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.
Impact of CT outcomes on time to market and list price of orphan medicines in France
More innovative medicines have a shorter assessment time by the CT ASMR rating achieved vs. average time from EMA approval to outcome of CT assessment Key...