There is a method to starting treatments, often seen in schools and insurance companies, to make sure that their resources are being used wisely. This is sometimes referred to as Step Therapy.
An insurance company, for example, will not cover medications unless the patient tries the cheaper options first. This is intended to save money for both the company and the patient.
Schools use a similar technique. They do not offer intervention until the child has demonstrated a significant need (fails), with their reason being that if the child doesn't show a need for these additional services, it would be an unwise use of resources to shower anyone who asks with them.
Mary Opinion Time: The Step Therapy technique started out with good intentions. This model is designed to save costs for everyone involved, and get high level help to the few who truly need it. And honestly, for someone who is as boring, medically-speaking, as I am, it works out just fine. However, there are significant flaws in this protocol for the "zebra population", and I want to review why this model may not be beneficial, particularly for patients with rare diseases and students with more severe developmental disabilities.
(Note: Zebra in the medical community refers to a patient who has a rare disease. The story goes that if you hear hooves behind you, everyone will assume it is a horse. But sometimes, it is a zebra. We are the zebras)
Our Seizure Medication Story
When my daughter Chelsea was almost five years old, she was diagnosed with a seizure disorder. I went down to the neurologist, and they prescribed a seizure control medication. Perfect! I thought, and off I went to the pharmacy to get it filled.
Turns out that the medication they put her on mildly controlled the seizures (and we were grateful for the improvement, though it didn't control them fully), but also came with the side effect of loss of bladder control. After about 6 months of ripping my hair out trying to figure out why I was having zero success in potty training my child (IDD aside, that is), I went back to the neurologist.
They put her on a new medication, but we didn't last long on that one, as she broke out into painful hives all over her body.
Cue medication number 3! This one controlled the seizures well, but came with the side effect of violent outbursts and aggressive behavior. We were told that those were the three medications our insurance would cover, so we had to choose- loss of bladder control, painful hives, or violent tendencies. Or do none and have more seizures.
Oh goodie, right? We ended up going with the violent tendencies drug, as Chelsea is tiny and was still easily manageable during aggressive episodes. As long as I worked out, I could handle her, though it broke my heart to see her angry and miserable.
Later, we moved and got a new neurologist. They recommended for us to trial a different, higher-level, drug when I explained our concerns. We got Chelsea on the medication, and saw an INSTANT improvement. ALL of the side effects vanished, and her seizures were controlled perfectly. She was back to our happy girl.
Now, here comes the kicker. Insurance REFUSED to pay for any of it (after appeals and everything) because cheaper options were available, and the medication cost close to $1,100 every month. Not only would insurance not help with the costs, they wouldn't even count it toward our deductible/ out of pocket max because it "wasn't approved."
THIS is the danger of Step Therapy.
Let's move on now to how this looks in the public school system. For an easy example, let's look at a common problem with Extended School Year (summer school) for a child with developmental disabilities.
The scenario: Suzy, 10 years old, has moderate Intellectual Disability, cannot read or write, cannot count beyond 10, but is a cheerful girl who is well-behaved in school and gets along with her peers. Her parents, concerned that she is not making any progress, contacts the school to look into summer school. The school replies that Suzy did not regress at any point during the school year, and that they only offer services to children after they fail/ regress significantly.
I have worked with families for YEARS, and I cannot even describe how often this scenario plays out in real life. I would guess more than 90% of the time.
Why is that an issue? Let's examine the performance of the child who IS offered summer school. Tommy is also 10 years old, but over Christmas break, forgot how to multiply two digit numbers, and failed the next six weeks of math. He gets summer school because he regressed, and the school exclaims that they need to get him back up to where he was performing before.
Wait a second! Suzy never had multiplication skills to begin with, so why is another child being offered help when she was denied services??
Welcome to one of the many injustices of Special Education. The school will not step in until the child fails. Suzy's goals are designed so that she can easily succeed, so she does not fail anything.
I do not say this to be unkind or judgmental toward teachers. They are overworked and underpaid, and want their students to be successful. The schools are underfunded, service availability is limited... It boils down to dollars. No administration wants to pay for extras unless they are mandated by the state. Often, if a child's IEP goals are not aggressive, it is easy for them to "pass" their classes, showing that they are making progress.
The issue I have with this methodology is that students who need habilitation services are denied when students in need of RE-habilitative services are approved. They BOTH need the help! There are a plethora of other examples in the public school systems in America, but I will leave that for another day.
So What Do We Do?
Now that we know the problem, what can WE as patients and patient advocates DO to change things?
On the insurance part of things, it is essential that rare disease patients participate in the drug development process early on. One story that stuck with me was a patient I spoke with who had a severe, cyclical vomiting syndrome (I forget the exact diagnosis). She talked about how she worked with a drug company to participate in the development of a treatment for her condition. The created a medication that stopped the vomiting, but that caused chronic diarrhea. The company had planned on releasing the drug, but it wasn't until she got a group of patient advocates together and said no, that wasn't a good option from the patient perspective, that they revisited it.
What is important to insurance and drug development companies is not necessarily what is most important to the patients dealing with the disease/ disorder, and if we don't speak up, how will they know what is most important to us? What may be a minor side effect to insurance companies could be life altering for patients.
I carry that same mentality into policymaking- if politicians never hear from us, they will assume that everything is working fine. So SPEAK UP! Your story is important!
As for schools, that would be a series of blog posts on its own! But the bottom line is still the same- you need to be clear and concise in what you are asking for and what your child needs and why. Early diagnosis and effective treatment is crucial for the success of the patient!
That basically covers it- a rough overview of Step Therapy and that dangers it presents to the rare disease population!