Table 2.

General advantages and challenges of adaptive trials

Advantages

Challenges

Improved flexibility More efficient use or allocation of available resources (e.g., financial or administrative) Improved statistical efficiency that can provide greater statistical power to detect a true drug effect Ethical considerations may be more readily addressed Ability to answer broader questions, that may be refined as the trial progresses, relative to nonadaptive designs Stakeholders may be more willing to support studies with adaptive elements because of the added flexibility

Advanced and specific analytical methods need to be used to avoid type I error rate inflation (i.e., identifying an ineffective intervention as effective) and control bias in estimates Gains in efficiency generally represent a tradeoff with other trial components (e.g., interim analyses may decrease expected sample size at the expense of an increase to the maximum sample size) Logistics to ensure appropriate trial conduct and integrity Adaptation may be limited by scientific or clinical constraints or make interpretation more challenging