In the past decade, a great deal of new knowledge about the molecular underpinnings of cancer has accumulated, and the unique array of molecular aberrations in each individual tumor can now be assessed through genomic sequencing and other high-throughput tests. The rationale for and the feasibility of developing molecularly targeted therapies for cancer has never been stronger, and there are hundreds of candidate targeted drugs in the development pipeline. However, many challenges remain in effectively and efficiently developing new targeted therapies for cancer, as well as in implementing them appropriately in clinical practice.
Through panel discussions and invited presentations, this NCPF workshop examined recent trends in the development and implementation of molecularly targeted cancer therapies and considered potential policy actions to improve the process. Workshop participants examined the challenges in developing molecularly targeted therapies and the associated biomarker tests within the traditional research and development paradigm as well as the challenges in implementing treatments and biomarker tests in clinical practice. A major goal of the workshop was to facilitate discussion about potential strategies and actions to address those challenges.
Participants were invited to discuss topics that included:
• clinical trial designs, the role of registries, and the N-of-1 approach to evidence development,
• evidentiary needs, regulatory processes, and ethical/legal considerations,
• the logistics and funding of multisite studies, including patient participation and coverage of experimental costs (biospecimen acquisition, biomarker test and drug) in clinical research, and
• clinical decision-making tools for appropriate implementation in clinical practice.
Workshop Planning Committee
Adrian Senderowicz, Ignyta, Inc., Chair
Kenneth Anderson, Harvard University
Michael Caligiuri, Ohio State University Comprehensive Cancer Center
Barbara Conley, National Cancer Institute
Carol Hahn, Duke University
Roy Herbst, Yale University
Samir Khleif, Georgia Regents University Cancer Center
Robert McDonough, Aetna
Lisa McShane, National Cancer Institute
John Mendelsohn, The University of Texas MD Anderson Cancer Center
Richard Pazdur, Food and Drug Administration
Richard Schilsky, American Society of Clinical Oncology
Confirmed Speakers
Garnet Anderson, Fred Hutchinson Cancer Research Center
Dane Dickson, Palmetto, Teton Cancer Institute
David Eberhard, The University of North Carolina at Chapel Hill
Bill Gradishar, Northwestern University
Neil Hayes, The University of North Carolina at Chapel Hill
Roy Herbst, Yale University
Matthias Holdhoff, Johns Hopkins University
Bruce Johnson, Dana-Farber Cancer Institute
Mia Levy, Vanderbilt University
David Litwack, FDA
Karen Long, Abbott Molecular
Patricia Lo Russo, Yale University
Jennifer Malin, Wellpoint
Anne-Marie Martin, GlaxoSmithKline
Donna Messner, CMTP
Federico Monzon, Invitae
Kathryn Phillips, University of California, San Francisco
Richard Schilsky, American Society of Clinical Oncology
Adrian Senderowicz, Ignyta, Inc.
Lilian Siu, Princess Margaret Hospital, Ontario Cancer Institute
David Solit, Memorial Sloan-Kettering Cancer Center
Sean Tunis, CMTP
Mickey Williams, National Cancer Institute