Controlling off-label medication use.

Area of Science:

• Pharmacoeconomics
• Health Policy
• Biotechnology Drug Regulation

Background:

• Off-label prescribing offers benefits like novel uses and avoids lengthy FDA labeling changes.
• However, unapproved indications raise patient safety concerns for toxic drugs and economic issues for costly medications.

Purpose of the Study:

• To propose a controlled method for managing off-label use of expensive and potentially risky medications.
• To ensure patient safety and economic viability in the utilization of biotechnology drugs.

Main Methods:

• A two-step regulatory process is proposed, mirroring device regulation.
• Drugs exceeding a cost benchmark (e.g., $12,000) would undergo Centers for Medicare & Medicaid Services (CMS) National Coverage Determination post-FDA approval.

Main Results:

• The CMS would determine coverage for off-label uses based on evidence through its National Coverage Determination process.
• This approach aims to control the use of high-cost, high-risk off-label drugs, primarily biologics.

Conclusions:

• The proposed CMS-driven evidence-based coverage determination can manage off-label use of expensive and risky drugs.
• This framework is expected to be adopted by other insurance companies, promoting safer and more cost-effective prescribing.