Priority setting for orphan drugs: an international comparison

Area of Science:

• Health economics
• Pharmaceutical policy
• Drug evaluation

Background:

• Orphan drugs, such as Cerezyme and Fabrazyme, address rare diseases.
• Priority setting for orphan drug reimbursement is complex and involves multiple stakeholders.

Purpose of the Study:

• To describe and understand the priority-setting process for Cerezyme and Fabrazyme in Canada, Australia, and Israel.
• To identify areas for improvement in orphan drug reimbursement decision-making based on stakeholder perspectives.

Main Methods:

• Qualitative case studies of three independent drug advisory committees.
• Interviews with 22 informants, including committee members, patient groups, and industry representatives.

Main Results:

• Reimbursement recommendations for orphan drugs were based on clinical evidence, cost, and cost-effectiveness.
• Committee members favored their current processes but noted fairness concerns for orphan drugs.
• Stakeholders suggested incorporating values like lack of alternatives and exploring alternative funding mechanisms.

Conclusions:

• Drug priority setting is not purely technical; it encompasses broader values.
• Understanding current reimbursement decision-making for orphan drugs can inform improvements for future orphan drugs.