The legal imperative for treating rare disorders

Area of Science:

• Legal and Health Policy Analysis
• Pharmaceutical Reimbursement Law
• Rare Disease Treatment Access

Background:

• Orphan drugs, vital for rare diseases, are highly expensive, straining healthcare budgets.
• Governments face pressure to reduce funding for these treatments, impacting patient access and causing distress.
• Reimbursement and pricing models for orphan drugs are under re-evaluation in the UK and EU.

Purpose of the Study:

• To present novel legal arguments compelling governments to provide orphan medicinal products.
• To analyze existing legal frameworks that mandate the provision of treatments for rare diseases.
• To counter arguments limiting access to orphan drugs and support patient rights.

Main Methods:

• Case study utilizing United Kingdom legislation.
• Examination of disability legislation, national constitutions, judicial review, tort law, and human rights law.
• Directly addressing and refuting potential objections to mandatory orphan drug provision.

Main Results:

• A compelling legal case demonstrates that existing laws mandate the treatment of orphan diseases.
• The analysis provides a robust legal foundation for the provision of orphan medicinal products.

Conclusions:

• The developed legal framework empowers doctors and patients to ensure continued access to orphan drugs.
• This analysis can guide the drafting of funding guidelines for orphan drugs across the EU.
• The legal right to treatment extends beyond rare diseases, potentially impacting broader healthcare budget allocation.