With hundreds of experimental gene therapies in the pipeline and development, Eric Tichy, PharmD, division chair at Mayo Clinic and an expert in the pharmacy supply chain, told Becker's he wonders who will pay for these drugs that cost seven figures each.
Since its first gene therapy approval in 2017, the FDA has approved 28 cell and gene therapies. One of them, hemophilia B drug Hemgenix, costs $3.5 million. Although many others cost millions of dollars each, they offer a one-and-done, curative regimen that can save patients and providers money.
Multiple hospital pharmacy leaders, including Dr. Tichy, have told Becker's they're excited to see the disruption power gene therapies can have on healthcare. But, questions are still swirling about how this pricey class of drugs will be bought, used and paid for.
"How are these actually going to be delivered to patients and what is the payment model going to look like?" Dr. Tichy said. "No one wants to be left holding the bag in a situation where a payer decides they're not going to cover it. The cost is so extreme, the timeliness of how payments occur can also be financially material for organizations. There's a lot of concerns around those aspects. That needs to get ironed out."
Health systems, standalone hospitals and other organizations are still working to operationalize gene therapy use because no one — payers, providers and patients alike — wants to take the risk of a multimillion-dollar drug and lose the bet.
Dr. Tichy said the ideal payment model for these therapies is ensuring timely payments after acquiring a drug so it's not "sitting on the financial books" and ensuring fair reimbursement for providers who deliver the therapies.
Gene therapies usually target rare and very rare conditions — like sickle cell disease, which has at least two gene therapy candidates approaching an FDA approval decision date — so providers might be navigating a different practice standard for each drug and each payer.
There's room for hope alongside these lingering worries, Dr. Tichy said: "The reason why it's exciting is you're actually curing a disease as opposed to managing the symptoms or giving something that will temporarily alleviate the condition. That's what makes it really special."