Considerations on use of real-world evidence in regulatory decision-making for rare diseases
ZHANG Xiao-min
LIU Xiao-xi
HUANG Zhe
Abstract：Real-world evidence(RWE)has been actively discussed in terms of utilization for regulatory decision-making on the benefit/risk assessment of a drug.Regulatory agencies from multiple countries around the world have released some specific policies and guidelines which are related to RWE,to actively evaluate and explore ways to optimize the utility of RWE to support regulatory decision-making.For rare diseases,it may not always be feasible to conduct traditional randomized control clinical trials because of the characteristics of rare diseases,such as low morbidity,small number of patients,and complex and serious life-threatening conditions.Thus,the evidence derived from actual clinical practices could provide a new approach to rare disease research.This paper reviewed the current situation on the use of RWE in regulatory decision-making for rare diseases,analyzed the issues of RWE in regulatory decision-making,and proposed some reasonable suggestions to provide references for the development of drugs for rare diseases.
Keywords：real world evidencerare diseaseorphan drugregulatory decision-making
Publication Date：2024-11-30
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：4( 2314-2317 )