President's Message
When your child gets sick, you hope there will be a treatment available for them.
For many patients with rare disorders, those treatments don’t exist. This situation is made even more tragic when the science exists to develop a treatment, but the cost and complexities of development have slowed or even stopped progress. Despite the advances made in the last 10 years for some rare diseases, there are hundreds of others where the science exists for a treatment, but the next step of development will not happen.
I created the Kakkis EveryLife Foundation to CureTheProcess and focus on fixing the problems associated with developing treatments for rare disorders.
Our goal is to improve the regulatory environment surrounding the approval of effective treatments. We plan to effect change in regulation of rare disease treatments by insightful scientific analysis and dialogue, grass roots support and political sponsorship, complementing but not competing, with the role of patient societies and research foundations.
We believe:
- No disease is too rare to deserve treatment.
- We already have the science we need to treat more rare disease patients.
- We need an improved process with new study designs and disease measures to accelerate the development of new treatments.
- We need the right people in both industry and FDA to make these changes effective.
- All new drugs for rare diseases should be safe.
The Kakkis EveryLife Foundation will be focused primarily on analyzing the problems and providing solutions that can accelerate the development process for rare disease treatments. We know it will be hard and complex, but the science is here and we have many patients depending on us, to CureTheProcess and treat them.
Thank you for your support,
Emil D. Kakkis, M.D. Ph.D.
President, Kakkis EveryLife Foundation