Both Prime Therapeutics (Prime) and Academy of Managed Care Pharmacy (AMCP) value nurturing the next generation of managed care pharmacy professionals, which was a key objective of the student session “A beginner's guide to decoding cell and gene therapy” that kicked-off the second day of AMCP Nexus 2024 in Las Vegas, Nevada.

Led by Prime’s Sneha Sharma, director of specialty clinical solutions, and Michael Szatkowski, product development director of specialty, the session offered timely takeaways to a packed house of both students and professionals eager to learn, or at least get a refresher on the complex landscape of cell and gene therapy.

Sharma began with a high-level review of the current state of genetic disorders, noting that there are 10,000 known, distinct,  rare diseases today — 80% of which have a genetic origin. A staggering 30 million Americans have a rare disease, and 50% of those affected are children. Worldwide, there are 80 approved cell and gene therapies to help manage a range of conditions, including sickle cell disease, hemophilia and more. And today, there are more than 1,500 clinical trials on cell and gene therapy in progress, Sharma noted.

“While we’ve made so many strides in this category, there’s so much more we can do,” Sharma said.

She added that it can take up to five years and seven clinicians to diagnose a patient who may require cell and gene therapy and several months after that to tailor and administer treatment.

In one example, a young girl, identified as Misty, was diagnosed at 3 months old with a form of blindness. By the age of 3, she was legally blind. It wasn’t until she was 11 years old that she was diagnosed with Leber’s congenital amaurosis, which impacted Misty’s ability to see in low light. One year later, she was treated with voretigene neparvovec-rzyl (Luxturna^®) in each eye. Though she remains legally blind, Misty — now at 19 years old — is a horse trainer. 

As Szatkowski described, a key issue is the cost of these therapies. The average wholesale acquisition cost (WAC) of cell and gene therapy can range from $2–4 million for gene therapy and $300,000–600,000 for cell therapy.

“As a managed care pharmacist, two things come to mind. The future is bright and exciting … as these can be life-changing therapies,” Szatkowski said. “The second is how we manage the cost of care to make sure patients can get therapies that they need.”

As cited in the latest Prime Medical Pharmacy Trend Report, 79% of health plans cover current gene therapies, while 7% do not. For plans that don’t cover these therapies, managed care pharmacy professionals can utilize value-based contracts, improve access to data and ease provider collaborations to manage cost, risk and administrative burden to improve access to therapy, Szatkowski explained.

“As a patient, you can’t navigate this by yourself,” Sharma said. “As a pharmacist, your impact will be here.”

Stay tuned for more AMCP Nexus 2024 in focus content throughout the week in the Prime newsroom.