An evaluation framework for funding drugs for rare diseases

Area of Science:

• Health Policy
• Pharmacoeconomics
• Rare Disease Treatment

Background:

• Generating robust clinical trial data for rare diseases is challenging, complicating drug funding decisions.
• Enzyme replacement therapies for inherited metabolic enzyme deficiencies exemplify this funding dilemma.
• The Ontario Public Drug Programs established a working group to address rare disease drug assessment.

Purpose of the Study:

• To develop a transparent, consistent, and adaptable policy for assessing drugs for rare diseases.
• To address the unique aspects of rare condition treatments from a public payer perspective.
• To incorporate principles of "accountability for reasonableness" in drug funding evaluations.

Main Methods:

• A seven-step policy framework was developed by the Drugs for Rare Diseases Working Group.
• Decision modeling was employed to estimate the clinical effectiveness of treatments.
• The framework was tested using idursulfase for mucopolysaccharidosis II (Hunter disease) as a case study.

Main Results:

• The developed framework successfully informed funding recommendations for idursulfase.
• A funding agreement with the manufacturer was reached, enabling idursulfase access in Ontario.
• The case study demonstrated the practical application and effectiveness of the policy framework.

Conclusions:

• The policy framework provides a structured approach to the challenges of funding rare disease drugs.
• The framework is intended for future use in assessing other rare disease medications.
• The policy is expected to evolve with experience and may serve as a valuable resource for other policymakers.