Leaders in health care are addressing critical access gaps for advanced treatments, particularly life-saving cell therapies and transplants. At the third annual TIME100 Health Leadership Forum in New York City on September 10, experts discussed the challenges patients face in receiving these transformative therapies, even as scientific breakthroughs continue.
"The biggest barrier was the fact that there were not donors to match," said Amy Ronneberg, chief executive officer of NMDP, a global nonprofit connecting patients with cell therapies and transplants. "As more patients have a donor, we now are facing issues around referral. Thousands of patients are not getting referred to transplant centers in a timely manner, those that are oftentimes are facing major barriers from a financial perspective."
Dr. Eneida R. Nemecek, director of Pediatric Cell Therapy and Transplant & Cancer Clinical Research at Oregon Health & Science University, identified the design of health systems as a significant obstacle. "When we're designing systems for healthcare, we forget that we're designing the systems for the patient. The system designs the system for itself, and then we try to adjust it to the patient," Nemecek stated.
Specialized therapies like CAR-T, often developed and tested at major academic institutions, create natural bottlenecks for wider dissemination. Dr. David L. Porter, director of the Center for Cell Therapy and Transplant at Penn Medicine, explained that these advanced treatments are frequently performed as proof of concept in large urban centers. Consequently, many individuals in the country lack proximity to these facilities and must travel considerable distances for care, a process that requires specialized expertise and technology.
"The barrier is actually collecting the cells," Porter said, referring to the specialized process required for treatments like CAR-T therapy. "It's a very specialized process."
Beyond the logistical and financial hurdles, the treatment process itself causes significant disruption. The weeks-long regimen of testing and treatment can lead patients and caregivers to consider less effective, but more accessible, suboptimal therapies. To combat this, initiatives are underway to bring these high-end therapies closer to patients. Porter and his colleagues have piloted a program at the University of Pennsylvania that implements CAR-T cell therapy in community settings, successfully treating over 60 patients who likely would not have traveled to a major city for care.
Ronneberg emphasized the importance of "education and awareness" for communities and the need for "funds to travel," drawing from her own experience as a cancer survivor who found navigation difficult despite having personal resources. NMDP's focus has broadened from donor identification to ensuring patients reach the appropriate treatment locations, whether academic or community-based.
To address these disparities, Nemecek and her colleagues are involved in projects like the ACCESS Initiative, collaborating with NMDP to train community centers and provide them with the accreditation needed for cell collection. "With valuable telehealth, with use of technology," Nemecek noted, "that's really where the future of our field is."