Repurposing Medicines Toolkit – Guidance for navigating the process

Published by MRCLife Arc


This toolkit aims to provide a guide for researchers, charities and others embarking on repurposing projects. It highlights common activities, issues and considerations at each development stage and signpost to other available resources. The content is mostly styled as questions – what should I be considering at each development stage? It is intended to stimulate thinking and not as a step-by-step guide, which reflects the variable nature of repurposing.

View this resource Bookmark this resource

MDC Connects: A Guide to Drug Discovery

Published by Medicines Discovery Catapult

Drug DiscoveryTarget IdentificationSmall Molecules

This guideline is a summary presentation from the Medicines Discovery Catapult (MDC) webinars. The aim was to create a resource of modern drug discovery knowledge to support small companies who are developing their own medicines. The guideline is focused on preclinical research with an emphasis on small molecules in the following topics:
• What makes, and how to identify, a good target
• Methods to identify the chemical compound which would become the drug
• How to optimise that drug
• How to test it works in models of disease
• And how to demonstrate that it would be safe to dose in humans

View this resource Bookmark this resource

Through a combination of in-house and external partnerships with a range of regulatory experts and groups, EATRIS can provide regulatory support for most types of product including: Expert opinion, Orphan Drug Designation and Scientific Advice application at the EMA, Pre-clinical and clinical plan development, Informal scientific advice with selected national competent authorities, for highly complex projects,
access to EATRIS Regulatory Database (free of charge) that contains information about the regulatory requirements, guidelines and legislations from 27 EU countries (as well as Norway, Switzerland, Turkey and Israel) regarding drug and medical device development derived from the application of European legislation.

View this resource Bookmark this resource

Early Drug Discovery and Development Guidelines: For Academic Researchers, Collaborators, and Start- up Companies

Published by EATRIS

Research and Drug DevelopmentDrug Discovery GuidelineSmall MoleculeDrug RepurposingTarget Validation Drug DevelopmentNCATSNIH

This manual contains guidelines to develop therapeutic hypotheses, target and pathway validation, proof of concept criteria and generalized cost analyses at various stages of early drug discovery. Various decision points in developing a New Chemical Entity (NCE), description of the exploratory Investigational New Drug (IND) and orphan drug designation, Drug Repurposing and drug delivery technologies are also described and geared toward those who intend to develop new drug discovery and development programs.

View this resource Bookmark this resource

Assay Development and Screening Technology (ADST)

Published by NCATS

Small MoleculeNCATSHigh Throughput Screening

One of the first steps in the drug development process is creating test systems — called assays — on which researchers assess the effects of chemical compounds on cellular, molecular or biochemical processes of interest. At NCATS, the experts in the Assay Development and Screening Technology (ADST) program work to optimize assays requested or submitted by the biomedical research community for high-throughput small-molecule screening

View this resource Bookmark this resource

Drug discovery and development: Role of basic biological research

Published by Alzheimers Dement (N Y).

Drug DiscoveryTarget Validation

This article provides a brief overview of the processes of drug discovery and development. The aim is to help scientists whose research may be relevant to drug discovery and/or development to frame their research report in a way that appropriately places their findings within the drug discovery and development process and thereby support effective translation of preclinical research to humans.

View this resource Bookmark this resource

Genetic-Driven Druggable Target Identification and Validation

Published by Trends Genet.

Target IdentificationGWAS AssociationGenetic ToolboxExpression Quantitative Trait Locus

This article reviews the opportunities and challenges, and infer criteria for the optimal use of genetic findings in the drug discovery pipeline. Choosing the right biological target is the critical primary decision for the development of new drugs. Systematic genetic association testing of both human diseases and quantitative traits, along with resultant findings of coincident associations between them, is becoming a powerful approach to infer drug targetable candidates and generate in vitro tests to identify compounds that can modulate them therapeutically

View this resource Bookmark this resource