Facing the practical problems of gene therapy | AMCP Annual 2026
Providers and payers certainly face some formidable challenges when it comes to gene therapy, but it pales in comparison to what patients go through.
“It is a huge commitment to go down the path of gene therapy,” said Brian MacDonald, Pharm.D., senior director, specialty clinical strategy and programs at Prime Therapeutics.
There are pretreatment workups and often the need to go through what is essentially chemotherapy to condition cells, notes MacDonald, and then postadministration follow-up and monitoring that, depending on the product, could be a several months to almost a yearlong process. “And then with that comes just all of the real-world factors and considerations — time off of work, travel, lodging, caregiver support … psychosocial factors that have to be weighed. It’s a huge decision for patients certainly to take on.”
MacDonald and his colleague, Soumya Vishwanath, Pharm.D., director, formulary management, at Prime, shared practical insights into gene therapy at a
Vishwanath said that health plans have responded to the difficulties that patients face with case management programs. The plans are taking a “holistic approach,” not just focusing on the drug, and are identifying different care coordination teams to make sure “the patient has an appropriate treatment journey and that they're working with the manufacturer to make sure the drug is given to them at the right place, at the right time.”
MacDonald and Vishwanath discussed the operational challenges for providers that include staffing, meeting manufacturer requirements and FDA-required risk evaluation and mitigation strategy (REMS) programs.
Vishwanath said health plans are taking a tiered approach to utilization management of gene therapy that starts with the FDA label but also factors in inclusion and exclusion criteria used in clinical trials.
MacDonald noted that payers also need to track how long the clinical benefits of a gene therapy last and follow patients even if they have a lapse in insurance coverage or wind up getting covered by another health plan.
The patient, provider and payer challenges with gene therapy overlap, MacDonald said. “This is a three-circle Venn diagram where there are shared concerns all over the place,” he said.
