对美国FDA适应性设计指导原则的介绍
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摘要
美国食品药品监督管理局(FDA)于2018年9月发布了《药物和生物制品临床试验适应性设计》的指导原则草案,该指导原则讨论了适应型设计的范围、动机、原则、类型、注意事项等几个方面。我国目前尚未专门制订适应性临床试验的指导原则,通过本文介绍,希望对我国使用适应性设计药物的研发和审评工作提供参考。
The US Food and Drug Administration(FDA) issued the guidance for industry on adaptive designs for clinical trials of drugs and biologics in Sep 2018.This guidance discusses the scope,motivation,principles,types and special considerations of adaptive designs and so on.As there is no specific guidance on this in China currently,this article introduces the US guidance to provide some reference for researchers and reviewers on adaptive designs in China.
The US Food and Drug Administration(FDA) issued the guidance for industry on adaptive designs for clinical trials of drugs and biologics in Sep 2018.This guidance discusses the scope,motivation,principles,types and special considerations of adaptive designs and so on.As there is no specific guidance on this in China currently,this article introduces the US guidance to provide some reference for researchers and reviewers on adaptive designs in China.
引文
[1]FOOD AND DRUG ADMINISTRATION(FDA).Adaptive designs for clinical trials of drugs and biologics guidance for industry[EB/OL].Washington(America):FDA,2018-09[2019-03-26].https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm201790.pdf.
[2]FDA.Adaptive designs for medical device clinical studies guidance for industry and food and drug administration staff[EB/OL].Washington(America):FDA,2016-07-27[2019-03-26].https://www.fda.gov/ucm/groups/fdagov-public/@fdagovmeddev-gen/documents/document/ucm446729.pdf.
[3]O’BRIEN P.A multiple testing procedure for clinical trials[J].Biometrics,1979,35(3):549-556.
[4]POCOCK S J.Group sequential methods in the design and analysis of clinical trials[J].Biometrika,1977,64(2):191-199.
[5]DEMETS L D L.Discrete sequential boundaries for clinical trials[J].Biometrika,1983,70(3):659-663.
[6]PROSCHAN M A,HUNSBERGER S A.Designed extension of studies based on conditional power[J].Biometrics,1996,51(4):1315-1324.
[7]DENNE J S.Sample size recalculation using conditional power[J].Stat Med,2001,20(17-18):2645-2660.
[8]CUI L,WANG H S J.Modification of sample size in group sequential clinical Trials[J].Biometrics,1999,55(3):853-857.
[9]CHOW S C,CHANG M.Adaptive design methods in clinical trials[J].Orphanet J Rare Dis,2011,3(1):1-13.
[10]TANNER M A.Tools for statistical inference:methods for the exploration of posterior distributions and likelihood functions[M].3rd edition.New York:Springer,1999:89-93.
[2]FDA.Adaptive designs for medical device clinical studies guidance for industry and food and drug administration staff[EB/OL].Washington(America):FDA,2016-07-27[2019-03-26].https://www.fda.gov/ucm/groups/fdagov-public/@fdagovmeddev-gen/documents/document/ucm446729.pdf.
[3]O’BRIEN P.A multiple testing procedure for clinical trials[J].Biometrics,1979,35(3):549-556.
[4]POCOCK S J.Group sequential methods in the design and analysis of clinical trials[J].Biometrika,1977,64(2):191-199.
[5]DEMETS L D L.Discrete sequential boundaries for clinical trials[J].Biometrika,1983,70(3):659-663.
[6]PROSCHAN M A,HUNSBERGER S A.Designed extension of studies based on conditional power[J].Biometrics,1996,51(4):1315-1324.
[7]DENNE J S.Sample size recalculation using conditional power[J].Stat Med,2001,20(17-18):2645-2660.
[8]CUI L,WANG H S J.Modification of sample size in group sequential clinical Trials[J].Biometrics,1999,55(3):853-857.
[9]CHOW S C,CHANG M.Adaptive design methods in clinical trials[J].Orphanet J Rare Dis,2011,3(1):1-13.
[10]TANNER M A.Tools for statistical inference:methods for the exploration of posterior distributions and likelihood functions[M].3rd edition.New York:Springer,1999:89-93.