Next week, ASCO’s annual meeting in Chicago will bring together thousands of oncology leaders to discuss the tremendous progress we have achieved in oncology research over the past year.  In the midst of that excitement, the field continues to face significant challenges in bringing ground-breaking treatments to patients. Clinical research continues to often be inefficient and unproductive.

One of the ongoing challenges for academic investigators, industry and foundations is the extreme time and cost of developing and commercializing new therapies. In the emerging era of precision oncology medicine, clinical studies often focus on increasingly small patient populations and subsets of indications. Other studies often employ powerful immune approaches with complex mechanisms of action. These studies are leading to incredible advances in targeted and immune therapies and durable patient responses.  However, the clinical studies required for these therapeutic approaches often target rare, difficult to recruit, patient subpopulations or survey multiple drug combinations and indications in large phase I/II studies. These studies often involve many amendments, large numbers of screened patients who may not qualify for the studies or face competing studies and ultimately studies that fail to meet their primary objectives. One response to these challenges is the Kraft Precision Medicine Accelerator and the Clinical Solutions Team at the Harvard Business School created to advance the use of master protocols in oncology.

One protocol: many treatments

A master protocol may use a variety of approaches to leverage a single, over-arching protocol to more efficiently evaluate multiple treatments, combinations, indications or patient cohorts   within a single trial.  The potential of a master protocol to accelerate the research process, and enable rapid learning and decision-making has been recognized for some time. The use of master protocols in various therapeutic areas has been enabled in recent years by advances in analytics, which have allowed research teams to analyze large study data for rapid decision-making enhancing our ability to identify the most promising therapies. Past and ongoing oncology master protocol studies have demonstrated the feasibility of large learning platform studies or sub-study master protocols to test multiple therapies. However, the number of drug approvals and changes in standard of care resulting from master protocols may be improved in the future through better dissemination of the knowledge gained from these studies. The Kraft Precision Medicine Accelerator Clinical Solution Team was formed to leverage the knowledge of leading cancer researchers with master protocol experience to support the design and execution of this approach in oncology. The study leaders, as well as operational managers, of a number of model master protocol studies currently in development or operation have volunteered their time and energy to support this initiative. The team is working with the Harvard Business School and the Kraft Accelerator leadership to disseminate best practices, critical paths, project performance metrics and study templates in future publications. 

Over the past year, IQVIA has participated in the Kraft Precision Medicine Accelerator to develop tools as well as best practices for these types of studies. IQVIA shares the Accelerator’s goal to identify and address challenges that slow the advancement of precision medicine, and to disseminate best practices that will speed commercialization of high-impact innovations. A key component of this effort is to encourage collaboration among cancer organizations, to better understand the most productive applications of master protocols to reduce the time, cost and redundancies in oncology research. Academic groups, foundations as well as industry leadership must come to the table to advance oncology research.

To support this goal, IQVIA and the Accelerator first conducted a deep dive into the master protocol landscape, researching past and current trials using master protocols to identify experiences, best practices and insights for future improvements. The output of that survey formed the basis for a meeting in the fall of 2017 with the FDA’s Oncology Center of Excellence to consider opportunities for better education and coordination. Multiple stakeholders, including the FDA, play a critical part in better utilization of master protocols for early signal-finding studies as well as seamless or registration studies. The Kraft Clinical Solution team continues to further develop our knowledge of master protocol studies with ongoing model master protocol study reviews focused on both organizational and study design and execution.

The Accelerator team believes that master protocol studies could be a game changer for oncology research. However, master protocols are often complex and may require lengthy startup. The study design, organizational and regulatory questions, especially for a new researcher and organization, raise potential risks that should be identified and addressed. We, at IQVIA, look forward to continuing to share learning with our colleagues on the Kraft Solution team and at ASCO in June, discussing how we can collaborate with other leaders to promote this important research, and to drive adoption of master protocol best practices in the years to come.

More about Brad Smith

Brad Smith, VP, Translational Medicine at IQVIA, is an executive leader with experience in the development and execution of biomarker and Dx strategies. Smith leads biomarker and companion diagnostic strategy and planning deliverables throughout the organization, including clinical, laboratory, consulting and commercial projects. He has more than 20 years of experience in biological research in academia, biotechnology and clinical services including over five years in planning and design of clinical development plans and protocols.