Adaptive Trial Design for Phase I, Phase II

Adaptive trial design is a hot issue in the drug development community. Adaptive conferences and web seminars abound, and suddenly, every consultant or vendor to the industry has become an adaptive expert. The FDA and EMEA are also much more receptive to adaptive trials than they were a few years ago.

Ranganath Nayak Adaptive Trial DesignHowever, the picture is a shade different from what the industry had expected. Originally, much of the focus was on adaptive phase III trials as well as on seamless phase II/III trials. Those are, indeed, successfully being implemented today. But the real action is in phase II dose-finding trials and even in phase I trials for safety.

In today’s new issue of Peer Perspectives in Oncology, Medelis talks with Ranganath Nayak, Ph.D., the CEO of Cytel, Inc., about the benefits, opportunities, and challenges presented by adaptive trial design in phase I and phase II studies. Cytel has designed more adaptive trials for industry sponsors than any other service provider and in virtually every therapeutic area, including medical devices.

Dr. Nayak begins by discussing why adaptive trial design is gaining so much momentum in early stage studies. “First, adaptive trials are most helpful when you do not know enough at the beginning of a trial to design it with confidence,” he says. “This is much more likely to be true in phases I and II than in phase III.

“Second, the best way to make expensive phase III trials more successful is to do more thorough work in phases I and II,” he continues. “Getting the dose right through well-designed phase I and phase II trials is the best way to maximize success in phase III, which then leads to a higher rate of NDAs. “

Dr. Nayak offers insight and expertise on a variety of issues surrounding adaptive trial design, including:

  • Commercial, ethical, and budgetary benefits of the adaptive trial model;
  • Statistical and data issues;
  • Changing to the adaptive model across multiple sites;
  • Safety issues to consider;
  • Budgeting for an adaptive trial;
  • Dealing with independent decision-making bodies;
  • Patient consent and communication issues;
  • Medical supply logistics;
  • Other best practices and planning issues.

View the abstract here.