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Title: Benefits, challenges and obstacles of adaptive clinical trial designs
Authors: Chow, S.-C 
Corey, R
Keywords: clinical trial (topic)
computer program
computer simulation
dose calculation
drug research
food and drug administration
phase 1 clinical trial (topic)
phase 2 clinical trial (topic)
phase 3 clinical trial (topic)
statistical analysis
United States
dose response
drug industry
sample size
treatment outcome
Clinical Trials as Topic
Data Interpretation, Statistical
Dose-Response Relationship, Drug
Drug Industry
Endpoint Determination
Research Design
Sample Size
Treatment Outcome
United States
United States Food and Drug Administration
Issue Date: 2011
Publisher: BMC
Citation: Chow, S.-C, Corey, R (2011). Benefits, challenges and obstacles of adaptive clinical trial designs. Orphanet Journal of Rare Diseases 6 (1) : 79. ScholarBank@NUS Repository.
Rights: Attribution 4.0 International
Abstract: In recent years, the use of adaptive design methods in pharmaceutical/ clinical research and development has become popular due to its flexibility and efficiency for identifying potential signals of clinical benefit of the test treatment under investigation. The flexibility and efficiency, however, increase the risk of operational biases with resulting decrease in the accuracy and reliability for assessing the treatment effect of the test treatment under investigation. In its recent draft guidance, the United States Food and Drug Administration (FDA) expresses regulatory concern of controlling the overall type I error rate at a pre-specified level of significance for a clinical trial utilizing adaptive design. The FDA classifies adaptive designs into categories of well-understood and less well-understood designs. For those less well-understood adaptive designs such as adaptive dose finding designs and two-stage phase I/II (or phase II/III) seamless adaptive designs, statistical methods are not well established and hence should be used with caution. In practice, misuse of adaptive design methods in clinical trials is a concern to both clinical scientists and regulatory agencies. It is suggested that the escalating momentum for the use of adaptive design methods in clinical trials be slowed in order to allow time for development of appropriate statistical methodologies. © 2011 Chow and Corey; licensee BioMed Central Ltd.
Source Title: Orphanet Journal of Rare Diseases
ISSN: 1750-1172
DOI: 10.1186/1750-1172-6-79
Rights: Attribution 4.0 International
Appears in Collections:Staff Publications

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