Presentation (CTI)

OBJECTIVES: Approximately 900 orphan-designated drugs were authorized in Europe between 2020 and 2024. Among them, 53% were small molecules, 45% were biologics or oligonucleotides, and 8% were advanced therapy medicinal products (ATMPs). This review aims to assess recent policy developments in Europe and Türkiye in light of this evolving treatment landscape.
METHODS: Following PRISMA guidelines, a systematic review was conducted using PubMed to identify peer-reviewed articles published between 2020 and 2025 discussing orphan drug policy in EU countries and Türkiye. Two reviewers independently screened titles, abstracts, and full texts based on predefined inclusion (e.g., policy recommendations) and exclusion criteria (e.g., disease-specific or non-drug-focused articles). A narrative synthesis was undertaken based on the Popay J. framework, emphasizing thematic similarities and differences across jurisdictions.
RESULTS: Out of 253 records screened, 82 met the inclusion criteria. Three main themes emerged:

1. Access disparities: Despite a growing number of EMA-approved orphan drugs, access and reimbursement remain inconsistent across Europe.
2. Regulatory and HTA reform: The EU’s evolving pharmaceutical and HTA regulations aim to standardize assessments, but many OMPs still lack sufficient evidence to demonstrate added benefit.
3. National strategy differences: Most EU countries aligned with the Rare 2030 roadmap. Türkiye’s 2021-2023 Action Plan focused on infrastructure, yet only 71 of 105 EMA-approved OMPs were accessible, and just 48% were reimbursed.

CONCLUSIONS: While Europe and Türkiye have made progress in rare disease policy and regulatory alignment, major gaps persist in equitable access. Tailored HTA approaches and improved cross-country coordination are essential to close implementation gaps and ensure timely access to orphan therapies.