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Orphan Drugs in Europe: Pricing, Reimbursement, Funding and Market Access Issues, 2013 Edition

Published: Jun 2013 | No Of Pages: 208 | Published By: JustPharmaReports.com
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.
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2.5 Orphan Designation Withdrawn 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
3. Issues for Debate
3.1 Concerns of Payers
3.1.1 Inadequate Premarketing Data
3.1.2 Growing Numbers of Orphan Drugs
3.1.3 Old Medicines Repurposed as Orphan Drugs
3.1.4 High Prices/Costs of Orphan Drugs 
3.1.5 Funding Needs Often Life-long with Cost Increases Over Time
3.1.6 Orphan Drugs are Rarely Cost-effective
3.1.7 Clustering of Treatment for the Same Rare Disease
3.1.8 Indication Creep
3.1.9 Extended Marketing Exclusivity 
3.1.10 Expensive Brands Replace Much Cheaper Compounded Forms
3.1.11 Entry by Major Pharmaceutical Companies
3.1.12 Orphan Drug Regulation Not Fair Balance
3.2 Response to Criticism by Industry
3.3 Value Assessment 
3.4 Cost Forecast to 2020
4. Assisting the Market Access Process
4.1 Role of Patient Advocacies
4.2 Use of Patient Registries
4.3. Pre-approval Access
4.3.1 Compassionate Use
4.3.2 Named/Individual Patient Supply
4.4 Managed Entry Agreements
4.5 Off-label Use
5. Availability and Prices of Orphan Drugs
5.1 Current Access Situation 
5.2 Channels of Distribution
5.3 Prices of Orphan Drugs
5.4 Factors Influencing Uptake
6. Situation in EU-5
6.1 France
6.1.1 Compassionate Use
6.1.2 Pricing & Reimbursement 
6.1.3 Health Economic Considerations 
6.1.4 Managed Entry Agreements
6.1.5 Hospital Funding
6.1.6 Special Incentives for Orphan Drugs
6.1.7 P&R Results with Orphan Drugs
6.1.8 Market Access Situation
6.2 Germany
6.2.1 Compassionate Use
6.2.2 Pricing & Reimbursement
6.2.3 Health Economic Considerations
6.2.4 Hospital Funding
6.2.5 Special Incentives for Orphan Drugs
6.2.6 P&R Results with Orphan Drugs Community market Hospital market
6.2.7 Market Access Situation 
6.3 Italy 
6.3.1 Compassionate Use
6.3.2 Pricing & Reimbursement
6.3.3. Health Economic Considerations
6.3.4 National Register of Orphan Drugs
6.3.5 Managed Entry Agreements
6.3.6 Special Incentives for Orphan Drugs 
6.3.7 Market Access Situation
6.4 Spain
6.4.1 Compassionate Use
6.4.2 Pricing & Reimbursement
6.4.3 Health Economic Considerations
6.4.4 Managed Entry Agreements
6.4.5 Hospital Funding
6.4.6 Special Incentives for Orphan Drugs
6.4.7 Market Access Situation
6.5 United Kingdom
6.5.1 Compassionate Use
6.5.2 Pricing & Reimbursement
6.5.3 Health Economic Considerations NICE SMC AWMSG Northern Ireland
6.5.4 Managed Entry Agreements
6.5.5 Outcome of HTA
6.5.6 Hospital Funding High cost PbR exclusions Pass through payments Specialist commissioning Devolved administrations
6.5.7 Special Incentives for Orphan Drugs
6.5.8 Market Access Situation
7. Situation in Other European Countries
7.1 Austria
7.2 Belgium
7.2.1 Compassionate Use 
7.2.2 Pricing & Reimbursement
7.2.3 Managed Entry Agreements
7.2.4 Special Orphan Incentives
7.2.5 Market Access Situation
7.3 Bulgaria
7.4 Croatia
7.5 Czech Republic
7.6 Denmark
7.7 Greece
7.8 Hungary
7.9 Ireland
7.10 Latvia
7.11 Netherlands
7.11.1 Pricing & Reimbursement
7.11.2 Special Orphan Incentives
7.11.3 Funding Provisions
7.11.4 Market Access Situation
7.12 Norway
7.13 Poland
7.14 Portugal
7.15 Romania
7.16 Russia
7.17 Serbia
7.18 Slovakia
7.19 Sweden
7.19.1 Pricing & Reimbursement
7.19.2 Managed Entry Agreements
7.19.3 Market Access Situation
7.20 Switzerland
8. Opportunities and Challenges from Personalised Medicine
8.1 Glivec and other TKIs
8.2 Kalydeco
8.3 Glybera
9. EU Initiatives
9.1 Community Action Programmes
9.2 Orphanet
9.3 Pharmaceutical Forum
9.4 Community Framework Programmes
9.5 EU Committee of Experts on Rare Diseases
9.6 National Plans
9.8 Registries
9.9 Health Technology Assessment
9.10 Collaborative Efforts to Improve Orphan Drug Access
9.10.1 MEDEV
9.10.2 CAVOD 
9.10.3 CAVOMP 
9.10.4 Mechanism of Co-ordinated Access to Orphan Medicinal Products
9.11 Differential Pricing 
9.12 Directive on Cross-border Healthcare
10. Future Prospects
10.1 Orphan Drug Regulation
10.2 Utilitarianism vs Rule of Rescue
10.3 Evidence Burden
10.4 Prices
10.5 Market Access
10.6. Competition from Generic and Biosimilar Versions
10.7 Success of EU-wide initiatives
Case Studies
Refusal of authorisation for Elelyso
Commission’s decision on Orphacol annulled by European Court
Data file for approval of Carbaglu 
Ilaris in gouty arthritis
Compilation of patient costs by AKU Society
Early access programme for Shire’s Elaprase
Early access programme for Gentium’s defibrotide
AMNOG assessment of Esbriet in Germany
Payment by results agreement for Esbriet in Italy
UK ministers reject AGNSS advice to fund Soliris in aHUS
OEP’s negotiating stance with CML drugs in Hungary
Coverage with evidence development for Duodopa in Sweden
How costly is treatment for chronic myeloid leukaemia?
Kalydeco in cystic fibrosis with G551D mutation 
Glybera, the pioneering gene therapy
1.1 World’s top revenue generating orphan drugs
2.1 Recent history of EMA fee reductions for designated orphan drugs
2.2 Examples of criteria for orphan designation
2.3 Overview of EU orphan drug designation procedure, 2000-2012
2.4 Distribution of EU orphan drug designation by disease prevalence
2.5 Breakdown of approved orphan drugs by therapeutic category
2.6 Breakdown of approved orphan drugs by MA holder
2.7 US orphan drug designations and approvals, 2000-2012
2.8 Orphan drugs with EU marketing authorisation by INN
2.9 Orphan drugs with EU marketing authorisation by brand name
3.1 Examples of repositioned orphan drugs
3.2 Common disease/rare disease price comparison for Belgium 
3.3 Previously available therapeutic alternatives for orphan drugs
3.4 Multiple treatment choices for some rare diseases
3.5 Examples of new orphan indications for previous orphan drugs
3.6 Cost comparison of using commercial and compounded forms
3.7 Proposed criteria for evaluation of orphan drugs
4.1 Umbrella rare disease patient groups at national level
4.2 Leading social media influencers in orphan drugs
4.3 Comparison of registries and RCTs
4.4 Number of rare disease registries by country
4.5 Rare disease registries by medical speciality
465 Early access programme check list
4.7 Timescales for five compassionate use programmes 
4.8 Strategic considerations on whether to charge for pre-approval supply
4.9 Scope of managed entry agreements by country
4.10 Drugs commonly used off-label in rare disease
5.1 Public funding situation with 64 orphan drugs in 15 countries, July 2013
5.2 Principal distribution channels for orphan drugs by country
5.3 Public prices of orphan drugs in EU-5, July 2013
5.4 Examples of hospital prices actually paid in Italy
5.5 Estimated MSPs for 32 orphan drugs in 8 countries, July 2013
6.1 Types of ATU in France 
6.2 ASMR scores/Prescrire evaluation for orphan drugs in France 
6.3 Changes in MSP with Tracleer in France since launch
6.4 Reimbursement status of orphan drugs in France, July 2013
6.5 Results of AMNOG benefit assessment & discount negotiations
6.6 Orphan drugs on ZE and NUB lists in Germany, 2013
6.7 ZE rates by dose for Glivec in Germany, 2013
6.8 ZE rates by dose for Yondelis in Germany, 2013
6.9 Examples of orphan drugs subject to PI competition in Germany
6.10 Cost to NHS of NICE-approved orphan drugs
6.11 Key steps in former AGNSS review process
6.12 Orphan drugs not recommended at NHS expense in parts of UK
6.13 High cost orphan drug exclusions from PbR in England, 2013-14
7.1 Expenditure of Austrian social insurance on orphan drugs
7.2 Reimbursable orphan drugs in Belgium
7.3 Publicly-funded orphan drugs in Bulgaria
7.4 Orphan drugs and similar products available in Greece
7.5 Results of rapid HTA review of orphan drugs in Ireland
7.6 Costliest orphan drugs in the Netherlands
7.7 Intramural and extramural orphan drugs reimbursed in Netherlands
7.8 Examples of rare diseases recognised in Russia
7.9 Coverage of ‘seven nosologies’ programme in Russia
7.10 Swedish cost effectiveness estimates for reimbursed orphan drugs
7.11 Orphan drug sales in Sweden, 2001-2011
8.1 Change in estimated MSP for Glivec 100mg, 2008-2013
8.2 Top-20 indications for EU orphan drug designation
9.1 EU-funded rare disease projects by country
9.2 EU transparent value framework for assessing new orphan drugs
9.3 Elements of the MoCA-OMP pilot
2.1 EU orphan drug marketing approvals by year
3.1 Change in annulised cost of ultra-orphan drugs by age (0-5 years)
3.2 Change in annulised cost of ultra-orphan drugs by age (0-40 years)
5.1 Total number of orphan drugs launched by country, 2001-09
5.2 Sales of orphan drugs per 100,000 inhabitants by country 
5.3 Share of orphan drugs in total market sales by country
5.4 CAVOMP information flow
1. Cross regional comparison of orphan drug policies
2. Approved medicines for rare diseases without EU orphan designation
3. EU designated orphan drugs with centralised marketing approval
4. Cross regional comparison of orphan drug MSPs 
Glossary of Abbreviations and Acronyms
Sources used for Product Availability & Price Information
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