April 13, 2018
The availability of orphan drugs with high unmet need
HTA Consulting assessed the availability of orphan drugs with high unmet need (non-oncological drugs, the only one with orphan status registered after 2012 by EMA). Results from 32 countries were obtained – all EU and EFTA countries were analyzed except Cyprus (as of 10/04/2018)
Average number of reimbursed orphan drugs:
| Western Europe | CEE |
| 10.3 | 3.0 |
- Most reimbursed drugs in Germany: 19 out of 23
- Average number of reimbursed drugs per country: 5,6
- The most often reimbursed medicine is Ofev (nintedanib) for the treatment of idiopathic pulmonary fibrosis.
- Strimvelis in the treatment of composite immunodeficiency resulting from adenosine deaminase deficiency (ADA-SCID) is not reimbursed in any of the analyzed countries.
Source: own study