Adaptive Trial Design in Clinical Research

Abstract

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, and retrospective adaptive designs. An adaptive design is flexible in modifying trial and/or statistical procedures of on-going clinical trials. However, it is a concern that the actual patient population after the adaptations could deviate from the originally target patient population. 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 entry, a formal definition of an adaptive design by PhRMA is given. Depending upon the adaptations applied, several commonly considered adaptive designs in clinical development are introduced. Regulatory and statistical perspectives are provided. Basic considerations and major obstacles and challenges when implementing an adaptive design in clinical trials are also discussed.