title: Navigating through orphan medicinal product regulations in EU and US--similarities and differences.
abstract: Rare diseases as the name suggests are the diseases which occur in a very small population due to which the development of medicinal products for these diseases is sidelined as it is anticipated that the cost of development will never be recovered from the sales. It has been estimated by National Institute of Health (NIH) that globally around 7000 rare diseases are there, many of which are of genetic origin. This paper aims to analyze the basic similarities and differences between the rules and regulations put forth by regulatory agencies of US and EU for development of medicinal products for rare diseases, also called orphan medicinal products. The basic purpose was to carve out the loopholes as well as positive aspects of each of these acts and regulations so as to have a clear understanding on the subject. It was to understand that how these legal instruments have stimulated the growth of the drug products for rare diseases and what other things can be done in order to achieve a better impact. This article also provides an overview of the various incentives offered as well as challenges and hurdles faced by each of these regulatory agencies while implementing these regulations.
authors.affiliation: Glenmark Pharmaceuticals Ltd., Glenmark House, HDO - Corporate Building, BD Sawant Marg, Chakala, Andheri (E), Mumbai 400 099, Maharashtra, India. Electronic address: jyoti.tiwari@glenmarkpharma.com.
authors.forename: Jyoti
authors.initials: J
authors.lastname: Tiwari
mesh_terms: D005062:European Union; D006077:Government Agencies / Q000331:legislation & jurisprudence; D006801:Humans; D007880:Legislation, Drug; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence*; D035583:Rare Diseases / Q000188:drug therapy; D014481:United States; D014486:United States Food and Drug Administration / Q000331:legislation & jurisprudence
keywords: Committee for Orphan Medicinal Products; Office of Orphan Products Development; Orphan Drug Act; Orphan designation; Orphan drugs; Rare diseases