Cell and Gene Therapy: Becoming More Ordinary. Still Extraordinarily Expensive

Familiar drugs such as Humira (adalimumab) and Stelara (ustekinumab) still top the list when it comes to drug costs. The glucagon-like peptide-1 agonists such as semaglutide, sold as Ozempic for diabetes and Wegovy for weight loss, have joined their ranks.

But now many ultraexpensive cell and gene therapies are in late-stage development, and a number of them are on the brink of FDA approval. Healthcare systems and payers are bracing themselves for an era when cell and gene therapy will be an everyday clinical reality rather than a clinical rarity or a promising research project.

Many different parts of a healthcare system — finance, pharmacy, medical and social work, to name just a few — need to work together to administer cell and gene therapies because of their inherent complexity and price tags that can run into the millions of dollars for a single dose, notes Carina Dolan, Pharm.D., M.S.Pharm., associate vice president, clinical oncology, pharmacoeconomics and market insights at Vizient, a group purchasing organization headquartered in Irving, Texas.

Carina Dolan, Pharm.D., M.S. Pharm.

“It is really a very holistic approach to therapy because these therapies are so complex. No one department in a health system can do it alone,” says Dolan.

Earlier this year, IQVIA published a report about global trends in pharmaceutical research and development that said there were 631 trials of cell and gene therapies started in 2023, with the majority (64%) sponsored by industry. IQVIA’s tally shows that while nonindustry trials have remained relatively flat over the last decade, industry-sponsored ones have shot up 276% from 2013 and 34% from five years ago. Much of that surge, according to IQVIA, comes from the increase in the number of CAR-T therapy trials. Just four industry-sponsored trials were started in 2013, compared with 150 starts in the three-year span of 2021-2023. According to IQVIA’s tally, 114 gene therapy trials were startedin 2023, a large majority (88 of 114, or 77%) of which were sponsored by industry.

Vizient convened its first-ever symposium on cell and gene therapy earlier this year, Dolan said 120 health systems, payers and manufacturers attended to discuss the operational and other challenges of implementing cell and gene therapy. Some academic health centers have experience with the therapies because their faculty members were investigators involved in clinical trials, but Dolan says commercialization brings its own set of challenges, including the negotiation of single-case payment agreements with payers for individual patients. “A single-case agreement means that one time — one patient, one drug,” Dolan says. Negotiations for those agreements need to start well before the patient is treated.

The Vizient report lists nine treatments for genetic disorders that could be approved by the FDA this year and next. Although they work at the genetic level, not all of the therapies require special handling or clinical administration.

Among the drugs listed by Vizient list are Miplyffa (arimoclomal), a treatment for Niemann-Pick Disease Type C with an estimated wholesale acquisition cost (WAC) price of $250,000 to $500,000; n-acetyl-l-leucine, also a treatment for Niemann-Pick Disease Type C with an estimated WAC price of $250,000 to $500,000; Upstaza (eladocagene exuparvovec), a one-time intravenous therapy for patients 18 months and older with aromatic L-amino acid decarboxylase deficiency with an estimated WAC price of more than $3 million; govorestat, a treatment for galactosemia with an estimated WAC price of $250,000 to $500,000. The Vizient experts said in the report that they anticipate 35 cell and gene therapies to be on the market by 2026 and expenditures to reach $25 billion.