16-18 November 2021
The FDA published a new guideline on Adaptive Design Clinical Trials for Drugs and Biologics. The document provides guidance to sponsors on the appropriate use of adaptive designs for clinical trials (CTs) to provide evidence of the effectiveness and safety of a drug or biologic. The guidance describes important principles for designing, conducting, and reporting the results from an adaptive CT. In addition, it advises sponsors on the types of information to submit to facilitate FDA evaluation. According to the agency, the concepts provided are also useful for early-phase or exploratory CTs as well as trials conducted to satisfy post-marketing requirements.
The guidance defines an adaptive design as a CT design that allows for prospectively planned modifications to one or more aspects of the design based on accumulating data from subjects in the trial.
According to FDA, adaptive designs can provide a variety of advantages. These advantages arise from the fundamental property of CTs with an adaptive design: they allow to adjust the trial to information that was not available when the trial began. The specific nature of the advantages depends on the scientific context and type of adaptation considered, with the following potential advantages:
The FDA strongly recommends that access to comparative interim results be limited to individuals with relevant expertise who are independent of the personnel involved in conducting or managing the trial and have a need to know. It has to be ensured that patients, investigators and their staff as well as sponsors do not have access to comparative interim results. Knowledge of accumulating data by trial investigators can adversely affect patient accrual, adherence, retention, or endpoint assessment, compromising the ability of the trial to reliably achieve its objective in a timely manner. According to FDA, a clinical trial with an adaptive design should include the following:
More information can be found in FDA´s Guidance for Industry: Adaptive Designs for Clinical Trials of Drugs and Biologics.