The mainstreaming of cell and gene therapy and what that means for their price and distribution | Asembia AXS26 Summit
Key Takeaways
- Manufacturing scale-up may force simplification of custody and release processes because duplicative safeguards that work at low volume become cost- and time-prohibitive at higher throughput.
- Pricing and contracting frameworks will need overhaul as treated populations expand; at $3 million per dose, 50,000 eligible patients implies roughly $150 billion in potential spend.
Cell and gene therapies are currently niche products, even within specialty pharmacy, drugs that, by most definitions, are treatments for a smaller number of patients. But at a session on cell and gene therapy at the 2026 Asembia specialty pharmacy meeting in Las Vegas, panelists discussed a future of many more cell and gene therapies that would scramble how the therapies are currently distributed, priced and paid for.
The existing payment and distribution are not broken, said Stephanie Wirkes, Diplom-Kauffrau, head of distribution strategy and execution at Bayer Pharmaceuticals U.S. “Do the models break the moment we start to scale, so the moment it’s not one patient per month, one patient in six months? What if it’s 80 per six months, 100 per month? When we get to scale, how do these existing models break or not break?” Bayer has a gene therapy for Parkinson’s disease in development that would, if it proves to be safe and effective, pose some of the problems of scale that Wirkes talked about.
“I think from a manufacturing side, we were very concerned,” Wirkes continued. “These are like our little babies, and we don’t want anything to break. We don’t want anything to get stuck somewhere, so we put a lot of protective layers on it. But it also creates a lot of redundancies. Well, once you’re scaling, you don’t have that luxury of having redundancies or duplications and things like that. So how do we have to actually change the models to really make sure that it’s really scalable?”
The panelists talked about a wide range of issues and challenges in cell and gene therapy in both its current state and scale and in a future posited to have a multitude of such therapies. Will Pih, Pharm.D., co-founder of Two Labs Pharma, a commercialization and access company, said the FDA has approved about 50 cell and gene therapies, and “there’s a ton of drugs in the pipeline,” fueled by ample investment. By disease, two of the leading categories are drugs for hematologic cancers and neurology, he said, but there are also therapies for retinal diseases and heart conditions. “It’s an amazing time. It’s like the invention of penicillin and the polio vaccine all happening at one time,” he said
The themes included what is likely to be a larger role for specialty pharmacies and changes to price and value assessments as cell and gene therapies move into the mainstream. Jennifer Lospinoso, managing director and consulting lead
“With these new therapies, we’ll have to be thinking about the comparison against an already-established, standardized care, so there’s a lot of work to do on the pricing front … to make sure, from a market access perspective, that these new cell and gene therapies are successful,” Lospinoso told the Asembia audience.
During the session and in a brief interview with Managed Healthcare Executive immediately afterward, Morgan Olson, senior vice president of biopharma business development for Orsini, spoke about the role that specialty pharmacies have in gene therapy. Orsini, a specialty pharmacy headquartered in Elk Grove, Illinois, specializes in rare diseases. Olson said the company currently distributes 12 cell and gene therapies, 10 of which are gene therapies.
Manufacturers of cell and gene therapies typically prize experience when looking for specialty pharmacies to work with, according to Olson. That experience requirement is, she acknowledged, a “chicken and egg” problem: The only way to have experience is to get experience. Olson also noted that many of these therapies are covered on the medical benefit, which may be a hurdle for some specialty pharmacies.
“When you think about a true specialty pharmacy, many of them are only contracted on the pharmacy benefit side of the house,” she said.
In contrast, her company “grew up, as Orsini, in the medical benefit, and then we added our pharmacy benefit over time, so we’ve had 30 years in the medical benefit space and understanding the dynamics of how to process insurance claims.” Orsini “started to refine that muscle uniquely to the cell and gene portfolio,” she said. “Over the last 10 years, we’ve built a repository of how to navigate the payer contracting on the cell and gene side,” she said.
Pih outlined the two prevailing ways of distributing cell and gene therapy. In the “flash title model,” the manufcturer owns the therapy and bears the risk until it is delivered to the site of care. An intermediary, such as a drug wholesaler, may be involved, but specialty pharmacies are not included.
The “specialty pharmacy drive-by model” does, as the name implies, involve specialty pharmacies, but specialty pharmacies do not physically take possession of therapy. They do, however, pay the manufacturers for it. According to information that Pih presented on a slide, the specialty drive-by model shifts the cash outlay and inventory risk away from the treatment centers. It is catching on, according to Pih, as cell and gene therapy becomes more common.
Olson said that manufacturers have used different strategies, some choosing to launch products through a specialty pharmacy, while others work directly with treatment centers that buy the therapy and then bill the payer — “the buy-and-bill” channel, Olson called it.
But executives at the manufacturers who work directly with treatment centers are changing their minds, she said.
“We’re getting calls from a lot of the manufacturers that launched only with the buy-and-bill channel,” she said. They want to outsource to a specialty pharmacy. “They want to have a specialty pharmacy partner for certain therapies and/or certain patients, and so they’re looking to add us as a channel after the fact,” Olson said.
Olson added that “especially as outpatient administration continues to grow, I think we’re going to continue to see a rise in the number of cases that go through specialty pharmacy as those outpatient centers, gain their understanding, figure out if they want to be a true cell and gene dispenser, buy and biller, or if they want to just manage the outpatient specialty pharmacy.”
Because Orsini is taking on the financial risk, it has an incentive to manage access and execution of cell and gene therapy well, Olson observed.
“We want to make sure that that patient gets on therapy, and the insurance claim is getting paid so we get paid as well. Otherwise, we’re taking a huge risk. As you know, the average gene therapy is $3 million.”
