Impact of Real-World Data on Market Authorization, Reimbursement Decision & Price Negotiation

• Published in: Therapeutic innovation & regulatory science 2021, Vol.55 (1), p.228-238
• Main Authors: Pulini, Alfredo Aram, Caetano, Gabriela Martins, Clautiaux, Henri, Vergeron, Laure, Pitts, Peter J., Katz, Gregory
• Format: Article
• Language: English
• Subjects: Clinical trials; Drug development; Drug Safety and Pharmacovigilance; Literature reviews; Medicine; Negotiations; Pharmacology; Pharmacotherapy; Pharmacovigilance; Pharmacy; Review
• ISSN: 2168-4790; 2168-4804
• DOI: 10.1007/s43441-020-00208-1

Real-world data (RWD) and real-world evidence (RWE) appear now as complementary to traditional randomized controlled trials (RCT), that remain the gold standard of the evidence. This review aims to illustrate how health authorities in France, United States (USA) and United Kingdom (UK) can integrate RWD and RWE in market authorization discussions and in new pathways of price and reimbursement negotiations. We conducted a review from the literature, online investigations and interviews. RWD and RWE can be valuable in the context of market access, reimbursement decisions, price negotiation, pharmacovigilance and positive patient outcomes. While RWD could open new areas of innovative approaches and improve the efficiency of health systems, they have methodological limitations requiring further analysis to reach a sufficient level of proof. Moreover, misleading use of “RWD” and “RWE” terms is very frequent and even the definitions used by stakeholders (when they have one) are heterogenous. Because of the intrinsic characteristics of each product, the value given to these RWD may differ a lot, making them a useful tool more than an indispensable one. In sum, RWD and, more precisely, RWE have the potential to bring value to the health system at every step of the drug development process, from the discovery to the pharmacovigilance phase.