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16 December 2014 EMA/452415/2012 Rev. 1 1 Human s Research and Development Support Sponsors applying for orphan for a medicine under Article 3(1) a paragraph 1 of Regulation (EC) No 141/2000 on orphan
16 December 2014 EMA/452415/2012 Rev. 1 1 Human s Research and Development Support Sponsors applying for orphan for a medicine under Article 3(1) a paragraph 1 of Regulation (EC) No 141/2000 on orphan medicinal products are requested to provide authoritative references to demonstrate that condition, for which medicine is intended, does not affect more than 5 in 10,000 people in EU at time application is made. Possible sources include relevant scientific literature and databases. After more than 10 years of implementation of Orphan Regulation Agency has accumulated a considerable amount of data on sources of prevalence of rare diseases from applications for orphan. In most cases those sources are publicly available but not easily accessible. The Agency has decided to make information collected so far publicly available. This will decrease administrative burden for applicants for orphan and thus encourage development of medicines for rare diseases. The information is provided in table below will be updated regularly. The information provided herewith does not replace obligation for sponsor under legislation to establish prevalence (see Regulation (EC) No 141/2000 and Guideline on format and content of applications for as orphan medicinal product, ENTR/6283/00). Sponsors are still obliged to submit an original, up-to-date prevalence calculation supported by data with ir orphan application. In Points to consider on calculation and reporting on prevalence of a condition for orphan, COMP/436/01, Agency provides guidance for sponsors in establishing prevalence for conditions and suggests sources of data, review methods and how to present results. Of note, references cited are not intended to substitute any epidemiological review of prevalence of rare diseases and y have to be assessed by Committee for Orphan Medicinal Products (COMP) in regulatory context of orphan medicinal product. In this sense, it has to be stressed that sometimes conclusions adopted reflect worst case scenarios that are adequate for regulatory purposes. It must also be emphasised that prevalence that sponsor is required to demonstrate is prevalence at time of application for orphan, whereas prevalence can change over time. If you have any comments on table on prevalence sources do not hesitate to contact us on 1 Updated format, footer and links 30 Churchill Place Canary Wharf London E14 5EU United Kingdom Telephone +44 (0) Facsimile +44 (0) Send a question via our website An agency of European Union European s Agency, Reproduction is authorised provided source is acknowledged. Acromegaly 0.6 Publications: Alexander, L., et al., Epidemiology of acromegaly in Newcastle region. Clin. Endocrinol (Oxf), 1980;12(1):71-9 Bengtsson, B.A., et al., Epidemiology and long-term survival in acromegaly. A study of 166 cases diagnosed between 1955 and Acta. Med. 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