Should rare diseases get special treatment?

Area of Science:

• Health Policy
• Drug Development
• Rare Diseases

Background:

• Orphan drug policies often prioritize rare diseases, leading to potential inequities.
• Rarity is the primary criterion for special treatment in current drug policies.

Purpose of the Study:

• To argue for a shift in orphan drug policy from rarity to disease severity as the primary funding criterion.
• To demonstrate that disease severity, not prevalence, influences judgments about overlooked treatments for rare diseases.

Main Methods:

• Philosophical argumentation and ethical analysis.
• Derivation of implications for drug development and policy.

Main Results:

• Prioritizing severity avoids issues associated with rarity-based prioritization.
• A severity-based approach is compatible with various ethical frameworks.
• This view highlights neglect of common diseases in developing countries.

Conclusions:

• Reforming orphan drug policy to focus on severity is ethically justifiable and practically beneficial.
• A severity-based framework can lead to more equitable drug development and funding.