Characteristics of pre-approval efficacy evidence for US accelerated approval and European conditional marketing authorization during 2015-2020
Objectives:To describe and compare the characteristics of the pre-approval efficacy evidence for the US accelerated approval(AA)and European conditional marketing authorization(CMA)from January 1,2015 to December 31,2020.Methods:All the drugs approved through AA and CMA pathway and their pre-approval studies were collected and analyzed based on the characteristics of their pre-approval efficacy evidence from public databases of US and Europe.Results:From 2015 to 2020,US FDA approved 122 and EMA approved 35 drugs and its indications(DAIs)through the AA/CMA pathway.The pre-approval efficacy evidence was mainly based on the surrogate endpoint(AA,93.3％;CMA,91.4％).The median of the sample size which provided the pre-approval efficacy evidence for AA and CMA were 88 and 124,with the size in AA was smaller than that in CMA(P =0.015).Most of the pre-approval evaluations were based on the response rate(AA,77.3％;CMA,60.0％)in single-arm study.There were 21.7％DAIs in AA and 4.7％in CMA had a response rate lower than 30.0％(P =0.005).Most of the pre-approval pivotal efficacy studies were PhⅡ(AA,65.6％;CMA,60.0％)single-arm(AA,79.8％;CMA,65.7％)study.Conclusion:Most of the AA and CMA DAIs were granted based on the response rate of surrogate endpoint from phase Ⅱ single-arm studies.The AA's strength of the pre-approval efficacy evidence is lower than CMA's.The difference came from the different policies of AA and CMA.
accelerated approvalconditional marketing authorizationdrug and indicationefficacy evidence
NETL
NSTL
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