Multi-year Budget-Impact Analysis of One-Time Genome-Editing and Somatic Cell Therapies Under Constrained Payer Envelopes

One-time genome-editing and autologous somatic cell therapies concentrate substantial acquisition costs in a single fiscal year, while avoided transfusion and complication offsets accrue over decades across fragmented public insurers. In Iran, where an extensive transfusion-dependent -thalassemia burden is concentrated along endemic belts, evaluating such advanced therapies requires accounting for explicit domestic cash envelopes and hard-currency import restrictions. We developed a ۱۰-year multi-payer budget-impact model across the Iran Health Insurance Organization, Social Security Organization, Armed Forces Medical Services Insurance Organization, and the Fund for Special and Incurable Diseases. Annual treated volume was modeled as the minimum of clinical demand, accredited-centre capacity (۵۰–۸۰ patients/year), an annual import quota, and a rial budget envelope (۳. ۲ thousand billion toman). At internationally referenced finished-product tariffs (۱۵۴ billion toman), a launch cohort of ۵۰ patients incurs a net year-one cost of ~۷. ۸۱ thousand billion toman—absorbing over ۶۰% of the Special Fund's entire annual outlay for ۱۳۰+ disease groups and restricting envelope-feasible access to just ۲۰ patients. Transitioning to domestic fill-finish and a ۵-year annuity substantially relaxes the rial envelope constraint, enabling treated volume to reach physical capacity limits. Crucially, installment models do not alleviate feasibility bottlenecks if vectors and closed-system consumables remain tied to the year of manufacture under strict foreign-exchange quotas. Furthermore, ۱۰-year clinical offsets recover only a fraction of acquisition costs, with ۳۵% of savings leaking due to cross-payer migration. Sustainable coverage necessitates domestic input substitution, multi-year payment installments, provincial equity quotas, and cross-payer outcome tracking.

کلیدواژه ها:

Budget-Impact Analysis ، Iran ، Β-Thalassemia ، Genome Editing ، Special and Incurable Diseases Fund.