Market Access for Orphan Drugs: assessing the global landscape

Maximum Purchase:
1 unit
Publication Date:
Decemeber 2013
Adding to cart… The item has been added

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