Gene therapy may be a once-in-lifetime treatment. That gives some patients and providers pause and a willingness to ‘wait it out’ until a better therapy comes along | AMCP Annual 2026

While gene therapy for hemophilia has struggled to catch on, the gene therapy for spinal muscular atrophy “seems to be a real success story.” said Brian MacDonald, Pharm.D.

That difference and others, said MacDonald, senior director of specialty clinical strategy and programs at Prime Therapeutics, makes “it hard to apply lessons from one condition or disease state to the others.” Still, MacDonald discussed some of the factors that might explain the low utilization of the gene therapies for hemophilia in an interview with Managed Healthcare Executive (MHE).

MacDonald and his colleague, Soumya Vishwanath, Pharm.D., director, formulary management at Prime, spoke yesterday at the 2026 Academy of Managed Care Pharmacy Annual Meeting at a session focusing on practical insights into gene therapy. MacDonald and Vishwanath were interviewed by MHE via Zoom before the meeting, which started Monday and ends tomorrow. Approximately 4,000 pharmacists and other healthcare professionals are attending the meeting, which is being held at the Music City Center in Nashville, Tennessee.

In his conversation with MHE, MacDonald spoke about the great potential of gene therapy and the help that he, Vishwanath, and other members of his groups provide to the 18 Blues plans that use Prime as their pharmacy benefit manager in managing the delivery and cost of gene therapies, many of which are priced in the millions of dollars. He noted, though, that some gene therapies have had lower utilization than expected and named the gene therapies for hemophilia as a prime example.

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, off the market. Both companies cited limited demand as a reason for the withdrawals.

“In hemophilia, what we saw in talking to providers was a situation where patients can often be hesitant to change treatment when they are stable and when their bleeds have been well managed on their current therapy,” MacDonald said.

Providers and patients have also held back, he said, because they view gene therapy as a single opportunity. “There may be a chance, from a coverage standpoint, that they only get one bite at the apple,” MacDonald said.

Providers and patients also want to be sure that gene therapy’s effect lasts if there is only one opportunity for such a treatment, MacDonald said.

“For the provider and for the patient, they wanted to really feel like when they chose to go down the path of pursuing gene therapy administration, that it was going to be a product that would be durable and would provide that sustained benefit for that patient,” he commented. “And so I think there have been providers and patients who were sort of willing to wait it out and wait for the second generation, sort of iterations of some of these products, to come to the market that may provide perhaps incremental boosts in either clinical effect or safety or both.”