Net present value cost analysis for treating retinitis pigmentosa

Area of Science:

• Ophthalmology
• Health Economics
• Genetics

Background:

• Retinitis pigmentosa (RP) is a group of inherited disorders that cause progressive vision loss.
• The financial burden of RP includes direct medical and nonmedical costs, as well as opportunity costs.
• Understanding these costs is crucial for evaluating the economic impact of potential treatments.

Purpose of the Study:

• To quantify the financial burden and opportunity cost of vision loss due to RP in the US.
• To model the potential economic benefits of novel gene therapies for RP using net present value (NPV).

Main Methods:

• Developed a NPV model incorporating economic, medical, and demographic assumptions.
• Utilized data from federal health expenditure tables, claims databases, and mortality rates.
• Performed scenario analyses on the impact of gene therapy at different treatment ages (15, 35, 55).

Main Results:

• The NPV opportunity cost of not treating RP was substantial, ranging from $2,091,032 (treated at age 55) to $3,973,909 (treated at age 15).
• A significant portion of these costs represented lost government benefit accrual and government program expenditures due to visual impairment.
• Earlier treatment, particularly during adolescence, resulted in higher overall savings.

Conclusions:

• Novel gene therapies for RP have the potential to offset considerable government costs related to disability payments.
• The economic benefits, in terms of reduced government spending, are maximized when gene therapy is administered at a younger age, such as adolescence.