Net present value cost analysis for treating retinitis pigmentosa.

Objectives: To provide insights into the financial burden and opportunity cost of vision loss from retinitis pigmentosa (RP) in the US by using net present value (NPV) of direct medical and nonmedical costs.

Methods: Assumptions, including economic (discount rate, median income, cost-of-living, Social Security and Medicare taxes, public insurance/supplemental benefits, nutrition assistance, and prescription drug assistance), medical (federal National Health Expenditure tables, a recent retrospective claims analysis, and Optum Health claims database) and demographic (mortality rate, increase in mortality due to visual impairment, progression of blindness, probability of survival, retirement rate, rate of disability, and RP diagnosis probability) were made to develop a NPV model. Scenario analyses were performed on benefits and costs arising from patients with RP, if vision could be preserved via novel gene therapies.

Results: Based on modeled assumptions, for a patient with RP treated with gene therapy at age 15, the NPV opportunity cost of not treating was $3,973,909 ($1,027,358 in lost accrual of government benefits throughout employment and $2,946,551 paid by government programs due to visual impairment). For a patient treated at age 35, the NPV opportunity cost of not treating was $2,866,964 ($719,926 in lost accrual of benefits and $2,147,038 paid by government programs). For a patient treated at age 55, the NPV opportunity cost of not treating was $2,091,032 ($758,445 in lost accrual of government benefits and $1,332,587 paid by government programs).

Limitations: As it was not possible to include all potential factors, the model may under- or overestimate true NPV. Potential treatment costs were included as hypothetical estimates.

Conclusions: Novel gene therapies for RP likely offset significant government costs toward disability payments; these savings may be even greater when administered during adolescence.