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The United States of America is famous for medical innovation. Our country leads the world in the number of new drugs and medical devices that are given the green light by our government. Unfortunately, this does not mean that every approved new therapy makes it easily to the patients who need it. The complexity of our health care system can create a frustrating and even intimidating process for patients and their families to get access to a medication that has been prescribed by a physician. That’s where people like me come in — as Head of US Value & Access at ITF Therapeutics, my job is to support people as they navigate the process of getting a prescription filled, starting treatment, and continuing on therapy as long as medically appropriate.
My team works with insurance companies to help them understand the value that rare disease medications can bring to patients and the role of new therapies in the evolving DMD treatment paradigm. Speaking for all of ITF Therapeutics, and my team in particular, we are committed to appropriately supporting patients and their caregivers as they navigate the often-confusing insurance process. This includes supporting communication with insurance companies, providing educational resources and financial assistance, and – when appropriate – supplying drug without charge to eligible patients facing insurance challenges.
After more than 20 years working in patient access, I know it may feel discouraging when a treatment is available and prescribed by your physician, yet instead of ordering it up for immediate delivery you have to wait and sometimes even fight for access. But do not despair. The DMD community is incredibly strong and supportive, and lots of help is available.
Little Hercules Foundation is a nonprofit organization dedicated to helping families access FDA-approved therapies. To quote Founder Kelly Maynard, whose son Jackson has Duchenne, “Sick kids should not be getting denied an approved treatment.” The foundation has an incredible team working to help educate payors and assist families in getting access. Contact the foundation if you’ve gotten an initial denial from your insurance company or if you just want to learn more about the process of obtaining access to treatment.
You can also find helpful information related to treatment access and coverage in this resource center created by the nonprofit organization Parent Project Muscular Dystrophy.
With newly approved treatments, many patients get denied coverage on their first try. A denial does not mean the insurance company does not believe the treatment may help you, or that they ultimately will not cover it for you. It’s just the way our system works – there are often multiple steps involved to getting an individual on therapy. Stay strong and seek help – remember that you have an empowered community at the ready to help guide you through the process.
C-DUV-US-0088-2 12/2025
