Health Systems Urged to Prioritize Pharmacy Leadership in Cell and Gene Therapy Programs

Starting a cell or gene therapy program is about much more than offering a new treatment. Behind each therapy is a complex system of contracts, safety rules, payer agreements and detailed workflows that all have to work together. Health system leaders are learning that clinical success is only part of the equation, and without strong financial and operational planning, patients can face delays before therapy even begins.

In the study, "From Vision to Viability: Developing Infrastructure for Cell and Gene Therapy Programs," Zahra Mahmoudjafari, Pharm.D., MBA, and her coauthors shared what health systems need to build safe and sustainable programs. Mahmoudjafari, clinical pharmacy manager for hematologic malignancies and cellular therapeutics at the University of Kansas Cancer Center, said that strong planning and early pharmacy involvement are essential to making these programs succeed.

Mahmoudjafari said she believes pharmacy leaders should be involved before a single patient is referred. She added that pharmacy is typically brought in too late, after a therapy has already been approved and key decisions have been made. Mahmoudjafari suggested pharmacies should help guide site selection, contract review and workflow design from the start.

"We're not just about dispensing product anymore," Mahmoudjafari said. "We're really quite anchored in safety, continuity and then overall sustainability across the program."

She said that pharmacists play a much larger role in these programs than many people realize. They help coordinate how products are handled, make sure the center meets regulatory requirements and support the management of serious side effects. Pharmacy teams also educate staff and help oversee the financial side of these high-cost treatments. Because cell and gene therapies are complex and expensive, missing these steps can put both patients and the program at risk.

Cell and gene therapies are expensive and tend to require detailed prior authorization. Mahmoudjafari said health systems should prepare early and work closely with both government and commercial insurers before treating their first patient.

"I think this space in particular requires partnership, partnership with our industry colleagues but then also with our payer colleagues. And really looking to educate," Mahmoudjafari said. "Health systems are responsible for that education piece also because our payers are looking to catch up with the innovation."

She expressed that successful centers clearly define coverage pathways, sites of care expectations and appeal strategies in advance. That means understanding the differences between Medicare and commercial plans, aligning coding and billing processes and modeling best- and worst-case financial scenarios.

While payers can still be challenging, she said they are no longer a "black and white box." Early communication, transparency and strong documentation can improve collaboration and reduce delays for patients.

Mahmoudjafari also discussed how health systems are organizing these services. Cell and gene therapies are often talked about together, but many systems are now separating them in practice. CAR T-cell therapy is usually offered within cancer centers and often builds on existing transplant programs. Gene therapy, however, may involve different diseases, patient groups and long-term follow-up needs. Some hospitals may choose to focus only on CAR T-cell therapy if they do not treat certain rare diseases or pediatric patients.

She noted that gene therapies can require more long-term monitoring and come with ongoing payer uncertainty. For some centers, it may not make sense to build a full gene therapy program if only a few patients would qualify each year.

Mahmoudjafari also spoke about sickle cell gene therapies, which have rolled out more slowly than many expected. She noted that innovation often comes with "bumps in the road." Over the next few years, she expects changes in volume, more therapies moving into outpatient settings and increased competition among products. Clinical trials are also expanding into non-oncology diseases, which could bring many more patients into the system.

Due to this uncertainty, she said health systems need flexible staffing and scalable workflows. Programs should not be built around a single product. Instead, leaders should design systems that can adjust as therapies and demand change.

"I've always used the statement of 'write everything in pencil,' because you're going to have to re-back track and understand that this is just a space that's moving quickly but also kind of slowly in a lot of ways too," she said.

Her advice for executives is straightforward: invest early in payer engagement, build adaptable systems and plan for change. The science may be groundbreaking, but long-term success depends on careful coordination across pharmacy, finance and operations. Mahmoudjafari concluded by saying that health systems should focus on who they serve and what they can do well. As these therapies continue to grow, she stressed that strong infrastructure and early pharmacy involvement are key to turning medical breakthroughs into real patient care.