This manual is a comprehensive resource for early drug discovery, providing guidelines for developing therapeutic hypotheses, validating targets and pathways, and establishing proof of concept criteria. It includes analyses of costs at various stages of drug development. The content encompasses critical decision-making points in New Chemical Entity (NCE) development, exploratory Investigational New Drug (IND) processes, and orphan drug designation. Furthermore, it delves into Drug Repurposing and drug delivery technologies, making it an invaluable guide for professionals initiating new drug discovery and development programs.

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Exploring the new horizons of Drug Repurposing: A vital tool for turning hard work into smart work

Published by European Journal of Medicinal Chemistry

Research and Drug DevelopmentClinical TrialDrug DiscoveryDrug RepurposingLead Molecule

This review provides different approaches for Drug Repurposing and addresses recent developments and future scope of drug repositioning strategies. In addition provides a list of repurposed drugs and repurposing resources.

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Discovering New Therapeutics Uses for Existing Molecules (NTU) program, National Center for Advancing Translational Sciences (NCATS)

Published by IRDiRC

Orphan DrugDrug RepurposingNCATSResearch and Drug DiscoveryNTU-NCATSFunding

The 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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The use or generation of biomedical data and existing medicines to discover and establish new treatments for patients with rare diseases – recommendations of the IRDiRC Data Mining and Repurposing Task Force

Published by Orphanet J Rare Dis.

Data MiningDrug Repurposing

This article reviews the past successes of data mining for Drug Repurposing, and planning for future biomedical research capacity, the DMR Task Force identified four strategic infrastructure investment areas to focus on in order to accelerate rare disease research productivity and drug development: (1) improving the capture and sharing of self-reported patient data, (2) better integration of existing research data, (3) increasing experimental testing capacity, and (4) sharing of rare disease research and development expertise. Additionally, the DMR Task Force also recommended a number of strategies to increase data mining and repurposing opportunities for rare diseases research as well as the development of individualized and precision medicine strategies.

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The use or generation of biomedical data and existing medicines to discover and establish new treatments for patients with rare diseases – recommendations of the IRDiRC Data Mining and Repurposing Task Force

Published by Orphanet J Rare Dis.

Data MiningIRDIRCDrug Repurposing

(IRDiRC) set up the multi-stakeholder Data Mining and Repurposing (DMR) Task Force to examine the potential of applying biomedical data mining strategies to identify new opportunities to use existing pharmaceutical compounds in new ways and to accelerate the pace of drug development for rare disease patients.

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