The successful introduction of orphan products in Europe remains problematic for the developers, although they can seek to alleviate some of the issues by carefully developing a launch strategy.
The first marketing authorisations for orphan drugs in Europe took place a decade ago. Since then there have been over 60 designated orphan products. These products now account for annual sales of more than $6 billion.
Traditionally the development and marketing of orphan drugs has not been an area of interest for major pharmaceutical companies, reflecting the view that orphan drugs were specialty products with limited market potential thus offering a poor fit for companies based upon the blockbuster model. However, 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. This has seen a number of changes in the orphan drug landscape and further developments are likely, especially from some of those not yet active in the field.
The 302 drugs that are approved for the treatment of 373 orphan indications only over a fraction of the approximately 7,000 rare diseases categorised as rare by the National Institute for Health (US).
The products Glivec and Tracleer show, it is possible for orphan drugs to generate substantial returns in Europe clearly demonstrating the commercial viability of Europe as a market for orphan drugs.
Orphan Drug Markets in Europe - Key developments and the strategies of major pharmaceutical companies is a unique report from URCH Publishing that offers the reader a comprehensive overview of the state of the orphan medicines market in Europe. 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.
Some key findings from the report include:
Within Europe sales of approved orphan products account for approximately $6bilion.
Novartis’ Glivec (imatinib mesylate) sales are over $1.5 billion.
Since January 2000 there has been a steady increase in applications for orphan designation with the Committee for Orphan Medicinal Products (COMP) averaging ten positive recommendations per month.
Five companies generated major revenues from the marketing of orphan drug products in 2010, four primarily by supplying biological products while just Actelion supplied small molecule therapeutics.
Big pharma has started to take orphan drugs seriously and a number have formed dedicated business units to explore opportunities.
Alexion’s Soliris shows that small patient populations are not incompatible with commercial success.
Although central approval covers 30 European countries, it does not necessarily provide for national availability as each national authority has to agree labelling and reimbursement.
The increasing collaboration between the FDA and EMA has led to the adoption of a common application form should facilitate developers’ efforts to exploit orphan drugs on a more international basis.
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