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BioPortfolio | Donald Macarthur | Orphan Drugs in Europe: Pricing, Reimbursement, Funding and Market Access Issues

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

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 panEuropean 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% and 12% of all new marketing applications to the EMEA in 2006 and 2007 respectively. 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 inmarket 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 startups

as well as mediumsized corporations with interest in orphan diseases and their treatment."

LarsUno

Larsson, CEO, Swedish Orphan

Author: Donald Macarthur

First published: April 2008

Length: 105 pages

Price: PDF £1450 / $2295

Format: PDF
Table of Contents
Price: $2,295.00 / €1,535.36

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