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Updated: Jan 9, 2026
Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
Published on: August 7, 2015
MODELING NATURAL HISTORY AND COST-EFFECTIVENESS OF GENE-AGNOSTIC TREATMENT IN RETINITIS PIGMENTOSA
Patrick W Sullivan1, Albert I Wertheimer2, Sandeep Grover3
1College of Pharmacy, Nova Southeastern University, Davie, Florida.
Purpose:
Treatment options for retinitis pigmentosa (RP) have been limited. A new generation of innovative but potentially expensive treatments under development may halt or reverse disease progression.
Methods:
Using an existing database, a closed-cohort microsimulation model of all genetic subtypes of RP was conducted from a U.S. societal perspective. A hypothetical gene-agnostic therapy was compared with standard care at age 30. Outcomes included VF and VA progression; incremental cost-effectiveness ratio (ICER: dollars per quality-adjusted life-year (QALY) gained); and lifetime medical and productivity-related costs.
Results:
Baseline cohort (age 30, N = 381; mean VA logMAR, 0.26 (20/36); mean VF sum total degrees (III4e), 749). By age 45, 79% of patients with untreated RP reached legal blindness or worse, compared with significantly delayed progression with treatment. ICER of gene-agnostic therapy was $158,554/QALY, yielding better outcomes compared with untreated RP (20.5 vs. 16.7 QALYs) and substantial indirect cost savings ($691,961) if a drug price of $1.5 million was assumed.
Conclusion:
A gene-agnostic treatment for RP with drug price of $1.5 million (both eyes) may be cost-effective for most types of RP, particularly with earlier initiation. These findings can inform clinical, economic, and health policy decisions, potentially supporting early intervention strategies and resource allocation for novel RP treatments.
Insights
A new gene-agnostic therapy for Retinitis Pigmentosa (RP) shows cost-effectiveness, potentially halting disease progression. Early intervention with this treatment may offer significant benefits for patients and healthcare systems.
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.
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