Thursday, September 26, 2013

Global Orphan Drugs In Europe Market - Industry Analysis, Size, Share, Growth, Trends and Forecast 2013

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 cost effectiveness?

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.

Orphan Drugs in Europe: Pricing, Reimbursement, Funding & Market Access, 2013 Edition is a fully revised and greatly enlarged version of Justpharmareports best-selling 2008 and 2011 reports, and the author’s sixth major title on the subject.

Click here to download detail report :

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.

Executive Summary

1. Introduction
1.1 Orphan Drugs
1.2 Rare Diseases 
1.3 Business Opportunities with Orphan Drugs

2. EU Orphan Drug Regulation.
2.1 Objectives
2.2 Qualifying Criteria
2.3 Procedure/Timetable
2.4 Incentives
2.4.1 Protocol Assistance 
2.4.2 Marketing Approval Assistance Priority review/fast track assessment Lower regulatory fees
2.4.3 Marketing Exclusivity Issues Relating to similarity
2.5 Orphan Designation Withdrawn 
2.6 Reduced Exclusivity for ‘Sufficiently Profitable’ Orphan Drugs
2.7 Comparison with US and Other Orphan Drug Policies
2.8. Results of EU Regulation
2.8.1 Designation
2.8.2 Marketing Approval 
2.9 Paediatric Regulation
2.9.1 Paediatric Orphan Drugs
2.10 Advanced Therapies Regulation

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