Gene therapy brims with promise. The reality has been less rosy for some therapies | AMCP Annual 2026

To prepare for his presentation at the 2026 Academy of Managed Care Pharmacy (AMCP) Annual Meeting, Brian MacDonald, Pharm.D., tallied up the number of gene therapies that had been approved by the FDA.

An even 20 have crossed the approval finish since the FDA approved Luxturna (voretigene neparvovec), the first gene therapy for an inherited disease, in December 2017, by MacDonald’s count.

With all those approvals “comes the potential clinical benefits that gene therapy promises, the idea of, hopefully, durable clinical impacts for these patients who may have little or no other treatment options,” said MacDonald, senior director, specialty clinical strategy and programs, at Prime Therapeutics, a Minnesota-based pharmacy benefit manager.

MacDonald and his colleague, Soumya Vishwanath, Pharm.D., director, formulary management, at Prime, spoke yesterday at the AMCP annual meeting at a session about practical insights into gene therapy. They spoke to Managed Healthcare Executive via Zoom before the meeting, which is being held at the Music City Center in Nashville, Tennessee.

Vishwanath noted that one of the misconceptions about gene therapy is that they are all single-dose cures. "There are different ways that these gene therapies work. Some of them are single administrations. However, others are multidose administrations," she said, adding that multidose administration can add a layer of complexity.

Prime is the pharmacy benefit manager for 18 Blues plans. MacDonald said he, Vishwanath and their colleagues help the plans prepare for gene therapy delivery and claims by reviewing the inclusion and exclusion criteria from clinical trials that led to the therapies' approval, some claims-based analysis, actuarial data, and other information. He described it as “kind of the art and the science” to help the plans estimate how people that they cover might be eligible for gene therapy.

For certain diseases, he said, “the volume and the utilization certainly have been pretty low," and his group also spends time figuring out the reasons for that. “What was the miss? What was the disconnect? Was it on the patient end? Is it on the provider end? Depending on the disease state, I think the answer varies,” MacDonald said.

“There was a lot of work that went into trying to figure out who these members might be that would seek gene therapy, and then, ultimately, seeing several years down the road that, you know, maybe the members and the providers haven't sought it out quite like we thought,” MacDonald continued.

The hemophilia gene therapies are one example of the uptake falling short of the forecasts, MacDonald said. Pfizer announced in February 2025 that it was taking Beqvez (fidanacogene elaparvovec), its gene therapy for hemophilia B, off the market, and BioMarin is pulling Roctavian (valoctocogene roxaparvovec), its gene therapy for hemophilia A. Both companies cited limited demand as a reason for the withdrawals.