Cost-effectiveness analyses and utility weights
Published cost-effectiveness analyses and health-state utility weights that a model of this product could cite, adapt, or compare against, grouped by how each article relates to the product and its indication. Every value is quoted from the article it cites, and section 3 lists the searches that found the articles.
10 cost-effectiveness analyses and 4 studies reporting utility weights · 11 sources
Overview
| Item | Detail |
|---|---|
| Literature searched | Tufts Medical Center Cost-Effectiveness Analysis Registry, searched October 21, 2026; the queries are listed in section 3. |
| Cost-effectiveness analyses | 10 |
| Studies reporting utility weights | 4 |
Cost-effectiveness analyses
Analyses of Tinlarebant (LBS-008)
No evidence found.
Treatments for the same indication
No evidence found.
The same disease in other populations or settings
No evidence found.
Related diseases and analogous treatments
Voretigene neparvovec versus standard of care in biallelic RPE65-mediated retinal disease, United States: ICER final evidence report (2018)
Relevance: Evaluates voretigene neparvovec against standard of care in biallelic RPE65-mediated retinal disease, an inherited retinal disease, from United States health care system and modified societal perspectives.
Voretigene neparvovec versus standard of care in biallelic RPE65-mediated inherited retinal disease, United States: Zimmermann et al. (2019)
Relevance: Evaluates voretigene neparvovec against standard of care in biallelic RPE65-mediated inherited retinal disease over a lifetime horizon from United States healthcare system and modified societal perspectives.
“We modeled the cost-utility of VN compared with standard of care (SoC).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A 2-state Markov model, alive and dead, with a lifetime horizon.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“VN led to an improvement in VA and VF that we assumed was maintained for 10 years followed by a 10-year waning period.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The cost of VN was $850 000, and other direct medical costs for depression and trauma were included for a US healthcare system perspective. A modified societal perspective also included direct nonmedical costs and indirect costs.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“VN provided an additional 1.3 QALYs over the remaining lifetime of an individual.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The average total lifetime direct medical cost for individuals treated with VN was $1 039 000 compared with $213 400 for SoC, leading to an incremental cost-effectiveness ratio (ICER) of $643 800/QALY from the US healthcare system perspective.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Direct nonmedical costs totalled $1 070 900 for VN and $1 203 300 for SoC, and indirect costs totalled $405 400 for VN and $482 900 for SoC, leading to an ICER of $480 100/QALY from the modified societal perspective.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Voretigene neparvovec-rzyl versus standard care in RPE65-mediated inherited retinal disease, United States: Johnson et al. (2019)
Relevance: Evaluates voretigene neparvovec-rzyl against standard care in RPE65-mediated inherited retinal disease over a lifetime horizon in the United States, with health states defined by levels of visual impairment.
“In this economic analysis, a health state transition model based on visual acuity and field with a lifetime horizon was developed to estimate the cost-effectiveness of voretigene neparvovec-rzyl.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Indirect costs, including educational attainment, productivity, caregiver burden, and governmental programs, were estimated using published literature and data analysis of public national surveys.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Costs and benefits were discounted at 3.0% annually.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The model population included 70 patients with RPE65 -mediated IRD, with a mean age of 15 years; 42 of 70 patients (60%) were female.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Voretigene neparvovec-rzyl had higher QALYs (18.1 vs 8.6) and lower total lifetime costs ($2.2 million vs $2.8 million) than SC (ie, was dominant) when including indirect costs, resulting in cost-savings and QALY increases. When indirect costs were excluded, the cost per QALY was $79 618.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Voretigene neparvovec-rzyl remains cost-effective if at least 8.8% of the long-term treatment effect continues after year 3 when including indirect costs and 43.3% when excluding indirect costs, assuming a cost threshold of $150 000 per quality-adjusted life-year.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Intravitreal pegcetacoplan versus sham in geographic atrophy, United States: Patel et al. (2024)
Relevance: Evaluates intravitreal pegcetacoplan against sham control in geographic atrophy in the United States, a model of a treatment that slows macular atrophy, the process the DRAGON trial measures as definitely decreased autofluorescence lesion growth.
“DESIGN: Cost analysis based on data from a published study.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“SUBJECTS: None; based on data from published sham control compared with 2 treatment groups in the index study.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“METHODS: Costs were based on 2022 Medicare reimbursement data. Specific outcomes were extrapolated from the DERBY and OAKS trials. Assumptions were made for the lifetime analysis based on a theoretical logistic growth model of the atrophy.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The costs to treat GA in every month (EM) and every-other-month (EOM) treatment groups over the 2 years as reported were $70 000 and $34 600, respectively.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The modeled lifetime costs were $350 000 (EM) and $172 000 (EOM), or $309 000/mm2 (EM) and $180 000 (EOM) /mm2.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The modeled time to 95% atrophy at 13 years was delayed by 2.5 years (EM) and 2.1 years (EOM).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The costs/quality-adjusted life-year gained based on modeled visual loss with 95% atrophy were $706 000 (EM) and $397 000 (EOM).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Voretigene neparvovec versus best supportive care in biallelic RPE65-mediated inherited retinal dystrophies, United Kingdom: Viriato et al. (2020)
Relevance: Evaluates voretigene neparvovec in biallelic RPE65-mediated inherited retinal dystrophies in the United Kingdom, with health states defined by levels of visual impairment.
“A Markov state transition model was constructed, with five alive health states determined based on the worst of VA and VF (i.e., whichever of VA or VF would assign the patient to a more severe health state) and death (Fig. 1)” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Costs were considered from the perspective of the National Health Service (NHS) and Personal Social Services in England and Wales, with costs falling outside of the healthcare system included in scenario analyses” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“A lifetime time horizon was considered, and costs and outcomes were discounted at an annual rate of 3.5% in line with current NICE guidance” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“A cost-effectiveness analysis was conducted comparing VN with BSC in individuals with biallelic RPE65-mediated IRD who have sufficient viable retinal cells.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Phase III trial data were used to inform transition probabilities up to year 1, after which the treatment effect was assumed to be maintained for 40 years, followed by a decline in vision.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“At list price, VN is associated with incremental costs of £612,404 and incremental QALYs of 6.4, resulting in an incremental cost-effectiveness ratio (ICER) of £95,072 per QALY gained.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“For example, in 52% of simulations, VN was associated with an ICER of < £100,000 per QALY.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The proportion of simulations considered cost-effective when accounting for the additional QALY weighting for treatments with significant QALY gains (i.e., > 10) was 71%.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Voretigene neparvovec versus standard of care in biallelic RPE65-mediated inherited retinal disease, Switzerland: Bhadhuri et al. (2022)
Relevance: Evaluates voretigene neparvovec in RPE65-mediated inherited retinal disease in Switzerland, with health states defined by levels of visual impairment.
“We adapted a previously published Markov cohort model for IRD. An annual cycle length, life-long time horizon, discount rate of 3% for cost and health outcomes, and Swiss health system perspective were used.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“SoC is best supportive care provided to patients with visual impairment.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The population of interest were IRD patients with a biallelic RPE65-mutation and sufficient viable retinal cells.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“For the base-case, VN resulted in incremental costs per patient of CHF 764’402 (VN: CHF 901’654, SoC: CHF 137’252), incremental blindness-free years of 7.67 (VN: 28.32, SoC: 20.65) and incremental QALYs of 6.73 (VN: 18.35, SoC: 11.62), leading to an incremental cost-effectiveness ratio of CHF 113’526 per QALY gained.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“In probabilistic sensitivity analysis, the cost-effectiveness of VN was better than CHF 100,000 per QALY gained in 41% of iterations.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“For the scenario analysis in which a societal perspective was adopted and for which a 50% work-related productivity loss from blindness was assumed, incremental costs of CHF 423,837 and an ICER of CHF 62’947 per QALY gained were produced.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Voretigene neparvovec versus annual physician checks and supportive care in biallelic RPE65-mediated inherited retinal degeneration, Germany: Uhrmann et al. (2020)
Relevance: Evaluates voretigene neparvovec-rzyl against annual physician checks and supportive care in RPE65-mediated inherited retinal degeneration over a lifetime horizon in Germany.
“We developed an individual patient sampling model to simulate patients over their lifetime. In a Monte Carlo analysis, 1000 simulations are performed. Cycle length of the two-state Markov model is 1 year.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“We assume SoC to consist of annual physicians’ checks plus supportive care according to the patient's individual state of visual impairment. Each patient is individually tracked over a lifetime horizon.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“To reflect a societal perspective in Germany, we adapt the model to reflect indirect costs in proportion to the degree of visual impairment.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“In the base case scenario, costs are discounted by 3%, as recommended by the German Institute for Quality and Efficiency in Health Care.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“On average, VN provides 4.82 additional QALYs for each patient over their lifetime” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Additional overall costs for VN are €755,566. The ICUR per additional QALY gained is €156,853” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“This decreases additional QALYs comparing VN with SoC to 2.89 QALYs. Additional overall costs increase to €1,169,403 and the ICUR to €404,774.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Immediate versus delayed basic low vision rehabilitation in age-related macular degeneration or diabetic retinopathy, Portugal: Hernández-Moreno et al. (2023)
Relevance: Evaluates immediate against delayed low vision rehabilitation with refractive correction, optical reading aids, and training, a form of the low-vision care listed in section 2 as the supportive care for Stargardt disease.
“The trial recruited patients diagnosed with age-related macular degeneration or diabetic retinopathy (DR) and visual acuity in the range 0.4–1.0logMAR in the better-seeing eye. Participants were randomised to one of the study arms consisting of immediate intervention and delayed intervention.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The intervention included: new refractive correction, optical reading aids, in-office training and advice about modifications at home.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The trial compared the outcomes 12 weeks after the start in both arms.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Economic analysis was conducted from the healthcare perspective. All costs are reported in euros for year 2020.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The mean cost of the intervention was €118.79 (SD = 24.37). The incremental cost-effectiveness ratio using the EQ-5D-5L was 30,421€/QALY and 1186€/QALY when using near acuity.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Figure 2C shows that the probability of the intervention to be cost-effective is 49% for a threshold of ~ €20,000 (Portugal per capita GDP) and Fig. 2D shows that the probability of the intervention to be cost-effective is 100% from a threshold of €2400.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Portable electronic vision enhancement systems added to optical low vision aids versus optical low vision aids alone in visual impairment, United Kingdom: Bray et al. (2017)
Relevance: Compares portable electronic vision enhancement systems added to optical low vision aids with optical low vision aids alone, the low-vision care listed in section 2, in a randomized crossover trial-based evaluation in the United Kingdom.
“An AB/BA randomized crossover trial design was used. Eighty‐two participants completed the study.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The trial intervention was the addition of a p‐EVES device to the participant's existing optical LVA(s) for 2 months, and the control intervention was optical LVA use only, for 2 months.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“All economic analyses were conducted from a societal perspective to take account of the financial burden of carer time. All costs are in £ sterling for 2014.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Discounting was not undertaken as the length of interventions did not exceed 12 months.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The mean difference in total costs between the p‐EVES plus optical LVA intervention arm and the optical LVA only arm (£417.19, CI £366.04 to £465.89) was divided by the mean effect (0.567 NV‐VFQ‐15 points), producing an estimated ICER of £735.77 (CI £481.03 to £1525.18) per unit change in ‘near vision’ visual function.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“At a threshold of £1000 per point improvement, the NV‐VFQ‐15 results demonstrate an 84.4% probability of cost‐effectiveness.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Mean difference in total costs (£398.95, CI £336.69 to £460.15) was divided by the mean difference in effect between the two interventions (0.006, CI −0.009 to 0.019), yielding an estimated cost per QALY of £66 490: over double the upper NICE cost threshold of £30 000 per QALY.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“At a threshold of £30 000 per QALY/YFC gain, probability of effectiveness was 21.4% using the EQ‐5D‐5L and 9.4% using the ICECAP‐A.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Argus II retinal prosthesis versus usual care in retinitis pigmentosa, Eurozone: Vaidya et al. (2014)
Relevance: Evaluates the Argus II retinal prosthesis against usual care in retinitis pigmentosa from a healthcare payer perspective over a lifetime horizon, with costs in euros.
“A multi -state transition Markov model was developed to determine the cost-effectiveness of Argus II versus usual care in RP from the perspective of healthcare payer.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“A hypothetical cohort of 1000 RP patients aged 46 years followed up over a (lifetime) 25-year time horizon.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Health outcomes were expressed as quality adjusted life years (QALYs) and direct healthcare costs expressed in 2012 €. Results are reported as incremental cost per ratios (ICERs) with outcomes and costs discounted at an annual rate of 3.5%.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Argus II Retinal Prosthesis fitted patients incurred a discounted incremental cost of 42,455 Euros in comparison to the patient given ‘CAU’ over the 25 years of time. Progressive improvement in the visual acuity of the Argus II fitted patients resulted in gain of 2.91 incremental QALYs (discounted). Incremental cost-effectiveness ratio for Argus II was calculated to be 14,603 Euros per QALY.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The Probabilistic Incremental Cost-Effectiveness Ratio (ICER) was estimated to be 14,482 Euros per QALY.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“In Figure 3 the CEACs show that Argus II becomes 100% cost effective at a WTP of 31000 Euros.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights
Utility weights from analyses of Tinlarebant (LBS-008)
No evidence found.
Utility weights for the same indication
No evidence found.
Utility weights for the same disease in other populations or settings
No evidence found.
Utility weights for related diseases and analogous treatments
Utility weights by level of visual impairment, RPE65-mediated inherited retinal disease: Johnson et al. (2019)
Relevance: Utilities by level of visual impairment, from moderate impairment to hand motion or no light perception, used in a U.S. model of an inherited retinal disease.
“A health utility vignette study specific to RPE65 -mediated IRD was used for health utility inputs.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Health utility derivation in this study was based on vignettes describing the health states.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights for moderate, severe, and profound visual impairment, counting fingers, and hand motion to no light perception, biallelic RPE65-mediated inherited retinal disease: Bhadhuri et al. (2022)
Relevance: Utilities by level of visual impairment, from moderate impairment to hand motion or no light perception, used in a Swiss model of an inherited retinal disease.
“Health state utilities were derived from a study where EQ-5D-5L estimates for IRD patients with biallelic RPE65-mutation were elicited from clinicians” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights for retinitis pigmentosa by visual acuity state, serious adverse events, and explantation, retinitis pigmentosa: Vaidya et al. (2014)
Relevance: Utilities for retinitis pigmentosa by visual acuity state, after serious adverse events, and after explantation, from a model of the Argus II retinal prosthesis.
“Brown et al. has published utility values for minimal light perception, light perception, counting fingers and reading letters. These utility values are elicited by using time trade off method in patients with ocular diseases” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Patients who experienced Serious Adverse Events (SAEs) after the Argus II implantation were assigned a utility reduction of 0.16 which is equivalent to the lost utility value estimated for severe dry eye in an article by Schiffman et al.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights for blindness and visual impairment, retinitis pigmentosa: Schwander (2014)
Relevance: Utilities for blindness and for visual impairment, averaged from published estimates in a NORC report, in an analysis of retinitis pigmentosa.
“The effect estimates are based on published health related quality-of-life estimates for the health states ‘Blind’ and ‘Visual Impaired’. According to a recent NORC report” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“the average health utility is 0.61 for the health state ‘Blind’ (based on 6 publications with the following utility values: 0.39, 0.51, 0.55, 0.60, 0.79 and 0.80), and 0.77 for ‘Visual Impaired’ (based on 8 publications with the following utility values: 0.62, 0.65, 0.73, 0.76, 0.78, 0.79, 0.87 and 0.96); a health utility of zero was applied for the state ‘Death’.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Search strategy
| Dataset | Query | Results |
|---|---|---|
| Ratios | voretigene OR "inherited retinal" | 11 |
| Ratios | "gene therapy" AND ("retinal" OR "ocular" OR "eye") | 5 |
| Methods | tinlarebant OR "LBS-008" | 0 |
| Ratios | Stargardt OR "ABCA4" | 14 |
| Ratios | "geographic atrophy" OR pegcetacoplan OR avacincaptad | 5 |
| Ratios | "retinitis pigmentosa" OR "retinal dystrophy" OR "retinal degeneration" | 4 |
| Ratios | "low vision" OR "vision rehabilitation" | 19 |
| Weights | Stargardt OR "ABCA4" OR "macular dystrophy" | 37 |
| Weights | "inherited retinal" OR voretigene OR "retinitis pigmentosa" OR "retinal dystrophy" | 26 |
| Weights | "geographic atrophy" | 1 |