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Off-label use of targeted therapies in oncology

Dominique Levêque1

  • 1Dominique Levêque, Division of Pharmacy, hôpital Hautepierre, 67000 Strasbourg, France.

World Journal of Clinical Oncology
|April 16, 2016
PubMed

Insights

Off-label drug use in oncology, especially for targeted therapies, is common but underdocumented. While potentially more rational due to molecular profiling, it faces challenges in clinical support and reimbursement.

Area of Science:

  • Oncology
  • Pharmacology
  • Genetics

Background:

  • Off-label prescribing involves using marketed drugs outside approved indications.
  • In oncology, this includes targeted therapies for tumors with matching molecular targets.
  • Anti-angiogenic drugs and immunotherapies also have potential for alternative uses.

Purpose of the Study:

  • To document and analyze the off-label use of targeted therapies in oncology.
  • To compare off-label prescribing patterns of targeted therapies versus older drugs.
  • To identify challenges associated with off-label targeted therapy use.

Main Methods:

  • Literature review and analysis of existing data on off-label drug prescriptions.
  • Comparison of prescribing trends for targeted therapies and conventional agents.
  • Assessment of factors influencing off-label use, including molecular profiling and cost.

Main Results:

  • Off-label use of targeted therapies is documented but appears to be within the range of older drugs.
  • Prescribing variations exist among different targeted agents.
  • Off-label use of targeted therapies may be more rational due to tumoral genotyping.

Conclusions:

  • Off-label targeted therapy use, while potentially rational, faces limited clinical support.
  • High drug pricing and the costs of molecular profiling present significant reimbursement challenges.
  • Further research and policy adjustments are needed to support evidence-based off-label prescribing.
Keywords:
Enzyme inhibitorMonoclonal antibodyOff-label anticancer drug useReimbursementTargeted therapy

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