Over the last seven years, the number of gene and high-cost novel therapies has accelerated. Many of the recently approved gene therapies are for pediatric diseases for which there are minimal treatment options.
Offering these complex therapies at Monroe Carell Jr. Children’s Hospital at Vanderbilt has required extensive coordination across multiple teams including providers, pharmacy, revenue cycle, finance, managed care contracting and risk.
The care is driven by the Pediatric Gene Therapy Workgroup, led by Hunter McIlwain, CPhT, MMHC, program director of Gene and Complex Therapies at Monroe Carell; Jonathan Soslow, MD, MSCI, professor of Pediatrics, co-director of the Gene Therapy Clinic, co-director of the Duchenne Muscular Dystrophy Clinic and director of Clinical Research for Pediatric Cardiology; and W. Bryan Burnette, MD, MS, professor of Clinical Pediatrics and Neurology, co-director of the Duchenne Muscular Dystrophy Clinic, director of the Vanderbilt CureSMA Care Center and interim chief, Division of Pediatric Neurology.
For their collaboration and commitment to delivering superior personalized health care, the Pediatric Gene Therapy Workgroup was honored with the Team Award during the September 2024 VUMC Leadership Assembly.
“The team’s work is a great example of Defining Personalized Care, which is what we strive for at VUMC,” McIlwain said. “There are many individuals across multiple departments working on each patient’s case. Each person I’ve worked with on the team has a desire to get it right and be part of something special for our patients.”
In May 2019, the first gene therapy for spinal muscular atrophy (SMA) — onasemnogene abeparvovec (Zolgensma) — was approved by the Food and Drug Administration. The first dose of the drug was administered at Monroe Carell in October 2019. Eleven patients have been treated to date.
In June 2023, the first gene therapy for Duchenne muscular dystrophy — delandistrogene moxeparvovec (Elevidys) — was granted accelerated approval by the FDA. The first dose was administered at Monroe Carell in November 2023, and five patients have been treated so far.
With multiple novel gene therapies with pediatric indications in the pipeline for FDA approval, the establishment of the workgroup at Monroe Carell, which consists of more than 30 members, enables efficient and effective response to these and other therapies to come.
“The focus and dedication of this team has driven the early success of our program and has put us at the forefront among centers offering these innovative therapies across the country,” Burnette said.
“This team has worked through an incredibly complex process to ensure that these medications are administered in the safest possible way,” Soslow said. “Despite the many challenges, all members of the team are focused on providing this life-changing care to our patients.”
One of the critical roles of this workgroup was the creation of therapy-specific workflows. Establishing a workflow for each individual therapy provides a framework for the complex process. Each key step, from identification of the patient requiring therapy through approval, administration of the dose, and confirmation of reimbursement, is defined and assigned to a specific team member, according to the nomination.
“There are a lot of approvals and complexities involved with gene therapies given their novel and expensive nature,” McIlwain said. “Gene therapy medications are not all created equal. Each of them has specific clinical guidelines and operational and financial considerations, so we take each one and create a very specific workflow of the steps and key team members involved. Our workgroup puts all the pieces together to make sure it’s a smooth process for our patients to get the specialized care they need. It’s especially important that our patients and families stay informed along the way as we get all the ducks in a row for their treatment.”
One nominator wrote about a child who received gene therapy for Duchenne muscular dystrophy.
“The Gene Therapy team at Monroe Carell is a multidisciplinary team of compassionate and dedicated individuals who work hard to support our Duchenne muscular dystrophy patients and their families,” the nomination read. “Without the efforts of every member of this team, our families would be challenged with finding other options for their child,” the nomination read.
“One specific case involved significant challenges with obtaining payer approvals while meeting the hopes of the family of an infusion date that aligned with the holidays,” the nominator wrote. “The combined efforts of the provider, managed care contracting, pharmacy leadership and pharmacy procurement pushing through the barriers resulted in the successful treatment of this patient and his ‘Christmas miracle.’”
McIlwain said the process developed by the workgroup can be applied to any future gene therapies that come to market. “We’re closely monitoring gene therapy pipelines so we know what’s coming and can be prepared. Once a new gene therapy hits the market that our providers and patients need access to, we’ll be ready to go. These therapies are groundbreaking and have the potential to change a person’s life in a big way. I’m glad I get to be part of it and work with such a great team.”
Watch the Leadership Assembly video about the Pediatric Gene Therapy Workgroup’s Elevate Team Award.
If you are a VUMC employee, you can nominate a colleague for an Elevate Credo Award, Five Pillar Leader Award, or Team Award. Visit the Elevate website to fill out a nomination form. Employees demonstrate credo behaviors when: they make those they serve the highest priority; respect privacy and confidentiality; communicate effectively; conduct themselves professionally; have a sense of ownership; and are committed to their colleagues. Elevate award nominations are accepted year-round. If a nomination is received after the cut off for an award selection period, the nomination will be considered for the next period. VUMC VOICE will post stories on each of the award winners in the weeks following their announcement.
