As gene therapies move beyond clinical trials, a difficult question follows patients into routine care: Who is responsible for documenting whether treatment remains safe and effective years after infusion? During a discussion entitled, “Industry–Academic Interface,” which was held at the 2026 American Society for Transplantation and Cellular Therapy (ASTCT) Gene Therapy Summit in Boston, health care professionals, industry leaders, and regulatory experts debated how to share that responsibility.1
“Rather than putting the entire burden on the sponsor and asking them to do it, I think you’ve got to move this discussion out of the box a little bit and start thinking about it more globally,” said Akshay Sharma, MBBS, MSc, of St. Jude Children’s Research Hospital.1 His comments captured a central tension: regulatory follow-up establishes whether an approved product meets its obligations, but health care professionals need broader evidence about patients’ long-term health.
Beyond Eliminating Pain Crises
Bill Hobbs, MD, PhD, of Vertex Pharmaceuticals, described why freedom from severe vaso-occlusive crises became a useful endpoint for sickle cell gene therapy. Earlier treatments generally sought to reduce episodes; with potentially transformative approaches. “The goal was not to reduce them, but to eliminate them,” he explained.1
Sharma argued that this achievement should not define the outer limits of success. “I would suggest that the bar needs to be higher,” he said, pointing toward organ function, persistent complications, and longer survival.1 These outcomes require years of observation. Fewer pain crises alone do not establish that kidney, cardiac, or pulmonary damage present before treatment has reversed.
The discussion also exposed a genuine disagreement over the evidence needed before treatment. Nicole Verdun, MD, a former FDA cell and gene therapy regulator, argued that established knowledge of disease genetics should play a greater role when deciding whether patients can receive promising therapies earlier.
“I actually think that we need to do more to rely on what we know about the science,” Verdun said.1 Her argument was not to abandon outcome monitoring, but to balance the urgency of treating severe disease against the time required to document long-term benefit.
The 2026 ASTCT and International Society for Cell & Gene Therapy practice recommendations similarly identify surveillance of organ complications, conditioning-related adverse effects (AEs), gene modification risks, and quality of life as important elements of ongoing care.2
Who Owns Long-Term Follow-Up?
Verdun described the difficulty of retaining patients in extended follow-up after they begin feeling healthy. Families may reasonably question annual assessments when the treatment was presented as potentially curative. Programs also change ownership, and regulatory follow-up commitments remain associated with specific sponsors.
