The potential for gene therapies is rapidly expanding, but not without barriers

The use of gene therapies may turn from a trickle into a torrent over the coming years, prompting the need for hospitals to consider relevant patient populations, policies guiding safe use and payer strategies, among other key issues.

Kyle Herndon, PharmD, MBA, BCPS

“The first and most important factor driving this focus is how gene therapies may benefit the patient, since these therapies are designed to treat diseases that have been historically incurable,” says Kyle Herndon, PharmD, MBA, BCPS, the PGY-2 Corporate Pharmacy Leadership Resident in Pharmacy Services for HealthTrust.

Realizing unmatched potential

Defined as the transfer of genetic material to a patient to treat disease (by replacing a faulty gene or adding a new one), gene therapy is meant to be a one-time dose, unlike small-molecule drugs or biologics that are taken regularly for chronic diseases. Such therapies are in development to treat rare diseases ranging from spinal muscular atrophy to scleroderma, as well as common conditions such as heart failure and osteoarthritis.

Only two gene therapies are currently available in the market, Herndon says, not including the widelyheralded CAR-T cell therapy for certain blood cancers. But by 2025, former Commissioner of the Food and Drug Administration (FDA) Scott Gottlieb, M.D., predicts that:

10 to 20 new gene therapy products
could be approved each year

“While gene therapy isn’t heavily utilized now,” Herndon says, “there are many in phase 3 clinical development, so we can expect use to increase as more products become approved and as more standardized policies regarding their use are developed over the next five years or so.”

Removing regulatory roadblocks

Gene therapy technology is currently outpacing the regulatory structure surrounding its use. Herndon notes that the FDA hasn’t yet provided guidelines regarding safe handling of gene therapies. And while the National Institutes of Health has stratified viral vectors (which deliver genetic material into a part of the body) into risk groups, guidance on minimizing exposure and risk is largely incomplete.

Fortunately, a handful of regional academic medical centers that have emerged as early adopters of gene therapy have created their own policies and procedures guiding operational use, some of which are available online. These include the University of North Carolina and the University of Kentucky.

“If another institution was going to become adept in the gene therapy field and start using these products,” Herndon adds, “a good place to start is to look at what peer hospitals are doing.” Meanwhile, the FDA released a slate of policies in January 2020 related to assessing gene therapies, which arrived as more than 900 investigational new drug applications were ongoing for gene and cell therapy clinical studies.

“The FDA has acknowledged that we’re now in a pivotal time for gene therapy, and they’re supporting it,” Herndon says.

Considering cost

As a one-time treatment, gene therapy is expensive, due to high costs for manufacturing and research and development. With a per-patient cost reaching several hundreds of thousands of dollars—or even into the multimillion-dollar range—for just a single dose, Herndon urges hospitals to consider various payer strategies. Among others, Herndon says, these could include annuity-based payment, outcomes-based payment or an outcomes-based rebate.

“There’s no real consensus in practice right now surrounding the best and most applicable payment methodology,” Herndon explains, adding that gene therapy developer Spark Therapeutics has experimented with various mechanisms of payment and has engaged with the Centers for Medicare & Medicaid Services (CMS) to explore the economic impacts of patient installment payments and higher rebates tied to clinical outcomes.

The most viable option in Herndon’s mind is the outcomes-based metric. “I think whatever evolves with payment will probably be based on patients meeting prespecified outcomes,” Herndon says. “Because these treatments are one-time doses intended to last the entirety of the patient’s life, there may be checkpoints: In five years, how is this patient doing? Have they relapsed?”

It’s clear we’re on the brink of discovering what the future holds. “If the FDA is supporting the innovation, it certainly means it’s going to become a reality,” says Herndon.

Learn more about considerations in gene therapy by accessing a webinar Herndon delivered recently for HealthTrust members.

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