Core Module

Introduction to Drug Repurposing « Back

The path from an idea to a repurposed drug being made available to patients is not always linear, though there are often some common milestones:

Identifying an appropriate drug to repurpose, i.e., a repurposing candidate
Establishing the basic science behind a repurposing candidate (e.g. the drug’s mechanism of action)
Proving that the drug is effective in a relevant in vivo disease model
Proving that the drug is safe and effective for the new indication in human clinical trials
Determining an effective and appropriate route to market
Ensuring fair and equitable access to the drug for patients

Throughout a drug repurposing project, the following should be considered:

How to fund each drug development stage
How to build the right development team with the right expertise for each development stage
How and when to involve and engage with patients and patient representatives
How to maintain effective relationships with all stakeholders (including regulators, Health Technology Assessment (HTA) experts, clinical teams, payers, etc.)
How to manage a complex project to a high standard

It is important at each stage to keep the end goal in mind – a new, safe and effective treatment reaching patients.

Drug Repurposing Explained: Webinar from Beacon; for rare diseases (formerly findacure). 70 minutes. See below.