How Pharmacists Can Lead the Transition as Gene Therapy Moves Into Mainstream Care

As gene and cell therapies move beyond specialized academic centers and into broader clinical practice, pharmacists are stepping into a far more expansive role. Alex Wolff, PharmD, BCOP, FHOPA, explains how the shift from “cell therapy” to “drug product” is transforming pharmacy operations, budgeting, verification workflows, and patient education. She outlines what pharmacists need to know now—especially as ex vivo and in vivo therapies introduce new storage, handling, and safety challenges—and why pharmacy expertise is crucial to supporting patients and providers in this rapidly evolving therapeutic landscape.

Q: Gene therapy is still a relatively unfamiliar space for many practicing oncology and hematology pharmacists. How do you see the pharmacist's role evolving as these therapies move from specialized academic centers into broader clinical settings, and what competencies should pharmacists be prioritizing right now?

Alex Wolff, PharmD, BCOP, FHOPA: Yeah, I think it's something that we've been sort of peripherally involved in with stem cell transplant. You know, largely we are involved with the drugs surrounding it and not the actual cells. What's different now is that the cell products are being operationally used as drugs. So things like chimeric antigen receptor (CAR) T-cell therapy that have come out in the last 6 years or so—and now, with gene therapies coming out that are cell-based products—they come with a National Drug Code (NDC), which means they're built into electronic medical records as a drug and not a cell therapy product.

That means that they are purchased through a pharmacy department budget, which means they have to be accounted for in budgeting because they're very expensive, and then their billing goes that way. So the logistical handling of the product through the system, whether it's ordering verification, requires a pharmacist verification if it's built as a direct product into the charge dropping, and sort of connecting the authorization piece to the revenue cycle on the back end is now shifting to pharmacy budget. So it's a little more administrative than it is necessarily day-to-day physically handling the product.

Some of those cell therapy aspects—and I know we're talking about storage later—are still physically handled within our cell lab, because they have those resources. The cells come frozen in liquid nitrogen. They require, for the ex vivo gene therapies, something like negative 150 degrees to be frozen, and that capability has lived within the cell processing lab, not necessarily in pharmacy. Some systems, though, if they anticipate pharmacy taking that portion over, are going to have to outfit those with some capital expenses and purchasing and training to be able to handle and process those cells.

So it is a little bit different, but I think it's coming out, like I said, largely from the billing and the organization of how they're shifting these to calling them drug products. When they come out that way, they're classified slightly differently. They're classified as biologics, but they're categorized under drugs, and so that shifts them into different buckets in the hospital departments in these large systems.

As pharmacists—as clinical pharmacists—I think that our role is expanding in education. We've always been very involved in education. I think we need to take more of a role in educating patients as to what's involved. It's a cell therapy, but it comes with inherent risks. It comes with things that are required to be monitored. Some gene therapies are one-and-done, and some may require multiple doses at different points, but even a one-and-done is going to have multiple months of monitoring for toxicities and potentially needing treatment and care for those toxicities.

In the case of ex vivo gene therapies, where we're using a cell-based product, they need chemotherapy beforehand. In the case of sickle cell disease, for example, it's not a cancer. It's not something that sickle cell–treating physicians, nurses, caseworkers, and patients are accustomed to—needing chemotherapy for a disease that's not a cancer. So coming in and educating on that piece of it, and why it's necessary in order for the gene therapy to work and take hold, and how the gene therapy is going to work to address the complications of their sickle cell disease, is important.

So I think there's a lot of gap in knowledge—in everybody: in the end user, which is the patient, in the prescribers. And as pharmacists, I think we're well positioned to provide a lot of that education, and that's going to be an invaluable piece too.

Q: From a pharmacy operations standpoint, what are the most meaningful day-to-day differences between managing an in vivo versus ex vivo gene therapy program? Are there handling, storage, or administration considerations that pharmacists are often caught off guard by?

Wolff:
Yeah, and I touched a little bit on that before with some of the in vivo products. With in vivo, your workflow is much more like a high-cost intravenous (IV) infusion drug. There's no preparative chemotherapy. There's no pretreatment required. Some of them require washout of the treatment that they were previously on; some don't. It just sort of depends on the disease state.

The storage and preparation vary based on product. Some of them require freezer temperatures, which we're generally used to. We freeze things like vaccines. We have some freezer capabilities. But the in vivo products—where you administer the vector to the patient—rarely require that liquid nitrogen freezing that's related to the ex vivo cell therapies. Those are cells that are coming to you, and so they do require that deep freeze. That capability is not something most pharmacies traditionally have. It's something that usually lives within the cell lab.

So whatever infrastructure your system has set up to receive cells, either from an allogeneic donor for a bone marrow transplant or for CAR T-cell therapy, it's a similar process and handling to that. There is a precedent in place as to how those are handled.

What is new, though, is that because it's labeled as a drug therapy, the way that most health systems will build that into their medical record is as a drug. And so you're not receiving and handling the product, but you still have to verify it in the system. The question has come up in several groups: Do your pharmacists physically lay eyes on the product? That's part of our job—visually verifying the product before you verify it.

Some places have taken to where, at my institution, the pharmacists who verify the order verify it against the report that goes into the system that the cell lab receives. So they verify that Patient A matches Patient A, that the dose that was sent is the dose that's prescribed, and that's their verification.

Other departments—I know that some of the hospitals in my system—will physically send the pharmacist to the floor to lay eyes on the product. Part of that is how the logistics are set up for your product to scan. A lot of hospitals use barcode scanning for enhanced safety, and how it's built to scan varies. Our system is built to scan the product label. So what comes from the manufacturer is what gets scanned as the verification of product. Other systems have it built to scan the pharmacy label. When you verify, it prints a label in your pharmacy, and that label is what needs to be scanned. So they have to take that to the product and verify it.

The reason we generally have to send the pharmacist to the floor in those cases is because there's a limited viability time post-thaw. You're generally limited, depending on the product, to a couple of hours; the shortest time is 20 minutes from thaw time to infusion time. And so that really limits the ability to send the product to pharmacy for verification and then back, because you run the risk of losing your viability window. It's a little bit of a workflow disturbance.

The in vivo products are a little bit logistically simpler, but they may require some manipulation. They may require being drawn from vials to syringes, from syringes into bags, or they may be available for direct injection. It just depends on the products. There's a couple different options available.