Benefit-Risk Assessment, Communication, and Evaluation (BRACE)

4E-06 - Patient Experience Data in regulatory decision-making: An analysis of non-interventional studies supporting marketing authorization applications submitted to the European Medicines Agency in 2018-2019

Monday, August 25, 2025

5:30 PM - 5:45 PM ET

Presenting Author(s)

Diogo Almeida

Faculdade de Farmácia, Universidade de Lisboa, Lisbon, Portugal
Lisbon, Portugal

Co-Author(s)

DU

Denise Umuhire

European Medicines Agency, Amsterdam, The Netherlands, Netherlands
CJ

Carla Jonker

European Medicines Agency, Amsterdam, The Netherlands, Netherlands
KP

Kelly Plueschke

European Medicines Agency, Amsterdam, The Netherlands, Netherlands
RG

Rosa Gonzalez-Quevedo

European Medicines Agency, Amsterdam, The Netherlands, Netherlands
BS

Bruno Sepodes

Faculdade de Farmácia, Universidade de Lisboa, Lisbon, Portugal
Lisbon, Portugal
CT

Carla Torre

Faculdade de Farmácia, Universidade de Lisboa, Lisbon, Portugal, Portugal

Background: By capturing patients’ perspective and preferences, Patient Experience Data (PED) offer valuable insights for medicines’ development, evaluation and monitoring. Whilst the use of PED is well-characterized in interventional studies, their role in non-interventional studies remains underexplored.

Objectives: This study characterizes how PED are used in non-interventional studies submitted to the European Medicines Agency (EMA) as part of marketing authorization applications (MAAs).

Methods: Initial MAAs and Extensions Of Indication (EOIs) containing real-world data (RWD), submitted to the EMA between 2018 and 2019, were systematically screened for PED in non-interventional studies. European public assessment reports, risk management plans, and post-authorization study protocols were reviewed. Predefined variables were documented in Microsoft Excel, and a descriptive analysis was performed.

Results: Thirteen out of 63 MAAs (20.6%) and three out of 28 (10.7%) EOIs containing RWD identified in a prior EMA study, included PED. These 16 applications contained 26 studies featuring PED, including 23 (88.5%) post-authorization studies, all of which corresponded to additional pharmacovigilance activities. Twenty-four distinct PED instruments were identified. Quality of life was the most assessed domain among the identified studies (12, 46.2%), with EQ-5D being used in 8 different studies.

Conclusions: PED use in non-interventional studies for regulatory purposes remains limited, despite its potential. Periodic analysis of such use of PED can help EU regulators evaluate their impact on medicines evaluation and monitoring, to ensure safe and effective treatments for patients.