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NCCN Policy Summit: Designing Clinical Trials in the Era of Multiple Biomarkers and Targeted Therapies

By Jessica K. DeMartino, PhD, Manager, Health Policy Programs

On April 25, 2014, the National Comprehensive Cancer Network® (NCCN®) hosted the NCCN Policy Summit, titled Designing Clinical Trials in the Era of Multiple Biomarkers and Targeted Therapies, in Bethesda, Maryland to present the findings of the NCCN Clinical Trials Work Group and discuss broader policy implications. With cancers increasingly recognized as being comprised of many subtypes differentiated by specific molecular profiles, defining appropriate patient subsets, selecting agents active against specific targets, predicting effective therapeutic combinations, and appropriately interpreting clinical trials results are significant challenges for clinicians, statisticians, pharmaceutical companies, and regulators. In order to meet the needs of people with cancer, these concerns must be addressed.

Alan Venook, MD, UCSF Helen Diller Family Comprehensive Cancer Center, chairman of the NCCN Clinical Trials Work Group, started the program by presenting the Work Group’s conclusions and recommendations. Dr. Venook discussed current adaptive designs for conducting clinical trials, including platform design, bucket trials, and umbrella design. Issues with biomarker specimens, including sample collection, processing, and storage, were addressed. Also discussed was the education gap that currently exists in the oncology space as it relates to biomarkers and how oncologists use the information. His presentation was followed by two roundtable discussions moderated by Clifford Goodman, PhD, The Lewin Group.

The first roundtable, Strategies for Implementation of Clinical Trials in the Era of Small Subsets and Multiple Agents, covered many topics including accruing patients for trials, clinical trial endpoints, regulatory requirements, data collection, and costs of conducting clinical trials. Roundtable panelists included David Flockhart, MD, PhD, Indiana University; Ramaswamy Govindan, MD, Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine; Al Benson III, MD, Robert H. Lurie Comprehensive Cancer Center of Northwestern University; Amy McKee, MD, US Food and Drug Administration; Eric Slosberg, PhD, Novartis; and Dr. Venook. By the end of the roundtable, the panelists agreed many improvements and scientific advancements are needed to reach the full potential biomarkers offer in developing drug therapies and, in turn, appropriately treating patients.

The day concluded with a second roundtable, Identifying and Defining Standards of Care for Cancer Subtypes, featuring panelists Michael Deininger, MD, Huntsman Cancer Institute at the University of Utah; Dr. Flockhart; Dr. Govindan; Rosemarie Hakim, PhD, Centers for Medicare and Medicaid Services; Jennifer Malin, MD, PhD, WellPoint; Lisa McShane, PhD, National Cancer Institute; and Asif Velani, MBA, Genentech. Topics for discussion included coverage and reimbursement of biomarker testing, value of biomarkers, and inclusion of biomarkers in guidelines. Ultimately, the panel struggled to resolve the divide that currently exists between the science of biomarkers and the coverage and reimbursement of biomarker testing.

A full length summary of the policy summit will be available in June 2014.

For more information about the NCCN Policy Program, visit NCCN.org or email healthpolicy@nccn.org.