Assessment of the Lifetime Costs of Severe Visual Impairment Due to Retinitis Pigmentosa

Introduction: Retinitis pigmentosa (RP) is a rare genetic eye disease characterized by progressive retinal degeneration, leading to blindness. RP-associated blindness and subsequent disability can generate substantial societal costs that far outweigh direct healthcare costs; however, prior studies have primarily focused on medical costs without quantifying lifetime indirect costs associated with RP-related disability. This study used actuarial models to examine lifetime excess costs resulting from RP-related disability. Results may advance the understanding of potential lifetime benefits of gene therapy for the treatment of RP. Methods: We used actuarial techniques to model three archetype patients with RP who become disabled at ages 50, 35, and 18, respectively, and quantified the costs associated with healthcare, social security and disability benefits, lost income, transportation, and supportive services. These costs were compared with scenarios without disability, assuming employment until retirement age. Results: Aggregate lifetime costs incurred by patients, payers, government, and employer programs vary by age and employment status at disability onset. We estimated the present value of lifetime costs following disability for a patient with RP at 1, 741, 141, 2, 586, 819, and 3, 072, 550 when disabled at ages 50, 35, and 18, respectively. These estimates represent lifetime excess costs, defined as the difference between lifetime costs with and without RP-related disability, of 1, 222, 496 (236%), 1, 948, 063 (305%), and 2, 303, 667 (300%), respectively. Lost income opportunity and costs incurred from employer and government sponsored disability benefits were the largest contributors to excess costs. Conclusion: This actuarial approach to assessing the burden of RP-related disability shows how costs, both direct and indirect, accrue over a lifetime following blindness. Projected lifetime disability costs are expected to far exceed all-cause healthcare costs, imposing an escalating burden on society. Gene therapies have the potential to prevent blindness, preserve income, and reduce disability-related costs, thereby reducing the societal cost burden associated with RP-related disability. Plain Language Summary: Retinitis pigmentosa (RP) is a rare inherited eye disease that gradually leads to blindness. Patients with RP often become disabled, incurring lifetime costs not only for healthcare services but also from lost income and the need for government or employer support. Using actuarial models, we estimated each patient with RP who becomes disabled will incur lifetime costs of 1. 2 million to 2. 3 million, depending on the age at disability. Most of these lifetime costs are associated with lost income and disability benefits. This study highlights the substantial societal costs of RP-related blindness and shows how interventions, such as gene therapies, could help preserve vision and income while reducing lifetime costs associated with disability. Keywords: disability, retinitis pigmentosa, blindness, gene therapy, inherited retinal diseases