“If you have two caregivers, and one stops working, you lose funds. They have to travel back and forth to their center, also taking care of their home needs as well,” she said.¹
Previously, the program reimbursed qualifying travel costs, but families still needed money upfront. Some could not cover those expenses while awaiting repayment. Fooks-Parker described working with lodging providers, home-center social workers, and outside foundations when financial assistance became more limited.
She also questioned unnecessary repeat visits: “Why can’t we consent to collection in the same trip so we reduce the trip by one?”¹
That seemingly small scheduling change can matter when families must travel across the country.
These concerns extend beyond any single center. Researchers have identified substantial geographic, infrastructure, and financial barriers to sickle cell gene therapy.³ In a 2026 survey of 82 evaluable families with children who had sickle cell disease, 60% reported household material hardship, and only 40% reported that they could access a hypothetical curative therapy with their existing resources. The survey measured reported access, not actual uptake of approved gene therapies.⁴
Making Coordination Visible to Patients
At Cohen Children’s, Halac described separate clinical, manufacturing, insurance, and family-readiness workflows that must converge before treatment. Standardized checklists helped identify tasks, but they could not anticipate each patient’s circumstances. Her team now reviews changes in clinical status, product availability, and family readiness across multidisciplinary meetings, with named responsibility for follow-through.¹
“It is important for us to give them one point of contact. If that person doesn’t know the answer, for sure they’re going to find the answer,” Halac said.¹
The approach also requires honest discussion of what patients and families understand about the next step. A published consensus statement on psychosocial readiness for sickle cell gene therapy recommends assessing practical support needs and coping resources to prepare patients for treatment rather than using psychosocial concerns as automatic exclusion criteria.⁵
Pharmacists can contribute by reconciling medication plans after schedule changes, verifying conditioning readiness, coordinating infection prophylaxis, and clarifying medication instructions when care crosses departments. Those are pharmacy practice implications of the speakers’ operational concerns, not tasks they assigned exclusively to pharmacists.
Building Follow-Up That Lasts
Ivory White, APRN, FNP-C, of Texas Children’s Hospital, argued that the demands of gene therapy continue long after discharge.
“The current model works clinically, but it just asks the patients to do everything, right?” she said, describing patients juggling hematology, transplant, fertility, pain, and psychological services.¹
Her team is developing a coordinated follow-up model with shared visits, a nurse navigator, and flexible scheduling.
“We want to have one care home, one team, one longitudinal plan, and one goal,” White said.¹
The proposed pathway extends to 15 years, with monitoring tailored to treatment phase and clinical stability, including organ complications, immune recovery, and delayed safety concerns.
FDA guidance recommends risk-based long-term observation for gene therapy products with potential delayed adverse events; not every product carries the same follow-up requirement.⁶ White’s model seeks to make surveillance realistic for patients who live far from their treatment centers, not merely available on a schedule.
Programs must therefore assess more than successful infusions. As Halac reminded attendees, “Every decision you make, every change will affect their quality of life.”¹
REFERENCES
Fooks-Parker S, Halac A, Diamond Y, White I. Designing a gene therapy program that patients can actually use. Presented at: ASTCT Gene Therapy Summit; November 5, 2026; Boston, MA.
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
Sharma A, John TD. Dismantling cost and infrastructure barriers to equitable access to gene therapies for sickle cell disease. Lancet Haematol. 2024;11(8):e556-e559. doi:10.1016/S2352-3026(24)00175-3
C. A. Kelly, M. A. Paul, M. Griffin, et al. “ Out of Reach: Understanding Resource Barriers to Gene Therapy for Pediatric Patients With Sickle Cell Disease.” Pediatric Blood & Cancer 73, no. 10 (2026): e70536.
https://doi.org/10.1002/1545-5017.70536 Hardy SJ, Crosby LE, Porter JS, et al. Assessing Psychosocial Risk and Resilience to Support Readiness for Gene Therapy in Sickle Cell Disease: A Consensus Statement. JAMA Netw Open. 2024;7(8):e2429443. Published 2024 Aug 1. doi:10.1001/jamanetworkopen.2024.29443
US Food and Drug Administration. Long term follow-up after administration of human gene therapy products: guidance for industry. Published January 2020.
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/long-term-follow-after-administration-human-gene-therapy-products
