By Raffaele Pereno – PhD, MBA
BASEL, Switzerland – Reporting from the Festival of Biologics 2025, CheckOrphan attended a thought-provoking keynote panel featuring Dr. Marco Traub, Founder of the Trans European Stem Cell Therapy Society (TESCT), and Dr. Pedro Cruz, Chief Technical Operations Officer at iBET – Instituto de Biologia Experimental e Tecnológica.
The Price of a Cure
The discussion centered on one of modern medicine’s most pressing challenges: the accessibility and affordability of curative gene and cell therapies.
The panel opened with a sobering fact—CAR-T therapies, once hailed as revolutionary, can now cost up to $2 million per patient.
“Such pricing reflects the extraordinary complexity and safety considerations involved,” explained Dr. Cruz. “But it also limits access. A therapy cannot be called curative if only a fraction of patients can receive it.”
CAR-T cell therapy remains an autologous process—each treatment is tailored from the patient’s own immune cells. This personalized approach drives costs and limits scalability.
Technical and Logistical Barriers
Dr. Traub noted that only a handful of hospitals in Europe currently have GMP-grade facilities and trained personnel to produce CAR-T products.
“The process requires T-cell extraction, viral-vector transfection, expansion, and reinfusion—all under strict GMP conditions,” he said. “Very few centers can perform this safely and effectively.”
One possible solution, he suggested, is centralized manufacturing in a limited number of specialized facilities while maintaining patient care locally. However, this would rely on a robust cold-chain logistics system similar to the one used during the COVID-19 vaccine rollout—an expensive and complex undertaking.
Another challenge is the distribution of viral vectors, which remains tightly regulated and difficult to decentralize under current EU rules.
Divergent Regulatory Paths
Dr. Cruz contrasted the U.S. FDA’s pragmatic approach, which allows “GMP-like” flexibility for hospital-based CAR-T production, with the stricter European framework, where full GMP certification is required even for small-scale manufacturing.
“This rigidity slows innovation,” he said. “Europe needs greater harmonization and flexibility to stay competitive in advanced-therapy manufacturing.”
Ethical and Policy Challenges
In some countries, such as Slovenia, every patient has a legal right to access the best available therapy. In practice, however, this creates immense pressure on physicians.
“Doctors fear the legal consequences of approving such costly treatments,” said Dr. Traub. “Many prefer to refuse therapy rather than risk liability—leaving patients to seek help from organizations like TESCT.”
According to Dr. Traub, about 80% of TESCT’s patients are individuals whose doctors declined to prescribe advanced therapies for financial or regulatory reasons.
Toward Accessible Innovation
Dr. Cruz emphasized that lower-cost CAR-T production models already exist.
“In Spain, we’ve shown that hospital-based CAR-T manufacturing can be both compliant and cost-efficient,” he said. “It proves that accessibility doesn’t have to compromise safety or quality.”
Both speakers agreed that the next decade must focus on making gene and cell therapies sustainable and equitable, not just technically feasible.
“Patients want access to the latest advanced technologies,” concluded Dr. Traub. “Our responsibility is to ensure those therapies reach everyone—not only those who can afford them.”
About the Speakers
Dr. Pedro Cruz is the Chief Technical Operations Officer at iBET, a non-profit R&D institution in Portugal dedicated to advancing bioprocessing, vaccine development, and cell- and gene-therapy innovation.
Dr. Marco Traub, PhD, is the Founder and President of the Trans European Stem Cell Therapy Society (TESCT), established in 2005 in Switzerland to accelerate the translation of regenerative medicine from the laboratory to clinical practice.