Challenges with Novel Clinical Trial Designs: Master Protocols.

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Cecchini M1Rubin EH2Blumenthal GM3Ayalew K4Burris HA5Russell-Einhorn M6Dillon H7Lyerly HK8Reaman GH9Boerner S10LoRusso PM10.

The 2018 Accelerating Anticancer Agent Development (AAADV) Workshop assembled a panel of experts for an in-depth discussion session to present Challenges with Novel Clinical Trial Designs. This panel offered assessments of the challenges faced by industry, the Food and Drug Administration, investigators, institutional review boards, and patients. The panel focused on master protocols, which include umbrella trials, platform trials, and basket trials. Umbrella trials and platform trials share many commonalities, while basket trials are more distinct. Umbrella and platform trials are generally designed with multiple arms where patients of the same histology or other unifying characteristics are enrolled into different arms and multiple investigational agents are evaluated in a single protocol. In contrast, basket studies generally enroll patients with different tumor types based on the presence of a specific mutation or biomarker regardless of histology; these trials may include expansion cohorts. These novel designs offer the promise of expedited drug assessment and approval, but they also place new challenges on all the stakeholders involved in the drug development process. Only by identifying the challenges of these complex innovative clinical trial designs and highlighting challenges from each perspective, can we begin to address these challenges. The 2018 AAADV Workshop convened a panel of experts from relevant disciplines to highlight the challenges that are created by master protocols, and where appropriate, offer strategies to address these challenges.

  1. Internal Medicine (Medical Oncology), Yale University
  2. VP Clinical Oncology, Merck Research Laboratories.
  3. Center for Drug Evaluation and Research, United States Food and Drug Administration.
  4. Office of Compliance, Food and Drug Administration.
  5. Department of Medicine/Medical Oncology, Sarah Cannon Research Institute.
  6. Chief Compliance Officer and IO, Advarra.
  7. Program and Education, Cancer Support Community.
  8. Surgery, Duke University Medical Center.
  9. Pediatrics, The George Washington University School of Medicine and Health Sciences.
  10. Yale Cancer Center, Yale University.