Sign in to our library of over $5 million worth of researchclose

Two Quick Steps

* Required Fields

Click Your Industry



Registered Customers

If you have an account with us, please log in.

* Required Fields



Subscribe today
Sign in to our library of over $5 million worth of researchclose

Two Quick Steps

* Required Fields

Click Your Industry



Registered Customers

If you have an account with us, please log in.

* Required Fields



Subscribe today

We deliver Mobile Wallet Market Research and SWOT Analysis from Alternative Energy to Pharmaceuticals and Healthcare Market Research Reports to Digital Media.

Skip to Main Content »

Search Site
Researchers Currently Online:1376

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

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

Double click on above image to view full picture

Zoom Out
Zoom In
Additional Information
Price $2,333.00
Meta Description Orphan Drugs in Europe: Pricing, Reimbursement, Funding & Market Access Issues, 2011 Edition
Publisher Justpharmareports
Date of Report Jan 6, 2011
Quick Overview
Orphan Drugs in Europe: Pricing, Reimbursement, Funding & Market Access Issues, 2011 Edition
Table of Contents
Table of Contents

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
1.4.2.1 Priority review/fast track assessment
1.4.2.2 Lower regulatory fees
1.4.3 Marketing Exclusivity
1.4.3.1 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1.5 Orphan Designation Withdrawn
1.6 Exclusivity Withdrawn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
5.5.3.1 NICE
5.5.3.2 SMC
5.5.3.3 AWMSG
5.5.3.4 Northern Ireland
5.5.3.5 Outcome
5.5.4 Hospital Funding
5.5.4.1 High cost PbR exclusions
5.5.4.2 Pass through payments
5.5.4.3 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

Tables
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

Appendices
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
Sources used for Product Availability & Price Information
References




...