Pharmacists Take the Lead on Advanced Therapeutics

At the 2025 American Society of Health-System Pharmacists (ASHP) Pharmacy Futures meeting, 4 pharmacy leaders from major health systems gathered for a candid panel discussion about the rapidly evolving role of pharmacists in managing advanced therapeutics, including cell and gene therapies.

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Cell and gene therapies represent a new frontier in medicine, offering the potential for 1-time, curative treatments for diseases ranging from genetic disorders to cancers. Gene therapies work by delivering genetic material into a patient’s cells to replace missing or malfunctioning genes, whereas cell therapies often involve modifying a patient’s own cells or donor cells to treat or even cure disease.¹

These therapies come with unique challenges for health systems, including ultra-sensitive storage and handling requirements, complex patient selection criteria, rigorous financial planning, and uncertain long-term outcomes. Their high cost and intricate manufacturing also mean that pharmacy teams must manage not only clinical pathways but also logistics, legal agreements, and payer strategy.²

Widespread Use, Complex Systems

Advanced therapeutics are no longer theoretical. These treatments are already in widespread use across health systems, from Mayo Clinic’s 9 active and 5 pending to University of California (UC) San Diego Health’s 12 active and 5 under review. At Children’s Hospital of Philadelphia (CHOP), eligible patients can access 12 advanced therapeutics and 8 more in the pipeline. Even community-based pediatric centers like Cook Children’s in Texas are delivering gene therapies, with 4 currently offered and 2 more under review.²

Pharmacists at the Center of Complex Innovation

One theme was clear: pharmacists are uniquely positioned to lead in the implementation of these high-impact therapies. Ashley Dalton, PharmD, MHA, associate chief pharmacy officer at UC San Diego Health System, emphasized the central role pharmacists play.

“Pharmacy is the ultimate middleman in every therapy… We are the clinical side, and we are the operational side. We’re the best person for this job in all of our health systems.”²

Still, as Heidi Trinkman, PharmD, BCOP, advanced therapeutics program manager at Cook Children’s, pointed out, there are significant learning gaps. The implementation of these therapies is challenging, requiring knowledge not just in clinical science but also in finance, operations, and logistics.

“You really need to have that whole overarching vision of the process and benefits and risks… You really need to be able to step back and see the whole picture of the process,” Trinkman said.²

Training the Future Workforce

The panelists agreed that existing pharmacy education often lacks the operational training necessary for advanced therapeutics. Richard Dyke, PharmD, BBA, DPLA, director of pharmacy business operations at CHOP, noted the importance of understanding contracting and economics and how these will impact ethical decisions.

“Who gets these therapies? How do you do that in a sustainable way?” he asked.²

The panelists all agreed that residency programs will likely evolve to integrate these non-clinical elements—perhaps as components within disease-specific residencies or in new business-focused pathways.

“We have to figure it out first before we can teach it,” Trinkman said.²

Governance and Multidisciplinary Collaboration

All 4 panelists described how their health systems are establishing governance structures to guide the implementation of these therapies. For example, UC San Diego Health has a high-cost drug committee that is evolving to include more clinical perspectives. CHOP and Cook Children’s have created executive-level steering committees focused on both research and business considerations.

Still, confusion remains over the role of pharmacy and therapeutics (P&T) committees in this process.

“This governance issue is a very hot topic,” Eric M. Tichy, PharmD, MBA, vice chair of pharmacy formulary for Mayo Clinic, explained. “It’s one of the top things people ask me for… This is what it is today, and it’s not exactly perfect.”²

The group agreed on the value of multidisciplinary collaboration, particularly with physicians. Often, physicians and other stakeholders may be unaware of the significant time it takes to establish these treatments within a health system, creating frustration and confusion.

“I find that [physicians] are a lot more patient after having gone through the process and have a deeper appreciation once they’ve been through it,” Trinkman said.²

Surprising Realities at the Point of Care

Despite the promise of these therapies, the panelists admitted they’ve encountered unexpected challenges in clinical practice. Dalton shared her surprise about how variability in manufacturing affects the patient experience, given that some products created using the patient’s cells may not always be usable or up to FDA standards.

“The product returned was going to be out of [specification]… It’s been a real wake-up call to start that conversation of out-of-[specification] product early in the process,” she said.²

For Dyke’s team, balancing research and commercial pathways have created gray areas. Often, teams are working to get authorization for an advanced therapeutic while the patient is also eligible for an investigational pathway, and it can be surprising when the patient chooses the investigational route.

Trinkman noted the hesitancy some patients have shown toward gene therapies, emphasizing the need for greater education about how these therapies work.

“I’m coming at it from the perspective of understanding the science behind it… When you talk about manipulating genes, that definitely tends to put some people off,” she said. “I just wasn’t really ready for the ‘No, thank you,’ because I couldn’t comprehend why [patients] wouldn’t just jump on this.”²

Even the durability of some gene and cell therapies remains uncertain, Tichy added, noting that patients may worry about a future relapse. That uncertainty contributes to hesitancy and complicates budgeting and forecasting for health systems.

Research as a Strategic Advantage

The panel closed with a discussion about research and its role in shaping future offerings. All agreed that being involved in trials provides institutions with a head start.

“It’s incredibly helpful if you were a study site,” Dyke said, emphasizing how trial experience familiarizes teams with unique preparation and handling procedures.²

However, Dalton noted that some procedures can change once a treatment is FDA-approved, with complications from Risk Evaluation and Mitigation Strategy programs as well as the occasional surprise on an FDA label.

As the field of advanced therapeutics continues to grow, the panel made 1 thing clear: pharmacists aren’t just part of the implementation team—they’re leading it. But doing so requires a new kind of expertise that spans science, strategy, and systems thinking.

REFERENCES

1. Cellular and Gene Therapy Products. FDA. Updated March 20, 2023. Accessed June 9, 2025. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products

2. Tichy EM, Dalton A, Dyke R, Trinkman H. Management of Advanced Therapeutics Across the Healthcare Continuum. Presented at: American Society of Health-System Pharmacists 2025 Pharmacy Futures. Charlotte, NC; June 9, 2025.