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Orphan Drugs in Europe: Pricing, Reimbursement, Funding and Market Access Issues, 2011 Edition

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?

The orphan drug Regulation 141/2000, resulting in 724 designated products and 62 marketing approvals in the first 10 years, has certainly been one of the most successful legislative measures by the EU. Encouraging development and accelerating regulatory approval are very important, but alone do not ensure orphan drugs are actually available for use. Rare disease patients need timely access to therapies that are fully funded by social health insurance/national health services. Hurdles are found at national, regional and local level. Discover what these are and the strategies employed by successful orphan drug companies.

Orphan Drugs in Europe: Pricing, Reimbursement, Funding & Market Access, 2011 Edition is a fully revised and greatly enlarged version of the best-selling 2008 report, and the author’s fifth major title on orphan drugs.

Updated to May 2011, it includes:

- Availability, price and reimbursement status for all 60 EU-designated orphan drugs with centralised marketing authorisations up to end-2010.
- How orphan drug policies differ across Europe. Expanded sections on each EU-5 country. Situation in 20 countries described in total.
- Key role of special ‘high cost’ funding systems explained.
- Impact of health technology assessment.
- The myths and realities of orphan drug costs.
- How risk sharing schemes and patient registries can help bridge the data gap.
- Ways to provide pre-approval early access.
- 12 case studies.
- The potential for EU collaboration on clinical added value of orphan drugs.
Executive Summary

1. EU Orphan Drug Regulation
1.1 Objectives
1.2 Qualifying Criteria
1.3 Procedure/Timetable
1.4 Incentives
1.4.1 Protocol Assistance
1.4.2 Marketing Approval Assistance Priority review/fast track assessment Lower regulatory fees
1.4.3 Marketing Exclusivity Issues relating to similarity
1.5 Orphan Designation Withdrawn
1.6 Exclusivity Withdrawn
1.7 Comparison with US and Other Orphan Drug Policies
1.8 Results
1.8.1 Designation
1.8.2 Marketing Approval
1.9 Paediatric Regulation
1.9.1 Paediatric Orphan Drugs

2. Concerns Raised by Payers
2.1 ‘There are so many rare diseases’
2.2 ‘A tidal wave of new orphan drugs is coming’
2.3 ‘Funding needs are often life long’
2.4 ‘Clinical data are very limited’
2.4.1 Questions on efficacy
2.4.2 Questions on dosage
2.4.3 Questions on safety
2.5 ‘Patient numbers are uncertain’
2.6 ‘Prices are arbitrary and too high’
2.7 ‘Market exclusivity stifles competition’
2.8 ‘Orphan drugs are not cost effective’
2.9 ‘Funds are diverted from treating more common conditions’
2.10 ‘Another orphan indication soon follows the first’
2.11 ‘Industry pursues a low-risk rare disease strategy’
2.12 ‘Orphan drugs may turn into blockbusters’
2.13 ‘Increased interest in orphan development by Big Pharma’
2.14 ‘Expensive products replace cheaper alternatives’

3. Response by Industry and Patient Advocacy Groups
3.1 No Excessive Pricing
3.2 No Soaring Costs
3.3 No Market Monopoly
3.4 No Flood of New Orphan Drugs
3.5 No Lack of Clinical Trials
3.6 Risk Sharing Schemes
3.6.1 Italy
3.6.2 UK
3.7 No Particular Safety Issues
3.8 Registries Provide Post-marketing Evidence
3.9 Public Support for Orphan Drug Funding

4. Market Access
4.1 Early Access Schemes
4.2 Commercial Distribution
4.3 Orphan Drug Availability
4.4 Orphan Drug Prices
4.5 Eurordis 2010 Survey
4.6 Orphan Drug Utilisation

5. Situation in EU-5
5.1 France
5.1.1 Compassionate Use
5.1.2 Pricing & Reimbursement
5.1.3 Health Economic Considerations
5.1.4 Hospital Funding
5.1.5 Incentives for Orphan Drugs
5.1.6 P&R Results with Orphan Drugs
5.1.7 Market Access Situation
5.1.8 Rare Disease Patient Groups
5.2 Germany
5.2.1 Compassionate Use
5.2.2 Pricing & Reimbursement
5.2.3 Health Economic Considerations
5.2.4 Hospital Funding
5.2.5 Incentives for Orphan Drugs
5.2.6 P&R Results with Orphan Drugs
5.2.7 Market Access Situation
5.2.8 Rare Disease Patient Groups
5.3 Italy
5.3.1 Compassionate Use
5.3.2 Pricing & Reimbursement
5.3.3 Health Economic Considerations
5.3.4 Incentives for Orphan Drugs
5.3.5 Market Access Situation
5.3.6 Rare Disease Patient Groups
5.4 Spain
5.4.1 Compassionate Use
5.4.2 Pricing & Reimbursement
5.4.3 Health Economic Considerations
5.4.4 Hospital Funding
5.4.5 Incentives for Orphan Drugs
5.4.6 Market Access Situation
5.4.7 Rare Disease Patient Groups
5.5 United Kingdom
5.5.1 Compassionate Use
5.5.2 Pricing & Reimbursement
5.5.3 Health Economic Considerations NICE SMC AWMSG Northern Ireland Outcome
5.5.4 Hospital Funding High cost PbR exclusions Pass through payments Specialist commissioning
5.5.5 Incentives for Orphan Drugs
5.5.6 Market Access Situation
5.5.7 Rare Disease Patient Groups

6. Situation in Other European Countries
6.1 Austria
6.2 Belgium
6.2.1 Pricing & Reimbursement
6.2.2 Orphan Incentives
6.2.3 Special Funding Provisions
6.2.4 Market Access Situation
6.3 Bulgaria
6.3.1 Special Funding Provisions
6.3.2. Market Access Situation
6.4 Croatia
6.5 Czech Republic
6.5.1 Pricing & Reimbursement
6.5.2 Market Access Situation
6.6 Denmark
6.7 Greece
6.7.1 Orphan Incentives
6.7.2 Special Funding Provisions
6.8 Hungary
6.9 Ireland
6.10 Netherlands
6.10.1 Pricing & Reimbursement
6.10.2 Orphan Incentives
6.10.3 Special Funding Provisions
6.10.4 Market Access Situation
6.10.5 Proposals for Change
6.11 Poland
6.11.1 Pricing & Reimbursement
6.11.2 Special Funding Provisions
6.11.3 Market Access Situation
6.12 Romania
6.13 Russia
6.14 Sweden
6.14.1 Pricing & Reimbursement
6.14.2 Hospital Funding
6.15 Switzerland

7. EU Initiatives
7.1 Community Action Programmes
7.2 Orphanet
7.3 Pharmaceutical Forum
7.4 Community Framework Programmes
7.5 EU Committee of Experts on Rare Diseases
7.6 National Plans
7.7 Collaborative Efforts to Improve Orphan Drug Access
7.8 Directive on Cross-border Healthcare

8. Conclusions

Case Studies
Co-exclusivity for agalsidases in Fabry disease
C1 esterase inhibitors for hereditary angioedema: what is unique?
Withdrawal of Thelin
Europe’s most expensive orphan drug
Is a price premium for commercial amifampridine justified?
Filling the void in Gaucher Therapy
Patient access schemes with Velcade
Named patient distribution of Ceplene
Second generation tyrosine kinase inhibitors for CML
Re-evaluation of PAH treatments in France
Varying HTA results for Myozyme in UK
Evidence development agreement with Duodopa in Sweden

1.1 Overview of EU orphan drug procedure, 2000-10
1.2 Distribution of positive COMP decisions by therapeutic area
1.3 Marketing authorisations for orphan designated medicines, 2000-10
1.4 Approved treatments for main lysosomal storage disorders
1.5 Distribution of orphan MAs by therapeutic area, 2000-10
1.6 Orphan drugs with marketing approval, by INN
1.7 Orphan drugs with marketing approval, by brand name
2.1 Size of pre-marketing clinical trials with approved orphan drugs
2.2 Rare diseases with the most orphan drug approvals in US
2.3 Previously available therapeutic alternatives for orphan drugs
3.1 Examples of risk-sharing agreements with orphan drugs
3.2 Number of rare disease registries by country
4.1 Some comparisons of CEE countries with Germany
4.2 Strategic considerations in charging for pre-approval sales
4.3 Principal distribution channels for orphan drugs
4.4 EU-5 availability of reimbursed orphan drugs by country
4.5 Public prices of orphan drugs in EU-5
4.6 Maximum MSPs of selected orphan drugs by country
4.7 Eurordis 2010 survey of orphan drugs by country
4.8 No. patients with access to orphan drugs by therapeutic area
5.1 ASMR scores for orphan drugs in France
5.2 Reimbursement status of orphan drugs in France
5.3 Orphan drugs on ZE and NUB lists for hospital payment in Germany
5.4 ZE rates by dosage for Glivec in Germany
5.5 Orphan drugs not recommended for NHS in devolved parts of UK
5.6 High cost orphan drug exclusions from PbR in England
6.1 Reimbursable orphan drugs in Belgium
6.2 Orphan drugs reimbursed in Bulgaria
6.3 Maximum MSPs and reimbursement limits for orphan drugs in Czech Republic
6.4 Intramural and extramural orphan drugs reimbursed in Netherlands
6.5 Orphan drugs reimbursed under therapeutic programmes in Poland
6.6 Coverage by ‘seven nosologies’ programme in Russia
6.7 Swedish cost effectiveness estimates for reimbursed orphan drugs
7.1 Rare diseases with highest numbers of clinical trials in Europe

1. Examples of orphan medicines predating the EU Regulation
2. EU designated orphan drugs with centralised marketing authorisations
3. Cross-national comparison of orphan drug policies

Glossary of Abbreviations and Acronyms
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