My FDA speech

"Good morning, and thank you for having us. I, along with my husband, stand before you today to share the still unfolding story of our daughter Ella's journey with infantile spasms.

In May of 2009, while not yet 5 months old, Ella began to have odd staring spells. Up to this point, she was on track developmentally, but, being a nurse, I feared right away that something very serious was wrong with our baby. Within the week, her spasms became more pronounced and began to cluster in short succession several times a day. By mid June, after an EEG that was determined to be "very abnormal and worrisome" by our neurologist, Ella was admitted to Children's Hospital for treatment of what was presumed to be infantile spasms.

We had already researched the possible causes of what we were seeing and were familiar with the term. And its devastating prognosis. I will never forget one of the first things I read about this catastrophic seizure disorder: that the incidence of mental retardation associated with it is estimated at 75 to 90 percent. There were also grim statistics about shortened life expectancy that I quickly banished from my mind.

After a few days of EEG monitoring and observation, Ella was officially diagnosed with infantile spasms. Quickly, the neurology team decided that ACTH would be the best course of action for our baby; we weighed the risks and agreed, and she started treatment the very next morning.

The case manager immediately began communicating with our insurance company to cover the prohibitive cost of the medication, knowing it would be a hardship on our family.

Because Acthar is not FDA-approved for use in infantile spasms, our insurance company denied coverage, calling it "experimental." An appeal was also denied for the same reason.

But the National Organization of Rare Disorders, along with the Acthar Support and Access Program, had already come on board to help us get this medication our daughter so desperately needed. Through their combined efforts, even though our insurance company never approved our claim, we were given Ella's entire six-week-course of treatment free of charge and without delay. We will be forever grateful for their assistance.

On her eighth day of treatment with ACTH, and exactly a month after her "staring spells" began, Ella had her last spasm, well over 10 months ago. 

Now 15 months old, our daughter is a happy, beautiful, active, loving little girl who charms everyone she meets. She attends a full-time early intervention program that provides her with daily physical and speech therapies, and, though she is delayed, we have great hope that she will continue to progress and will some day reach her optimal development.

We take nothing for granted, knowing that hers could have easily been a very different story. We believe that ACTH gave our daughter back to us. We hope that you would strongly consider approving Acthar for use in the treatment of infantile spasms, so that many more children like Ella would have access to this life-giving medication."

♦