The guidance outlines the main steps to take in preparation for submission and a list of documents required for the main1 types of paediatric submissions
View this resource Bookmark this resourceRegulatory 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.
European Paediatric Translational Research Infrastructure (EPTRI)
Published by EPTRI
Drug DevelopmentpaediatricRegulatory Affairsresearch infrastructureTranslational ResearchThe EPTRI Centralised Services are provided by the EPTRI CMO and dedicated experts. These services include:
*Providing access to paediatric patients’ cohorts
*Providing access to paediatric samples and related data
*Advice on in-vivo / in-silico non-clinical study design
*Advice on in-vivo / in-silico clinical study design
*Advice and document preparation for regulatory and ethics procedures
*Advice on preparing HTA reports for paediatric medicines, diagnostics, and medical devices
*Advice on data and sample sharing preparation
*Support with applications to public and private funding opportunities
DIRECTIVE 2004/9/EC OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 11 February 2004 on the Inspection and Verification of Good Laboratory Practice (GLP).
Published by Official Journal of the European Union
Chemical SubstancesChemical UseDirectiveEthical Use of AnimalsGood Laboratory Practice (GLP)This Directive is to verification of the organisational processes and the conditions under which laboratory studies are planned, performed, recorded and reported for the non-clinical testing, carried out in accordance with the rules and regulations, of all chemicals in order to assess the effect of such products on man, animals and the environment.
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EU Guidance on Preclinical Pharmacological and Toxicological Testing of Vaccines (CPMP/SWP/465/95).
Published by The European Agency for the Evaluation of Medicinal Products, EMA
Animal ModelsEMAGuidelinePreclinical TestingToxicityVaccineThis is a note for guidance on the preclinical evaluation of new vaccine products including combined vaccines, vaccines are those containing antigens not yet described in European Pharmacopoeia monographs or in WHO requirements and vaccines containing live organisms. It indicated the suitable animal models and ethics issues for vaccine preclinical testing.
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WHO Guidelines on Non-Clinical Evaluation of Vaccines (2005)
Published by WHO Technical Report Series
GuidelineNonclinical StudiesToxicityVaccineThis document provides guidance to national regulatory authorities (NRAs) and vaccine manufacturers on the nonclinical evaluation of vaccines by outlining the international regulatory expectations in this area.
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DIRECTIVE 2004/10/EC OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 11 February 2004 on the Harmonisation of Laws, Regulations and Administrative Provisions Relating to the Application of the Principles of Good Laboratory Practice and the Verification of Their Applications for Tests on Chemical Substances (Codified Version)
Published by Official Journal of the European Union
Chemical SubstancesChemical UseDirectiveEthical Use of AnimalsGood Laboratory Practice (GLP)This directive indicates harmonisation of laws, regulations and administrative provisions relating to the application of principles of good laboratory practice and the verification of their applications for tests on chemical substances.
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COMMISSION RECOMMENDATION of 18 June 2007 on Guidelines for the Accommodation and Care of Animals Used for Experimental and Other Scientific Purposes
Published by Official Journal of the European Union
Animal CareAnimal FacilitiesEthical Use of AnimalsGuidelinesProtection of AnimalsRecommendationThis directive is to protect animals used for experimental and other scientific purposes to ensure that any possible pain, suffering, distress or lasting harm inflicted as a consequence of procedures being conducted upon them, shall be kept at a minimum. It provides guidelines for the accommodation and care of animals.
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DIRECTIVE 2010/63/EU OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 22 September 2010 on the Protection of Animals Used for Scientific Purposes?
Published by Official Journal of the European Union
DirectiveEthical Use of AnimalsProtection of AnimalsIt is a directive for acknowledged the importance of the protection and welfare of animals used for scientific purposes at international level.
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European Medicines Agency. EMA’S GUIDE ON ADVANCED THERAPY MEDICINAL PRODUCTS. Non-Clinical Development Flowchart. Version 1.0 – 29 November 2021
Published by EMA
Gene TherapyMedical ProductsNon-Clinical Development SchemeEMA’s guide to help developers of gene therapy medicinal products (GTMPs) and cell-based medicinal products (CBMPs) navigate the most important regulatory requirements during the non-clinical development phase
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European Medicines Agency. Committee for Advanced Therapies (CAT). Procedural Advice on the Provision of Scientific Recommendation on Classification of Advanced Therapy Medicinal Products in Accordance with Article 17 of Regulation (EC) No 1394/2007. EMA/CAT/99623/2009 Rev.2. 5 November 2021
Published by EMA
Comittee for Advanced TherapiesScientific RecommendationThe purpose of this procedure is to allow applicants to clarify whether a given product based on genes, cells or tissues meets the scientific criteria which define ATMPs, in order to address questions of borderline with other areas such as cosmetics or medical devices, which may arise as science develops.
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