With many gene therapies in development for an increasing number of indications, their curative promise presents a unique competitive dynamic as they enter the market over the coming years. A one-time treatment with the potential to cure patients could create a “winner takes all” dynamic, whereby second and later entrants for the same indication would have significantly smaller patient populations, such as treatment-resistant or inappropriate patient segments.
Rare disease PMA trends in France: Price achieved vs. disease prevalence
In this installment, we examine the relationship between launch prices in France and disease prevalence as reported in the Haute Autorité de Santé assessments.