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.

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  2. Regulatory Science
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European Commission. Good Practice on the Assessment of GMO-Related Aspects in the Context of Clinical Trials with Human Cells Genetically Modified. Version 5. November 2021

Published by European Commission (EC)

Clinical TrialsGMOGood PracticeTranslational Research

Guideline on Clinical trials with medicinal products that contain or consist of GMOs must also comply with applicable requirements under Directive 2001/18/EC on the deliberate release into the environment of genetically modified organisms

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European Medicines Agency. Committee for Medicinal Products for Human Use (CHMP). Guideline on Adjuvants in Vaccines for Human Use. 2005 (EMEA/CHMP/VEG/134716/2004).

Published by The European Medicines Agency Evaluation of Medicines for Human Use ,

Animal ModelsEMAGuidelineNon-Clinical TestingToxicityVaccine

This Guideline addresses the quality, non-clinical and clinical issues arising from the use of new or established adjuvants in vaccines. The applicability of this guideline to established adjuvants will vary on a case-by-case basis. 

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ANIMPACT – An Ethical, Legal and Practical Perspective on the Impact of a New Regulatory Framework for the Scientific Use of Animals on Research and Innovation. Final Publishable Summary Report. 1.OCT.2013 – 31.Dec.2016

Published by CORDIS EU Research Results

DirectiveEthical Use of AnimalsProtection of AnimalsRegulations

The project ANIMPACT – An ethical, legal and practical perspective on the impact of a new regulatory framework for the scientific use of animals on research and innovation have addressed external and internal regulation mechanisms regulating animal research in Europe. 

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European Medicines Agency. Committee for Medicinal Products for Human Use. ICH Guideline S11 on Nonclinical Safety Testing in Support of Development of Paediatric Pharmaceuticals. Step 5. 31 March 2020. EMA/CHMP/ICH/616110/2018.

Published by European Medicines Agency (EMA)

Animal ModelsEMAGuidelineNon-Clinical TrialsPaediatricsPharmaceuticals Toxicity

This guideline recommends an approach for the nonclinical safety evaluation of pharmaceuticals intended for development in paediatric populations. This can include products with prior adult use, as well as products being considered for initial human use in paediatrics 

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European Medicines Agency. Committee for Medicinal Products for Human Use (CHMP). Guideline on the Need for Non-Clinical Testing in Juvenile Animals of Pharmaceuticals for Paediatric Indications. 2008 (EMEA/CHMP/SWP/169215/2005).

Published by European Medicines Agency (EMA)

Animal ModelsEMAGuidelineJuvanile AnimalsNon-Clinical TestingPaediatric Indications

This document provides guidance on the need for, role and timing of studies in juvenile animals in the non-clinical safety evaluation of medicinal products for paediatric use. It is applicable to initial medicinal products applications and also to authorised medicinal products being further developed to include paediatric indications. It outlines potential safety concerns that cannot be adequately assessed in the adult population, in standard non-clinical studies, or in clinical trials. 

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UNESCO. International Declaration on Human Genetic Data, 2003.

Published by Unesco

Genetic DataUNESCO

The purpose of the Declaration is to ensure the respect of human dignity and protection ofhuman  rights  and  fundamental  freedoms  in  the  collection, processing, use  and  storage  ofhuman genetic data, in keeping with the requirements of equality, justice and solidarity

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Commission of the European Communities. COMMISSION DIRECTIVE 2009/120/EC of 14 September 2009 amending Directive 2001/83/EC of the European Parliament and of the Council on the Community code relating to medicinal products for human use as regards advanced therapy medicinal products.

Published by European Commission (EC)

Advanced Therapy RegulationMedicinal Products

This Directive addresses the definitions and detailed scientific and technical requirements for gene therapy medicinal products and somatic cell therapy medicinal products should be updated

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Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004 (Text with EEA relevance)

Published by European Commission (EC)

Advanced Therapy RegulationMedicinal Products

This regulation addresses issues related to the definition and  the development of advanced therapies, such as gene therapy, somatic cell therapy, and tissue engineering

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