Rare disease therapies can transform lives — but getting them reimbursed is one of the hardest challenges in market access. Small populations, limited data and high prices collide with payer budgets and cost-effectiveness thresholds.
In the EU, orphan designation is granted under Regulation (EC) No 141/2000 for conditions affecting no more than 5 in 10,000 people, bringing incentives including ten years’ market exclusivity. But designation is not reimbursement — access still depends on national HTA and pricing decisions.
The evidence Catch-22
With so few patients, large randomised trials are often impossible. Orphan dossiers frequently rest on single-arm studies, surrogate endpoints or small, immature datasets. The result is a familiar bind: rapid approval on limited evidence, followed by high clinical uncertainty when payers assess value.
Delays and inequity
The average time from EU marketing authorisation to actual patient access for orphan medicines is roughly 18 months, and it varies enormously between countries — leaving patients with the same condition facing very different access depending on where they live.
Bridging the gap
High per-patient prices, needed to recoup R&D across tiny populations, sit uneasily with cost-effectiveness thresholds. The main tools to bridge the uncertainty are managed-entry and risk-sharing agreements and greater use of real-world evidence to confirm value after launch. From January 2028, orphan medicines also enter the scope of the EU Joint Clinical Assessment, which may harmonise the clinical evaluation — though pricing and reimbursement stay national.
The Greek picture
Greece gives orphan medicines some eased pricing rules — for example, they can be priced even where reference prices exist in only two Eurozone Member States, and are exempt from the usual requirement to be reimbursed in two-thirds of Member States. Reimbursement runs through EOPYY and the HTA Committee, with an annual horizon-scanning process in place since 2022. Clawback and rebate obligations still weigh heavily on high-cost products.
How PQRA helps
PQRA builds orphan-drug value dossiers, designs managed-entry proposals and manages the EOPYY reimbursement route — turning limited evidence into a defensible case for access.
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