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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The Assay Guidance Manual

Published by EATRIS

Research and Drug DevelopmentDrug Discovery GuidelineSmall MoleculeDrug RepurposingTarget Validation Drug DevelopmentNCATSNIH

This manual is a comprehensive resource for early drug discovery, providing guidelines for developing therapeutic hypotheses, validating targets and pathways, and establishing proof of concept criteria. It includes analyses of costs at various stages of drug development. The content encompasses critical decision-making points in New Chemical Entity (NCE) development, exploratory Investigational New Drug (IND) processes, and orphan drug designation. Furthermore, it delves into Drug Repurposing and drug delivery technologies, making it an invaluable guide for professionals initiating new drug discovery and development programs.

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Joint EMA-FDA Scientific Advice

Published by IRDiRC

Regulatory AffairsEarly Access SupportScientific AdviceJoint EMA-FDA Scientific Advice

The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services have a program to provide parallel scientific advice (PSA) to sponsors. The goal of the PSA program is to provide a mechanism for EMA assessors and FDA reviewers to concurrently exchange with sponsors their views on scientific issues during the development phase of new medicinal products (i.e., new human drugs and Biologics).

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Rare diseases, orphan drugs, and their regulation in Asia: Current status and future perspectives

Published by IACMHR Co., Ltd.

Orphan DrugRegulatory Science

this paper describes the current status of the regulation of rare diseases and orphan drugs in Asia and we comparatively analyze the regulation of rare diseases and orphan drugs worldwide in order to examine the challenges to and future perspectives on promoting research on rare diseases and development of orphan drugs in China and other Asian countries.

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ICH: Safety

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

Nonclinical Safety in Paediatric MedicineCarcinogenicity StudyGenotoxicity StudyToxicokinetics and PharmacokineticsRepeat-Dose ToxicityReproductive ToxicologyBiotechnological ProductSafety Pharmacology StudyImmunotoxicology StudyTherapeutic Area-SpecificPhotosafety Evaluation

The European Medicines Agency publishes scientific guidelines on human medicines that are harmonised by the International Council for Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH)

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