Expert Tips for Handling an Insurance Claim Denial for Gene Therapy

When Alison Joseph and William Small’s two youngest sons were diagnosed with Duchenne muscular dystrophy (DMD) in 2017, they were told there were no treatment options.

“Because of their specific mutation, they didn’t qualify for exon-skipping or gene therapy trials. We were just doing standard-of-care steroids, hoping for something new,” Alison says.

In 2024, hope arrived when the US Food and Drug Administration (FDA) approved a new gene therapy — Elevidys, developed by Sarepta Therapeutics — for ambulatory individuals at least 4 years old with DMD. They wanted to pursue it right away.

A difficult decision

The Small brothers, Hunter, 11, and Noah, 9, share the same exon-5 deletion — a rare mutation in DMD that had previously excluded them from clinical trials. During initial testing, doctors noticed that Hunter had cardiac abnormalities, while Noah’s heart looked healthy. Alison and William decided not to dose Hunter at the time, believing that was the safest choice.

Noah’s insurance approval for Elevidys came through in November 2024 — no denials, no appeals. Next, the family waited for the hospital to finalize a payment agreement with Blue Cross Blue Shield and order the therapy. This is a normal part of the process for new therapies, and it can involve weeks or months of waiting. Noah finally received his infusion in March 2025.

“He did really well,” Alison says. “Just some nausea early on, but cardiac and liver-wise, he was great.” The difference was immediate and striking. “Before gene therapy, he’d complain about end-of-day fatigue and leg pain several times a week. Since the infusion, not once. He has more energy, more endurance — people who haven’t seen him in months can’t believe the change.”

The denial no one expected

A few months later, the family repeated Hunter’s testing and met with their care team. By this point, more boys had been safely dosed with Elevidys, and combined with how well Noah was doing, the team felt confident it was the right time to move forward with Hunter’s treatment.

The family thought it would be a familiar process. “Same disease, same mutation, same insurance,” Alison says. “We thought it would be straightforward.”

It wasn’t. Their claim was denied, their first appeal was denied, and the following peer-to-peer review was also denied.

Determined not to give up, Alison crafted a strong appeal letter addressing the insurance company’s stated reasons for denial. She also contacted her employer’s HR department, Arkansas state officials, and the Office of Personnel Management in Washington, DC. The family even appeared in a national TV news segment highlighting their case, and shortly after it aired, they got the call they’d been praying for.

“When I saw ‘Blue Cross Blue Shield Federal’ pop up on my phone, I almost passed out,” Alison remembers. The denial was overturned, and after three months of back-and-forth, they were finally able to move forward.

A challenging system

New, single-dose gene therapies are the most expensive drugs on the market, with Elevidys priced at $3.2 million. This makes them out of reach for most people without insurance coverage.

“We were even looking into selling our house,” Alison says. “It just didn’t seem fair that I have insurance, and we would have to bankrupt ourselves to get an FDA-approved treatment.”

Unfortunately, Alison’s story is one that many families in the neuromuscular community can relate to. Insurance denials are a common hurdle, and the challenges often multiply as children age into adulthood and take on their own health and coverage decisions.

Research from Texas Children’s Hospital and Baylor College of Medicine found that many teens and young adults with chronic conditions feel unprepared to handle insurance on their own as they move into adult care. Even those with coverage often struggle to understand benefits, face limits on which doctors they can see, and encounter unexpected costs that delay or derail treatment. The study concluded that health insurance “is a complex system that directly affects young people’s ability to manage their health and transition successfully.”

These findings echo what families like Alison’s face: insurance literacy and access can make or break the path to timely care.

4 steps to insurance approval

According to Lindsey McKinnon, Senior Specialist in MDA’s Resource Center, navigating health insurance approvals for gene therapy can be overwhelming and challenging, often requiring patience and careful organization.

“There are proactive steps families can take to stay organized and improve their chances for approval,” she says.

To help make the process feel a little less overwhelming, Lindsey breaks it down into four practical steps:

Step 1: Understand your coverage.

Request your entire insurance policy — not just the summary. Review benefit exclusions, deductibles, and what is considered investigational.

Step 2: Request a case manager.

Having one consistent point of contact at the insurance company streamlines communication and helps you track next steps.

Step 3: Work closely with your care team.

Make sure your healthcare provider compiles clear, well-documented medical evidence. Ask who on your care team handles prior authorizations and appeals.

Step 4: Build a strong letter of medical necessity.

Structure your letter in a two-column format: one column lists the insurer’s stated criteria; the other shows how your case meets each point. “Keep it factual, concise, and tied directly to their language,” Lindsey says.

The Texas Children’s Hospital study found that fewer than 1 in 5 patients ever had insurance explained by their pediatric provider. Families are largely left to figure it out alone — which is why MDA’s Access to Coverage workshops and one-on-one guidance through the Resource Center fill such a critical gap.

Common denial reasons — and what you can do

Denials can still happen, even with a strong application or appeal. Understanding common reasons, which are listed below, and how to respond, can help you take the next steps.

Denial: “Does not meet plan criteria.”

Insurers may rely on narrow clinical trial criteria rather than the broader FDA-approved label. Ask your doctor to highlight what’s actually in the drug’s label.

Denial: “Investigational or experimental.”

Even FDA-approved treatments sometimes receive this denial. You or your doctor can submit a request for an external review by an independent neuromuscular specialist. Setting clear response deadlines at this stage can help prevent further delays.

Denial: “Benefit exclusion.”

Some insurance plans omit gene therapy entirely. In this case, families can explore other options:

• If you have health insurance through your employer, ask Human Resources if an override is possible.
• Check if your child may qualify for Medicaid as secondary coverage.
• Review a spouse’s plan or Health Insurance Marketplace (HealthCare.gov) options during open enrollment.
• Rally community support or engage local media and elected officials.

In combating denials, persistence and escalation are key.

Understanding the appeals process

When an insurance denial happens, it sets in motion a structured appeals process — one designed to ensure your case receives a fair review. To follow this process effectively, it helps to know how it works.

Internal appeal: Reviewed within your insurance company

• Level 1: Peer-to-Peer Review. Your doctor speaks directly with the insurance company’s medical reviewer to explain the medical need for treatment.
• Level 2: Medical Director Review. A new reviewer within the insurance company re-examines the decision.

External appeal: Reviewed by an independent third party

• Level 3: Independent External Review. A specialist outside the insurance company, often with neuromuscular expertise, makes a final determination. You or your doctor can request this review.

Families usually have 60 days to request an external appeal, which can be requested after a Level 1 or Level 2 denial. Insurers typically have up to 30 days to respond to each internal appeal, meaning the full process often takes two to three months. However, expedited appeals can be decided within 72 hours if the condition is considered life-threatening.

“MDA can help families at any stage,” Lindsey adds. “We have an insurance worksheet and an Access to Coverage workshop that walks through the process step by step. Our Gene Therapy Support Network is available to answer questions, review policy documents, and offer guidance.”

Her parting advice mirrors Alison’s lived experience: Be persistent, and don’t take no for an answer.

Gratitude and hope

Hunter received his gene therapy infusion in October 2025, and today, the brothers are playing with newfound strength. The family knows there are still uncertainties. Elevidys can only be administered once, and long-term safety data only dates back to 2018. Still, Alison says the goal is clear: preserve her sons’ strength and mobility as long as possible.

After years of advocating for her boys, Alison wants systemic change, so fewer families have to wage the same battle.

“Fighting the disease is hard enough,” Alison says. “You shouldn’t have to fight for your care.”

Emily Blume is a freelance journalist living in Washington state.

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Insurance and Gene Therapy Resources

These resources from MDA can help you navigate the health insurance process and learn about new therapies.

• Access to Coverage Workshops: On-demand online educational sessions on approved treatments, the appeals process, and other insurance issues.
• Health Insurance Worksheet: Helps families compare and choose the right health insurance plan. Find it under “General Neuromuscular Disease Resources.”
• MDA Advocacy: Learn how to contact your elected representatives and advocate for access to therapies.
• MDA Gene Therapy Support Network: Trusted guidance, access to care, and support for individuals and families at every step.
• MDA Resource Center: MDA Specialists can provide support and resources to families every step of the way. Call 833-ASK-MDA1 or email ResourceCenter@mdausa.org.
• MDA Toolkit: Checklists and resources to keep you in control and organized as you navigate insurance and healthcare landscapes.
• Navigating Insurance: Gene Therapy: A guide to understanding coverage, approvals, and appeals. Find it under “Gene Therapy.”