Despite recent advances in deciphering the molecular pathways that trigger cancer and its progression, progress in developing therapies that effectively target those pathways has been hampered by their complexity. Researchers are finding that many cancer pathways have backup or bypass pathways that foster drug resistance. Consequently, targeted therapies that initially are quite effective in certain patients eventually stop working. In addition, many therapies appear to work best when they are used in combination with various treatment modalities.
Combining investigational cancer therapies early in their development is thought to be a promising strategy for identifying cancer treatments that will be effective in the long term, especially when a combination targets multiple pathways and byways in the development or progression of cancer, or more than one step in a pathway, potentially conferring greater benefit than a therapy directed at a single target. However, this approach to drug development presents several unique challenges, including developing and applying appropriate preclinical tests and clinical trial designs, prioritizing which combination therapies should be tested, avoiding toxicity of multiple agents, and overcoming legal and cultural barriers that impede collaboration among pharmaceutical companies, and between pharmaceutical companies and academic or government research institutions.
To help further the development of innovative combination cancer therapies, the National Cancer Policy Forum of the Institute of Medicine held a workshop entitled “Facilitating Collaborations to Develop
Combination Investigational Cancer Therapies” in Washington, DC, on June 13–14, 2011. The workshop agenda can be found in Appendix B of this report. The workshop convened experts to identify barriers that may be impeding the development of combination investigational cancer therapies as well as to offer ways to overcome those barriers. Workshop presentations and discussions included those that addressed:
• Scientific challenges and opportunities in the codevelopment of investigational therapies;
• Regulatory environment for codevelopment, including the recent Food and Drug Administration (FDA) draft guidance on this topic;
• Cultural and legal issues that affect collaboration;
• Lessons learned from codeveloping human immunodeficiency virus and cancer combination therapies; and
• Current examples that can serve as possible models of collaboration to develop combination cancer therapies.
A key goal of this workshop was to identify potential solutions to improve collaboration and accelerate the development of promising combination investigational cancer therapies. This document is a summary of the workshop. The views expressed in this summary are those of the speakers and discussants, as attributed to them, and are not the consensus views of the workshop participants, nor of the members of the National Cancer Policy Forum.