GRANDRx will focus on rare and neglected diseases, which collectively affect millions of people throughout the world.
Therapy development for rare and neglected diseases needs transformation driven by collaboration.
Fewer than 200 rare and neglected diseases have an available therapy and there is limited likelihood of future treatments. The path to developing therapies for any type of disease is filled with challenges. It is particularly true for rare diseases that companies have a difficult time recovering their costs for research, development, and manufacturing, either due to small patient numbers or diseases occurring in countries that do not have the ability to cover costs.
GRANDRx Solutions
We are mapping a route forward to develop a robust system for translating basic science discoveries into safe and effective therapeutics. For all who have been waiting for medical research to offer treatments for a wide range of rare and neglected diseases, collaborations and new systems will pave the way.
When we work together in the coming months and years, we will construct gateways through three walls in the current system: assay development, preclinical development and clinical development.
GRANDRx will bring together the rare and neglected disease community through initiatives, partnerships, programs and projects to further characterize and address the barriers in the system, as well as work with federal institutes and agencies on informed regulatory and policy solutions.