The definition and acceptability of an orphan condition is pivotal for the assessment of European orphan medicinal product designation applications, and consequently the eligibility for incentives. Here, based on the experiences of the Committee for Orphan Medicinal Products, we discuss how to define orphan conditions in the context of the European regulatory framework.
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References
European Commission. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on Orphan Medicinal Products (Official Journal of the European Communities, 1999).
European Commission. Commission Notice on the Application of Articles 3,5 and 7 of Regulation (EC) No 141/2000 on Orphan Medicinal Products (EC, 2016).
European Commission. Guideline on the Format and Content of Applications for Designation as Orphan Medicinal Products and on the Transfer of Designations from One Sponsor to Another, 27.03.2014 (EC, 2014).
Ayme, S. et al. Rare diseases in ICD11: making rare diseases visible in health information systems through appropriate coding. Orphanet J. Rare Dis. 10, 35 (2015).
Hunter, N. L., Rao, R. G. & Sherman, R. E. Flexibility in the FDA approach to orphan drug development. Nat. Rev. Drug Discov. 16, 737–738 (2017).
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Supplementary Figure
Flow chart for designation of an orphan condition in the European Union
Supplementary Table
Minutes of COMP discussions on accepted and unaccepted orphan conditions
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O’Connor, D.J., Sheean, M.E., Hofer, M.P. et al. Defining orphan conditions in the context of the European orphan regulation: challenges and evolution. Nat Rev Drug Discov 18, 479–480 (2019). https://doi.org/10.1038/nrd.2018.128
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DOI: https://doi.org/10.1038/nrd.2018.128
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