When my daughter, Taylor, was diagnosed with Batten disease, I couldn’t accept “no treatment, no cure.” I vowed not to “sit and watch” as the disease stole my child. The monster would not win without a fight. 

Since that day, Taylor’s Tale has made a significant financial contribution to treatment development, but funding is not the only obstacle standing in the way of a cure. That’s exactly why Taylor’s Tale has endorsed the CureTheProcess campaign.

CureTheProcess, led by the Kakkis EveryLife Foundation, promotes science-driven public policy to support the development of treatments for rare diseases. A related goal of the campaign is to give even the rarest diseases access to an accelerated approval process and meet the original purpose of the Orphan Drug Act.

 

Specific efforts include: 

• Establishing a new Office of Drug Evaluation for Genetic and Biochemical Diseases, 

consolidating expertise to ensure safe, effective and timely patient access to needed treatment.

• Creating a new standard for the surrogate and biomarker endpoints used for rare disorders, allowing treatments for these diseases to have full access to the accelerated approval pathway.

• Devising new clinical study design paradigms for rare diseases that properly account for clinical heterogeneity and disease complexity to properly capture treatment effects.

Should the intentions of the CureTheProcess campaign be fulfilled, the results will be monumental for the 30 million Americans who suffer from over 7,000 rare diseases. Timelines for the development of rare disease therapeutics will be shortened, and more patients will get earlier access to the treatments they need.  Certain rare disease treatments that are now untouched will become targets of drug development, and more early-stage biotech companies will get financial backing. Finally, should the campaign be successful, the FDA review process will be improved and become more specialized. 

When our family learned that Taylor was born with a rare genetic disease for which there is currently no treatment, we refused to accept the status quo. We vowed first to save Taylor, but along the way, we realized that our cause goes far beyond our little girl. So, when we founded Taylor’s Tale, we made a second vow: to do everything in our power to ensure that every child gets the happy ending they deserve. Today, we stand beside the Kakkis EveryLife Foundation and their efforts to CureTheProcess – and make happy endings possible for everyone who suffers from a rare disease.

To learn more about the CureTheProcess campaign, click here. 

Sharon King

Board of Directors, Taylor’s Tale

Mother/Fighter/Believer