Orphan Drugs in Europe

Pricing, Reimbursement, Funding and Market Access Issues

Publication Date	April 2008
Publisher	JustPharmaReports
Product Type	Report
Pages	105
ISBN Number	not applicable
Product Code	JPR00002

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Summary

In order to raise public awareness, 29 February 2008 was dedicated the first European rare disease day ('a rare day for very special people'), but European payers need no reminding of the significance of rare disease treatments, generally known as orphan drugs.

Eight years earlier, EU Regulation 141/2000 was enacted to encourage R&D in rare diseases and increase orphan drug output. By any standard the Regulation has been a major success. Almost 800 designation applications have been made, with 44 orphan medicines from 31 sponsoring companies receiving pan-European marketing approval. An estimated 1.6 million patients with 38 different rare diseases have potentially benefited, and the surge of interest in the area has offered hope to many more. Orphan drugs accounted for 23% of all new marketing applications to the EMEA in 2006 alone.

Rare diseases and their treatments may still be emotive territory, with a strong patient voice and genuine unmet need, but the pendulum is swinging from a guarantee of 'special status' when it comes to public reimbursement to issues of budgetary impact and cost effectiveness - not only because of the high cost of orphan drugs themselves, but the growing number of target diseases and treatments, associated new costs of medical education and diagnosis, and the need for chronic disease management.

Questions answered by this report include:

What is the actual in-market status of all EU designated orphan drugs with marketing authorisations up to the beginning of 2008?
Which drugs are covered by reimbursement, which are not?
Are special funds available for orphan drugs?
Where is health technology assessment required?
How large is the European price corridor for orphan drugs?
Is there a relationship between target patient population and price?
How do orphan drug policies differ across the leading European countries?
Do payers view all orphan drugs as unique?

Content

Executive Summary

1. Introduction
1.1 What are Orphan Drugs?
1.2 What are the P&R Issues 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 Information
       2.4.2 R&D
       2.4.3 Protocol Assistance
       2.4.4 Marketing Approval Assistance
           2.4.4.1 Priority review/fast track
           2.4.4.2 Lower regulatory fees
       2.4.5 Market Exclusivity
           2.4.5.1 Exclusivity withdrawn?
   2.5 Comparison with US Orphan Drug Act
   2.6 Results
       2.6.1 Designation
       2.6.2 Marketing Approval
       2.6.3 Medical Benefit

3. Compassionate Use

4. Paediatric Regulation

5. Market Access

6. Pricing and Sales
   6.1 Public Prices
   6.2 Manufacturer Selling Prices
   6.3 Sales

7. Distribution

8. Advantages and Disadvantages of Orphan Designation
   8.1 Pros
       8.1.1 Incentives
   8.2 Cons
       8.2.1 Most National Incentives Still Apply
       8.2.2 Some Help from EU Still Available
       8.2.3 Limitation to Market Exclusivity
       8.2.4 Initial fee Waivers May Not be Permanent
       8.2.5 Information Disclosure to Authorities
       8.2.6 Information Disclosure to Competitors
       8.2.7 No Regulatory Flexibility

9. Future EU Developments
9.1 New Communication
9.2 Common HTA, Single EU Price for Orphan Drugs?

10. National Situation
10.1 EU-5
   10.1.1 Germany
       10.1.1.1 Orphan incentives
       10.1.1.2 Patient access
       10.1.1.3 Funding provisions
       10.1.1.4 Impact on P&R
       10.1.1.5 Health economic considerations
   10.1.2 France
       10.1.2.1 Orphan incentives
       10.1.2.2 Patient access
       10.1 2.3 Funding provisions
10.1.2.4 Impact on P&R
10.1.2.5 Health economic considerations
10.1.3 UK
   10.1.3.1 Orphan incentives
   10.1.3.2 Patient access
   10.1.3.3 Funding provisions
   10.1.3.4 Impact on P&R
   10.1.3.5 Health economic considerations
10.1.4 Italy
   10.1.4.1 Orphan incentives
   10.1.4.2 Patient access
   10.1.4.3 Funding provisions
10.1.4.4 Impact on P&R
10.1.4.5 Health economic considerations
10.1.5 Spain
   10.1.5.1 Orphan incentives
   10.1.5.2 Patient access
   10.1.5.3 Funding provisions
   10.1.5.4 Impact on P&R
   10.1.5.5 Health economic considerations
10.2 Other European Countries
   10.2.1 Austria
   10.2.3 Belgium
   10.2.4 Bulgaria
   10.2.5 Czech Republic
   10.2.6 Ireland
   10.2.7 Netherlands
   10.2.8 Poland
   10.2.9 Portugal
   10.2.10 Slovak Republic
    10.2.11 Sweden
   10.2.12 Other Nordic Countries
   10.2.13 Switzerland

11. Pricing Case Studies
   11.1 Chronic Myeloid Leukaemia
   11.2 Pompe Disease
   11.3 Pulmonary Arterial Hypertension

12. Conclusions

13. Strategic Implications

List of tables
1.1 Level of evidence in selected orphan drug appraisal dossiers
2.1 Examples of orphan medicines predating the orphan drug Regulation
2.2 Advice to orphan drug sponsors from EMEA
2.3 Orphan drug regulations compared
2.4 Overview of EU procedures for orphan drug designation and approval
2.5 EU designated orphan drugs with centralised marketing authorisations
2.6 National marketing authorisations granted for EU-designated orphan drugs
2.7 Main therapeutic classification of approved orphan drugs
2.8 EU designated orphan drugs with previous non-orphan use
4.1 Marketed orphan drugs with supporting data in paediatric populations
5.1 European availability of orphan drugs by product
5.2 European availability of orphan drugs by country
5.3 First launch quarters for centrally authorised orphan drugs
5.4 Reimbursement status of Aldurazyme across EU
6.1 Public prices of EU orphan drugs
6.2 Estimated manufacturer selling prices of orphan drugs (EU-5, Swiss & US)
7.1 Main distribution channels for orphan drugs
10.1 ZE rates for Glivec in Germany
10.2 ASMR scores for orphan drugs in France
10.3 Status of orphan drugs in Scotland after SMC review
10.4 Status of orphan drugs in Wales after AWMSG review
10.5 Costs per QALY for ultra orphan drugs in UK
10.6 Certified rare disease centres in Italy
10.7 Treatment costs with selected orphan drugs in Italy
10.8 Reimbursement status of orphan drugs in Belgium
10.9 Actual reimbursed expenditure on orphan drugs in Belgium
10.10 Intramural and extramural orphan drugs reimbursed in the Netherlands
10.11 NFZ points value and MSP of reimbursed orphan drugs in Poland
10.12 Swedish cost effectiveness estimates for orphan drugs
10.13 Swiss orphan drugs with simplified marketing authorisations

List of figures
6.1 Estimated manufacturer selling prices across Europe for Glivec
6.2 Orphan target population vs monthly adult treatment cost