Kakkis Every Life Foundation's CureTheProcess Campaign
In July the Wilson Disease Association signed on to endorse the Kakkis Every Life Foundation's CureTheProcess campaign for rare disease treatment development. The campaign strives to inspire science-driven public policy to increase the predictability of the regulatory process for rare disease treatments. The WDA supports Kakkis Foundation's goal of giving even the most rare diseases access to the accelerated approval process and fulfill more completely the original intentions of the Orphan Drug Act.
Their goals are to:
- Establish a new Office of Drug Evaluation for Genetic and Biochemical Diseases, consolidating expertise to ensure safe, effective and timely patient access to needed treatment.
- Create a new standard for the surrogate and biomarker endpoints used for rare disorders, to allow treatments for these diseases to have full access to the accelerated approval pathway.
- Devise new clinical study design paradigms for rare diseases that properly account for clinical heterogeneity and disease complexity to properly capture treatment effects.
For more information please visit http://www.curetheprocess.org/ to learn more about how you can take action to support this important initiative.