Riluzole has been around since 1995 as the only drug to treat ALS. Riluzole is not a cure for ALS. Its purpose, as I understand it, is to prolong the median stage of the disease for a person living with ALS (PALS) by about three months. Some PALS decide not to take the Riluzole for a lot of different reasons. Cost of the drug is one of those reasons. Drugs for rare or orphan diseases typically cost a fortune and can bankrupt a family.
I chose to take Riluzole immediately upon being diagnosed. I don’t remember what my own motivation was at that point, but my husband wanted those extra three months. I had private insurance at the time and the copay was $385 for a month’s supply. I found a coupon online at GoodRX that lowered the cost to $185 per month as long as I did not use my private insurance. Another pharmacy we tried wanted $999 for the same medicine. I can never understand how that works. It seems to me that drugs, like politics, is just another game. Maybe I am just naive.
Not too long ago I took part in a Patient Advisory Panel for ALS patients and caregivers. The panel was designed to get input on a new sublingual form of Riluzole. Biohaven Pharmaceuticals made the drug and was writing a protocol for FDA approval. Imagine that! A pharmaceutical company who wanted to hear from the end user, the ALS patient.
This discussion was being facilitated by Easy Access Care. Their brochure explains how the FDA works with pharmaceutical companies under the Expanded Access Program as follows:
Under its expanded access programs, the US Food and Drug Administration (FDA) works with companies to allow access to investigational drugs outside of a clinical trial to patients with serious or life threatening illness for whom there are no comparable satisfactory alternate therapies (EAC).
According to Biohaven Pharmaceuticals and Early Access Care, (Biohaven, February 17, 2018) expanded access is intended to be a form of treatment for “a patient with a serious disease disease or condition“ (Biohaven, February 17, 2018). EAC’s expertise allows the drug companies to focus on research and development of new treatments and drugs while they take care of the paperwork and red tape. You know there has to be red tape if it involves getting governmental approval.
Biohaven’s sublingual riluzole offers advantages over the usual tablet form. The obvious advantage is that it can be put under the tongue. For patients who have difficulty swallowing this is a huge plus. It does not require any liquid to be taken with it and it dissolves quickly. Fasting is not required in the sublingual form as it is with the tablet form (Biohaven, February 17, 2018).
In order for me to try the new sublingual medicine, my doctor had to complete forms, obtain approval from Duke Health Systems, and apply for the medication from Biohaven. My doctor handled a lot of the paperwork himself because there was no budget that would cover the program cost. This is what your doctor will do for you if he is a true ALS Advocate. I was the first, and to date, the only patient at Duke to make the request for the drug. I was interested in taking sublingual riluzole because under the expanded access program it would be free to me. I am on hospice now and they will not provide a drug that will prolong my life. So even though all of my other medications and supplies are provided free, riluzole is not. If I choose to take it, I pay out of pocket.
I was on the sublingual form of riluzole for about a month before I had to stop. I knew during the panel discussion that I was never a fan of mint, but that was the flavor chosen for the sublingual. I took the initial dose and did not expect such a strong flavor of peppermint. I pretty much hate peppermint. The first dose gave me a coughing and choking fit that lasted for 15-20 minutes. I also had the stinging and numbing that I recognized from my past experience taking the tablet form by mouth. I don’t know if I would have been able to take the sublingual if it came in another flavor. I had even resorted to adding the new form into a slurry of my other meds that are put into my feeding tube. That helped me to avoid the coughing and choking. I realized, however, that a sublingual and transdermal medication shouldn’t be taken this way. It would not be effective.
Those PALS who are peppermint lovers and currently taking riluzole, but have swallowing or economic problems may be interested in trying this sublingual version. If you are a patient at the Duke ALS Clinic will be a little bit ahead in the application process. I hope it works well for you if you decide to try it.
Biohaven Pharmaceuticals and Early Access Care, February 17, 2018, “Patient advisory panel: ALS patients and care providers”
Early Access Care (EAC), (n. D.), BHV-0223 “Expanded access protocol: For people with amyotrophic lateral sclerosis”