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Research and Drug DevelopmentOrphan DrugPublicFunded ProgrammeNIH InitiativeNIH 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
View this resource Bookmark this resourceA Priority Review designation results in an abbreviated PDUFA mandated goal date for a Biologics License Application (BLA) or a New Drug Application (NDA), from 10 months (under standard procedure) to 6 months.
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FundingNTU-NCATSResearch and Drug DiscoveryNCATSDrug RepurposingOrphan DrugThe 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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Special Protocol AssessmentRegulatory AdviceClinical Trial DesignClinical DevelopmentRegulatory AffairsFDA-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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Research and Drug DevelopmentClinical DevelopmentNIHCRNClinical Trial ReadinessRare Disease Clinical Research NetworkCinical Trial DesignThe 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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Research and Drug DevelopmentRegulatory AffairsClinical DevelopmentEndpointBiomarkerAnimal ModelQualification ProcessDrug Development ToolCenter for Drug Evaluation and ResearchThis 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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Regulatory AffairsEarly Access SupportAdvanced TherapyRegenerative Medicine Advanced Therapy DesignationThe RMAT Designation is analogous to the Breakthrough Designation designed for traditional drug candidates but applies to regenerative medicine treatments, and allows companies to interact with the FDA more frequently during the clinical development of the therapy. An RMAT-designated therapy is eligible for priority review and accelerated approval.
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