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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European Commission funded programs and resources

Published by IRDiRC

Funded Programme Orphan Drug Development Public

List of different (co-) funding initiatives of the European Commission, a number of which are dedicated to orphan drug development

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Crowd funding

Published by IRDiRC

Crowdfunding Private Rare Disease

Multiple websites/platforms provide crowd funding services or describes how to initiate crowdfunding for rare diseases

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NIH funded programs and resources

Published by IRDiRC

Funded Programme NIH Initiative Orphan Drug Public Research and Drug Development

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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AMED funded programs and resources

Published by IRDiRC

Public

AMED provides seamless funding from basic research to practical application and adequate implementation of clinical studies/trials, in order to promote drug development in Japan

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Conditional and Time-limited Authorization of Regenerative Medical Products

Published by IRDiRC

Advanced Therapy Procedure CTARP Marketing Authorisation MHLW or PMDA Regenerative Medical Products PMD Act Regulatory Affairs

A regulatory framework for the approval of regenerative medical products to benefit the patients with unmet medical needs under The Law on Securing Quality, Efficacy and Safety of Products Including Pharmaceuticals and Medical Devices (PMD Act)

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FDA Expedited Program for serious conditions – Fast Track Designation

Published by IRDiRC

Early Access Support Expedited Programme Fast Track Designation Rare Disease Regulatory Advice Regulatory Affairs

Process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

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Humanitarian Use Device

Published by IRDiRC

Early Access Support Humanitarian Use Device Medical Device Regulatory Sciences Safe Medical Devices Act

the Safe Medical Devices Act to create a new regulatory pathway for device products intended for diseases or condition that affect small (rare) populations that defined HDEs and HUDs.

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US Orphan Drug Designation

Published by IRDiRC

Early Access Support Orphan Drug Designation Orphan Drug Incentive Regulatory Advice Regulatory Affairs

The ODA is intended to provide incentives for Orphan Drug development and to defray the costs of qualified clinical testing expenses incurred in connection with the development of drugs for rare diseases and conditions.

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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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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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