Who Should Track Gene Therapy Outcomes Long Term? ASTCT Panel Debates Shared Responsibility

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.

She identified a separate weakness in routine postapproval monitoring. “We have a very passive reporting system post market, which has a lot of flaws in it inherently.”¹ Spontaneous AE reports may not provide the systematic longitudinal information required to detect some delayed effects or determine treatment durability.

FDA guidance recommends extended, risk-based observation for certain gene therapies because delayed AEs may emerge years after administration. Requirements depend on the product and its characteristics; a single surveillance schedule does not apply to every therapy.³

The panel did not propose transferring manufacturers' legal responsibilities to hospitals. Instead, members debated whether additional research infrastructure could answer questions that sponsor-specific studies were not designed to address.

Building Evidence Across Institutions

Sonali Chaudhury, MD, of Ann & Robert H. Lurie Children's Hospital of Chicago, argued for a stronger role for academic treatment centers. Regarding broader outcomes collection, she said, “…it needs to move from the sponsors to the academic institutes to own that.”¹

Chaudhury leads ReGenT, the Real World Experience Consortium for Commercial Gene Therapy Products in Non-Malignant Disorders. The collaboration includes more than 55 treatment centers internationally and works to coordinate real-world experience with commercial genetic therapies.⁴

“We need the willingness from sponsors, from regulatory bodies, to collect the information that's been missed and address it in real time,” Chaudhury said.¹ The challenge, she explained, is collecting useful information without duplicating patients' reporting obligations or asking individual institutions to reinvent established systems.

The Center for International Blood and Marrow Transplant Research already supports transplant and cellular therapy outcome collection.⁵ Under the Centers for Medicare & Medicaid Services Cell and Gene Therapy Access Model, it also helps gather clinical and patient-reported outcomes for eligible Medicaid recipients receiving sickle cell gene therapy, including pain, fatigue, and functioning.⁶

For pharmacists, structured long-term records could support surveillance of conditioning-related complications, medication changes, infections, and suspected delayed toxicities. Reporting clinically relevant findings and maintaining communication between gene therapy centers and local care teams are practical implications of the discussion, not responsibilities the panel assigned exclusively to pharmacists.

The Economics of Gene Therapy at Scale

The closing segment of the discussion raised another constraint: collecting robust evidence may be difficult to sustain if therapy costs prevent broader access. Responding to a question about publicly financed health systems, Sharma warned that the cost structure is “just unsustainable” when many increasingly available therapies are considered together.¹

He cited an institution-specific example of gene-edited sickle cell therapy manufacturing at St Jude: “our cost for manufacturing is $60,000.”¹ He acknowledged that grant funding and institutional resources subsidize the program. That figure is not a complete treatment cost and cannot be compared directly with commercial list prices, which also reflect development, regulatory, and business expenses. St. Jude has described its on-site investigational sickle cell gene-editing program.⁷

The panel reached no agreement on registry governance or payment reform. It did establish a practical challenge for the field: if gene therapies are expected to deliver decades of benefit, long-term evidence collection must be coordinated, useful to patients, and financially sustainable.

REFERENCES

1. American Society for Transplantation and Cellular Therapy. Industry–academic interface. Panel discussion presented at: ASTCT Gene Therapy Summit; November 5, 2026; Boston, MA.

2. Sharma A, Kassim A, Thompson A, et al. Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy. Transplant Cell Ther. 2026;32(7):778-791. doi:10.1016/j.jtct.2026.03.019

3. Long term follow-up after administration of human gene therapy products: guidance for industry. News release. FDA. Updated January 30, 2020. Accessed November 6, 2026. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/long-term-follow-after-administration-human-gene-therapy-products

4. Leading international consortium on gene therapy outcomes. News release. Ann & Robert H. Lurie Children's Hospital of Chicago. December 17, 2025. Accessed November 6, 2026. https://www.luriechildrens.org/en/news-stories/leading-international-consortium-on-gene-therapy-outcomes/

5. Data collection. Center for International Blood and Marrow Transplant Research. Accessed November 5, 2026. https://cibmtr.org/
   Centers for Medicare & Medicaid Services. CGT Access Model frequently asked questions. Accessed November 5, 2026. https://www.cms.gov/priorities/innovation/cgt-access-model-frequently-asked-questions

6. St. Jude Children's Research Hospital. SAGES1: CRISPR/Cas9 gene editing for sickle cell disease. Accessed November 5, 2026. https://www.stjude.org/care-treatment/clinical-trials/sages1-sickle-cell.html