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

Hannah Sievers,Mickaël Hiligsmann,Angelika Joos

doi:10.1017/s0266462321000131

Hannah Sievers, Mickaël Hiligsmann + Show 1 more

Open Access

https://doi.org/10.1017/s0266462321000131

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Abstract

This 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. Eleven 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. All 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. This 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.

Highlights

The pharmaceutical industry, regulators, and health technology assessment bodies (HTAbs) are increasingly exploring the potential of real-world data (RWD) as a complementary source to generate evidence regarding the effectiveness and safety of medicines in routine clinical practice (1;2)
RWD collection is often linked to managed entry agreements and conditional marketing approvals
real-world evidence (RWE) is rarely submitted by pharmaceutical companies as RWD does not exist at the time of benefit assessment after marketing approval

Summary

Introduction

The pharmaceutical industry, regulators, and health technology assessment bodies (HTAbs) are increasingly exploring the potential of real-world data (RWD) as a complementary source to generate evidence regarding the effectiveness and safety of medicines in routine clinical practice (1;2). The European Medicines Agency (EMA) aims to increase the use of RWE in regulatory decisions and has been relying on RWD collected on an “ad-hoc” basis for postmarket safety surveillance and noninterventional postapproval safety and efficacy studies (PASS, PAES) to generate additional evidence on drug utilization and adverse outcomes in practice (4–7). RWD collection requirements can become mandatory in Germany following the new regulation for more safety in drug supply (GSAV) (8) Under this regulation, the Federal Joint Committee (G-BA) may request RWD collection from pharmaceutical companies to be submitted to compulsory indication-based registries for the national benefit assessment of new medicines that can only show “limited scientific evidence” such as orphan drugs, drugs conditionally approved, or those approved under exceptional circumstances (9– 11).

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