1

Introduction

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



The National Academies | 500 Fifth St. N.W. | Washington, D.C. 20001
Copyright © National Academy of Sciences. All rights reserved.
Terms of Use and Privacy Statement



Below are the first 10 and last 10 pages of uncorrected machine-read text (when available) of this chapter, followed by the top 30 algorithmically extracted key phrases from the chapter as a whole.
Intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text on the opening pages of each chapter. Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.

Do not use for reproduction, copying, pasting, or reading; exclusively for search engines.

OCR for page 1
1 Introduction 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 complex - ity. 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 develop - ment is thought to be a promising strategy for identifying cancer treat- ments that will be effective in the long term, especially when a combi- nation 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. How - ever, this approach to drug development presents several unique chal- lenges, 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 com - panies, and between pharmaceutical companies and academic or govern- ment research institutions. To help further the development of innovative combination cancer therapies, the National Cancer Policy Forum of the Institute of Medi- cine held a workshop entitled “Facilitating Collaborations to Develop 1

OCR for page 1
2 COLLABORATIONS TO DEVELOP COMBINATION CANCER THERAPIES 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: • cientific challenges and opportunities in the codevelopment of S investigational therapies; • egulatory environment for codevelopment, including the recent R Food and Drug Administration (FDA) draft guidance on this topic; • ultural and legal issues that affect collaboration; C • essons learned from codeveloping human immunodeficiency L virus and cancer combination therapies; and • urrent examples that can serve as possible models of collabora- C tion 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 com- bination 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.