CTTI will host a public webinar on Tues., Oct. 13 to announce new resources that provide a roadmap for designing and running master protocols.

A master protocol approach allows researchers to answer multiple scientific questions within a single clinical trial infrastructure. With a flexible design and highly centralized operational features, the master protocol platform can eliminate several challenges, resources, and expenses associated with developing and running individual protocols.

“With the success of master protocols in COVID-19 trials, there’s never been a better time to rethink the way you’re doing clinical trials in other disease areas,” said Pamela Tenaerts, executive director at CTTI. “In many cases, a well-designed and well-planned master protocol approach is an ideal way to get answers to scientific questions faster and serve patients better. Our new set of resources give sponsors, non-profit groups, and others a starting point and pathway for designing and conducting a successful master protocol study from start to finish in your disease area.”

CTTI will release a complete master protocol design and implementation roadmap, case studies, and other resources including a:

• 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 development of an FDA engagement strategy

“Due to the complexity of master protocol trials and the potential regulatory impact, a master protocol needs to be well-designed and well-conducted to ensure patient safety and quality data,” said Nicholas Richardson, clinical reviewer at the Center for Drug Evaluation and Research (CDER) at the U.S. Food and Drug Administration (FDA). “This requires significant upfront planning and resources, and CTTI’s new resources outline considerations and instructions for planning and executing a successful master protocol study.”

Through a high-level landscape review, a series of expert meetings, and a public open comment opportunity, CTTI created these resources to help organizations develop well-designed master protocols and create a network to facilitate their implementation.

“We need functioning resources that address obstacles in master protocol design and how to overcome them,” said Marianne Chase, director of research operations at the Neurological Clinical Research Institute (NCRI), Massachusetts General Hospital. “With CTTI’s new offerings, we now we have a roadmap to follow—we can hit the ground running with development and, eventually, start to reap the many benefits that a master protocol approach has to offer.”

The free webinar will begin at 11 a.m. ET and will be led by Richardson; Chase; Abby Bronson, Edgewise Therapeutics; and Daniel Millar, Janssen R&D.

Amid the COVID-19 pandemic, 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. CTTI invites members from across the clinical trials ecosystem to provide feedback on its draft resources to help drive the appropriate use of master protocols in diverse therapeutic areas.

Master protocols studies have the potential to more efficiently ask and answer scientific questions – ultimately, getting the right drugs to patients more quickly. However, many organizations lack experience in running master protocol studies, presenting major study design and operational challenges.

CTTI is addressing this gap by developing publicly available resources to support sponsors, patient advocacy groups, and others that want to design a master protocol trial. Public feedback will help shape the final materials that CTTI makes available to the public this summer.

The resources, developed by experts and other stakeholders across the clinical trials ecosystem, include:

• Master protocols roadmap
• FDA engagement tool
• Business & operations plan template
• Protocol development template
• Vendor assessment tool

Feedback is welcome until April 24 at 11:59 a.m. ET.

Despite a growing enthusiasm for the use of master protocol studies, expertise is critically limited and few resources exist to guide planning and implementation. Working to fill this gap, CTTI held its first Master Protocol Studies expert meeting on Oct. 22-23 to identify strategies, tools, and resources that will help drive appropriate and efficient use of master protocols.

Attendees discussed challenges and potential solutions to planning and running master protocol studies. They also uncovered and agreed on some important common themes:

• The Value is Clear…Master protocol studies are growing in popularity. Their flexible design and highly centralized operational features have the potential to more efficiently ask and answer scientific questions.
• …But a Roadmap is Needed…However, many organizations lack experience in running master protocol studies, presenting major study design and operational challenges.
• …As is Support for Non-Traditional Drug Developers. Publicly available resources are needed to support organizations that want to design a master protocol trial, particularly for non-traditional drug developers such as patient advocacy groups and other nonprofits.
• Be Excited, but Realistic. Enthusiasm for a master protocol approach should be balanced with awareness of its complex infrastructure, long-ranging timelines, and significant upfront planning and resources.
• There are Keys to Long-Term Success. A strong cross-institutional infrastructure, including a robust site network and solid communications approach, are critical to achieving long-term sustainability of master protocols.

Based on these discussions and findings, attendees refined CTTI’s draft roadmap for the successful design and implementation of master protocol studies, and identified a broad set of tools and resources that can drive adoption of master protocols across diverse therapeutic areas.

Learn more about CTTI’s Master Protocol expert meeting and next steps with work to advance master protocol adoption.