EU Project
SIMPATHIC

Accelerating drug repurposing for rare diseeses

SIMPATHIC is the abbreviation for “accelerating drug repurposing for rare neurological, neurometabolic and neuromuscular disorders by exploiting SIMilarities in clinical and molecular PATHology”. 

What does that mean exactly? The researchers focus on expediting the drug repurposing process and enhancing every aspect of it. The goal is to make it faster, ensure that drugs reach patients as swiftly as possible and increase patients’ well-being. Traditionally, medication is developed one disease at a time.

Reducing timelines

The consortium will fast-track the testing of drug safety and effectiveness in humans, via intelligent trial design. We will combine patients with different diseases into one ‘basket trial’. These are trials testing treatments that target specific issues in various diseases that have similar genetic changes or other shared risk factors. While basket trials are not entirely new, they are groundbreaking in the context of rare diseases. 

With this approach the goal is to decrease the timeline from 12 years for new drugs to 8 years for repurposed drugs, with the eventual aim of achieving a timeline as short as 4 years at an average cost of several millions of euros.