DIA's Adaptive Design Scientific Working Group (ADSWG): Best Practices Case Studies for "Less Well-understood" Adaptive Designs

Therapeutic Innovation & Regulatory Science
Eva MillerYannis Jemiai

Abstract

Adaptive design (AD) clinical trials use accumulating subject data to modify the parameters of the design of an ongoing study, without compromising the validity and integrity of the study. The 2010 US Food and Drug Administration (FDA) Draft Guidance on Adaptive Design Clinical Trials described a subset of 7 primary design types as "less well-understood." FDA defined these designs as those with limited regulatory experience. To better understand the properties of these less well-understood ADs and to promote their use when applicable, the Best Practices Subteam for DIA's Adaptive Design Scientific Working Group conducted an extensive nonsystematic search and reviewed trials from multiple sponsors who had employed these designs. Here, we review 10 specific case studies for which less well-understood ADs were employed and share feedback about their challenges and successes, as well as details about the regulatory interactions from these trials. We learned that these designs and associated statistical methodologies can make difficult research situations more amenable for study and, therefore, are needed in our toolbox. While they can be used to study many diseases, they are particularly valuable for rare diseases, small population...Continue Reading

References

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Citations

Jan 1, 2017·Therapeutic Innovation & Regulatory Science·Weili HeMin Lin
Jul 29, 2020·Journal of Clinical Medicine·Ferdinando D'AmicoLaurent Peyrin-Biroulet

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Clinical Trials Mentioned

NCT00547898
NCT00543543
NCT00268476
NCT01191801
NCT00047775
NCT01156571
NCT01010061
NCT01998880
NCT02053610
NCT01000961

Software Mentioned

PHOENIX
PLATFORM
RAPTOR
CHAMPION
MAMS
STAMPEDE
Procysbi

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