Early Acces Support

In this section you will find relevant information for the early access to medicines for Rare Diseases such as the compassionate use of medicines.

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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)

DistributionHuman Tissues and CellsPreservationProcessingDonationQuality and Safety

This 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 Advice

The 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).

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Commission Notice Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another 2022/C 440/02

Published by European Commission (EC)

Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another

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ICH Clinical investigation of medicinal products in the paediatric population E11 (R1)

Published by International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH)ECRIN

Clinical Trials Toolkit

Provides an outline of critical issues in pediatric drug development and approaches to the safe, efficient, and ethical study of medicinal products in the pediatric population. The purpose of this addendum is to complement, provide clarification and current regulatory perspective on topics in pediatric drug development.

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European Medicines Agency Guidance for Applicants seeking scientific advice and protocol assistance

Published by ECRINEuropean Medicines Agency (EMA)

Clinical Trials Toolkit

This guidance document addresses a number of questions that users of the scientific advice or protocol assistance procedures may have. It provides an overview of the procedure to obtain scientific advice or protocol assistance and gives guidance to Applicants in preparing their request. This guidance document also explains the scope and nature of scientific advice and protocol assistance. It will enable Applicants to submit requests which are in line with Scientific Advice Working Party (SAWP) requirements and which can be validated and evaluated quickly and efficiently.

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