Medicaid can't opt out of gene therapies, so planning matters more, says Katelyn Meyer, Pharm.D. | AMCP Nexus 2026

Medicaid programs face a different set of problems than commercial plans when new cell and gene therapies come to market. They can't leave a drug off the formulary, according to Katelyn Meyer, Pharm.D., of MassHealth. Many of the rare genetic diseases these therapies target affect children, a group Medicaid covers in large numbers.

Meyer, a clinical pharmacy manager with MassHealth at the Massachusetts Executive Office of Health and Human Services, explored these issues in a session titled “Ahead of the Curve: Emerging Specialty Therapies and Managed Care Readiness,” this week at the AMCP Nexus 2026 conference in San Diego. In this interview with Managed Healthcare Executive, she explained what makes the specialty pipeline hard for Medicaid to plan for and how plans can prepare.

Coverage without a choice

Medicaid must cover drugs from any manufacturer that takes part in the Medicaid Drug Rebate Program, Meyer said. That includes drugs that earned accelerated approval or carry questions about how long their benefit lasts. This makes it hard to predict how many patients will need a therapy.

She also pointed to a trend of FDA approvals that reach beyond the patients studied in trials, such as a therapy tested only in children being approved for adults.

“For Medicaid, that puts us in a tough position because there's no data,” she said.

Paying for an uncertain benefit

Some manufacturers are willing to sign outcomes-based or value-based agreements tied to real-world results, Meyer said. Others aren't, and payers often have no lever to change that. Newer companies whose first product is a cell or gene therapy may not have the capital to pay out $1.5 million if a drug fails. She called the agreements worth pursuing but imperfect and “often administratively very challenging to actually do.”

Start with the standard of care

To prepare for therapies arriving in the next year or two, Meyer recommended a landscape review. In hemophilia, MassHealth hasn't seen much interest in gene therapy because patients already do well on existing treatments. A therapy that extends survival in a fatal disease fills a larger unmet need and will likely see more uptake. Centers that took part in clinical trials can also offer insight into real-world use.

However, she cautioned against doing too much too early.

“The FDA has been fairly unpredictable as of late,” she said, noting that expected approvals have been rejected and vice versa.