Abstract
Twenty years of orphan regulation in Europe have now elapsed, with almost 2,400 orphan designated medicinal products and more than 190 orphan products authorised in the EU. Alongside the evolution in understanding of rare diseases, considerable regulatory knowledge has also been accumulated regarding the level of evidence that would support inclusion of products into the framework. This article reviews publications and regulatory documents pertaining to orphan medicinal product designation in the EU and discusses the general expectations in submitted applications as reflected in the current regulatory practise. Important elements to recommend granting a European orphan designation are the key considerations of orphan condition, medical plausibility, seriousness, and prevalence, while significant benefit is also assessed when there are authorised medicinal products for the sought indication. This review attempts to clarify the specific concepts currently used in that regard and discusses how the available data can be used to justify the criteria for designation. Moving away from theoretical expectations or assumptions, it stresses that the applications have to be complemented with nosological and epidemiological justifications pertaining to the proposed condition, as well as relevant data in specific non-clinical in vivo models or in affected patients to support inclusion into the orphan scheme.
Highlights
Regulatory Standards in Orphan Medicinal Product Designation in the EUStelios Tsigkos 1*, Segundo Mariz 1, Maria Elzbieta Sheean 1, Kristina Larsson 1, Armando Magrelli 2,3 and Violeta Stoyanova-Beninska 3,4
Article 5 of Regulation (EC) No.141/2000 describes the procedure for orphan designation and removal from the Register of Orphan Medicinal Products [1]
The effort to incentivise development in genuinely rare diseases may raise a concern that non-rare diseases could benefit from the incentives after being subdivided into smaller groups of patients, each falling below the legal definition of rarity
Summary
Stelios Tsigkos 1*, Segundo Mariz 1, Maria Elzbieta Sheean 1, Kristina Larsson 1, Armando Magrelli 2,3 and Violeta Stoyanova-Beninska 3,4. Specialty section: This article was submitted to Regulatory Science, a section of the journal
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