OPENing Doors for Ashleigh
The Story Behind The OPEN Act
Ashleigh Pike is an inspiration to many in our community and across the nation. Her story touches patients, families, advocates, and health care professionals alike.
Ashleigh is one of the main driving forces behind important legislation passed by the House last year, my OPEN Act.
I had the pleasure of meeting Ashleigh during a roundtable I hosted in my district to gather patient perspectives, ideas, and advice as part of the 21st Century Cures initiative. Ashleigh shared her story with me, and thus inspired the OPEN Act…
Three years ago, Ashleigh’s life changed dramatically. As she explained during our roundtable, “…prior to August 2, 2013 I was a healthy 25-year-old about to begin my 5th year teaching elementary school. I loved to dance and was a regular at the gym.”
During a vacation to Washington, D.C. in 2013, Ashleigh became acutely ill, requiring a wheelchair for her flight home. Two days later, she suffered a grand mal seizure from hyponatremia.
Once an active, enthusiastic elementary school teacher, Ashleigh is now unable to teach or enjoy life’s simple pleasures like taking a walk on the beach. She is unable to even stand for long periods of time.
Ashleigh struggles with many things we take for granted on a daily basis:
· Hindered communication due to small nerve neuropothy and violent coughing attacks;
· A malfunction in her autonomic nervous system which involves breathing, blood pressure, sweating, digestion, etc.;
· Issues with blood pressure as her system cannot regulate itself;
· Trouble standing for very short periods of time due to blood pooling at her feet, unable to make it back to her heart, brain, etc.; and
· Fainting and a dramatic passing out which appears to be seizures due to her body’s attempts to circulate blood to required regions.
Many of these symptoms fall under the category of dysautonomia — an umbrella term used to describe conditions that cause a malfunction in the autonomic nervous system.
For most dysautonomia patients, diagnosis can take up to six years. Ashleigh considers herself luckier than some patients — her mother, who works in the medical field, and her father have been incredible advocates. Ashleigh also credits the help of her community, which is dedicated to educating and helping patients and doctors dealing with this condition.
I share Ashleigh’s praise for our supportive community which provides her comfort and strength. Stakeholders in Florida have been extremely valuable in encouraging research and raising awareness for medical conditions. Dysautonomiaproject.org, which was founded by Kelly Freeman in Clearwater, is working tirelessly to educate patients and doctors on how to diagnosis an autonomic dysfunction and make appropriate referrals to specialized facilities for more pinpointed diagnoses.
For Ashleigh though, it was still a battle trying to get doctors and the correct medical care. This is largely due to the fact that the medical community has never before seen such a rare form of dysautonomia.
Once it was clear that local hospitals were limited in their ability to help, Ashleigh and her parents made their way to Vanderbilt Autonomic Research Center, University of Texas Southwestern, the Mayo Clinic in Jacksonville, FL, and the National Institutes of Health where Ashleigh serves as a recurring research patient for testing and analysis.
Ashleigh’s medical team is still searching for an accurate diagnosis and efficient treatment methods.
In the meantime, Ashleigh found hope in an orphan (or rare) drug Northera (Droxidopa), a drug meant to reduce dizziness and lightheadedness. She is also benefiting from other promising treatments that have not yet been approved for her condition.
These orphan drugs are not easy to get. Ashleigh explained during my roundtable, “Many times insurance companies won’t cover them because most of the medicines are used ‘off label.’ Meaning they are not tested or approved for our disease, but they are our only option.”
This is why the OPEN Act is crucial. Ashleigh and her mother made this important plea in their testimony during my roundtable, “We need Congress’s help to ensure patients have access to affordable treatments!”
My OPEN Act will help these orphan diseases that are not easily treated due to their rare status. This bill will incentivize drug makers and innovators to “repurpose” major market drugs for life-threatening rare diseases such as Ashleigh’s. This opens the door to the development of hundreds of safe, effective, and affordable treatments for rare disease patients.
The OPEN Act will allow Ashleigh to gain access to potential cures and treatments for her condition that she would otherwise struggle to receive. This bill will help millions of people by ensuring medications are safe and effective for rare conditions and can be reimbursed through insurance coverage.
As Ashleigh says, “The OPEN Act will allow those with rare diseases a chance at medicines that could be helpful when currently these medicines are otherwise not being developed, or are not approved for us to even try. It would decrease the cost of orphan drugs as well. What a gift Congressman Bilirakis has given this community!”
My OPEN Act passed the House last year as part of the 21st Century Cures Act. Now, it is up to our Senate colleagues to advance this important legislation. We must get this done for the millions of patients like Ashleigh, the millions who are struggling each and every day.