Assessing the economic challenges posed by orphan drugs

Area of Science:

• Health Economics
• Pharmaceutical Policy
• Rare Disease Research

Background:

• Rare disease drug development historically underserved by commercial interests.
• Legislation incentivizes orphan drug creation, but high costs persist.
• Standard health technology assessment (HTA) often deems orphan drugs not cost-effective, limiting access.

Purpose of the Study:

• Evaluate the adequacy of current HTA methods for orphan drugs.
• Explore societal preferences regarding orphan drug value.
• Propose a research agenda for orphan drug development, funding, and access.

Main Methods:

• Literature review on orphan drug policy and HTA.
• Analysis of economic evaluation challenges for rare disease treatments.
• Discussion of societal value and ethical considerations.

Main Results:

• Standard HTA methods may not fully capture the societal value of orphan drugs.
• High costs and cost-effectiveness hurdles limit patient access.
• Current frameworks may not align with societal preferences for rare disease treatments.

Conclusions:

• Re-evaluate standard HTA methodologies for orphan drugs.
• Investigate societal value to inform funding and access decisions.
• Develop new frameworks to ensure equitable patient access to orphan drugs.