Practices and considerations for feasibility evaluations of real-world external control arm for antineoplas-tic drugs based upon electronic medical records
OBJECTIVE To summarize the practical experiences and considerations of evaluating the feasibility of external control arm of real-world studies of antineoplastic drugs based upon medical electronic medical records in real medical environ-ment.METHODS This study focused upon analyzing domestic and foreign regulatory policies and new drug or biologic licensing applications of patient-level real-world data(RWD)or other external data for contextualization of trial results.Publicly available regulatory documents of clinical and statistical reviews,advisory committee briefing materials were retrieved for each application to summarize the policy requirements and regulatory decision-making considerations.RESULTS Though accepting the evidence of real-world external control arm in the approval of new drugs,FDA evaluated the real-world evidence with caution,mainly consid-ering the comparability between external control arm and experimental arm.This study of medical electronic records emphasized the necessity of feasibility evaluation of external control arms in real-world study and proposed an analytical framework for feasibil-ity evaluations.CONCLUSION Contextualizing single-arm trials with patient-level RWD is an embodiment of regulatory sci-ence.Currently domestic policy supports the use of real-world evidence for regulatory decision-making and FDA accepts it for approval submissions.However,there are still challenges of RWD quality and relevance.Through feasibility evaluations,we can assess data quality,demonstrate the comparability of populations and endpoints,plan in advance to help reduce bias and finally boost the strength of real-world evidence.
external control armreal-world datafeasibility evaluationreal-world evidence
NETL
NSTL
万方数据
