Building cell and gene therapy programs takes early planning, strategic risk decisions and careful growth

Health system leaders face difficult choices when deciding how to build cell and gene therapy programs. These treatments can transform patient care, but they also require long timelines, upfront investment and careful planning. In the study, “From Vision to Viability: Developing Infrastructure for Cell and Gene Therapy Programs,” Zahra Mahmoudjafari, Pharm.D., MBA, and her coauthors outline a framework to help organizations think through those decisions in a structured way.

In a recent interview with Managed Healthcare Executive, Mahmoudjafari discussed two of the toughest questions leaders face: when to begin onboarding before FDA approval and how to think about volume and financial sustainability.

Becoming a qualified treatment center can take six to 18 months, according to Mahmoudjafari. Some institutions begin preparing before FDA approval while others wait. Mahmoudjafari said the right approach depends on each organization’s priorities and tolerance for risk.

“I mean, I think that all boils down to the center and what their priorities are. So waiting for FDA approval reduces that early risk of maybe working on something that then doesn't ultimately get approved,” she said. “But on the other hand, if you wait for that FDA approval, you may delay your institutional learning, where you can't roll out quite as quickly.”

She expressed that early participation in clinical trials can accelerate readiness because teams gain experience with workflows, staff training and sponsor expectations before commercial approval arrives. However, that path requires upfront investment and comfort with uncertainty.

“Of course, coming from an academic center that has access to clinical trials, I'm always going to advocate for clinical trial participation, not only because you're expanding access to patients, but it accelerates your readiness by building that experience with workflow, staff training, and then sponsor expectations up front. It does require some investment and tolerance for uncertainty. That kind of just is dependent again on that strategic planning for the system itself,” she said. “So for me, I consider it to be a strategic priority. Early trial involvement is an advantage and not a luxury.”

For systems that wait, she cautioned there may be consequences. She also added that financial sustainability is another complex challenge.

CGTs are expensive and require investments in staffing, compliance and quality oversight, she shared. Leaders often ask whether there is a minimum patient volume required to make a program viable.

Mahmoudjafari said there is no simple answer.

“Yeah, that's a trick question. There is no universal volume threshold that's determined by your systems and your capacity of your system,” she said, adding that higher volume can help spread fixed costs, but sustainability depends on more than numbers alone.

“There's no denying that these therapies are very expensive, and if you have and have the potential for high volumes, that can be really impactful,” she said. “So, economies of scale do matter. I would say understanding fixed costs, so staffing, compliance, quality oversight that becomes more manageable as volume grows, but sustainability depends more on your payer mix, your site of care, maybe some of your operational efficiencies.”

For smaller programs, success is still possible, she said, if leaders understand their regional demand and build carefully.

“That is a really difficult question to answer. I would say, for smaller programs, you can succeed if you're disciplined, selective and really understand your own regional demand,” Mahmoudjafari said. “I think trying to take such a big, gargantuan project and divvying it up to small, digestible pieces that will make it a lot more sustainable in the long run.”