FDA Qualification process – Drug Development Tools

Published by IRDiRC

Animal Model Biomarker Center for Drug Evaluation and Research Clinical Development Drug Development Tool Endpoint Qualification Process Regulatory Affairs Research and Drug Development

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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Japan Compassionate Use Programs

Published by IRDiRC

Clinical Development Compassionate Use Programme Early Access Support MHLW or PMDA Regulatory Regulatory Affairs

A system for patients with life-threatening conditions without effective a treatment to gain access to unapproved investigational medical products.

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FDA Expedited Program for serious conditions – Special Protocol Assessment

Published by IRDiRC

Clinical Development Clinical Trial Design Regulatory Advice Regulatory Affairs Special Protocol Assessment

FDA-SPA is a process in which sponsors may ask to meet with FDA to reach agreement on the design and size of certain clinical trials, clinical studies, or animal studies, to determine if they adequately address scientific and regulatory requirements for a study that could support marketing approval.

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EU Compassionate Use Programs

Published by IRDiRC

Clinical Development Compassionate Use Early Access Support EMA Compassionate Use European Union Orphan Drug Regulatory Affairs Research and Drug Development

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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Extrapolation of efficacy and Safety in medicine development

Published by IRDiRC

Adaptative Pathways Clinical Development Clinical Trial Design Extrapolation of Efficacy and Safety Regulatory Sciences Research and Drug Development Study Design Target Population

The EMA describes extrapolation as extending information and conclusions available from studies in one or more subgroups of the patient population (source population(s)), or in related conditions or with related medicinal products, in order to make inferences for another subgroup of the population (target population), or condition or product, thus reducing the amount of, or general need for, additional evidence generation (types of studies, design modifications, number of patients required) needed to reach conclusions.

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National programs for early access

Published by IRDiRC

Clinical Development Compassionate Use Regulatory Sciences

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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Alternative designs for Small Population Clinical Trials

Published by IRDiRC

Adaptative Pathways Clinical Development Clinical Trial Clinical Trial Design Rare Disease Regulatory Sciences Small Population

Guidance and general recommendations are available to select the most efficient study design for each medical condition or trial, and on potential adaptions of conventional designs to the low sample size scenario.

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Adaptive Pathways

Published by IRDiRC

Adaptative Pathway Clinical Development Clinical Trial Design Early Access Support Regulatory Affairs

The adaptive pathways approach is part of the European Medicines Agency’s (EMA) efforts to improve timely access for patients to new medicines

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AMED Clinical Innovation network – Diseases-related Registries

Published by IRDiRC

Agency For Medical Research and Development Cinical Trial Clinical Development Clinical Innovation Network Initiative on Rare Diseases Japan Patient Registry Research and Drug Development

Japan Agency for Medical Research and Development (AMED) leads the Initiative on Rare and Undiagnosed Diseases (IRUD) program. The program provides patients across Japan suffering from undiagnosed diseases with a diagnostic strategy that involves genetic analysis

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Clinical Research Networks

Published by IRDiRC

Cinical Trial Design Clinical Development Clinical Trial Readiness CRN NIH Rare Disease Clinical Research Network Research and Drug Development

The RDCRN is made up of multiple individual, Rare Disease Clinical Research Consortium (RDCRC) and a Data Management and Coordinating Center (DMCC). The RDCRCs are intended to advance the diagnosis, management, and treatment of rare diseases with a focus on clinical trial readiness

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The Innovation Management Toolbox was originally developed through funding from the EJP RD project, which received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No. 825575. The continued development and maintenance of the Toolbox are now supported by the ERDERA partnership, funded under the European Union’s Horizon Europe Research & Innovation programme, grant agreement No. 101156595.

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