A companion diagnostic is a medical device, often an in vitro device, which provides information that is essential for the safe and effective use of a corresponding drug or biological product.
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.
DIRECTIVE 2004/23/EC OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 31 March 2004 on setting standards of quality and safety for the donation, procurement, testing, processing, preservation, storage and distribution of human tissues and cells.
Published by European Commission (EC)
Quality and SafetyDonationProcessingPreservationDistributionHuman Tissues and CellsThis Directive sets the standards of quality and safety for the donation, procurement, testing, processing, preservation, storage and distribution of human tissues and cells.
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EMA Innovation Task Force
Published by IRDiRC
Regulatory AffairsEarly Access SupportScientific AdviceEMA Innovation Task ForceEarly Stage AdviceThe ITF is a multidisciplinary EMA group that includes scientific, regulatory and legal competences. It was set up to ensure coordination across the Agency and to provide a forum for early dialogue with applicants on innovative aspects in medicines development. Area of interest: innovative therapeutics and techniques, innovative development methods, borderline products.
From the first request by the applicant, the procedure lasts approximately 8-10 weeks (ITF Secretariats confirms eligibility criteria approx. within 2 weeks; when confirmed, then approx. 2 weeks about experts’ involvement + meeting date after 4-6 weeks).
a list of 1415 rare diseases that are related to EMMA strains. Please use the search bar to search for rare diseases, gene name, ORPHAcode or EMMA strain.
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European Orphan Drug Designation
Published by IRDiRC
Regulatory AffairsEarly Access SupportOrphan Drug DesignationEU orphan legislation offers important incentives to encourage the development of medicinal products for rare diseases and the success of the legislation has been demonstrate
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Guidelines 04/2021 on codes of conduct as tools for transfers
Published by European Data Protection Board English
General Data Protection RegulationGPDRInternational Transfers of DataThe aim of these guidelines is to specify the application of Article 40-3 of the GDPR relating to codes of conduct as appropriate safeguards for transfers of personal data to third countries in accordance with Article 46-2-e) of the GDPR.
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Guidelines 07/2020 on the concepts of controller and
processor in the GDPR
Published by European Data Protection Board
General Data Protection RegulationGPDRController ProcessorThe main aim is to clarify the meaning of the concepts and to clarify the different roles and the distribution of responsibilities between these actors.
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Quality, preclinical and clinical aspects of Gene Therapy medicinal products
Published by EMA
Advanced TherapyGene TherapyCell TherapyNon-ClinicalQualityGenetically Modified Cellsomatic cellclinicalGuide for the development and evaluation of Gene Therapy Medicinal Products (GTMPs) intended for use in humans and presented for Marketing Authorisation Application (MAA). Its focus is on the quality, Safety and efficacy requirements of GTMPs.
View this resource Bookmark this resourceThe Vienna Drosophila Resource Center (VDRC) is part of the Vienna Biocenter Core Facilities (VBCF), a publicly funded, non profit research infrastructure.
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WHO Guidelines on Non-Clinical Evaluation of Vaccines (2005)
Published by WHO Technical Report Series
GuidelineVaccineNonclinical StudiesToxicityThis 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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