Author: [AUTHOR] Published on 12/1/2025 12:00:00 AM
Noha Rayad, PhD, Director, AstraZeneca, Mississauga, ON
What does being a Rare Diseases (RD) Community Vice Chair mean to you?
It means being a steward of hope, contributing to building a future where every patient, no matter how rare their condition, benefits from precision, evidence-based treatments that are developed with scientific rigor and compassionate advocacy.
What is your favorite part of being the Rare Disease (RD) Community Vice Chair?
My favorite part is collaborating with all stakeholders—patient advocates, researchers, clinicians, regulatory agencies, and industry—to address the unique challenges of rare diseases, such as sparse and heterogeneous populations, the 6–8 years diagnostic odysseys, and the high cost of curative therapies like gene therapies. It is a blending of scientific expertise with advocacy to address those challenges.
Do you have a favorite tip or trick for clinical practice or research that you want to share with fellow members?
Engage in all endeavors/advocacy for rapid diagnosis (patients will not benefit from a new treatment if they are not correctly diagnosed) and keep pushing for the integration of advanced computational tools to optimize dosing strategies for rare disease therapies, where data are often sparse.
When you are not working, how do you spend your free time?
Light exercise, sleeping, spending quality time with family and friends, and traveling.
How do you keep focused and motivated?
Knowing that my work can accelerate drug development for the 90% of rare diseases lacking approved treatments. Also, knowing that 30% of children with rare diseases may not reach their fifth birthday fuels me to develop targeted therapies faster.
Dr Rayad has been a member of ASCPT since 2020.
