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Challenges with SMA Treatments

Last updated: April 2022

We asked our team of advocates: What challenges have you faced around SMA treatment options?

Portrait of SMA Advocate Allie Williams

Insurance approval challenges for adults with SMA

Allie: Due to how the approval of the medication process goes with the trials, which often do not go over the age of 21, sometimes older adults have issues getting insurance approval because of our age. The testing is also not made for minute improvements, quality of life, or even progression halting, which the medications are meant to provide.

Insurance wants to see huge numbers of gains on scales that many of us don’t even fit on, to begin with, and it can be a hassle getting pre-approved.

I was unable to get Spinraza due to insurance not approving it in my state, but also due to the lack of administrators that could actually give the med to an adult with a fused spine.

Portrait of SMA Advocate Samantha Przybylski

Age as a factor in accessing SMA treatments

Samantha: Some challenges I have faced around treatment options with SMA have been whether or not I would qualify due to age, how do I obtain the medication, and what are the requirements needed for insurance to approve me and pay for the medication.

As an adult with SMA, not all treatments are approved for those over a certain age. Often as people with SMA grow older, they age out of their pediatric resources.

Wanting to be on the treatment required me to find an adult neurologist familiar with SMA and the new treatments available. I had to come up with my own road map to get to my goal of treatment at the end.

Insurance approval can be difficult and, ideally, you need a physician who has jumped through those hoops previously to help you get approved. Do your research on the front end to help you get to your end goal of treatment.

Portrait of SMA Advocate Michaela Hollywood

Differences in treatment approvals in the UK

Michaela: As someone living in the UK, we are usually behind the US on approvals of new medications. I live in Northern Ireland and, sadly, we don’t really have equal access to clinical trial sites either, and to be able to get into clinical trials here usually means a weekly flight to another part of the UK or another European country!

However, the UK does have the NHS which provides free healthcare to citizens. That means that once a drug is approved for a license, and is evaluated for cost-effectiveness, we receive it for free via the NHS.

I’m lucky enough to receive Evrysdi on a Managed Access Agreement on the NHS. But before this was available, I also managed to access it before approval through an Early Access to Medicines Scheme - a new program where drug companies will provide treatment where there is severe unmet clinical need before it is licensed.

Accessing treatment can feel painfully slow. But it is possible. I have a background in lobbying and activism which definitely helped me in this, just as it does in other areas of my life!

Portrait of SMA Advocate Chaz Hayden

Treatment should be about stabilization

Chaz: The biggest challenge is insurance approvals, even after being on the medication for over a year. I think that insurance companies are convinced that SMA treatments will magically make me jump out of my wheelchair and run around and if I’m not doing that then in their eyes the medication isn’t working.

This is the farthest from the truth, especially for a patient at my age in their mid-twenties. Treatments are all about stabilization and the possibility of small, quality of life improvements like better speech and eating more efficiently.

Insurances need to get educated and it starts with drug manufacturers doing proper trials and explaining the benefits for each age group instead of lumping everybody together.

Portrait of SMA Advocate Ainaa Farhanah

Lack of treatment options in my country

Ainaa: Firstly I don’t have any option, living in Malaysia. I haven’t received the treatment.

Getting support from the medical team is very challenging. Especially for us adults. I tried to talk to the doctor regarding the treatment but the conversation was being cut off immediately. Maybe they assume that the fact we are already alive till now is good enough.

But deep down it hurts to hear it from the doctor themself. I think we all have the right to get treatment. If there is a treatment, we have the right to choose it. I wish all doctors will act with heart, compassion, and empathy. Not just by looking at the numbers. Adult patients might be little and rare to be seen but we deserve to be treated equally.

Portrait of SMA Advocate Mike Noon

Find the right treatment plan for you

Mike: I consider myself lucky in this department. I know insurance is the biggest barrier for us. I’ve been on Medicare for the last 10+ years. I have found Medicare very easy when it comes to approving treatments. This has been a huge decision when I think about trying to get back into the workforce.

I think it’s criminal that private insurance makes individuals who are dealing with a rare disease like SMA jump through hoops to get approval for treatments. These treatments can literally be the difference between life and death for some of us.

I want to leave you with one last piece of advice. Don't let someone else’s good or bad experiences on a treatment influence your treatment plan. We are all different, what works for me might not work for you. Find yourself a doctor who you feel works best for you. Discuss your goals with them and develop a treatment plan that is best suited for you.

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This article represents the opinions, thoughts, and experiences of the author; none of this content has been paid for by any advertiser. The SpinalMuscularAtrophy.net team does not recommend or endorse any products or treatments discussed herein. Learn more about how we maintain editorial integrity here.

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