How Do You Make Informed Decisions about Implementing Adaptive Designs?

Read more about this and the discussions that you missed at the MassBio Forum on Adaptive Designs in Clinical Research that took place last month.

Veristat’s President and Chief Biostatistician, John Balser, PhDmoderated a panel of industry leading adaptive design experts to tackle the questions of how, when and why to consider implementing adaptive designs in clinical development. The panelists represented the CRO, Sponsor and Physician’s point of view.   Below is a summary of what was discussed.

• Stella Stergiopoulous from Tufts Center For The Study Of Drug Development kicked off the presentations with an introduction to adaptive trial design, including an assessment and summary of the FDA guidance on adaptive design clinical trials. She highlighted that the implementation of adaptive designs can reduce the development timeline and save costs by stopping trials early and reassessing sample size mid-study.

• Dr. Mark Chang from Veristat presented his advice on strategic mistakes to avoid and how to increase the overall probability of success of clinical trial programs by use of adaptive designs and integrating sequential decision processes in drug development, selecting appropriate adaptive strategies for differing scenarios, and of how one can design efficient early phase adaptive trials when faced with limited information, heterogeneity of populations, and constraints of time and money.


• Cyrus Mehta from Cytel presented a case study of a confirmatory adaptive trial for acute myeloid leukemia that included a sample size re-estimation at the interim analysis. During the discussion Dr. Mehta illustrated the impact of adaptive design on the operating characteristics of trials.

• Deepak Bhatt from Brigham and Women’s Hospital Heart and Vascular Center provided a physician’s perspective on how the biotechnology and pharmaceutical industry may improve implementation of clinical trial. Dr. Bhatt highlighted the implementation of randomized registry trials in other countries and brought forward some ethical concerns common among physicians whose patients may be participating in clinical studies.

• Yi Liu from Takeda presented her recent experience implementing adaptive clinical studies, including one studies with sample size re-estimation with multiple doses and multiple endpoints, a seamless Phase II/III study design, and an adaptation on a study endpoint. Dr. Liu highlighted key factors when considering a seamless study design and how it can be implemented efficiently in a clinical study program.

During the moderated discussion, the panelists spoke of recent successes and failures in the implementation of adaptive design.

“The key advice for the implementation of adaptive designs was to plan early and spend time to choose the right design for the development program, to interact with the FDA early and often when selecting an adaptive study design, and to consider how patients and clinicians may be impacted by the trial design selected. "

A well-planned trial with a team that has maintained open communication with the FDA during design planning and has patient and clinician investment will have improved efficiency and ease of implementation.