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Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases

Published by NIH

Natural HistoryFDA

The purpose of this funding opportunity announcement (FOA) is to support efficient and innovative natural history studies that advance medical product development in rare diseases/conditions with unmet needs. Through the support of natural history studies with high quality and interpretable data elements, FDA expects to address critical knowledge gaps, remove major barriers to progress in the field, exert a significant and broad impact on a specific rare disease or multiple rare diseases with similar pathophysiology, and facilitate rare disease product development.

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Basket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare Diseases

Published by NIH

Clinical TrialEtiology

NCATS seeks to facilitate rare diseases research by enabling efficient and effective movement toward clinical trials in multiple rare diseases. The purpose of this NOFO is to provide support for basket clinical trials of drugs targeting shared molecular etiologies in more than one rare disease, and in the process to identify and overcome challenges in adapting the oncology basket trial model to rare diseases. Projects proposed for this NOFO will require individuals with expertise in carrying out clinical trials in rare diseases. Applicants are expected to collaborate with clinical investigators at academic institutions. Projects involving clinical investigators who are part of the Rare Disease Clinical Research Network (RDCRN) and which focus on diseases under study in the RDCRN are strongly encouraged. See https://www.rarediseasesnetwork.org/ for additional information.

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Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases

Published by NIH

Clinical Trial

The purpose of this funding opportunity announcement (FOA) is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions. Additionally, through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare diseases and exert a broad and positive impact on rare disease drug development.

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Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes

Published by NIH

Clinical Trial Readiness

This Notice of Funding Opportunity (NOFO) invites researchers to submit applications for support of clinical projects that address critical needs for clinical trial readiness in rare diseases. The initiative seeks applications that are intended to facilitate rare diseases research by enabling efficient and effective movement of candidate therapeutics or diagnostics toward clinical trials, and to increase their likelihood of success through development and testing of rigorous biomarkers and clinical outcome assessment measures, or by defining the presentation and course of a rare disease to enable the design of upcoming clinical trials.

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Preclinical Proof of Concept Studies for Rare Diseases

Published by NIH

PreclinicalProof of Concept

This notice of funding opportunity (NOFO) provides funding to conduct efficacy studies in an established rare disease preclinical model to demonstrate that a proposed therapeutic agent warrants further development. In addition to preclinical efficacy, accompanying pharmacodynamic and pharmacokinetic studies would be supported. Therapeutic agents include small molecules, biologics or biotechnology-derived products. The goal of this NOFO is to spur therapeutic development for rare diseases by advancing projects to the point where they would attract subsequent investment supporting full Investigational New Drug (IND) application development or progression to clinical trials in the case of repurposing or repositioning.

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Natural History, Clinical Outcome Assessment, and Biomarker Studies of Rare Neurodegenerative Diseases

Published by NIH

BiomarkerNatural HistoryClinical Outcome

The purpose of this funding opportunity announcement (FOA) is to support efficient natural history studies alone or in conjunction with the development and validation of clinical outcome assessments (COAs) and/or biomarker studies to address the unmet needs in rare neurodegenerative diseases for children and adults. Through the support of studies with high quality and interpretable data elements, FDA expects to address critical knowledge gaps, remove major barriers to progress in the field, exert a significant and broad impact on a specific rare neurodegenerative disease or multiple rare neurodegenerative diseases with similar pathophysiology, and facilitate rare disease product development.

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Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases

Published by NIH

NeuromuscularClinical Trial Readiness

This Funding Opportunity Announcement (FOA) invites researchers to submit applications for support of clinical studies that address critical needs for clinical trial readiness in rare neurological and neuromuscular diseases. These studies should result in clinically validated biomarkers and clinical outcome assessment measures appropriate for use in upcoming clinical trials. Through the support of trial readiness studies, NINDS expects to enhance the quality and increase the likelihood of success of clinical trials in these rare diseases.

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Pilot Projects Investigating Understudied Proteins Associated with Rare Diseases

Published by NIH

Druggable ProteinPilot

The purpose of this funding opportunity announcement (FOA) is to solicit applications for pilot projects to elucidate a role for understudied proteins associated with rare diseases. Awards will support generation of preliminary data and/or tools around eligible understudied protein(s). A list of eligible proteins is provided and are members of druggable protein families that have a known association with a rare disease. This FOA is intended to jumpstart research on understudied proteins that are associated with rare diseases and provide applicants with sufficient funding to perform basic biochemical and/or biological work to further the characterization of understudied proteins associated with rare disease.

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Ultra-Rare Gene-based Therapy (URGenT) Network Resource Access

Published by NIH

CMOContract Research OrganizationCROContract Medical Organization

The purpose of this FOA is to provide investigators with a mechanism to access contract research/medical organizations (CROs/CMOs) and subject matter experts (SMEs) within the NINDS Ultra-Rare Gene-based Therapy (URGenT) Network to support planning, manufacturing, and limited nonclinical therapeutic development efforts.

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Critical Path Public Private Partnerships

Published by NIH

Research Demonstration Cooperative Agreements

The FDA seeks an application to continue to maintain, manage existing consortia groups convened and established by the Critical Path Institute. This is a renewal of the cooperative agreement #5U18FD005320 as part of the Critical Path Initiative. This is in support of Critical Path Initiative introduced in Section 566 of the Federal Food, Drug, and Cosmetic Act. This includes developing innovative, collaborative projects in research, education, and outreach for fostering drug product innovation, enabling the acceleration of development, manufacturing, and translational therapeutics, enhancing safety, efficacy, quality, and performance. This funding opportunity will provide support, depending on availability of FDA funding.

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