O acordo de acesso gerenciado do onasemnogeno abeparvoveque trará economia para o Sistema Único de Saúde (SUS)? Uma análise de impacto orçamentário

Abstract

Introduction (with objective): In March 2025, a Managed Access Agreement (MAA) was formalized between the Ministry of Health and the company holding the registration for onasemnogene abeparvovec (OA) for the treatment of patients with Spinal Muscular Atrophy (SMA) in the SUS (Brazilian Public Health System). The agreement covers patients up to six months of age with a confirmed genetic diagnosis of 5q SMA type 1, with up to three copies of SMN2, who are not undergoing invasive mechanical ventilation for more than 16 hours a day. To mitigate the financial impact and clinical uncertainties, the agreement linked payment to the clinical performance of the therapy, defined as increased survival without permanent ventilation, achievement and maintenance of motor milestones. The model provides for 40% of the payment at the time of infusion and installments of 20% at 24, 36 and 48 subsequent months, conditional on achieving the outcomes [1]. Objective: To estimate the budgetary impact of implementing AAG for OA from the perspective of the SUS (Brazilian Unified Health System). Material and Method: An open cohort model was developed using the epidemiological method for incident cases [2,3,4]. The analysis encompassed direct medical costs, without applying discount rates, over a five-year time horizon. The baseline scenario estimated the costs of therapy without AAG. The alternative scenario estimated the cost of therapy according to AAG negotiation, in which the remaining payment installments would be canceled according to the patient's trajectory. It was assumed that 100% of therapy purchases would be made through AAG. Sensitivity analysis was performed to assess uncertainties. Results: The total accumulated cost over 5 years for installment payments and achievement of agreed results via AAG was R$ 1.3 billion, compared to R$ 1.9 billion in the baseline scenario with OA disbursement before AAG. The resulting incremental cost was -R$ 608,458,836. The projected avoided cost due to the suspension of payments in case of therapeutic failure was R$ 154,622,098. Sensitivity analysis demonstrated that the model is more sensitive to the clinical success rate at month 36 and to the scenario of a different source of evidence [4]. The parameters used in the base case considered the evidence available when the agreement was signed. The scenario analysis considering 100% event-free survival and achievement of milestones was equivalent to the postponement of installments, conferring the advantage of diluting the disbursement over time when direct medical costs are observed, with the caveat that 70% of the study population used other pharmacological therapies for SMA over time. Mitigation of uncertainties can be achieved through access to the AAG follow-up outcome records and understanding the operational costs to make it viable. Considering the administrative costs, the difficulty in generating aggregated clinical data, and the delay in access (maintaining litigation for a longer period), it is possible that performance-based agreements do not offer advantages that outweigh their limitations. Conclusions: Although the modeled AAG projects savings of R$ 154,000 in risks associated with therapeutic failures over 5 years, there are uncertainties to be addressed by monitoring the AAG's own registry of outcomes obtained. Postponing installments through AAG can promote the sustainability of incorporating advanced therapy into the SUS (Brazilian Unified Health System). Furthermore, it is necessary to consider whether the mitigation of financial risk with direct costs is offset by the administrative and operational viability of alternative payment models.