Payers are becoming increasingly strict. Four of the first seven orphan drugs to enter Germany's AMNOG procedure were considered to bring only minor additional benefit and for two others the benefit was deemed non-quantifiable. Discounts were required for all. Re-evaluation by the Transparency Commission in France has seen both SMR and ASMR scores downgraded with resultant price cuts. Half of all orphan drugs evaluated have been rejected by one or more HTA bodies in the UK.
Manufacturers of treatments for Fabry and Pompe diseases are currently negotiating discounts in the Netherlands to prevent delisting after results from a four-year conditional reimbursement PMS period disappointed. On a more positive note, September 2013 saw the start of the first pilot pan-EU assessment under the Mechanism of Co-ordinated Access to Orphan Medicinal Products (MoCA-OMP) initiative.
Updated to autumn 2013, it includes:
- Availability, price and reimbursement status for all 64 EU-designated orphan drugs as of June 2013 with centralised marketing authorisations granted prior to end-2012.
- How orphan drug policies differ across Europe. Expanded sections on each EU-5 country, Belgium, Netherlands and Sweden. 25 countries covered in total.
- Both hospital and out-of-hospital market segments.
- How reimbursement systems treat orphan drugs. High cost' funding systems explained.
- Impact of health technology assessment.
- How managed entry agreements and patient registries can help bridge the data gap.
- Key role of patient advocacy groups.
- 15 case studies.
- The potential for European collaboration to accelerate market access.
Key Topics Covered:
2. EU Orphan Drug Regulation
3. Issues for Debate
4. Assisting the Market Access Process
5. Availability and Prices of Orphan Drugs
6. Situation in EU-5
7. Situation in Other European Countries
8. Opportunities and Challenges from Personalised Medicine
9. EU Initiatives
10. Future Prospects
For more information visit http://www.researchandmarkets.com/research/w9xx7c/orphan_drugs_in
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