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.
World Orphan Drug Congress Europe 2024 Review: 3 Themes for sustainable and equitable access to medicines for RD patients
This year’s European World Orphan Drug Congress, attended by CRA’s Bhavesh Patel, Michele Pistollato, Charlotte Poon, Owen Male and Clara Zacharko in...