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Core Module

Introduction to Drug Repurposing « Back

The Drug Repurposing Pathway

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.


Learn More

‘Drug Repurposing Explained’ Webinar

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