Use of Real‐World Data and Evidence in Drug Development of Medicinal Products Centrally Authorized in Europe in 2018–2019

• DOI: 10.1002/cpt.2462
• Publication Date: 2021-10-24T13:43:50Z
• Authors: Sini Marika Eskola; Hubertus Gerardus...; Andrew Bate; Marie Louise De B...; Helga Gardarsdottir
• Keywords: Marketing authorization; Drug Development; Orphan drug

Real‐world data/real‐world evidence (RWD/RWE) are considered to have a great potential complement, in some cases, replace the generated through randomized controlled trials. By tradition, use of RWD/RWE postauthorization phase is well‐known, whereas published pre‐authorization medicines development lacking. The primary aim this study was identify and quantify role (RWE signatures) during phase, as presented initial marketing authorization applications new centrally evaluated with positive opinion 2018–2019 ( n = 111) by European Medicines Agency (EMA). Data for retrieved from evaluation overviews Public Assessment Reports (EPARs), which reflect scientific conclusions assessment process accessible EMA website. RWE signatures were extracted into an Matrix , including 11 categories divided over 5 stages drug lifecycle. Nearly all EPARs included discovery (98.2%) life‐cycle management (100.0%). Half them full (48.6%) supporting regulatory decisions at registration (46.8%), third (35.1%) early development. more often seen orphan conditionally approved medicines. Oncology, hematology, anti‐infectives stood out therapeutic areas most their phase. findings bring unprecedented insights about vast decision making.