09 December 2011
The number of medicines designated and marketed with an orphan status will grow steadily in Europe says a new report. Orphan Drug Markets in Europe - Key developments and the strategies of major pharmaceutical companies published by URCH Publishing, a business information provider, says that the commercial success of many orphans over the last ten years is boosting interest from Big Pharma as their blockbuster model becomes unsustainable. Already a number of major companies have reassessed their strategy with respect to orphan drugs and five of the top ten have made explicit commitments to the development of such drugs.
“Developing treatments for some of these diseases offers pharmaceutical companies the chance of improving their corporate images, which have become tarnished in recent years, as well as offering commercial opportunities that can help sustain companies’ revenues,” says Dr Peter Norman, the analyst and author of the report. “Recently we have seen GSK, Pfizer and Sanofi beef-up their rare disease research though acquisitions and collaborations,” notes Dr Norman.
However, the 60 page market report points out that the European orphan drug market presents more challenges to developers than does the smaller US market. Although central approval covers 30 different countries, it does not automatically provide for national availability. Each national authority has to agree labelling and reimbursement which not only leads to delays in products reaching the market but also in orphan products becoming available in most member states. Two surveys by EURODIS have suggested that only in a few countries, including France and Germany, are all (or nearly all) approved orphan products available. Thus any European launch strategy needs careful planning addressing reimbursement issues, potential patient numbers and distribution of patients within the EU. However, for the more prevalent rare diseases, a strategy focusing on early launch in the five major markets remains the preferred option as it is for non-orphan drugs.
In addition the increasing collaboration between the FDA and EMA departments that deal with orphan drugs has led to the adoption of a common application form should facilitate developers’ efforts to exploit orphan drugs on a more international basis. But, what may cause a greater problem in future, will be trying to match the rising cost of orphan drug development against increasing cost-containment in health services which may pose challenges in marketing some new orphan products.
Orphan Drug Markets in Europe - Key developments and the strategies of major pharmaceutical companies is available from URCH Publishing. The 30,000 word report will provide the reader with a first-rate review of orphan drugs in Europe since the introduction of legislation a decade ago. The carefully authored study provides expert insight into the market to date, regulatory challenges and future opportunities concerning orphan drugs. Particular attention is paid to the differences in regulation and market between Europe Japan and the US.