Abstract
Orphan drugs are drugs used in the treatment of life-threatening or chronic diseases that affect fewer than 1 out of 2000 persons in the European Union. Since the implementation of the European Regulation on Orphan Medicinal Products in 2000, 61 orphan drugs have been brought to market. One-third of these were granted their marketing authorisations based on non-comparative clinical research. Certain orphan drugs for extramural use will be transferred to the performance-based hospital financing system within the next few years. Unapproved orphan drugs are generally not reimbursed. In so-called compassionate use programmes, unauthorised orphan drugs can still become available to patients who do not participate in clinical trials. Compassionate use drugs are made available by the manufacturer.
| Translated title of the contribution | Orphan drugs: availability, reliability and reimbursement |
|---|---|
| Original language | Dutch |
| Pages (from-to) | A4252 |
| Journal | Nederlands Tijdschrift voor Geneeskunde |
| Volume | 156 |
| Issue number | 17 |
| Publication status | Published - 2012 |
Keywords
- Chronic Disease
- Compassionate Use Trials
- European Union
- Evidence-Based Medicine
- Humans
- Insurance, Health, Reimbursement
- Legislation, Drug
- Orphan Drug Production
- Rare Diseases