J Pharmacoepidemiol Risk Manage.  2022 Sep;14(2):133-143. 10.56142/jperm.22.0009.

Case Analysis in Drug Approval by FDA/EMA Using Real-World Data/Real-World Evidence

• Song J
• Kim J
• Park S
• Kim JH
• Noh Y
• Sung HG
• Shin JY

• KMID: 2534684
• DOI: http://doi.org/10.56142/jperm.22.0009

Abstract

In recent years, regulators have been willing to use real-world data (RWD) and real-world evidence (RWE) to support their regulatory decisions in various areas, such as initial marketing authorizations, expanding indications or patient groups for approved medicines and post-market surveillance. The purpose of this paper is to investigate how RWD/RWE has been used in drug approval process and introduce examples of ‘Food and Drug Administration (FDA)’ and ‘European Medicines Agency (EMA)’ regulatory decision making based on RWD/RWE. We investigated cases from the FDA, EMA, and Aetion, Inc. and reviewed the study design including data source, inclusion/exclusion criteria, endpoint definition, statistical method, efficacy assessment and regulatory decision. We selected the 8 cases of RWD/RWE used for generating evidence and classified our cases into 3 parts according to regulatory context: 1) primary approval, 2) off-label use and 3) label expansion. FDA and EMA are having big interest in RWD/RWE and currently using RWD/RWE for drug approval process; however, in Korea, the use of RWD/RWE has been limited to post-marketing surveillance and the need for using RWD/RWE in drug approval process is growing. By referring to overseas cases, we propose to establish drug review and approval systems and to develop guidelines to suit our situation so that RWD/RWE can support regulatory decision-making and their use can expand throughout the life-cycle of drugs.

Keyword

Real-world data; Real-world evidence; Drug approval process; Regulatory decision making