Optimizing Patient Access to Orphan Medicinal Products: Lessons from Central and Eastern Europe

Area of Science:

• Health Economics
• Pharmaceutical Policy
• Rare Disease Research

Background:

• Orphan medicinal products (OMPs) are crucial for treating rare diseases.
• Central and Eastern European (CEE) countries face unique challenges in OMP access compared to Western Europe.
• Healthcare systems in CEE have undergone rapid transformations, impacting OMP accessibility.

Purpose of the Study:

• To identify barriers and enablers for optimizing patient access to OMPs in CEE countries.
• To provide insights for sustainable and equitable OMP access strategies.
• To compare OMP access across seven CEE nations.

Main Methods:

• Mixed-methodology approach combining systematic literature reviews and in-depth interviews.
• Stakeholder engagement including policymakers, payers, industry, and patient groups.
• Analysis of CEE-specific initiatives and health technology assessment (HTA) frameworks.

Main Results:

• Sub-optimal OMP access observed in most CEE countries, characterized by delays and lower reimbursement rates.
• Slovenia and Czechia show notable exceptions in OMP access.
• Key barriers identified: limited awareness, inadequate HTA, insufficient financing, and fragmented patient engagement.

Conclusions:

• Addressing barriers in HTA, financing, and patient engagement is crucial for improving OMP access in CEE.
• Implementing rare disease policies and patient-inclusive decision-making can enhance OMP accessibility.
• Sustainable and equitable OMP access requires tailored strategies for the CEE region.