실사용데이터/실사용근거를 활용한 FDA와 EMA의 규제적 의사결정 사례 분석

• 영문명: Current Status and Examples of US FDA or EMA’s Regulatory Decision using Real World Data/Real World Evidence
• 발행기관: 대한약학회
• 저자명: 성희진(Hi Gin Sung) 박한희(Han-Heui Park) 정규원(Gyu-Won Jung) 신주영(Ju-Young Shin)
• 간행물 정보: 『약학회지』제64권 제2호(2020년), 136~155쪽, 전체 20쪽
• 주제분류: 의약학 > 기타의약학
• 파일형태: PDF
• 발행일자: 2020.04.30

영문 초록

Lately, the use of Real-World Data (RWD)/Real-World Evidence (RWE) have been actively expanded in regulatory decisions. We aimed to investigate how RWD/RWE were used in pursuit of supporting regulatory actions of FDA and EMA. We selected 9 examples of regulatory decision making with the support of RWD/RWE. To identify information included in RWE submission, we referred to the official website of US FDA or EMA. In addition, we used ClinicalTrials.gov and Medline database to collect information on study design using RWE and RWD source used to generate RWE. Our study drugs were classified into 3 parts according to regulatory context; primary approval, label expansion, and regulatory response to safety signal after marketing. Among these examples, 5 biologic products including 1 vaccine had gone through accelerated review supported by RWE. To summarize, we investigated several examples where RWD/RWE has already been used in the process of making regulatory decisions for assessing the safety of drugs as well as their effectiveness. We recommend further discussions on advancing the use of RWD/RWE in regulatory context to improve patient access to novel rare disease treatments and provide more efficient safety assessment.

목차

서 론(Introduction)

연구 방법(Research Methods)

결 과(Results)

고 찰(Discussion)

결 론(Conclusion)

감사의 말씀(Acknowledgment)

Conflict of Interest

References

키워드

Real world data real world evidence effectiveness safety rare disease regulatory decision making