Excerpt

The term “targeted therapies” refers to antineoplastic treatments designed not to kill cells but, more precisely, to attack growth factors, cell surface receptors, and intracellular proteins that mediate the cancer cell’s ability to proliferate, grow, or evade cell death. Examples of targeted therapies include small molecule inhibitors, monoclonal antibodies, and conjugated agents. Intended to damage or destroy cancer cells while minimizing the effects on normal cells, several targeted therapies have been successfully brought into routine clinical use, with approval from the U.S. Food and Drug Administration (FDA) for specific indications. The use of these FDA-approved agents has expanded to include multiple indications other than those for which they received FDA approval (i.e., “off-label indications”); in current clinical practice in oncology, off-label prescribing is common.

The primary purpose of this technology assessment is to evaluate the state of the evidence for/against the use of selected targeted therapies for off-label indications. Secondarily, the report also considers the practicality of the traditional systematic review approach, when applied to examine the evidence in rapidly evolving therapeutic areas such as targeted therapies for various cancers.

Contents

• Peer Reviewers
• Executive Summary
• 1. Background
  • Clinical Context
  • Technology Assessed
  • Policy Context
  • Scope of Study
• 2. Methods
  • Selection Criteria
  • Search Strategy
  • Data Abstraction
  • Quality Assessment
  • Data Synthesis
• 3. Results
  • Velocity of Evolution of the Literature
  • Systematic Literature Review for Drug/Disease Combinations
• 4. Discussion
• References
• Acronyms and Abbreviations
• Appendix Results of Systematic Literature Review for Specific Drug/Disease Combinations

Acknowledgements: The authors gratefully acknowledge the contributions of R. Julian Irvine, Project Co-ordinator; Rebecca Gray and Amanda McMillan, Editors; and Alice Thacher, Research Assistant.

This report is based on research conducted by the Duke Evidence-based Practice Center under contract to the Agency for Healthcare Research and Quality (AHRQ), Rockville, MD (Contract No. HHSA 290-02-0025). The findings and conclusions in this document are those of the author(s) who are responsible for its contents; the findings and conclusions do not necessarily represent the views of AHRQ. No statement in this article should be construed as an official position of the Agency for Healthcare Research and Quality or of the U.S. Department of Health and Human Services.

The information in this report is intended to help health care decision-makers; patients and clinicians; health system leaders; and policymakers make well-informed decisions and thereby improve the quality of health care services. This report is not intended to be a substitute for the application of clinical judgment. Decisions concerning the provision of clinical care should consider this report in the same way as any medical reference and in conjunction with all other pertinent information, i.e., in the context of available resources and circumstances presented by individual patients.

This report may be used, in whole or in part, as the basis for development of clinical practice guidelines and other quality enhancement tools, or as a basis for reimbursement and coverage policies. AHRQ or U.S. Department of Health and Human Services endorsement of such derivative products may not be stated or implied.

None of the investigators has any affiliations or financial involvement related to the material presented in this report.