Building the Plane While Flying It: Operational Realities of Cell and Gene Therapy Program Development
核心洞察
A panel of advanced therapeutics leaders discussed strategic planning, operational readiness, and financial sustainability for cell and gene therapy programs at academic medical centers.
Panelists emphasized that governance success depends on identifying institutional champions across pharmacy, managed care, financial clearance, legal, and clinical teams organized into a centralized steering structure.
Payer variability remains the most persistent challenge a decade into CAR-T (搜索) therapy, with prior authorization approval not guaranteeing payment and reimbursement models varying dramatically across payers.
A panel of advanced therapeutics leaders convened to explore the operational, financial, and governance realities of standing up cell and gene therapy programs, offering a candid look at what Zahra Mahmoudjafari, PharmD, MBA, BCOP, FHOPA, described as "building the plane while flying it." The discussion, which featured Mahmoudjafari alongside Jenny L. Craven, PharmD, BCPS, Elias Pittos, PharmD, BCOP, CSP, and Mark Duckworth, BS, moved through three critical areas: strategic planning, operational readiness, and financial sustainability.
Governance: Identifying Champions Across Every Lane
Panelists emphasized that success in launching cell and gene therapy programs depends less on clinical readiness than on identifying institutional champions across every stakeholder lane. Pharmacy, managed care, financial clearance, legal, and clinical teams must be organized into a centralized steering structure with clear accountability. The panel flagged ethicists and contracting teams as groups that frequently get left out during early planning stages, a gap that can create downstream complications.
Mahmoudjafari, director of pharmacy in advanced therapeutics at the University of Kansas City Health Center (搜索), and Craven, senior clinical pharmacist and pharmacy lead for emerging therapies at UC Davis Health (搜索), both stressed that governance structures must be built deliberately and inclusively from the outset.
Operational Readiness: The SOP Scalability Problem
On operations, the group addressed standard operating procedure (SOP) development and sustainability. The panel stressed that effective procedures are written by the people who actually follow them, revisited frequently, and supported by dedicated pharmacist leads. A systemic threat to scalability emerged in the discussion: the pipeline volume problem, which could potentially require a unique SOP per drug as the number of approved therapies grows.
Pittos, medical director of Cell, Gene, and Advanced Therapies at McKesson, and Duckworth, clinical research program manager in the Division of Oncology at The Children's Hospital of Philadelphia, contributed perspectives on how institutions can manage this growing operational burden without sacrificing quality or compliance.
Reimbursement: The Field's Most Persistent Challenge
The panel was notably candid about the financial landscape. A decade into CAR-T (搜索) therapy, payer variability remains the field's most persistent and underestimated challenge. Prior authorization approval does not guarantee payment, and reimbursement models vary dramatically across payers. Community centers face additional structural barriers that academic medical centers largely do not, creating disparities in patient access.
Looking Ahead: Allogeneic and In Vivo Therapies
Looking forward, the group expressed optimism about allogeneic and in vivo therapies streamlining delivery and expanding community access. However, they cautioned that volume growth and evolving reimbursement models may require rethinking the buy-and-bill paradigm entirely. The panelists encouraged institutions to engage pharmacy early, build connected relationships across departments, and not be intimidated by the complexity of bringing that first therapy on board.