실사용데이터/실사용근거를 활용한 FDA와 EMA의 규제적 의사결정 사례 분석

• Published in: Yaghag-hoi-ji Vol. 64; no. 2; pp. 136 - 155
• Main Author: 성희진(Hi Gin Sung), 박한희(Han-Heui Park), 정규원(Gyu-Won Jung), 신주영(Ju-Young Shin)
• Format: Journal Article
• Language: Korean
• Published: The Pharmaceutical Society Of Korea 30.04.2020; 대한약학회
• Subjects: 약학; effectiveness; Real world data; rare disease; real world evidence; regulatory decision making; safety
• ISSN: 0377-9556; 2383-9457
• DOI: 10.17480/psk.2020.64.2.136

Lately, the use of Real-World Data (RWD)/Real-World Evidence (RWE) have been actively expanded inregulatory decisions. We aimed to investigate how RWD/RWE were used in pursuit of supporting regulatory actions ofFDA and EMA. We selected 9 examples of regulatory decision making with the support of RWD/RWE. To identifyinformation included in RWE submission, we referred to the official website of US FDA or EMA. In addition, we usedClinicalTrials.gov and Medline database to collect information on study design using RWE and RWD source used togenerate RWE. Our study drugs were classified into 3 parts according to regulatory context; primary approval, labelexpansion, and regulatory response to safety signal after marketing. Among these examples, 5 biologic products including1 vaccine had gone through accelerated review supported by RWE. To summarize, we investigated several examples whereRWD/RWE has already been used in the process of making regulatory decisions for assessing the safety of drugs as wellas their effectiveness. We recommend further discussions on advancing the use of RWD/RWE in regulatory context toimprove patient access to novel rare disease treatments and provide more efficient safety assessment. KCI Citation Count: 0