With nearly 8,000 rare diseases, of which only 5% have an approved treatment, repositioning a drug is therefore a strategy of interest for rare diseases and a hope for patients.
A repositioned drug is generally approved more quickly (3-12 years) at a reduced cost (50-60%) and with a lower risk during development.
Is repositioning the right opportunity to find drugs for rare diseases at a lower cost and with less risk?
While there are some great success stories, other stories have the bitter taste of failure...
The Foundation For Rare Diseases has just published in the magazine Thérapie, an article which helps to see more clearly, to distinguish fiction from reality and to propose an answer to the question!