As we approach #RareDiseaseDay, our Development Head of Cell and Gene Therapy, Carolyn Sasse recently published an article on the importance of #patient voice in advancing therapies for #rarediseases. Read the full piece here: https://lnkd.in/gARR94JP
Absolutely although it does required early and extensive analysis of patients needs to eventually create an adapted PRO, potentially impact futur label and real needs
Shanta Sarfare
8mo
So true! The need for rare disease treatments is acute.
Really enjoyed this!
Leveraging real-world data can accelerate the identification of potential candidates for rare disease trials, enhancing patient recruitment strategies. This approach streamlines the process and ensures a more diverse participant pool, addressing the unique challenges of rare disease research. By integrating these insights, we can better understand patient needs and tailor treatments accordingly, ultimately advancing the development of transformative therapies.