Real-world evidence: perspectives on challenges, value, and alignment of regulatory and national health technology assessment data collection requirements

H. Sievers*, A. Joos, M. Hiligsmann

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review


ObjectiveThis study aims to assess stakeholder perceptions on the challenges and value of real-world evidence (RWE) post approval, the differences in regulatory and health technology assessment (HTA) real-world data (RWD) collection requirements under the German regulation for more safety in drug supply (GSAV), and future alignment opportunities to create a complementary framework for postapproval RWE requirements.MethodsEleven semistructured interviews were conducted purposively with pharmaceutical industry experts, regulatory authorities, health technology assessment bodies (HTAbs), and academia. The interview questions focused on the role of RWE post approval, the added value and challenges of RWE, the most important requirements for RWD collection, experience with registries as a source of RWD, perceptions on the GSAV law, RWE requirements in other countries, and the differences between regulatory and HTA requirements and alignment opportunities. The interviews were recorded, transcribed, and translated for coding in Nvivo to summarize the findings.ResultsAll experts agree that RWE could close evidence gaps by showing the actual value of medicines in patients under real-world conditions. However, experts acknowledged certain challenges such as: (i) heterogeneous perspectives and differences in outcome measures for RWE generation and (ii) missing practical experience with RWD collected through mandatory registries within the German benefit assessment due to an unclear implementation of the GSAV.ConclusionsThis study revealed that all stakeholder groups recognize the added value of RWE but experience conflicting demands for RWD collection. Harmonizing requirements can be achieved through common postlicensing evidence generation (PLEG) plans and joint scientific advice to address uncertainties regarding evidence needs and to optimize drug development.
Original languageEnglish
Article number40
Number of pages8
JournalInternational Journal of Technology Assessment in Health Care
Issue number1
Publication statusPublished - 24 Feb 2021


  • data collection
  • drug industry
  • health technology assessment
  • qualitative research
  • real-world evidence
  • Qualitative research
  • Drug industry
  • Health technology assessment
  • Data collection
  • Real-world evidence


Dive into the research topics of 'Real-world evidence: perspectives on challenges, value, and alignment of regulatory and national health technology assessment data collection requirements'. Together they form a unique fingerprint.

Cite this