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 Don Macarthur’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
Please email email@example.com for sample pages of this report. More information about this report can be found at http://www.arrowheadpublishers.com/