The FDA is seeking comments of a new draft guidance that it recently issued for industry called “Adaptive Designs for Medical Device Clinical Studies.” An “adaptive design” for a medical device clinical study is defined as a clinical trial design that allows for prospectively planned modifications based on accumulating study data without undermining the trial’s integrity and validity.
An adaptive study design can have several distinct advantages when compared to an unchanged (fixed) design. It can be more efficient, saving time, money, and resources. Adaptive designs can improve the chance of trial success by employing sample size reassessment. It can yield an improved understanding of the effect of the investigational treatment and a better understanding of benefit and risk. Adaptive design may facilitate transition from premarket to postmarket follow-up.
Adaptive designs can include a plan to modify the patient population during the study, converting what would otherwise be a failed study to one with, for example, a more targeted indication for which there are data to support both safety and effectiveness. This adaptation could help identify patients more likely to have a favorable benefit-risk profile from the use of a device.
Adaptive studies can also improve decision-making at milestones during product development or increase the chance of a successful study with the potential to improve time-to-market.
A sound clinical study requires extensive planning, with consideration given to all elements of the trial, from design to a plan for data analysis. With adequate preplanning, a sponsor can use the study’s accumulating data to modify various aspects of the study in a scientifically valid manner. An unplanned modification to the study may weaken its scientific validity and may not be approved or endorsed by the FDA. However, in some specific circumstances, study modifications after the trial begins can be scientifically valid if the trial design decision-makers have had no access to the outcome results by treatment.
This document addresses adaptive designs for medical device clinical trials and is applicable to premarket medical device submissions including Premarket Approval Applications (PMA), premarket notification (510(k)) submissions, de novo submissions (Evaluation of Automatic Class III Designation), Humanitarian Device Exemption (HDE) applications and Investigational Device Exemption (IDE) submissions. This guidance can be applied throughout the clinical development program of a medical device, from feasibility studies to pivotal clinical trials.
Comments on the guidance will be accepted until August 18, and may be submitted electronically to www.regulations.gov. For more information, visit www.medicaldesignbriefs.com/FDA_Adaptive .