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Master Protocol Studies

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overview icon Overview

Using Master Protocol Studies to Drive Medical Product Development

 

The value of master protocol studies has become clear: we need agile clinical trials that foster collaboration to address major public health threats and ongoing research challenges.

 

Working with stakeholders across the clinical trials ecosystem, CTTI developed a robust set of resources that guide the appropriate use of master protocols, including a:

  • Master Protocol Design & Implementation Guide: A high-level roadmap for the pre-planning, planning, and execution phases of a master protocol study
  • Value Proposition Guide: outlines key considerations for presenting the value proposition of the master protocol to stakeholders such as collaborators and vendors
  • Study Simulation Tool: provides an overview of key modules that should be simulated in order to optimize master protocol design
  • Protocol Development Map: describes the unique aspects of developing a master protocol from the academic or nonprofit organization perspective
  • Operations Partner Assessment Tool: outlines considerations for engaging and selecting vendors as partners in master protocols
  • FDA Engagement Tool: describes the formal mechanisms that facilitate early interaction with the FDA and provides a general timeline for the development of an FDA engagement strategy

Additionally, specific to the pandemic, CTTI led a panel discussion, “The Fastest Path to Effective COVID-19 Treatments: Using Master Protocol Studies," highlighting results from an analysis of data from ClinicalTrials.gov, as well as best practices and insights from those involved in COVID-19 treatment master protocols.

Recording Now Available: The Fastest Path to Effective COVID-19 Treatments: Using Master Protocol Studies Public Summit

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in detail icon In Detail

What is the issue?

Master protocol studies offer innovative opportunities in the design and conduct of clinical trials. The term master protocol is generally used to describe clinical trials that use one overarching protocol to guide multiple, simultaneously occurring sub-studies. Master protocol studies can be used to study multiple diseases, multiple therapies, or both.

 

While enthusiasm for master protocol studies is growing, the number of organizations that have sufficient experience designing and implementing master protocol studies remains critically low. Several organizations have provided opportunities for stakeholders to learn general information about master protocol studies. Efforts are needed to build off this important work to create practical, detailed resources to support the design and conduct of master protocol studies.

 

 

What CTTI Project has been initiated to address the issue?

Master Protocol Studies: Opportunities, Challenges, and Emerging Best Practices (“Master Protocol Studies”) (2019 – ongoing)

 

 

What are the objectives of CTTI’s work on master protocols?
  • Compare and contrast the distinct design and operational features of basket, umbrella, and platform trials.
  • Identify emerging best practices for the design and conduct of master protocol studies.

 

What is the anticipated impact of CTTI’s work on master protocols?

CTTI’s work will increase the appropriate use of master protocol studies.

 

 

How was evidence gathered to support our findings?
  • A landscape review – Expert interviews and a literature scan will be used to develop a high-level roadmap that details strategies to address common roadblocks that inhibit the development of master protocol studies at the pre-planning, planning, or execution stage.
  • An Expert Meeting series – This will be used to identify emerging best practices and prioritize the creation of tools to support stakeholders’ efforts to address common challenges related to the design and implementation of master protocol studies.

 

Project Team Members

Rolesort ascending Name Affiliation
Team Member Ritesh Jain EMD Serono
Team Member Nicholas Kenny Syneos Health
Team Member Barbara LeStage Individual Patient/Caregiver
Team Member Roger Lewis University of California, Los Angeles
Team Member Greg Licholai Yale University
Team Member Sharon Love University College London
Team Member Sumithra Mandrekar Mayo Clinic
Team Member Debra Michaels DIA
Team Member Sabrina Paganoni Harvard University
Team Member Jane Perlmutter Individual Patient/Caregiver
Team Member Dionne Price Food and Drug Administration
Team Member Paulina Selaru Pfizer, Inc.
Team Member Antonia Valakas EMD Serono
Team Member Gene Vinson Syneos Health
Team Member Alain Vuong GlaxoSmithKline
Team Member Kirsty Wydenback Medicines and Healthcare Products Regulatory Agency (MHRA)
Team Member Kert Viele Berry Consultants
Team Member Michelle Detry Berry Consultants
Team Member Elena Kovalenko Novartis
Team Member Christine Mayer-Nicolai EMD Serono
Team Member Abby Bronson Edgewise Therapeutics
Team Member Pamela Tenaerts Clinical Trials Transformation Initiative
Team Member Angie Botto-van Bemden Arthritis Foundation
Team Member Louise Brown University College London
Team Member Amy Burd Leukemia & Lymphoma Society
Team Member Paul Bycott Pfizer, Inc.
Team Member Karen Dimick Genentech - a member of the Roche Group
Team Member Hans-Juergen Fuelle Novartis
Team Member Emma Healy Novartis
Team Lead Rajeshwari Sridhara Food and Drug Administration
Team Lead Shenghui Tang Food and Drug Administration
Team Lead Marianne Chase Harvard University
Team Lead Daniel Millar Janssen Pharmaceuticals
Team Lead Nicholas Richardson Food and Drug Administration
Project Manager Kim Fisher* Clinical Trials Transformation Initiative
Project Manager Zachary Hallinan Clinical Trials Transformation Initiative

Organization affiliations are listed as active affiliations during the project.

*Indicates former project manager, team leader, or team member.