WASHINGTON—When Darlene Shelton’s first grandchild, Danny, was only 7 days old, he developed a bruise on his chest. Two days later, he had a second bruise. And 3 days after that, he got a third bruise.
At the age of 11 months, doctors properly diagnosed Danny with hemophilia B and immediately started him on coagulation factor IX (recombinant), marketed by Pfizer as BeneFIX. Now 12 years old, Danny has been on that medication his entire life.
By contrast, Shelton said, “with Lily, Danny’s 10-year-old sister, it took us 4 years and 4 doctors to get her tested and diagnosed, and then 6 more years to get her first dose of factor, because women are considered to be only carriers, and so their symptoms are not taken seriously.”
Soon after Danny’s diagnosis, a nurse began stopping by the house to give the boy his twice-a-week infusions. But what if Danny got into an accident and the home health nurse couldn’t come?
“We thought in an emergency that Danny would be protected, until late 2014 when we found out from a friend who has hemophilia—an adult who had a car wreck, broke his right wrist and hit his head, so he couldn’t self‑infuse,” she said. “He called 911. They came. He had his clotting factor there, in the original packaging, and he was still coherent. He was telling them what his dosage was. They said, ‘Sir, we cannot give you that medication because it’s not on our ambulance.’”
And the same would have gone for Danny, too. Shelton called local authorities in her hometown of Dexter, Missouri.
“We were informed that, although we had medical alert on his headrest in a car seat with his factor back attached to it with medical orders and dosing, the paramedics or ER doctors were not allowed to give it due to conflicting protocols and liability fears,” she said.
Outraged, Shelton decided to form her own nonprofit group, Danny’s Dose Alliance. Its mission, as stated on the group’s website, is “to change current outdated emergency treatment protocols that affect the proper care of special needs medical patients.”
Shelton: Arcane Laws Based on ‘Fear of the Unknown’
The nonprofit operates on a shoestring budget of $20,000 a year. Its 8-member board of directors as well as Shelton and her office assistant are all volunteers.
Shelton, speaking with Rare Disease Advisor during the recent 2022 Rare Diseases & Orphan Products Breakthrough Summit here, said 28 million Americans have 40 conditions that call for specialized emergency medical care outside of currently existing protocols (16 of which require the patients to carry their own specialty medications). Hemophilia A and B together account for only 52,000 of those 28 million patients.
“It’s really fear of the unknown. It’s a lack of education. There’s more of a fear that, ‘Oh, I don’t know this medicine, so I shouldn’t give it.’ Rather, the real fear should be, ‘If I don’t give this medication, this patient will be harmed.’”
Shelton said the main catalyst for starting her charity was to change state laws that prohibit people who aren’t doctors to give medication supplied by patients or their families.
“Basically, if it’s not on the ambulance, they can’t give it,” she told us. “The ER is the same. It’s a liability fear, because they’re so afraid of a patient bringing in their own medication. What if they didn’t store it correctly? What if it’s been tampered with? Of course, you have it in the original packaging, unopened and untouched.”
She added: “When I’ve been in these meetings, I tell them families take care of that medication; it’s your lifeline. You make sure that medication is taken care of, because it is what saves your loved one. It’s not something you play around with”—especially when that medication costs upwards of $50,000 per month.
Have a Treatment Plan Ready Just in Case
Due to her organization’s nationwide lobbying efforts, Shelton was able to get bills passed in her home state of Missouri as well as in Minnesota expressly allowing a paramedic to administer patient-carried, prescribed specialty medications. Arkansas passed similar legislation in 2021, and both Colorado and Louisiana are likely to follow suit, she said.
“We were very careful to leave out the word ‘lifesaving’ because these medications still need to be administered, even if you don’t think it’s life-or-death,” she said. “For instance, a muscle bleed is horrendous, and there are other conditions that affect different body functions. This is not going to be an over-the-counter medication in a pill bottle or a purse. These are specialty medications prescribed to the patient for their rare or chronic disease.”
In the meantime, Shelton has designed a yellow headrest cover made of cotton polyester that can be washed easily. It slips over the headrest of any car seat and has a drawstring to keep it in place, and a pocket that holds emergency orders.
“In Missouri, along with allowing the paramedics to administer patient‑carried meds, they need orders. A paramedic can only act upon the orders of a physician. The person who knows you best as the patient is your specialist,” she said.
“We teach our families that they need to get emergency orders from their specialist, so the paramedics can treat based on those orders. You have to have them readily available—and what better place than a pocket on the headrest of the car, because when they come on the scene, they’re always going to look in the face of the patient.”
Meanwhile, aside from protecting people with hemophilia from COVID-19 and waiting for a potential gene therapy that might cure the disease once and for all, Shelton has some advice for parents of children with bleeding disorders.
“Families must go meet with their local EMS service and say, ‘Hey, I live in your area and our family has this condition,’ and ask for a treatment plan. That means everybody is going to get together, you’re going to have meetings, they’re going to contact your specialist, and they’re going to develop a plan of action for when an emergency happens, and know exactly who your doctor is, know exactly the best emergency room to go to,” she said.
“It’s going to involve your school nurse. If any permissions need to be given, that can already be done. Then the day something happens, everybody is prepared and things won’t go south.”