Patient Experience Data in Non‑Interventional Studies Supporting EMA Marketing Authorisation Applications between 2018 and 2023

Study type

• Study topic:
  Other
• Study topic, other:
  Patient Experience Data
• Study type:
  Not applicable

Study design details

• Study design:
  Retrospective descriptive analysis
• Main study objective:
  The primary objective of this study is to characterise the use of PED in non‑interventional NIS submitted to support regulatory decision‑making within initial marketing authorisation applications (MAAs) and extensions of indication (EoIs) incorporating real‑world data (RWD) and assessed by the European Medicines Agency (EMA) between 2018 and 2023. This includes describing the regulatory lifecycle stage (pre‑ or post‑authorisation), study design, objectives, data sources, and the types of PED collected, as well as the instruments and methodologies used
• Setting:
  Not applicable
• Interventions:
  Not applicable
• Comparators:
  Not applicable
• Outcomes:
  Not applicable
• Data analysis plan:
  Regulatory documents of eligible submissions were screened to identify NIS including PED, as well as patient engagement (PE) activities. Data were extracted on study and PED characteristics. Descriptive statistics will be used to summarise the characteristics of PED use across the identified NIS. Quantitative variables will be analysed descriptively, including counts, percentages, means and medians. Statistical analyses will be performed using Excel
• Summary results:
  250 applications were screened. 42 (17%) submissions included PED in NIS or PE activities. Most of these submissions concerned antineoplastic and immunomodulating agents and frequently involved orphan medicinal products. Across all these submissions, a total of 47 NIS using PED and 6 PE activities were identified, with most NIS conducted in the post authorisation setting, often as additional pharmacovigilance activities. PROs were the most common form of PED, primarily assessing quality of life, while patient preference studies were less frequently used.