Pharmacists Lead Implementation of Advanced Cell and Gene Therapies Across Major Health Systems
核心洞察
Pharmacists are uniquely positioned to lead the implementation of advanced cell and gene therapies, serving as both clinical and operational experts in health systems managing these complex treatments.
Major health systems are already delivering widespread advanced therapeutics, with Mayo Clinic offering 9 active therapies, UC San Diego Health providing 12, and Children's Hospital of Philadelphia making 12 available to eligible patients.
Significant challenges include high costs, manufacturing variability, patient hesitancy toward gene manipulation, and the need for new governance structures and educational frameworks.
Cell and gene therapies are rapidly transitioning from experimental treatments to widespread clinical reality across major health systems, with pharmacists emerging as essential leaders in their implementation and management.
At the 2025 American Society of Health-System Pharmacists (搜索) (ASHP) Pharmacy Futures meeting, four pharmacy leaders from major health systems revealed the extensive reach of these advanced therapeutics. Mayo Clinic currently offers 9 active therapies with 5 pending, while University of California San Diego Health (搜索) provides 12 active treatments with 5 under review. Children's Hospital of Philadelphia (CHOP) makes 12 advanced therapeutics available to eligible patients with 8 more in the pipeline, and even community-based pediatric centers like Cook Children's (搜索) in Texas are delivering gene therapies, offering 4 currently with 2 more under review.
Pharmacists as Central Coordinators
Ashley Dalton, PharmD, MHA, associate chief pharmacy officer at UC San Diego Health System, emphasized the unique positioning of pharmacists in managing these complex therapies. "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," Dalton stated.
These therapies present unprecedented 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 mean pharmacy teams must manage not only clinical pathways but also logistics, legal agreements, and payer strategy.
Educational and Training Gaps
Despite their central role, significant learning gaps exist in preparing pharmacists for advanced therapeutics management. Heidi Trinkman, PharmD, BCOP, advanced therapeutics program manager at Cook Children's (搜索), highlighted the comprehensive knowledge required beyond clinical science.
"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 explained.
Richard Dyke, PharmD, BBA, DPLA, director of pharmacy business operations at CHOP, noted the importance of understanding contracting and economics in making ethical decisions about patient access. "Who gets these therapies? How do you do that in a sustainable way?" he questioned.
The panelists agreed that residency programs will likely evolve to integrate non-clinical elements, potentially as components within disease-specific residencies or through new business-focused pathways. "We have to figure it out first before we can teach it," Trinkman acknowledged.
Governance and Collaboration Challenges
Health systems are establishing new governance structures to guide implementation of these therapies. UC San Diego Health has evolved its high-cost drug committee to include more clinical perspectives, while CHOP and Cook Children's (搜索) have created executive-level steering committees focused on both research and business considerations.
Eric M. Tichy, PharmD, MBA, vice chair of pharmacy formulary for Mayo Clinic, described governance as "a very hot topic" and "one of the top things people ask me for," noting that current approaches are "not exactly perfect."
Multidisciplinary collaboration, particularly with physicians, remains crucial but challenging. Physicians may be unaware of the significant time required to establish these treatments within health systems, 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 observed.
Unexpected Clinical Realities
The implementation of advanced therapeutics has revealed surprising challenges in clinical practice. Dalton shared her surprise about manufacturing variability affecting patient experience, noting that some products created using patient cells may not always be usable or meet 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 explained.
Patient hesitancy toward gene therapies has also emerged as an unexpected challenge. Trinkman noted resistance from some patients when discussing gene manipulation: "When you talk about manipulating genes (搜索), that definitely tends to put some people off. I just wasn't really ready for the 'No, thank you,' because I couldn't comprehend why [patients] wouldn't just jump on this."
The uncertainty surrounding therapy durability further complicates patient decisions and health system planning. Tichy noted that patients may worry about future relapse, contributing to hesitancy and complicating budgeting and forecasting.
CRISPR Breakthrough and Access Challenges
The field achieved a significant milestone in 2023 with the approval of exagamglogene autotemcel (exa-cel; Casgevy; Vertex Pharmaceuticals), the first CRISPR-based medicine for treating sickle cell disease (搜索) and transfusion-dependent β-thalassemia (搜索). This CRISPR-Cas9 (搜索) gene-editing technology allows for precise alterations in patients' DNA sequences to correct disease-causing mutations.
However, the high costs of gene therapies create substantial access barriers. Many health plans either do not cover approved gene therapies or place restrictions on the number of patients who may receive treatment annually. The limited accessibility stems from significant development and research costs, compounded by the focus on rare or ultrarare diseases with small patient populations.
Research as Strategic Foundation
Participation in clinical trials provides health systems with crucial advantages in implementing advanced therapeutics. "It's incredibly helpful if you were a study site," Dyke emphasized, noting how trial experience familiarizes teams with unique preparation and handling procedures.
However, Dalton cautioned that procedures can change once treatments receive FDA approval, with complications from Risk Evaluation and Mitigation Strategy programs and occasional surprises in FDA labeling.
As advanced therapeutics continue expanding, pharmacists are positioned not just as implementation team members but as leaders driving these transformative treatments forward. Success requires developing new expertise spanning science, strategy, and systems thinking to navigate the complex landscape of personalized medicine.