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Orphan Drugs in Europe: Pricing, Reimbursement, Funding & Market Access Issues

DUBLIN, April 22, 2014 /PRNewswire/ --

Research and Markets ( has announced the addition of a new report "Orphan Drugs in Europe: Pricing, Reimbursement, Funding & Market Access Issues" to their offering.

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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

Media Contact: Laura Wood , +353-1-481-1716, [email protected]

SOURCE Research and Markets

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