Senate Bill 207 would ensure that all payments -- whether made by patients or on their behalf -- are applied to the patients’ out-of-pocket requirements, protecting families from having to decide whether to pay twice for their medications or pay for utilities, groceries or rent.
Pompe disease is a rare genetic disorder that disrupts the body’s ability to break down glycogen, causing it to accumulate in muscles including those that control breathing and heart function. This buildup leads to progressive muscle weakness, breathing problems, fatigue, and other serious complications. Without treatment, it can be fatal. Infantile-onset Pompe disease is the most severe form and is often fatal within the first year of life. Late-onset Pompe disease progresses more slowly and is debilitating and life-threatening if left untreated.
There is no way to know exactly how many people in Ohio have Pompe disease. Currently, there are at least 15 known pediatric cases and 5 adult cases, but the actual number is unknown because adult-onset diagnoses are often missed.
Our only approved therapy—enzyme replacement therapy (ERT)—is not optional and there are no generic options. It’s the only therapy that keeps people like me alive. ERT is complex, ongoing, and expensive. Copay assistance programs exist to help patients access life-saving treatments and therapies like ERT.
Recently, my insurer changed its policy on third-party copay assistance without notice. I only learned of this when I was told my ERT might no longer be covered. My experience highlights a broader reality: by exploiting copay assistance programs, insurers avoid paying their fair share for rare disease therapies, forcing patients into impossible financial choices.
This is not about luxury, it’s about survival. I’m not asking for special treatment. I’m asking for fair access to a medically necessary therapy that keeps me alive and functioning. Denying copay financial assistance is not just a financial decision, it’s a discriminatory denial of care by another name. Patients with rare diseases already face enough barriers: delayed diagnoses, limited specialists, and few treatment options. Financial gatekeeping should not be one of them.
Ohio can join more than two dozen other states in protecting vulnerable patients. Tens of thousands of Ohioans rely on copay assistance to afford their medications.
I urge the Ohio Legislature to act swiftly and vote yes on Senate Bill 207. Let’s ensure that no Ohioan is denied care simply because they cannot afford it, especially when help is available.
Heather Shorten is the founder and executive director of Pompe Alliance