Abstract
Real-world data/real-world evidence (RWD/RWE) are considered to have a great potential to complement, in some cases, replace the evidence generated through randomized controlled trials. By tradition, use of RWD/RWE in the postauthorization phase is well-known, whereas published evidence of use in the pre-authorization phase of medicines development is lacking. The primary aim of this study was to identify and quantify the role of potential use of RWD/RWE (RWE signatures) during the pre-authorization phase, as presented in the initial marketing authorization applications of new medicines centrally evaluated with a positive opinion in 2018–2019 (n = 111) by the European Medicines Agency (EMA). Data for the study was retrieved from the evaluation overviews of the European Public Assessment Reports (EPARs), which reflect the scientific conclusions of the assessment process and are accessible through the EMA website. RWE signatures were extracted into an RWE Data Matrix, including 11 categories divided over 5 stages of the drug development lifecycle. Nearly all EPARs included RWE signatures for the discovery (98.2%) and life-cycle management (100.0%). Half of them included RWE signatures for the full development phase (48.6%) and for supporting regulatory decisions at the registration (46.8%), whereas over a third (35.1%) included RWE signatures for the early development. RWE signatures were more often seen for orphan and conditionally approved medicines. Oncology, hematology, and anti-infectives stood out as therapeutic areas with most RWE signatures in their full development phase. The findings bring unprecedented insights about the vast use of RWD/RWE in drug development supporting the regulatory decision making.
| Original language | English |
|---|---|
| Pages (from-to) | 310-320 |
| Number of pages | 11 |
| Journal | Clinical Pharmacology and Therapeutics |
| Volume | 111 |
| Issue number | 1 |
| Early online date | 24 Oct 2021 |
| DOIs | |
| Publication status | Published - Jan 2022 |
Bibliographical note
Funding Information:S.M.E. conducts a part‐time professional PhD at Utrecht Centre for Pharmaceutical Policy and Regulation. In her daily work, she holds a position as Director and Team Leader of Regulatory Affairs, Drug Development, and Manufacturing at European Federation of Pharmaceutical Industries and Associations (EFPIA). This research is independent and not funded by EFPIA. A.B. is a full‐time employee and stock‐holder in GlaxoSmithKline. The views expressed in this article represent the author’s own thoughts and are independent of their employers. This research is independent and not funded by GSK. H.G.M.L., M.L.D.B., and H.G. are employed by Utrecht University conducting research under the umbrella of the Centre for Pharmaceutical Policy and Regulation. This center receives no direct funding or donations from private parties, including those in the pharmaceutical industry. Research funding from public–private partnerships (e.g., IMI, The Escher Project ( http://escher.lygature.org/ ), is accepted under the condition that no company‐specific product or company‐related study is conducted). The center has received unrestricted research funding from public sources, for example, the World Health Organization (WHO), Netherlands Organization for Health Research and Development (ZonMW), the Dutch National Health Care Institute (ZIN), EC Horizon 2020, the Dutch Medicines Evaluation Board (MEB), and the Dutch Ministry of Health.
Publisher Copyright:
© 2021 The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.