Does orphan drug legislation really answer the needs of patients?

Treatment with imiglucerase might cost as much as US$400000 per year for an adult patient.10 Although Gaucher's disease affects fewer than 20000 patients in the USA,11 Genzyme reportedly received more than $800 million in revenue in 2004 from this product alone.12 Some would like to see a cap p...

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Bibliographic Details
Published in:The Lancet (British edition) 2008-06, Vol.371 (9629), p.2041-2044
Main Authors: Haffner, Marlene E, Dr, Torrent-Farnell, Josep, MD, Maher, Paul D, MD
Format: Article
Language:English
Subjects:
Biotechnology
European Union
Health Services Needs and Demand
Humans
Immunogenicity
Internal Medicine
Legislation
Marketing
Metabolic disorders
Motivation
Orphan Drug Production - economics
Orphan Drug Production - legislation & jurisprudence
Pharmaceutical industry
Product development
Rare Diseases - drug therapy
United States
Urea
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Summary:Treatment with imiglucerase might cost as much as US$400000 per year for an adult patient.10 Although Gaucher's disease affects fewer than 20000 patients in the USA,11 Genzyme reportedly received more than $800 million in revenue in 2004 from this product alone.12 Some would like to see a cap placed on revenues from orphan drugs, shortening of exclusivity provisions, or review of exclusivity provisions when profitable.13 Orphan blockbuster drugs such as epoetin alfa (Epogen) and recombinant human-growth hormone (Genotropin, Humatrope, Nutropin) have been criticised because much of their post-market revenue has come from off-label use. Pegademase was the first instance of an enzyme-replacement therapy for a metabolic disease.34 After pegademase, five enzyme-replacement therapies were developed to treat Gaucher's disease, Fabry's disease, and enzyme deficiencies of the urea cycle.35 Pegademase also involved the first use of a polyethylene glycol delivery system to increase the half-life of a drug and decrease immunogenicity.22 This approach has had more widespread applicability in Pegasys, a pegylated interferon subsequently approved for the treatment of hepatitis C.36 From monoclonal antibodies such as rituximab, to conjugated monoclonal antibodies such as tositumomab, to small molecules such as imatimb mesylate, it is apparent that orphan legislation is not just supporting me-too products derived from advances in more prevalent disorders, but rather development of orphan products has been and is part of the discovery of innovative treatments.
ISSN:0140-6736
1474-547X
DOI:10.1016/S0140-6736(08)60873-9