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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 marksCarly pflaumPatrizia cavazzoniDrug administrationRare disease drug developersPilot programBiologics evaluationTrials advancing rareInvestigational new drugDrug evaluationFederal registerBiologics license applicationNew drug applicationEmpower rare disease drug developersDemonstrating substantial evidenceEffectiveness based

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 cavazzoniPeter marksRare disease drug developersPilot programUs department of healthHuman servicesDrug administrationFor immediate releaseBiologics evaluationTrials advancing rareInvestigational new drugDrug evaluationFederal registerBiologics license applicationNew drug applicationEmpower rare disease drug developers

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 leeOffice of translational sciencesOffice of new drugsRegulatory aspects of rare disease drug developmentDivision of rare diseasesRare disease drug developersRare diseases teamOffice of communicationsOffice of the centerRare neurodegenerative disease grant programDrug development programDrug administration centerOncology centerProgram future outreachRadiological healthPilot program

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 marksDisease grant programPilot programRare pediatric disease priority review voucher programRare disease drug developersOrphan drug tax creditBiologics evaluationDrug evaluationAccelerating rareEmpower rare disease drug developersRare disease endpoint advancementCritical pathRare neurodegenerative diseasesRare diseasesHealth careHealth care cost