The shortcomings of traditional study designs have come into sharp relief during the COVID-19 pandemic. Regulatory agencies and the research community alike have responded, acknowledging the need for different ways of performing clinical trials.
At Parexel Biotech, we view this as an opportunity to heighten the acceptance of novel trial designs. We believe that this growing openness heralds more expeditious delivery of solutions that address unmet patient needs while maintaining high data quality and integrity. With the statistical methodology well established, we have advocated for innovations such as basket, umbrella, and platform trials to align with the sponsor’s development strategy. While operational complexity is undoubtedly involved, we believe that the benefits outweigh the challenges and anticipate that this approach will soon become the norm.
As the adoption of novel and adaptive trials gains momentum, the use of master protocols offers an important option for the clinical research community. Master protocols employ a single infrastructure, trial design, and protocol to simultaneously evaluate multiple drugs and or disease populations in multiple sub-studies. This represents a significant departure from traditional trial designs, where a single drug is tested in a single disease population in a single trial.
Master protocols can more efficiently determine which therapy or combinations of therapies can generate the most robust response in diseases with the same overall clinical trial structure. When using a master protocol is an option, whether a basket, umbrella, platform, matrix, or other design, this approach has significantly accelerated decision-making processes.
Of course, there is a great deal for project teams to consider in planning and executing a successful master protocol design.
- Bringing together cross-functional experts to plan and agree upon a design that incorporates all points of view is recommended. These include medical, regulatory, biostatistics, project leadership, data management, clinical, logistics, medical communications, and real-world evidence perspectives.
- The evolution of clinical trial designs has led to the parallel introduction of new technologies. Rapidly evolving systems, tools, and processes support pharmacovigilance, risk management, decentralized trials, regulatory compliance, data integrity and completeness, clinical supply chain management, and much more. A thorough understanding of these tools, and judicious application at the appropriate stages, is foundational.
- While technology is a key component in complex trials, practical operational oversight, expert project management, and sound governance are essential. Master protocol trials involve numerous concurrent activities, requiring skillful resource planning to properly structure and organize teams and assign the right people at the right stages. Contingent team members might need to be available to manage variabilities in workload, and outside vendors brought in to assist with certain functional tasks requiring specialized expertise.
- Initial planning must encompass feasibility, regulatory submissions and approvals, site management and monitoring, data management, data availability, safety management, quality management, and all other components of the study lifecycle.
Parexel Biotech actively supports the evolution from traditional to innovative trial designs and embraces this paradigm shift in drug development. Our clinical development team has created a playbook focusing specifically on master protocol designs in tandem with multiple working groups and subject-matter experts. We offer this in-depth resource to help facilitate planning, operationalizing, and executing your research studies in our mutual quest to accelerate the delivery of new treatment options to patients.
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