Rare Pediatric Priority Review Voucher

Published by IRDiRC

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

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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Rare Pediatric Disease Designation

Published by IRDiRC

Early Access Support Orphan Drug Designation Paediatric Medicine Rare Paediatric Disease Designation Regulatory Affairs

The FDA grants rare pediatric disease designation for diseases with serious or life- threatening conditions that affect people from birth to 18 years of age, that affect fewer than 200,000 people in the U.S.

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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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Discovering New Therapeutics Uses for Existing Molecules (NTU) program, National Center for Advancing Translational Sciences (NCATS)

Published by IRDiRC

Drug Repurposing Funding NCATS NTU-NCATS Orphan Drug Research and Drug Discovery

The NTU program aims to improve the process of developing new treatments for diseases by finding new uses for existing therapies that already have cleared several key steps along the development path. Existing or partially developed therapeutic candidates can be repurposed for use in new disease indications

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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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NCATS Toolkit for Patients-Focused Therapy Development

Published by IRDiRC

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

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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Stakeholders interactions – Patients Affairs Staff

Published by IRDiRC

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