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Orphan Drugs in Europe: Pricing, Reimbursement, Funding and Market Access Issues
Just Pharma Reports, April 2008, Pages: 105
In order to raise public awareness, 29 February 2008 was dedicated the first European rare disease day (‘a rare day for very special people’), but European payers need no reminding of the significance of rare disease treatments, generally known as orphan drugs.
Eight years earlier, EU Regulation 141/2000 was enacted to encourage R&D in rare diseases and increase orphan drug output. By any standard the Regulation has been a major success. Almost 800 designation applications have been made, with 44 orphan medicines from 31 sponsoring companies receiving pan-European marketing approval. An estimated 1.6 million patients with 38 different rare diseases have potentially benefited, and the surge of interest in the area has offered hope to many more. Orphan drugs accounted for 23% of all new marketing applications to the EMEA in 2006 alone.
Rare diseases and their treatments may still be emotive territory, with a strong patient voice and genuine unmet need, but the pendulum is swinging from a guarantee of ‘special status’ when it comes to public reimbursement to issues of budgetary impact and cost effectiveness – not only because of the high cost of orphan drugs themselves, but the growing number of target diseases and treatments, associated new costs of medical education and diagnosis, and the need for chronic disease management.
Questions answered by this report include:
- What is the actual in-market status of all EU designated orphan drugs with marketing authorisations up to the beginning of 2008?
- Which drugs are covered by reimbursement, which are not?
- Are special funds available for orphan drugs?
- Where is health technology assessment required?
- How large is the European price corridor for orphan drugs?
- Is there a relationship between target patient population and price?
- How do orphan drug policies differ across the leading European countries?
- Do payers view all orphan drugs as unique?
This is what one highly informed reviewer thought of Donald Macarthur’s earlier report on orphan drugs, published by Scrip Reports in 2000:
“The author’s previous orphan drug report was an extremely useful instrument for Swedish Orphan to fully understand the global opportunities within the orphan disease and orphan drug area, and did serve as a stimulus to get the company started. I am convinced that this latest report equally will serve as a very useful document for academia with interest in drug development, small start-ups as well as medium-sized corporations with interest in orphan diseases and their treatment.”
Lars-Uno Larsson, CEO, Swedish Orphan
About the Author:
Donald Macarthur is an independent analyst, consultant and writer on international pharmaceutical business issues. Issues covered include drug cost containment policy worldwide, pricing and reimbursement, hospital access, European integration and enlargement, wholesale and retail distribution, mail order, homecare, orphan drugs, generics, parallel trade, Rx-to-OTC switching, and several aspects of the Japanese market and industry.
He has written over 50 major reports and also founded, edited and published for four years the world’s first periodical on drug pricing and reimbursement, Pharma Pricing Review..
Formerly with PriceSpective and also the first Secretary General of the European Association of Euro-Pharmaceutical Companies, consultancy clients include the European Federation of Pharmaceutical Industries and Associations - he was the very first consultant ever used by EFPIA - Pharmaceutical Research and Manufacturers of America (PhRMA), the European grouping of national full-line pharmaceutical wholesaling associations (GIRP), the UK’s Office of Fair Trading, other government agencies, and many major multinational biopharmaceutical manufacturers, legal and financial firms. Numerous presentations have been given at international conferences, and he has testified both at a US Senate Committee hearing and at the HHS Secretary’s task force hearing on prescription drug importation. His articles have appeared in many English language pharmaceutical business journals, with some translated into Japanese, German and Turkish.
Qualifying as a pharmacist from the University of London, Mr Macarthur's early career involved community and hospital pharmacy practice, 16 years in development, regulatory affairs and medical department functions in the pharmaceutical industry in the UK (Fisons, 1969-72; Parke-Davis, 1972-75; Roche, 1976-78; Serono, 1978-82; Lundbeck 1982-84), one year in Japan, and four years with PJB Publications (publishers of Scrip).
Product samples
Orphan Drugs in Europe Sample
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