CURETHEPROCESS™
ACCELERATING BIOTECH INNOVATION FOR RARE DISEASES

The Transverse Myelitis Association has become a partner organization of the Kakkis Everylife Foundation and has endorsed the 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 goal is to give even the most rare diseases access to the accelerated approval process and fulfill more completely the original intentions of the Orphan Drug Act.

The TMA has endorsed these efforts 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.

The following is a message from the Chairman of the Kakkis Everylife Foundation:

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 disease 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. 

Working together we will achieve the needed regulatory changes at the FDA to increase the predictability of the regulatory process and encourage biotech innovation for rare biochemical and genetic disorders. With your support we can give even the rarest diseases access to the accelerated approval process and put orphan treatments on the fast track.

Thank you for your support,

Emil D. Kakkis, M.D. Ph.D.
President, Kakkis EveryLife Foundation
77 Digital Drive, Suite 210, Novato, CA 94949
(415)884-0223
www.curetheprocess.org