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.
7 cost-effectiveness analyses and 6 studies reporting utility weights · 8 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 | 7 |
| Studies reporting utility weights | 6 |
Cost-effectiveness analyses
Analyses of Aqneursa (levacetylleucine and IB1001)
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
Hypothetical disease-modifying therapy versus usual care in spinocerebellar ataxia type 1, Netherlands: van Prooije et al. (2023)
Relevance: Models a hypothetical disease-modifying therapy against usual care in spinocerebellar ataxia type 1 in the Netherlands, a progressive cerebellar ataxia with health states by ataxia severity; levacetylleucine is approved for ataxia in A-T.
“A patient-level state-transition model was constructed to simulate current practice, as well as the possibilities and consequences of a hypothetical future therapy in individuals with SCA1. The model simulates the consequences of disease progression of individual patients through different health states of SCA1, over a lifetime horizon.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“These health states are based on SARA score: pre-ataxic (SARA 0–2), slight ataxia (SARA > 2–14), moderate ataxia (SARA > 14–26), severe ataxia (SARA > 26–34), and end-stage ataxia (SARA > 34)” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“future QALYs were discounted at 1.5% annually according to Dutch guidelines” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Where needed, costs were converted to the year 2020, using the consumer price index from Statistics Netherlands [ 30 ]. Costs per health state are presented in Table 2 . To take time preferences into account, future costs were discounted at a 4% rate annually” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“For SCA1 in the Netherlands, the threshold was calculated to be €80,000 per QALY [ 31 , 32 ]. Since the costs of the hypothetical therapy are not included in the model, the iNMB represents the maximum costs of the hypothetical therapy to be considered cost-effective.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Usual care €118,821 10.44 – €118,821 10.44 – Full treatment €5079 2.44 €190,165 €324 6.68 €534,274 Start at slight €6217 2.31 €178,648 €3,385 6.27 €498,372” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“This implies that if treatment is 50% effective, it is cost-effective if the total costs of therapy do not exceed €534,274. At 25% effectiveness iNMB was €190,165.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Miglustat versus symptomatic therapy in Niemann-Pick disease type C, Serbia: Gutić et al. (2022)
Relevance: Evaluates miglustat against symptomatic therapy in Niemann-Pick disease type C in Serbia; Niemann-Pick disease type C is the first approved indication of levacetylleucine (section 2).
“The perspective of the Serbian Republic Health Insurance Fund was chosen for this study, and the time horizon was eighty years.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The study was conducted through the generation and simulation of the Discrete-Event Simulation model. The model results were obtained after Monte Carlo microsimulation of a sample with 1000 virtual patients.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Treatment with miglustat was not cost-effective when compared with symptomatic therapy and was associated with negative values of net monetary benefit regardless of the onset of neurological manifestations (- 110,447,627.00 ± 701,614.00 RSD, - 343,871,695.00 ± 2,577,441.00 RSD, - 1,397,908,502.00 ± 23,084,235.00 RSD and - 2,953,680,879.00 ± 33,297,412.00 RSD) for early infantile, late infantile, juvenile and adolescent cohorts, respectively).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Hypothetical disease-modifying treatments versus natural history in Huntington disease, United States: Guzauskas et al. (2024)
Relevance: Models hypothetical disease-modifying treatments started at successive functional stages of Huntington disease against natural history from a U.S. societal perspective, a stage-based model of a progressive inherited neurodegenerative disease.
“We developed a health state transition model that separately analyzed 40-year-old individuals with prefunctional decline (PFD, HD Integrated Staging System [HD-ISS] stage <3, total functional score [TFC] 13), active functional decline Shoulson and Fahn category 1 (SF1, HD-ISS stage 3, TFC 13-11), and SF2 (HD-ISS stage 3, TFC 10-7).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“We used annual model cycles, a lifetime horizon, and a US payer perspective (i.e., focused on direct medical care costs [in 2023 US dollars 10 ] only) as well as a modified societal perspective incorporating indirect medical costs and productivity loss. We discounted all model outcomes by 3% per year to reflect their present value” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“(1) DMT A delays further functional decline by 3 years and reduces the rate of TFC progression by 25% (rate ratio applied to the constant TFC progression rate = 0.75); (2) DMT B delays further functional decline by 2 years and reduces the rate of TFC progression by 15% (rate ratio = 0.85); and (3) DMT C delays further functional decline by 1 year and reduces the rate of TFC progression by 5% (rate ratio = 0.95).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Prefunctional decline population a (discounted) Incremental costs −$35,220 −$142,904 to $20,972 −$21,808 −$92,426 to $25,927 −$14,335 −$69,357 to $33,443” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Shoulson and Fahn stage 1 population (discounted) Incremental costs $188,245 −$34,489 to $395,879 $115,352 −$64,427 to $308,235 $40,184 −$135,263 to $215,048” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Shoulson and Fahn stage 2 population (discounted) Incremental costs $279,724 −$72,371 to $550,523 $168,799 −$72,088 to $403,636 $62,875 −$132,939 to $274,364” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The life year and QALY gains were lower for the PFD population (1.48 and 1.42, respectively) compared with the SF1 (4.04 and 3.13, respectively) and SF2 (4.17 and 2.67, respectively) populations.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“However, while there were potential standard care and societal cost savings resulting from DMTs observed in the PFD population, standard care and societal costs were generally increased compared with natural history in the SF1 and SF2 populations because of prolonged time spent in the more expensive, lower quality-of-life health states.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Cerliponase alfa versus best supportive care in neuronal ceroid lipofuscinosis type 2, Canada: CADTH (2019)
Relevance: Evaluates cerliponase alfa against best supportive care in neuronal ceroid lipofuscinosis type 2 from a Canadian public health care payer perspective, a rare progressive pediatric neurodegenerative disease treated with a chronic intracerebroventricular infusion.
Lifelong immunoglobulin replacement therapy versus hematopoietic stem cell transplant in congenital agammaglobulinemia, United States: Sun et al. (2022)
Relevance: Evaluates lifelong immunoglobulin replacement therapy against hematopoietic stem cell transplant in congenital agammaglobulinemia in the United States from a societal perspective; immunoglobulin replacement is a supportive therapy for the immunodeficiency of A-T listed in section 2.
“This economic evaluation used Markov analysis to model the base-case scenario of a patient aged 12 months with congenital agammaglobulinemia receiving lifelong IRT vs MSD or MUD HSCT.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“One-way sensitivity and probabilistic sensitivity analyses evaluated uncertainty around parameter estimates performed from a societal perspective over a 100-year time horizon. The threshold for cost-effective care was set at $100 000 per quality-adjusted life-year (QALY).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“All costs were adjusted for inflation and are reported in 2020 US dollars with future costs and QALYs discounted at 3% annually with adjustment from 0% to 7% in sensitivity analysis.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“In this economic evaluation of patients with congenital agammaglobulinemia, lifelong IRT cost more than HSCT ($1 512 946 compared with $563 776 [MSD] and $637 036 [MUD]) and generated similar QALYs (20.61 vs 17.25 [MSD] and 17.18 [MUD]). Choosing IRT over MSD or MUD HSCT yielded ICERs of $282 166 per QALY gained over MSD and $255 633 per QALY gained over MUD HSCT, exceeding the US willingness-to-pay threshold of $100 000/QALY.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“When annual IRT price was reduced from $60 145 to below $29 469, IRT became the cost-effective strategy.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Home-based subcutaneous versus hospital-based intravenous immunoglobulin in primary immunodeficiency disease, Australia: Windegger et al. (2020)
Relevance: Compares home-based subcutaneous with hospital-based intravenous immunoglobulin replacement in primary immunodeficiency disease in Australia, the supportive immunoglobulin therapy listed for A-T in section 2.
“We developed a Markov cohort simulation model with six health states: PID without infection, PID with infection treated at home or hospital, bronchiectasis without infection, bronchiectasis with infection treated at home or hospital, bronchiectasis with chronic Pseudomonas aeruginosa infection, and death, from an Australian healthcare system perspective. A 10-year time horizon with weekly cycles was chosen” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“All cost and outcomes (QALYs) of patients in both groups were converted to present value using a discount rate of 5% annually, as per standard practice in Australia36, which was adjusted to weekly cycles for analysis.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The cumulative 10-year cost per patient was 297,547 Australian dollars (A$) with IVIg and A$ 251,713 for SCIg. IVIg resulted in 5.55 QALYs and SCIg 5.57 QALYs. Thus, SCIg appears to be a cost-saving option and possibly improves QALY from the Australian healthcare system perspective (i.e., the dominant treatment option).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“A probabilistic sensitivity analysis showed that the SCIg option is preferred in 93.2% of simulations given willingness to pay of A$ 50,000 per QALY gained.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Newborn screening for severe combined immunodeficiency versus no screening, Netherlands: Van der Ploeg et al. (2019)
Relevance: Evaluates newborn screening for severe combined immunodeficiency in the Netherlands; section 2 reports that this T-cell receptor excision circle screening also identifies infants with A-T.
“Lifetime costs and effects of newborn screening for SCID were compared to a situation without screening in the Netherlands in a decision analysis model. Model parameters were based on literature and expert opinions.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Due to earlier detection, the number of deaths due to SCID per 100,000 children was assessed to decrease from 0.57 to 0.23 and a number of 11.7 quality adjusted life-years (QALYs) gained was expected.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Total yearly healthcare costs, including costs of screening, diagnostics, and treatment, were €390,800 higher in a situation with screening compared to a situation without screening, resulting in a cost-utility ratio of €33,400 per QALY gained.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights
Utility weights from analyses of Aqneursa (levacetylleucine and IB1001)
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 ataxia stage, spinocerebellar ataxia type 1: van Prooije et al. (2023)
Relevance: Utilities for the pre-ataxic stage and for slight, moderate, severe, and end-stage ataxia in spinocerebellar ataxia type 1, for the progression of ataxia in A-T.
“Values are based on EQ-5D data from the EuroSCA dataset. The utility value for the pre-ataxic stage is based on the Dutch EQ-5D-3L population norm at age 35–44” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Disutilities for dysphagia, falls, loss of autonomy, and slurred speech, Niemann-Pick disease type C: Gutić et al. (2022)
Relevance: Disutilities for dysphagia, incoordination leading to falls, loss of autonomy, and slurred speech in Niemann-Pick disease type C, the first approved indication of levacetylleucine.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
Utility weights for untreated and treated Niemann-Pick disease type C: van Karnebeek et al. (2015)
Relevance: Utilities for untreated and treated Niemann-Pick disease type C, the first approved indication of levacetylleucine.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
“A discrete event simulation model was conducted to follow ID patients through the diagnosis and treatment of NP-C, forecast the costs and effectiveness for a cohort of ID patients and compare the outcomes and costs in two different arms of the model: plasma oxysterol screening and routine diagnosis procedure (anno 2013) over 5 years of follow up. Data from published sources and clinical trials were used in simulation model.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“It would result in lower total cost and would slightly improve patients' quality of life.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights by functional stage, Huntington disease: Guzauskas et al. (2024)
Relevance: EQ-5D utilities for the premanifest stage and the functional stages of Huntington disease, a staged progressive inherited neurodegenerative disease.
“The PFD population, for which we modeled a spectrum of symptom onset timing over 60 years based on the HD-ISS, was assigned a health state utility equivalent to the general US population that was obtained from a US-based EQ-5D-5L study of population norms.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“For the active functional decline health states (SF1-SF4), we used health state utilities obtained from the EQ-5D-5L outcomes of a related retrospective, cross-sectional study of the HD burden of illness conducted between September 2020 and May 2021 in France, Germany, Italy, Spain, the United Kingdom, and the United States” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights with and without infection and bronchiectasis, primary immunodeficiency disease: Windegger et al. (2020)
Relevance: Utilities with and without infection and with and without bronchiectasis in primary immunodeficiency disease, for the immunodeficiency and sinopulmonary disease of A-T.
“Utility weights calculated from the Assessment of Quality of Life utility instrument AQoL-6D (n=33)” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights for agammaglobulinemia with and without immunoglobulin replacement therapy and after stem cell transplant, agammaglobulinemia: Sun et al. (2022)
Relevance: Utilities for agammaglobulinemia with and without immunoglobulin replacement therapy, the supportive therapy for the immunodeficiency of A-T listed in section 2.
Search strategy
| Dataset | Query | Results |
|---|---|---|
| Ratios | levacetylleucine OR "acetyl-leucine" OR "acetyl-L-leucine" OR Aqneursa OR IB1001 | 0 |
| Methods | levacetylleucine OR "acetyl-leucine" OR "acetyl-L-leucine" OR Aqneursa OR IB1001 | 0 |
| Methods | "ataxia-telangiectasia" OR "ataxia telangiectasia" OR "Louis-Bar" | 0 |
| Ratios | ataxia OR ataxic | 3 |
| Weights | ataxia OR ataxic OR "Friedreich" OR spinocerebellar | 10 |
| Ratios | omaveloxolone OR "Friedreich" OR "Niemann-Pick" OR miglustat OR arimoclomol | 6 |
| Ratios | "immunoglobulin replacement" OR "primary immunodeficiency" OR "subcutaneous immunoglobulin" | 5 |
| Weights | "Niemann-Pick" OR "primary immunodeficiency" OR "immunoglobulin replacement" OR agammaglobulinemia | 41 |
| Ratios | cerliponase OR "Batten" OR "Rett syndrome" OR trofinetide OR "atidarsagene" OR "Huntington" | 36 |
| Ratios | "severe combined immunodeficiency" AND "newborn screening" | 1 |
| Weights | "ataxia-telangiectasia" OR "ataxia telangiectasia" | 0 |