The Future of Gene Therapies and Their Impact on Payers | AMCP Annual 2025

Gene therapies continue to evolve, with a number of promising treatments expected to receive FDA approval in the upcoming months.

Typically, gene therapies are considered one-time treatments, but with particular conditions, new approaches are on the rise that may require repeat administration, according to Maria Lowe, Pharm.D., BCPS, associate vice president of Pharmaceutical Intelligence at the Institute for Clinical and Economic Review (ICER).

Lowe discussed the latest developments in gene therapies at the AMCP 2025 annual meeting in Houston, where she highlighted the potential approval of many gene therapies this year, particularly in the areas of epidermolysis bullosa, Fanconi anemia and neurology.

“I think more than half of the 23 emerging products that I’ll be highlighting, more than half of them are already at the FDA with an FDA decision expected sometime this year,” Lowe said.

One of the therapies under review is pz-cel, a treatment for epidermolysis bullosa expected to receive an FDA decision by the end of April.

Although there are many gene therapies that are designed as single-dose treatments, pz-cel and similar therapies in this space may require repeat administration over time.

This represents a shift in the gene therapy model, raising concerns for payers regarding long-term costs, she mentioned.

Other therapies expected to reach the market include RPL-102 for Fanconi anemia, which has initiated its Biologics License Application (BLA) process.

In addition, Lowe discussed the challenges of pricing and coverage for repeat-use gene therapies, noting that traditional one-time treatments allow payers to accept high upfront costs by eliminating long-term treatment costs.

However, she mentioned that therapies requiring multiple doses create uncertainty about long-term expenses.

“I think the main challenge is we’re talking about a much more significant expense, or the potential for a more significant expense over time,” she said. “Instead of having a price for a treatment that you expect to be delivered once, and that’s it, it’s difficult to know how many rounds of treatment patients might require with these agents and for what period of time throughout their life.”