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.

  1. Showing 5 results for:
  2. Regulatory Science
All SubjectsClinical Development

Stakeholders interactions – Patients Affairs Staff

Published by IRDiRC

Research and Drug DevelopmentRegulatory AffairsEarly Access SupportPatient EngagementPatient AdviceFDA Regulatory AdvicePatient Affairs StaffPatient SupportClinical Development

At the FDA, the Patient Affairs Staff (PAS) is devoted to supporting cross-cutting patient engagement activities across the FDA. Its mission is to coordinate and support patient engagement activities across FDA to facilitate awareness and collaboration with patients, their advocates and the FDA

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EU Compassionate Use Programs

Published by IRDiRC

Research and Drug DevelopmentRegulatory AffairsClinical DevelopmentOrphan DrugEarly Access SupportEuropean UnionCompassionate UseEMA Compassionate Use

Compassionate use is a treatment option that allows the use of an unauthorised medicine. Under strict conditions, products in development can be made available to groups of patients who have a disease with no satisfactory authorised therapies and who cannot enter clinical trials.

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National programs for early access

Published by IRDiRC

Regulatory AffairsClinical DevelopmentCompassionate Use

Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available

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FDA Qualification process – Drug Development Tools

Published by IRDiRC

Regulatory AffairsResearch and Drug DevelopmentCenter for Drug Evaluation and ResearchClinical DevelopmentEndpointBiomarkerAnimal ModelQualification ProcessDrug Development Tool

This guidance provides a framework for interactions between the Center for Drug Evaluation and Research (CDER) and the entity proposing the DDT for qualification (the submitter). It also explains the kinds of data that should be submitted to support qualification of a DDT and creates a mechanism for CDER’s formal review of the data to ultimately qualify the DDT.

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