Policy Issues in the Development and Adoption of Biomarkers for Molecularly Targeted Cancer Therapies

A long-held goal in oncology has been to develop therapies that target the specific abnormalities in each patient's cancer rather than simply treating cancers based on the tissue of origin. In the past decade, advances in technology have enabled researchers to relatively quickly and inexpensively determine, in minute detail, the genetic makeup of tumors. Although relatively few targeted cancer therapies are currently available in the clinic and it is not yet clear whether all cancers are driven by genetic changes that can be targeted, there is widespread optimism in the cancer community that this new ability to assess the genetic abnormalities in tumors will ultimately lead to better cancer treatments and improved patient outcomes.

Policy Issues in the Development and Adoption of Biomarkers for Molecularly Targeted Cancer Therapies is the summary of a workshop convened in November 2014 by the Institute of Medicine's National Cancer Policy Forum to discuss recent trends in the development and implementation of molecularly targeted cancer therapies and explore potential policy actions to address specific challenges. This report highlights the presentations and discussions at the workshop.

Contents

• THE NATIONAL ACADEMIES
• WORKSHOP PLANNING COMMITTEE
• NATIONAL CANCER POLICY FORUM
• Reviewers
• Acknowledgments
• Acronyms
• WORKSHOP SUMMARY
  • INTRODUCTION
  • MOLECULAR BIOLOGY REVOLUTION IN CANCER DIAGNOSIS AND TREATMENT
  • CHALLENGES IN BIOMARKER TEST DEVELOPMENT
  • REGULATORY OVERSIGHT CHALLENGES
  • CLINICAL IMPLEMENTATION CHALLENGES
  • REIMBURSEMENT CHALLENGES
  • GATHERING THE EVIDENCE: INNOVATIVE CLINICAL TRIALS
  • GATHERING THE EVIDENCE: COVERAGE DECISIONS
  • GATHERING THE EVIDENCE: DATABASES AND REGISTRIES
  • EDUCATION AND CLINICAL DECISION SUPPORT NEEDS
  • RESEARCH NEEDS
  • CLOSING REMARKS
• REFERENCES
• Appendix Workshop Statement of Task and Agenda

Rapporteurs: Sharyl J. Nass, Jonathan Phillips, and Margie Patlak.

This project was supported by Contract No. HHSN261200900003C and Contract No. HHSN263201200074I (Task Order No. HHSN26300052) between the National Academy of Sciences and the Centers for Disease Control and Prevention and the National Cancer Institute/National Institutes of Health, respectively, and by the American Association for Cancer Research, the American Cancer Society, the American Society for Radiation Oncology, the American Society of Clinical Oncology, the American Society of Hematology, the Association of American Cancer Institutes, AstraZeneca, Bristol-Myers Squibb, the Cancer Support Community, C-Change, the CEO Roundtable on Cancer, EMD Serono, Helsinn Healthcare SA, the LIVESTRONG Foundation, the National Comprehensive Cancer Network, Novartis Oncology, the Oncology Nursing Society, and Sanofi Oncology. The views presented in this publication do not necessarily reflect the views of the organizations or agencies that provided support for the activity.

Suggested citation:

IOM (Institute of Medicine). 2015. Policy issues in the development and adoption of biomarkers for molecularly targeted cancer therapies: Workshop summary. Washington, DC: The National Academies Press.

NOTICE: The workshop that is the subject of this workshop summary was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine.