MODELING NATURAL HISTORY AND COST-EFFECTIVENESS OF GENE-AGNOSTIC TREATMENT IN RETINITIS PIGMENTOSA

Area of Science:

• Ophthalmology
• Health Economics
• Genetics

Background:

• Retinitis Pigmentosa (RP) presents limited treatment options, impacting vision and quality of life.
• Innovative gene-agnostic therapies are emerging, offering potential to halt or reverse RP progression.

Purpose of the Study:

• To evaluate the cost-effectiveness of a hypothetical gene-agnostic therapy for Retinitis Pigmentosa (RP) from a US societal perspective.
• To compare the economic and clinical outcomes of a novel RP treatment versus standard care.

Main Methods:

• A closed-cohort microsimulation model was used, encompassing all genetic subtypes of RP.
• A hypothetical gene-agnostic therapy was assessed against standard care, analyzing visual field (VF) and visual acuity (VA) progression.
• Key metrics included the Incremental Cost-Effectiveness Ratio (ICER) and lifetime costs.

Main Results:

• Untreated RP patients faced a 79% risk of legal blindness by age 45.
• The gene-agnostic therapy demonstrated delayed disease progression and improved outcomes (20.5 vs. 16.7 QALYs).
• The ICER was $158,554 per QALY gained, with significant indirect cost savings projected at $691,961 for a $1.5 million drug price.

Conclusions:

• A $1.5 million gene-agnostic RP treatment may be cost-effective for most RP types, especially when initiated early.
• Findings support early intervention and inform resource allocation for novel RP treatments.
• This research aids clinical, economic, and health policy decisions regarding RP management.