OBJECTIVES: Multiple innovative treatments for myasthenia gravis (MG) are expected to become available. Healthcare payers often rely on economic evaluations to determine access to treatments. This study demonstrates how real-world data can be used to identify patients with high economic burden, expediting public reimbursement and enhancing the value of new treatments.

METHODS: We utilized nationwide patient-level data from the Norwegian Patient Registry to identify incident MG patients during 2010-2021. Patients were divided into five groups based on treatment with intravenous immunoglobulin (IVIg) following diagnosis: 1) all IVIg patients; 2) patients treated with IVIg during the first year only; 3) patients treated with IVIg in the second year or later; 4) patients treated with IVIg as maintenance treatment; and 5) non-IVIg patients. Resource utilization and direct medical costs were estimated using Diagnosis-Related Group (DRG) cost weights.

RESULTS: Among 1,083 incident MG patients, 14.3% (N=155) were treated with IVIg, 4.9% (N=53) received IVIg in the first year only, 9.4% (N=102) were treated with IVIg in the second year or later, and 3.4% (N=37) received IVIg maintenance treatment. MG-related inpatient stays and outpatient encounters were 2.8-fold higher for IVIg patients compared with non-IVIg patients. IVIg patients had 3.3-fold higher direct medical costs during follow-up. Direct medical costs per patient were EUR 95,364 for all IVIg patients, EUR 71,934 for those receiving IVIg in the first year only, EUR 107,538 for patients treated with IVIg in the second year or later, EUR 111,148 for patients on IVIg maintenance treatment, and EUR 28,952 for non-IVIg patients.

CONCLUSIONS: IVIg treatment is a significant indicator of higher resource use and costs in MG. Real-world data can retrospectively identify patients with high economic burden that offer a greater scope to reduce costs, thereby facilitating access to innovation and optimizing healthcare spending. This approach can serve as a model for other conditions.