of all EMA approved orphan medicines are available in all 27 EU member states2
We apply intelligent new approaches to the issues around medicine affordability to broaden patient access to medicines.
Value for Health Systems & Payors
Our approach delivers value beyond medicines for patients, healthcare professionals and healthcare systems.
- Supporting patients to get the most from their medicines
- Supporting clinician-patient conversations
- Championing sustainable access to rare disease medicines
More rare disease treatments deliver medicine affordability
When orphan medicines lose exclusivity, we often assume generic competition will improve affordability and patient access. But does that happen in practice?
Our team conducted an analysis of 31 orphan medicines withdrawn from the European Community Register on expiration of orphan exclusivity between 2008 and 2020.
What did we find?
- Despite loss of exclusivity and patent expiration, only a minority of expired orphan medicines included in our analysis (9/31) had undergone genericisation.
- Generic entrants led to substantial price reductions.
- There was a clear correlation between the number of generic competitors and price decline, underscoring the potential for increased competition to drive further cost savings.
We believe that increased competition post patent expiration could play an important role in addressing the ongoing challenge of access of high-cost orphan medicines across Europe.
Review our poster for further details on our analysis.
1. Pharmaceutical Technology (2025) Orphan drug market access in Europe: HTA challenges and the growing potential of RWE. Available at: https://www.pharmaceutical-technology.com/sponsored/orphan-drug-market-access-in-europe-hta-challenges-and-the-growing-potential-of-rwe/ (Accessed: 14 March 2026).
2. EFPIA Patients W.A.I.T. Indicator 2024 Survey Published May 2025.
OTD-NP-GBR-00034 | August 2026