Adaptive design methods in clinical trials - a review

In recent years, the use of adaptive design methods in clinical research and development based on accrued data has become very popular due to its flexibility and efficiency. Based on adaptations applied, adaptive designs can be classified into three categories: prospective, concurrent (ad hoc), and...

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Bibliographic Details
Published in:Orphanet journal of rare diseases 2008-05, Vol.3 (1), p.11-11, Article 11
Main Authors: Chow, Shein-Chung, Chang, Mark
Format: Article
Language:English
Subjects:
Adaptive control
Analysis
Clinical trials
Clinical Trials as Topic - methods
Clinical Trials, Phase II as Topic
Clinical Trials, Phase III as Topic
Evaluation
Humans
Methods
Random Allocation
Rare diseases
Rare Diseases - drug therapy
Research Design
Review
Statistics as Topic
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Summary:In recent years, the use of adaptive design methods in clinical research and development based on accrued data has become very popular due to its flexibility and efficiency. Based on adaptations applied, adaptive designs can be classified into three categories: prospective, concurrent (ad hoc), and retrospective adaptive designs. An adaptive design allows modifications made to trial and/or statistical procedures of ongoing clinical trials. However, it is a concern that the actual patient population after the adaptations could deviate from the originally target patient population and consequently the overall type I error (to erroneously claim efficacy for an infective drug) rate may not be controlled. In addition, major adaptations of trial and/or statistical procedures of on-going trials may result in a totally different trial that is unable to address the scientific/medical questions the trial intends to answer. In this article, several commonly considered adaptive designs in clinical trials are reviewed. Impacts of ad hoc adaptations (protocol amendments), challenges in by design (prospective) adaptations, and obstacles of retrospective adaptations are described. Strategies for the use of adaptive design in clinical development of rare diseases are discussed. Some examples concerning the development of Velcade intended for multiple myeloma and non-Hodgkin's lymphoma are given. Practical issues that are commonly encountered when implementing adaptive design methods in clinical trials are also discussed.
ISSN:1750-1172
1750-1172
DOI:10.1186/1750-1172-3-11