A recent discussion at the HOPA Annual Conference highlighted how hemophilia and sickle cell disease offer important lessons about the future of gene therapy access. Although both conditions qualify only the most severely affected individuals for treatment, their patient populations and care needs differ significantly, and those differences shape how gene therapies can be delivered in practice.
For hemophilia, patients are typically accustomed to lifelong IV factor infusions and high‑cost therapies. Gene therapy introduces a new model: a single, expensive upfront treatment intended to replace ongoing prophylaxis. Liver‑related inflammation remains a key safety concern, with some individuals requiring months of steroid therapy to manage enzyme elevations.
For sickle cell disease, access challenges are more complex. The eligible population is larger and disproportionately affected by socioeconomic barriers, including reliance on public insurance and limited access to major transplant centres. Ex vivo gene therapies require chemotherapy, hospitalization, and frequent follow‑up, creating significant logistical and caregiving burdens for working‑age adults.
Across both conditions, long‑term safety monitoring is essential. While early results are promising, data beyond five years remain limited, and rare risks such as treatment‑related leukemia have been reported in some lentiviral‑based therapies.
Pharmacists play a critical role in navigating prior authorizations, coordinating bundled payment models, ensuring supportive medications are covered, and educating financial teams about the clinical steps required for each therapy. For centres just beginning to explore gene therapy programs, identifying a physician champion and building a multidisciplinary team are key first steps.
