Neurologists Share Tips for Securing Patient Access to Gene Therapies

Transformative gene therapies for neuromuscular diseases won’t make it into patients’ hands without the tenacious efforts of clinicians, hospital administrators, and other advocates, experts said at a session of the 2025 Muscular Dystrophy Association Clinical & Scientific Conference in Dallas, Texas, as they shared their advice for driving the change to enable patients to receive these advances.

Session chair Leigh Maria Ramos-Platt, MD, clinical professor of neurology and pediatrics at the University of Southern California Keck School of Medicine, gave an overview of the potential roadblocks to delivering life-changing gene therapies for conditions like Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). These could include insurance coverage (whether commercial or Medicaid), hospital policy, and state and national policies. She promised a session that would include 3 clinicians sharing “their experiences navigating these blocks, pushing boundaries that are put up, and changing policy and insurance in individual, state, and national levels,” as well as a “very brave” hospital administrator “to show that administration doesn’t have to be a block in getting some of our therapies done.”

Neurologists need to advocate for patients at the individual, state, and national levels to ensure they can access life-changing gene therapies. | Image Credit: © lordn - stock.adobe.com

According to Lamar Davis II, MD, associate professor of pediatrics at University of Mississippi Medical Center, prior authorization and insurance approval for these novel therapies is a “significant battle, and one that’s always changing.” As a pediatric neurologist, his top tips include showing the payer not just the patient’s condition, but also the progressive and fatal nature of the disease course and the ramifications of inappropriate treatment—for instance, the $143,705 annual cost of SMA per household before the advent of disease-modifying therapy.¹

Like the classic Lion King song, Davis said, one needs to “Be Prepared” by creating templates for letters of appeal and anticipating every possible aspect that could be nitpicked, from name typos to ensuring Medicaid coverage hasn’t lapsed. Taking a cue from Captain America, the mindset that “I can do this all day” is helpful when arguing to insurance companies that a drug must be covered for an eligible patient.

Neurologists need to advocate for their patients, Davis explained, because “insurance companies are not going to knock on our clinic doors asking us if we have patients” who need treatment. “It’s truly an effort of just not stopping, because the science is on our side…ethics is on our side in terms of treating these kids. We just have to be willing to put in work and not stop.”

Moving up a level from the individual patient-clinician relationship to the hospital decision to offer a gene therapy program, Malika Maddison, MHA, MBA, RT(T), vice president of service line strategy and operations, Children’s Hospital Los Angeles, explained what hospitals are looking for in assessing these therapies. They want to have a business plan, understand the return on investment, and know that it aligns with the organization’s mission and vision. They also must communicate with the manufacturer to understand the delivery process, patient population, financing, and coverage.

Prior experience with the drug is enormously helpful: “If we were a clinical trial site, then likely that means we’re already comfortable infusing the drug. We already know who we're going to use it [for]. We already know what the possible reactions are going to be—that just makes it easier for us,” Maddison said.

When aiming to get a hospital on board, an advocate needs to show administration that creating a high-cost drug program is the only solution, whether the objective is enabling access to high-quality care, ensuring equitable care, minimizing financial risk, or some combination. Financial rewards may not always result in generating revenue, Maddison explained; they could at times entail a cost-avoidance strategy of averting the long-term costs of not intervening in the disease course.

Overall, the key is “problem solving to identify the problems and find creative solutions. Every solution is not always going to be right in front of your face. You’re going to have to dig forward and you’re going to have to find stakeholders, and I would encourage a multidisciplinary team of stakeholders. Sometimes they’re not always bringing the solutions, but sometimes they’re asking the right questions, and sometimes their questions are the contributions to a solution,” Maddison concluded.

This determined persistence can also be applied to collaborations at the state level to secure gene therapy coverage, according to the next speaker, Chamindra Laverty, MD, neurologist at UC San Diego Health. She described 2 examples of this cooperation that have made a real difference in getting treatment into the hands of California patients.

First, she mentioned the 2016 approval of eteplirsen (Exondys 51; Sarepta), which was the first gene therapy for DMD but provoked concerns about equity in access for patients on Medicaid.² Laverty and other California providers huddled to create guidelines on eligibility for coverage, pitch them to the state Medicaid program director, and eventually saw them adopted—even though they were broader than the clinical trial eligibility criteria—because they showed their reasoning.

“That gave me some sense of confidence and also peace, and I knew that what I was doing wasn’t disparate from other physicians in the same state,” Laverty said. She can also now assure patients that the drug is likely to be approved, even if they are on Medicaid, “and interestingly, it actually facilitated the private payers to cover the drug for the same reasons.”

The group has since repeated that process each time there is a paradigm shift with approval of a new high-dollar gene therapy, and “we were able to shape California policy for the Medicaid program as a result of us actually collaborating with each other.”

Her second example required national collaboration to demonstrate to payers that early intervention is necessary for young patients with 4 copies of the SMN2 gene, who were being wrongly classed as having “adult-onset” disease. She and colleagues across 22 sites collected natural history data to show that this genotype does result in early loss of ambulation and that disease-modifying therapy can stabilize or improve their condition.

This was not an easy task, Laverty said, requiring many individuals’ time, willingness, data use agreements, and review board approval across universities. Even recording patients’ date of birth was a challenge because it’s considered protected health information. This experience led her to conclude “we need a better way to actually collaborate across the 50 states.”

“I do think that with advancements in technology, that link of extracting that information can be made easier, but it does require people and that requires money, and I’ve learned that nothing good is cheap,” Laverty said, calling for government funding to maintain a national neuromuscular disease registry but acknowledging that this funding may not be forthcoming in the current political landscape.

Finally, Nicholas Johnson, MD, MSCI, FAAN, associate professor of neurology at Virginia Commonwealth University, presented tips on how clinicians can advocate for their patients at the national level. Practitioners are suited to deliver this message because they know best what their patients need and are accustomed to finding common ground amid competing interests, Johnson said.

“Most advocacy issues start with a champion,” Johnson explained. “You have to find an elected official who’s willing to influence others and move your issues forward in the process,” which could be someone with a personal link to neurological disease. When meeting with this decision-maker, Johnson suggested humanizing the issue by telling a patient story, then providing the facts and figures in a leave-behind document or follow-up email. He also noted the importance of speaking like one would to a patient by avoiding medical jargon.

“These skills can be learned; you can nurture them over time. I highly encourage you to do that. That’s never been more important than now,” Johnson said.

As the session closed, a patient in the audience thanked the panelists for their efforts on behalf of the neuromuscular disease community. “Those of us with neuromuscular disease are also often called inspirational, and needlessly so for just living our lives, but the efforts you’re doing are truly inspirational… It's very eye opening and I really appreciate what you’re doing.”

References

1. Chambers GM, Settumba SN, Carey KA, et al. Prenusinersen economic and health-related quality of life burden of spinal muscular atrophy. Neurology. 2020;95(1):e1-e10. doi:10.1212/WNL.0000000000009715

2. What we're reading: Anthem will not cover first drug approved for Duchenne. AJMC^®. October 10, 2016. Accessed March 19, 2025. https://www.ajmc.com/view/what-were-reading-anthem-will-not-cover-first-drug-approved-for-duchenne