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.
Pricing implications for drugs included in China’s Rare Diseases Catalogue (RDC)
China’s Rare Disease Catalogue (RDC) plays a growing role in shaping awareness, investment, and access to therapies for rare diseases in China.