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FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies

FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies
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Peter-marks , Carly-pflaum , Patrizia-cavazzoni , Drug-administration , Rare-disease-drug-developers , Pilot-program , Biologics-evaluation , Trials-advancing-rare , Investigational-new-drug , Drug-evaluation , Federal-register , Biologics-license-application

FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies

The FDA is announcing the opportunity for a limited number of sponsors to participate in a pilot program allowing for more frequent communication with FDA staff to provide a mechanism for addressing late-phase product development issues.

Patrizia-cavazzoni , Peter-marks , Rare-disease-drug-developers , Pilot-program , Us-department-of-health , Human-services , Drug-administration , For-immediate-release , Biologics-evaluation , Trials-advancing-rare , Investigational-new-drug , Drug-evaluation

Accelerating Rare disease Cures (ARC) Program Emerges as a Conduit for Empowering Rare Disease Stakeholders

The ARC Program strives to increase the number of treatments by promoting innovative scientific design, providing a deeper understanding of regulatory policies, and engaging with patients and their advocates, and other rare disease stakeholders.

Kerry-jo-lee , Office-of-translational-sciences , Office-of-new-drugs , Regulatory-aspects-of-rare-disease-drug-development , Division-of-rare-diseases , Rare-disease-drug-developers , Rare-diseases-team , Office-of-communications , Office-of-the-center , Rare-neurodegenerative-disease-grant-program , Drug-development-program , Drug-administration-center

Why the underestimated economic burden of rare diseases could be costing the U.S. trillions of dollars

While rare diseases impact a small population, the cost to society is staggering. It may be in the range of $7.2-$8.6 trillion per year.

Peter-marks , Disease-grant-program , Pilot-program , Rare-pediatric-disease-priority-review-voucher-program , Rare-disease-drug-developers , Orphan-drug-tax-credit , Biologics-evaluation , Drug-evaluation , Accelerating-rare , Empower-rare-disease-drug-developers , Rare-disease-endpoint-advancement , Critical-path