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Market Access for Orphan Drugs: assessing the global landscape

  • December 2013
  • -
  • Firstword Pharma
  • -
  • 100 pages




Their development costs can be high and the market is relative small, yet orphan drugs represent a significant contribution to public health—and the pharmaceutical market.

In fact, analysts predict that orphan drugs will account for 15 percent of the global pharmaceutical market by 2018. Yet getting there will not be easy. While governments offer incentives for development, payers must also balance the needs of the majority with the high cost of orphan drugs. International and regional regulations are also hurdles to be overcome, despite harmonisations efforts by agencies.

In Market Access for Orphan Drugs: assessing the global landscape, FirstWord goes around the world to lay bare the market access issues affecting orphan drug developers in the US, Asia and Europe. Based on thorough research and European national drug policies translated specifically for FirstWord, the report reviews the challenges, offers insight into strategies being used and includes invaluable comparative tables. Filled with information on trends, patient access schemes and the role of patient organisations, the report is a must-read for orphan drug developers and manufacturers.








Key Report Features of Market Access for Orphan Drugs: assessing the global landscape include:


Comparative tables for orphan drug polices and incentives in the major markets
A look at the role of patient organisations and registries in orphan drug development
Pricing and reimbursement information for key markets
Overview of orphan drug trends by year, company and therapeutic focus





Key Benefits


Developing and marketing orphan drugs poses several challenges. Through Market Access for Orphan Drugs: assessing the global landscape, you will:


Understand the regulatory and policy frameworks that exist in key markets
Gain insight into the incentives available to orphan drug developers
Appreciate the role of patient groups as potential collaborators in new therapy development
Access information relating to which companies are successfully operating in the orphan drug arena



Getting Your Orphan Drug to Market answers key questions:


Which countries have orphan drug-specific regulations and how are they harmonised?
What incentives are offered to European and American orphan drug developers?
How does orphan drug designationvary from market to market?
How does pricing and reimbursement differ between the general drug market and orphan drugs?








Key facts

By 2020, the recently-formed International Rare Disease Research Consortium aims to be able to diagnose most rare diseases and to have developed 200 new therapies
Biomarkers and improved clinical diagnostics are increasing the possibility of developing targeted rare disease therapies
Currently, there are 6,000 to 8,000 rare diseases, with genetic research identifying more
Although the prevalence of rare disease is extremely low, they affect 30 million people in the EU


Content Highlights


As the orphan drug industry faces new opportunities and challenges, you will discover:

The importance of patient groups in both advocacy and collaborations
How regulations and incentives vary from country to country
The concerted efforts by regulatory agencies to harmonize certain aspects of orphan drug regulations in order to ease sponsors’ journey through requirements








Table Of Contents

Market Access for Orphan Drugs: assessing the global landscape
1.Executive summary
2.How rare is an orphan disease?
3.Orphan drug policy and regulation
3.1.Regulation in the US
3.2.Regulation in the EU
3.3.International harmonisation
3.4.EU national rare disease policies
3.4.1.France
3.4.2.First national rare disease plan
3.4.3.Second national plan for rare diseases
3.4.4.Cancer plan 2009-2013
3.4.5.Orphanet
3.5.Germany
3.6.Italy
3.7.Spain
3.8.Uk
4.Orphan drug policy and regulation in selected countries around the world
4.1.Japan
4.2.Australia
4.3.Taiwan
4.4Canada
4.4.1.Special Access Program
4.4.2.Reforms announced, 2012
4.5.Brazil
5.Incentives for orphan drug development
5.1.Incentives in the US
5.1.1.Market exclusivity
5.1.2.Grants for orphan drug development
5.1.3.Tax credits and fee exemption
5.1.4.Accelerated approval processes
5.2.Incentives in the EU
5.2.1.Market exclusivity
5.2.2.Protocol assistance
5.2.3.Fee reductions
5.2.4.Tax incentives
5.2.5.EU-funded research
6.Finding the right patients for clinical trials
Patient organisations
6.1.Patient registries
6.2.Biomarkers and companion diagnostics
6.3.Pricing and reimbursement
7.Pricing and reimbursement in the EU
7.1.France
7.1.1.Drug pricing
7.1.2.Orphan drug reimbursement
7.2.Germany
7.2.1.Drug pricing
7.2.2.Orphan drug reimbursement
7.3.Italy
7.3.1.Drug pricing
7.3.2.Orphan drug reimbursement
7.4.Spain
7.4.1.Drug pricing
7.4.2.Orphan drug reimbursement
7.5.UK
7.5.1.Drug pricing
7.5.2.Orphan drug reimbursement
7.6.Orphan Drug reimbursement in the US
8.Availability and access to orphan drugs
8.1.Patient access schemes for specific treatments
8.2.Cetuximab and sunitinib
8.3.Ranibizumab
8.4.Eculizumab
8.5.Egalsidase alfa and egasidase beta
8.6.Alglucosidase alfa
8.7.Access to medicines for rare diseases in the US
8.8.Access to medicines for rare diseases in the EU
8.9.France
8.10.Germany
8.11.Italy
8.12.Spain
8.13.UK
9.Trends in orphan drug development
9.1Orphan drugs in the US
9.2.Orphan drugs in the EU
10.Orphan drug commercial success stories
11.Conclusions

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