I’m infuriated when patients are denied enzyme replacement therapy

This past week has been heavy on my heart, with my mind full of anger and frustration. I just want to go to the driving range and hit a bucket of golf balls to release some of the stress. While these feelings are not about my journey, they are about other Pompe warriors.

When I found my tribe, I also found a new family. When they hurt and suffer, I do, too.

We live in a society that requires us to get referrals and approvals for many medical treatments and prescriptions. First, doctors diagnose a patient with a condition or disease. Then they prescribe a medicine or other treatment. You’d think it’d be smooth sailing after that, but in the U.S., our medical treatment and quality of life are usually put in the hands of health insurance companies.

Getting a diagnosis of Pompe disease is a hard enough pill for a patient to swallow, but it’s worse when you add to that a health insurance company denying the treatment, even if it’s been approved by the U.S. Food and Drug Administration (FDA) and prescribed by a doctor. For a newly diagnosed Pompe patient, this is heartbreaking.

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When denials are posted online

Pompe support groups on social media have their benefits. I get to meet and talk with people with Pompe from all over the world. They share their journeys and experiences while helping others. Many ask for advice on an issue or topic and are usually referred back to their medical professionals.

But when I read that insurance has denied a patient treatment and they need suggestions about what to do next, it doesn’t settle well with me.

Within the Pompe disease community, people are generally diagnosed by their neuromuscular specialist or a neurologist and get referrals for enzyme replacement therapy. But sometimes they share with the community that their insurance company has denied this treatment. How can insurance dictate whether a patient deserves a therapy that’s FDA-approved for their disease?

There’s no cure for Pompe disease, but a biweekly infusion with an FDA-approved drug can help slow the progression of muscle weakness. Yet in some cases, insurance companies still deny patients this treatment. It makes me angry and frustrated every time I see someone post about a denial. I wish I could do something to help. Why do we live in a society where this happens? Why isn’t there more care for the human being?

In my groups, Pompe patients have commented that they’ve needed two or three submissions to their insurance to finally get approval; in some cases, that took six months or longer. Through the process, the person has probably gotten weaker and the disease has demoralized them. This seems neither ethical nor fair.

Something needs to be done about it. How can we better advocate for others and ourselves?

Advocacy is the best response

As a Pompe disease patient, I have to advocate for my own treatments. I call, email, and video chat with doctors, including specialists, as much as possible, to the point where I probably annoy them with all my questions. But some people need more help in their corner. In those cases, drug companies sometimes assign case managers and workers to patients. I believe those workers need to be more involved to help advocate for the patients with their health insurance.

Health insurance companies in the U.S. need a wake-up call, and not just the customer service representative repeating a script they’ve been taught to deliver the bad news and then hanging up, as if the patient’s life doesn’t matter. Insurance companies need to know we won’t be silent. We’ll appeal their decisions and let them know that the Pompe disease community can’t afford to take no for an answer. I know I’m going to advocate for myself and every Pompe warrior out there.

Though the storm may beat me down, I’ll keep going. I have a passion not only to spread awareness about Pompe disease, but also to stand up for our community. This isn’t a sprint, but a marathon. Together we are stronger.

The journey “On the Road to Pompe” keeps going forward. It’s headed your way to more advocacy and positivity, and to more people understanding that Pompe patients’ voices do matter.

Note: Pompe Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Pompe Disease News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to Pompe disease.