This document is the IRIS guide to registration
Preliminary requirements for all IRIS submissions, including
substance and Research Product Identifier registration
Regulatory Science
The information proposed in this section provides key elements on the regulation of medicines for Rare Diseases in human and provides support in the:
Health Technology Assessment,
Drug repurposing,
Preclinical suties,
Early access support,
Clinical Development,
Marketing Authorization,
Ethic and legal issues,
Regulatory Agencies.
EMA_Procedural Advice Appeal Procedure Orphan Designation Review_2020
Published by EMA
AppealMarketing AuthorisationOrphan Medicinal ProductProcedure AdviceThis document describes procedural advice on appeal procedure for orphan
medicinal product designation or review of orphan
designation criteria at the time of marketing authorisation
This document summarises the conference “Medicines for Rare Diseases and Children: Learning from the Past, Looking to the
Future”
Joint evaluation of Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006 on medicinal products for paediatric use and Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products.SWD/2020/0163 final
Published by European Commission (EC)
Orphan Medicinal ProductsFirstly, it assesses the strengths and weaknesses of the two legal instruments, both separately and in combination with each other. It focuses on how they have catered for products for unmet medical needs, taking into account how pharmaceuticals are developed, science advances, and business models change. Secondly, it provides insights into how the various incentives and rewards for which the Regulations provide have been used, along with an analysis of the related financial consequences, both in general and by stakeholder group.
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Committee for Medicinal Products for Human Use (CHMP) Role of pharmacokinetics in the development of medicinal products in the paediatric population 2006 Doc. Ref. EMEA/CHMP/EWP/147013/2004.
Published by European Medicines Agency
AdolescentsChildrenDose FindingExtrapolationInfantsNewbornsPharmacokineticsToddlersThis document provides advice on the use of pharmacokinetic studies in paediatric drug development and on methodological issues concerning pharmacokinetic studies in paediatric patients.
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Committee for Medicinal Products for Human Use (CHMP) and Paediatric Committee (PDCO). Guideline on The Investigation of Medicinal Products In The Term And Preterm Neonate. 2009 Doc. Ref. EMEA/536810/2008.
Published by European Medicines Agency
Adverse EffectsClinical TrialsNeonateThis guideline addresses the considerations and requirements for the design and conduct of clinical trials in premature and term neonates using medicinal products of relevance for the use by this population. It includes background information on the maturation of organs and of body functions.
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ICH E11(R1) guideline on clinical investigation of medicinal products in the pediatric population. EMA/CPMP/ICH/2711/1999. Step 5.
Published by European Medicines Agency
Paediatric FormulationPaediatric Medicinal Product DevelopmentPharmacokineticThis document describes that paediatric drug development has evolved since the original ICH E11 Guideline (2000), requiring consideration of regulatory and scientific advances relevant to pediatric populations. This addendum does not alter the scope of the original guideline which outlines an approach to the safe, efficient, and ethical study of medicinal products in the pediatric population.
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Regulation (EC) 1901/2006 of 12 December 2006 on medicinal products for paediatric use and amending Regulation (EEC) No 1768/92, Directive 2001/20/EC, Directive 2001/83/EC and Regulation (EC) No 726/2004.OJL 378/1, 27.12.2006, as amended by REGULATION (EC) No 1902/2006.
Published by European Parliament and of the Council of the European Union
Medicinal ProductsPaediatric UseThis Regulation lays down rules concerning the development of medicinal products for human use in order to meet the specific therapeutic needs of the paediatric population, without subjecting the paediatric population to unnecessary clinical or other trials and in compliance with Directive 2001/20/EC.
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EMA_Guidance Procedural Advice Orphan Designation_2021
Published by EMA
Orphan Medicinal ProductSponsorThis document describes the procedural advice for orphan medicinal product
designation
Guidance for sponsors
This document is the COMMISSION REGULATION (EC) No 847/2000
of 27 April 2000
laying down the provisions for implementation of the criteria for designation of a medicinal
product as an orphan medicinal product and definitions of the concepts ‘similar medicinal product’
and‘clinical superiority’
