Step Therapy - Its Effect on Rare Disease Patients

Last week when I asked the question - if you had a one-on-one with your lawmaker what would you ask, one person mentioned eliminating step therapy. I thought that I would take some time to explain to others what step therapy is for those who may not understand it and also explain the ramifications of it for those who suffer with rare diseases. Step therapy is a big enough issue in the rare disease community that it is one of the nine categories that states are evaluated on by NORD (National Organization of Rare Disorders).

Step therapy, put quite simply, is when there are multiple treatments for a disorder that are FDA approved and insurance companies dictate the order that steps must be taken in order for them to pay for treatment. It is in place to save insurance companies money, which in turn would save us all money and that sounds like it is a good idea - we all know that prices of medical treatment are our of control. So, why would we not want to try treatments that are cost effective first?

If you just read that - you would wonder why almost everyone who advocates in the rare disease community is opposed to step therapy. The biggest problem here is who is making the decision - it is not you, it is not your doctor - it is your insurance company. It is not looking at your specific case, it is not looking at the rest of your medical complexities and co-morbid conditions necessarily. Step therapy is your insurance company looking at a list of approved medications for an illness and only looking at the money.

As I have talked about before, many rare diseases are a lot more complex than what you read about in a medical textbook or even in one or two studies that may have been done decades ago. Many of us go from doctor to doctor to find a doctor that works well with us specifically and that will find the treatment that fits what we are experiencing, not what is necessarily published. Can you imagine how frustrating it is then to be told once you find that treatment that works best for you only to be told by your insurance company that they will not pay for it until you fail at other medications that your doctor already knows will not be effective?

Think about how I have mentioned the diagnostic journey and the effects that can have on patients. The time it takes to get the diagnosis often delays effective treatment by years, possibly causing the effects and symptoms of the illness to increase. Then you get your diagnosis and it is one with treatments, but you have to go through step therapy to get the the one medication that your doctor knows is best for you. Again, your treatment is delayed, but this time you not only know your illness, but know what your best chance of treatment is. Many rare diseases are progressive - delaying treatment means you get worse, some are fatal, meaning that delaying treatment may mean dying earlier. Why are we allowing insurance companies to dictate these steps in place of the medical professional we have entrusted with out care?

Here is the link to the map of where you state is in Step Therapy according to NORD's latest report card https://rarediseases.org/policy-issues/step-therapy/. If you do not like where your state is on this issue, I will tell you the same thing that I have said before - contact those people who make the decisions. Call them, write them - whatever feels best to you, but tell them how you feel and more importantly how this impacts you or your loved one and the damage this policy is doing. It can seem scary to contact politicians at first, but remember when my congressman called and I thanked him for calling - he thanked me for paying his salary. They work for us and we need to tell them where we stand on these important issues.

If you are unsure what to say or how to say it, I am more than happy to help. It is summer and I am advocating full-time while school is out. I am more than happy to help draft a letter or a template that can be emailed or mailed to your legislators and help you figure out who is best to send it to. Together we can make these changes, but we need to tell them and we need to be clear and loud in our voices.