Novel approach to decision making for orphan drugs

Area of Science:

• Health Economics
• Public Health Policy
• Pharmaceutical Policy

Background:

• Limited access to orphan medicinal products (OMPs) in Czechia (59% available, 29% reimbursed) prompted policy change.
• Declining OMP availability and reimbursement rates over time highlighted patient access challenges.
• Public debate led to the development of a special pathway for OMP reimbursement in Czechia.

Purpose of the Study:

• To describe the newly adopted rigorous pricing and reimbursement procedure for OMPs in Czechia.
• To evaluate the potential of this novel approach in improving patient access to orphan drugs.
• To present Czechia's system as a real-world example of value-based decision-making in healthcare.

Main Methods:

• Legislation incorporates value assessment recommendations and societal perspective for rare diseases.
• Marketing Authorization Holders or Health Insurance Funds submit applications with clinical, cost-effectiveness, and budget impact analyses to the HTA agency.
• Professional associations and patient organizations participate, providing evidence and comments, followed by HTA agency assessment and an advisory body's binding opinion.

Main Results:

• A rigorous pricing and reimbursement procedure with strict timelines and methodology has been adopted.
• The process involves submission, evidence assessment, and evaluation by a multi-stakeholder advisory body.
• The new pathway aims to provide satisfactory patient access to orphan drugs.

Conclusions:

• The novel approach is expected to improve patient access to orphan medicinal products.
• This system serves as a practical demonstration of value-based decision-making.
• The crucial role of the advisory body in issuing binding opinions is highlighted.