DUBLIN, April 22, 2014 /PRNewswire/ --
Research and
Markets (http://www.researchandmarkets.com/research/t2f5mf/orphan_drugs_in)
has announced the addition of a new report "Orphan Drugs in
Europe: Pricing, Reimbursement, Funding & Market Access
Issues" to their offering.
(Logo:
http://photos.prnewswire.com/prnh/20130307/600769 )
The cost of treating rare disease in an age when austerity measures
are hitting total healthcare funding across Europe is a highly controversial, even emotive
subject. There is a clear and pressing clinical need, a strong
patient voice and the treatments themselves sometimes offer the
only hope. But is this enough to assure premium prices and special
status in an era of budget limitations, and growing demands for
evidence of clinical and economic value?
Orphan Drugs in Europe: Pricing,
Reimbursement, Funding & Market Access Issues, 2013 Edition, a
new publication from Justpharmareports, finds that if there is
discrimination by payers towards orphan drugs as a group it is
still in a positive way. The products are as diverse as the
diseases they treat and some are coming under greater scrutiny,
however.
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 by
GKV-Spitzenverband 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.
Other payer concerns include the numbers of marketed and pipeline
orphan drugs, the quality of clinical evidence, clustering of
treatments for the same rare disease, uncertainty on patient
numbers and dosage, the high prices being requested, diversion of
funds away from treating more common conditions, repurposed old
medicines, indication creep, displacement of cheaper alternatives,
and a failure to meet cost effectiveness thresholds.
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, the report 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:
Executive Summary
1. Introduction
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/t2f5mf/orphan_drugs_i
Media Contact: Laura Wood , +353-1-481-1716,
press@researchandmarkets.net