcomes for European Medicines Agency Regulatory Approval of Oncologic Drugs Between 2017 and 2020

Friday, August 26, 2022

8:00 AM – 6:00 PM CEST

Location: Hall C

Publication Number: 342

Presenting Author(s)

Carla Torre
– Faculty of Pharmacy University of Lisbon, Lisboa, Lisboa, Portugal

Co-Author(s)

Maria Manuel Teixeira
– Faculdade de Farmácia da Universidade de Lisboa, Lisbon, Portugal., Portugal
Fabio Cardoso Borges
– European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium, Belgium
Paula Barão, PharmD MSc PhD
– Pharmacovigilance Technician, Faculdade de Farmácia da Universidade de Lisboa, Lisbon, Portugal., Portugal
João Rocha
– Faculdade de Farmácia da Universidade de Lisboa, Departamento de Farmácia, Farmacologia e Tecnologias em Saúde, Lisboa, Portugal., Portugal
Bruno Sepodes
– Faculdade de Farmácia da Universidade de Lisboa, Departamento de Farmácia, Farmacologia e Tecnologias em Saúde, Lisboa, Portugal, Portugal

Background: Cancer diseases, and their treatment regimens, are associated with significant negative symptoms, side effects and functional limitations. Within this context, where quality of life may be more relevant than longevity, collection of patients reported outcomes (PRO) in clinical trials is of special interest and recommended by regulatory authorities. Within clinical trials framework, PRO may provide evidence to support drug approval, labelling and marketing claims.

Objectives: This study aims to analyse the evidence based on PRO of new oncology indications approvals by European Medicines Agency (EMA) between 2017 and 2020 and to identify PRO label claims granted.

Methods: Oncology drugs and indications approved by EMA between 2017 and 2020 were identified. European Public Assessment Report (EPAR) and Summary of Product Characteristics (SmPC) were reviewed for each drug to identify PRO use and PRO label claims.

Results: A total of 128 oncology indications were approved by EMA, and of these 100 (78.1%) included PRO in the confirmatory clinical trials. Thirty-seven indications (37.0%) were supported by randomized trials and the remainder 63 by open-label trials. PRO were defined as a secondary endpoint in 60 studies (57.7%), exploratory in 31 (29.8%) and as both in 13 (12.5%). In total, 56 different measures were used, of those 41 (73.2%) were disease-specific measures. Nevertheless, PROM selected relied on the EORTC (41.3%), FACIT (17.1%) and EQ-5D (29.2%) measures. A total of 76 indications (59.4%) had PRO reviewer comments included in the EPAR, however only 22 indications (17.8%) included label claims in the SmPC, being the majority related to solid tumours. The reasons identified in the EMA reviewers’ commentaries to exclude PRO claims were described for 34 indications (44.7%).

Conclusions: Despite growing recognition of the value of PRO data for the development of improved cancer therapies, PRO implementation remains challenging. The main reasons identified in our study are related with study design, missing data, study conduct and PROM selection