On December 26, 2016 the FDA approved Spinraza, the first ever therapy for Spinal Muscular Atrophy, the leading genetic cause of death in infants.
It was like a shot of adrenaline for a community that numbers about 350 families in Michigan and 15,000 nationwide.
“We’ve got something we never had before,” Kenneth Hobby, president of CureSMA.org says. “SMA has changed as a community. We’ve changed from just coping and having hope. We’ve got something we can actually do something practical with right now.”
But since that groundbreaking day in late December, very few patients have received this potentially life-saving drug. Across the country, Hobby estimates, there are only two or three hundred people in 243 states.
Here in Michigan there is only one patient currently on the drug - Channel 7 anchor Stephen Clark’s 4-year-old granddaughter Scarlet. And that happened because a small Muscular Dystrophy clinic in Farmington Hills stepped forward and offered to administer the drug.
“We understood the importance to all the SMA families,” Sonda Rossman of the Michigan Institute for Neurological Disorders says, “So we made it a priority to work on our processes and protocols and come up with a way to administer this. And as you know we successfully administered Spinraza to Scarlet on January 12th 2017.”
It was stunning news to Jennifer Petzke, who has been relentlessly imploring local hospitals to administer the drug.
“They’re just not ready yet is what I’ve been getting,” the Whitmore Lake teacher says. “There’s not much communication from any of the hospitals - its kind of hush-hush. Some of them have sent out letters saying they were only going to do certain types of kids, some of them say you just have to come get on the waiting list. There’s not really clear communication yet. Its very frustrating because you have a drug that could potentially help your kid and you can’t have access to it. Every day is less muscle for him and every day it progresses a little more.”
So why can a small private clinic do what our big hospitals are either unable or unwilling to do?
“We’ve never had an approved therapy in SMA before,” Kenneth Hobby points out. “So a lot of the hospitals that know SMA, know SMA patients, don't have the set up for an approved therapy.”
Plus Hobby says big institutions were caught flat-footed when the FDA approval came months sooner than expected and for a much broader type of patient.
As a result only a couple dozen sites in 24 states have started offering Spinraza. Here in Michigan there is only one so far.
And it is showing positive results in the early stage of treatment. Scarlet can sit unsupported and unassisted for short periods of time - something she’s never been able to do. And she is starting to show strength and movement in her arms and legs that disappeared as the disease began to take hold three years ago.
This is all welcome news to families like Jennifer Petzke’s who not only have hope that there is a drug that works, but now have at least one place where they may be able get it.
Statement from MIND Director Sonda Rossman regarding Spinraza:
We’re obviously not going to be able to accommodate all families. We’re going to have to see what the volume is. I know the need is out there. We want to do whatever we can but hopefully as the larger institutions get onboard and are able to administer then all families can be serviced in Michigan and elsewhere around the country.
To contact The Michigan Institute for Neurological Disorders Spinraza Hotline: (248) 553-6220
Statement from University of Michigan Medical Center regarding Spinraza:
Michigan Medicine prioritized the approval of Nusinersen/Spinraza to bring the medication as quickly as possible to our patients after a thorough evaluation that placed the safety and well-being of our patients at the forefront.
We plan to offer Nusinersen/Spinraza to patients who meet our treatment guidelines who have all types of SMA and are 14 years old and younger. The age distinction was made because there is no research into how this drug affects anyone older than 14 or what side effects it may cause. We are eagerly awaiting data that shows effectiveness and safety in patients over 14.
Statement from Beaumont Health regarding Spinraza:
Before offering a new medication or treatment for patients, Beaumont Health takes a careful, methodical approach to evaluate the drug or treatment’s safety and efficacy. Spinraza is currently in our evaluation process, which requires multiple levels of review and can take several months.
We examine safety and patient risks; compare the drug’s effectiveness with other medications; and review the drug’s cost and value for patients. We also establish guidelines for who will be eligible for treatment.
Once a drug is approved for use at Beaumont, we fully educate our staff regarding its administration.
We appreciate that patients are anxious to have access to a new drug that might help them. We have a responsibility to ensure any new drugs added to our formulary are safe, effective and will be of value for patients.
Statement from DMC Children’s Hospital regarding Spinraza:
Our pharmacy is still working through what the exact process/guidelines will look like and still in discussions on what the financial implications will be. Unfortunately, we are not in a position to make an official statement just yet.
Much has been said about the staggering cost of Spinraza - just over $1.1 million dollars in the first two years, $375,000 every year after that for life.
Kenneth Hobby, president of www.cureSMA.org, says that’s not too far out of line for “orphan” drugs, used to treat a relatively small number of patients.
Plus, he points out, the drug in trials has shown to be extremely effective, making it even more valuable.
Even though insurance companies, like hospitals, continue to study and debate the value of Spinraza, some insurers have already begun to approve the drug.
UnitedHealth group, America’s largest insurer will cover the drug in most cases. Anthem and Humana will only cover Spinraza for infants for now. But the company marketing Spinraza, Biogen, assures me that no patient will be turned away because they can’t afford the drug.
Below is the statement from Biogen regarding access to Spinraza and affordability:
SMA is a very challenging disease, and treatment with SPINRAZA can be complicated for some patients. Treatment frequently requires a well-coordinated, multi-disciplinary team, and hospitals often find they have to tailor an approach for each individual with SMA, many of whom are infants in fragile condition. In many instances we see multiple hospital departments working together in ways they have never had to before.
We are encouraged that each week we are seeing an increased number of sites treating patients with SPINRAZA. Yet we also recognize that we all have much work to do to achieve our common goal of making sure that every patient who needs SPINRAZA receives it. Biogen is committed to working with hospitals, healthcare professionals, patient advocacy groups and payors to tackle this challenge together and get this therapy to the people who need it.
As part of our ongoing commitment to the SMA community, Biogen has developed SMA360°™, a comprehensive patient assistance program to support SPINRAZA patients. Biogen’s SMA360° support provides certain services that address nonmedical barriers to access. These include logistical assistance, product education, insurance benefits investigations, and financial assistance. A complete list of the SMA360° offerings can be found at www.SPINRAZA-hcp.com/support