NCATS Toolkit for Patients-Focused Therapy Development

Published by IRDiRC

Research and Drug Development Rare Disease Toolkit for Patient NCATS Toolkit Patient-Focus Therapy Development

The NCATS Toolkit for Patient-Focused Therapy Development (Toolkit) was developed through a collaboration between NCATS and rare diseases patient groups. The patient groups had expressed an interest in exchanging knowledge and sharing best practices in therapy development

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Rare Pediatric Priority Review Voucher

Published by IRDiRC

Research and Drug Development Regulatory Affairs Paediatric Medicine Marketing Authorisation Orphan Medicine Regulatory Advice Rare Paediatric Priority Voucher

This program grants a voucher for priority review from the US Food and Drug Administration (FDA), which aim to render a decision within 6 months (in contrast to 10 for a standard review). The developed drug for which the voucher is awarded must be intended for a rare disease or condition and that primarily affect individuals from 0 to 18 years. The voucher can be utilized for any other drug development program.

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Stakeholders interactions – Patients Affairs Staff

Published by IRDiRC

Research and Drug Development Regulatory Affairs Clinical Development Early Access Support Patient Engagement Patient Advice FDA Regulatory Advice Patient Affairs Staff Patient Support

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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US Expanded Access Program

Published by IRDiRC

Research and Drug Development Regulatory Affairs Orphan Drug Early Access Support Orphan Medicine Expanded Accesss Programme

The Program allows patients with serious rare diseases to have access to pre-market drugs and devices, if the Sponsor agrees to this under their expanded access policy

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Single Patient Expanded Access

Published by IRDiRC

Research and Drug Development Regulatory Affairs Orphan Drug Early Access Support Orphan Medicine Single Patient Expanded Accesss

This FDA program allows early access to drugs and medical devices before marketing approval, under regulatory oversight with collection of Safety data, to individual patients with serious or immediately life-threatening diseases or conditions who lack therapeutic alternatives. This includes emergency and non-emergency expanded access.

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NIH funded programs and resources

Published by IRDiRC

Research and Drug Development Orphan Drug Public Funded Programme NIH Initiative

NIH is the largest public funder of biomedical research in the world, investing more than $32 billion a year to enhance life, and reduce illness and disability. The tool has its best use at very early phases of drug development

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Clinical Research Networks

Published by IRDiRC

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

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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BEST (Biomarkers, Endpoints, and other Tools) Resource

Published by FDA-NIH Biomarker Working Group

Research and Drug Development Clinical Trial Design Endpoint Biomarker

A biomarker discovery and validation guideline for clinical trial design and the drug development.

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Preclinical biomarker qualification

Published by the Society for Experimental Biology and Medicine

Research and Drug Development Biomarker Drug Development Tool Qualification Biomarker Regulatory Qualification Preclinical

An overview of the biomarker qualification process that provides information on current evidentiary considerations. It also provides new information on the evolution of the role that preclinical qualification plays in clinical qualification of biomarkers and the novel approaches that are being utilized to improve the process

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FDA Qualification process – Drug Development Tools

Published by IRDiRC

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

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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