Emerging Therapies Committee at UC Davis Hits the Accelerator Not the Brake | AMCP 2024

Creation of yet another committee is not usually the prescription for speedy action, but it worked out that way at UC Davis Health when it came to expensive cell and gene therapy.

When the Sacramento, California, health system relied on its traditional pharmacy and therapeutics (P&T) committee and the processes it had traditionally used to start the usage Spinraza (nusinersen), the new, expensive drug for spinal muscular atrophy, “it took months and months to that agent onboarded,” says Sarah Bajorek, Pharm.D., MBA, assistant chief pharmacy officer, ambulatory care, at UC Davis Health. There were multiple rounds of emails going back and forth and people waiting around to play their part in the admittedly complicated endeavor of inaugurating the use of a six-figure drug. “So we did a needs assessment and realized that we had a lot of waste and inefficiencies in the process,” Bajorek says.

From that understanding sprang UC Davis’ new emerging therapies committee, which could serve as the model for other healthcare systems grappling with high stakes issues and complexities posed by cell and gene therapies that have multimillion price tags.

Last year, UC Davis treated a patient with Elevidys (delandistrogene moxeparvovec) a $3.2 million gene therapy treatment for Duchenne muscular dystrophy, just 43 days after the FDA approved the drug in June 2023.

“We know there’s a lot of things in the pipeline that are coming,” says Jenny Craven, Pharm.D., who is in the newly created position of senior emerging therapies pharmacist at UC Davis Health. “We’re really trying to focus on how to make this reproducible and predictable and how to make sure there’s no surprises, as much as possible, when it comes to management of these agents.

Bajorek and Craven spoke on Tuesday about the emerging therapies committee and some of the lessons they have learned about delivering cell and gene therapy at the annual meeting of the Academy of Managed Care Pharmacy (AMCP) in New Orleans. Their talk was titled, “Taming Million Dollar Drugs – A Health Care System Emerging Therapies Program.” They were interviewed by Managed Healthcare Executive prior to the meeting.

Craven says the emerging therapies committee, which is subcommittee of the P&T committee, has 15-20 members and includes four or five people from the health system’s pharmacy department, others from procurement and finance, clinicians and an ethicist. Bajorek said the committee was initially set up to review and oversee the clinical, operational and financial aspects of the cell and gene therapies. But after some experience the clinical review was moved over to a separate clinical subcommittee. Craven says the emerging therapies committee is “nonclinical in nature, so most of the people who are on the committee are actually more operational.”

In another adjustment, the committee also set up a process that establishes a workgroup for newly onboarding drugs. “That workgroup is there to hopefully go through each of the little details that come up, just to make sure that the day-to-day operations have been thought about and that if there’s anything that needs to circulate back up to the leadership, then that’s identified early,” says Craven.

Picking a pharmacist to lead the onboarding and creating the emerging therapies pharmacist position makes sense, says Craven, pharmacists have experience interacting with the many parts of the health system that get involved when drug is priced at six figures and above. Craven said one of her responsibilities is to head off any problems with insurance coverage. “We start looking at payer information right away because we know that can be a rate-limiting step for the timeline for how things are going to progress,” says Craven. That means getting the contracting team involved, she says, and making sure that there are letters of agreement or single-case agreements in place or underway. There are also nitty-gritty issues such as whether the drug has an identifying number that allows a claim to be submitted.

“The whole point of the committee is that we have these experts on the committee, so we have the contracting experts — we leave that expertise to them,” says Craven. “As pharmacists, we don’t typically deal with letters of agreement or single-case agreements. That structure hasn’t changed. What we really want, though, is to make sure that the right people have the information as early as possible and that they understand where we are in the timeline.” With so many people in so many different areas involved, Craven says it has been important to have a good planning tool, and she is a fan of Microsoft Planner.

The criteria for drugs coming under the emerging therapies committee’s review include cost — the threshold is $300,000 a year — and special logistical needs. In addition to Elevidys and Zolgensma ( onasemnogene abeparvovec), a gene therapy for spinal muscular atrophy, the emerging therapies committee has reviewed several CAR-T therapies and Leqembi (lecanemab), a treatment for Alzheimer's disease that the FDA approved in 2023. Craven says the committee is currently working on Brineura (cerliponase alfa), a enzyme replacement treatment for a form of Batten disease.

"There's probably a few others that have maybe not received an extensive review, but that is kind of the point is that we're able to assess, does this need a full onboarding workgroup assessment?" says Craven.