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. 2006 Mar;61(3):355-60.
doi: 10.1111/j.1365-2125.2006.02579.x.

Orphan drug development is progressing too slowly

Roberta Joppi  1 , Vittorio Bertele, Silvio Garattini
Affiliations

Orphan drug development is progressing too slowly

Roberta Joppi et al. Br J Clin Pharmacol. 2006 Mar.

Abstract

Aims: To assess the methodological quality of OMP dossiers and to discuss possible reasons for the small number of products licensed.

Methods: Information about orphan drug designation and approval was obtained from the website of the European Commission-Enterprise and Industry DG and from the European Public Assessment Reports.

Results: Out of 255 OMP designations, only 18 were approved (7.1%). Their dossiers often showed methodological limitations such as inappropriate clinical design, lack of active comparator where available and use of surrogate end-points.

Conclusions: The paucity of European incentives for manufacturers and the poor documentation underpinning the applications may have limited the number of new OMP. The over 5000 rare diseases awaiting therapy are an important public health issue.

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Comment in

  • Rare diseases and orphan drugs.
    Aronson JK. Aronson JK. Br J Clin Pharmacol. 2006 Mar;61(3):243-5. doi: 10.1111/j.1365-2125.2006.02617.x. Br J Clin Pharmacol. 2006. PMID: 16487216 Free PMC article. No abstract available.

References

    1. Regulation (EC) 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. Official Journal. 2000;L 18:1–5. 22/1/
    1. Federal Drug Administration. Orphan drug act. [17 March 2005]. Available on the URL http://www.fda.gov/orphan/oda.htm (Last)
    1. Committee for Proprietary Medicinal Products. Note for Guidance on Repeated Dose Toxicity. 2000. [17 March 2005]. Available on the URL http://www.emea.eu.int/pdfs/human/swp/104299en.pdf (Last) July.
    1. Stolk P, Willemen M, Leufkens HG. Utrecht (NL): WHO Expert Committee on the Selection and Use of Essential Medicines, UISP; 2005. Rare essentials? Drugs for rare diseases on the essential medicines list.

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