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.
9 cost-effectiveness analyses and 1 study reporting utility weights · 9 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 | 9 |
| Studies reporting utility weights | 1 |
Cost-effectiveness analyses
Analyses of SKY-0515
No evidence found.
Treatments for the same indication
Hypothetical disease-modifying treatments versus natural history in Huntington disease, United States: Guzauskas et al. (2024)
Relevance: Evaluates hypothetical disease-modifying treatments against Huntington's disease natural history from United States payer and modified societal perspectives, by the disease stage at treatment start, the decision-modeling framework section 4.1 cites for future disease-modifying treatments.
“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
The same disease in other populations or settings
In vitro fertilization with preimplantation genetic testing versus unassisted conception with prenatal diagnosis in couples with one heterozygous Huntington disease individual, United States: Christensen et al. (2022)
Relevance: Evaluates in vitro fertilization with preimplantation genetic testing against unassisted conception with prenatal diagnosis in couples with a partner heterozygous for Huntington's disease in the United States, a reproductive setting of the same disease.
“Cost-effectiveness analysis in a theoretical cohort of 3,851 couples, where one individual is heterozygous for Huntington disease.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Outcomes included cost and quality-adjusted life years (QALYs) for both parents” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A willingness-to-pay threshold was set at $100,000/QALY.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“In vitro fertilization preimplantation genetic testing is lower in cost and higher in effectiveness compared to unassisted conception with prenatal diagnosis among couples with one heterozygous Huntington disease individual, making it the dominant strategy.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“In vitro fertilization preimplantation genetic testing was associated with 77 more QALYs and a cost savings of $46,394,268.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Most notably, in our theoretical cohort of couples, IVF preimplantation genetic testing resulted in 1,079 fewer Huntington disease-affected offspring.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Related diseases and analogous treatments
Hypothetical disease-modifying therapy versus usual care in spinocerebellar ataxia type 1, Netherlands: van Prooije et al. (2023)
Relevance: Evaluates a hypothetical disease-modifying therapy started in the pre-ataxic or the slight ataxia stage of spinocerebellar ataxia type 1 against usual care in the Netherlands, with health states defined by ataxia severity stage.
“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
Continuous, fixed-duration, and test-and-discontinue regimens of a hypothetical disease-modifying therapy versus best supportive care in early symptomatic Alzheimer disease, United States: Boustani et al. (2022)
Relevance: Evaluates continuous, fixed-duration, and test-and-discontinue regimens of a hypothetical disease-modifying therapy against best supportive care in mild cognitive impairment due to Alzheimer disease or mild Alzheimer disease from a United States health care sector perspective.
“We developed a Markov state-transition model to estimate the cost-effectiveness of a hypothetical DMT plus best supportive care (BSC) versus BSC alone among Americans living with mild cognitive impairment (MCI) due to AD or mild AD.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A hypothetical DMT was assumed to confer a 30% reduction in progression from MCI and mild AD.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The base case annual drug acquisition cost was assumed to be $56,000.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“From the health care sector perspective, continuous treatment with a hypothetical DMT versus BSC resulted in an ICER of $612,354 per QALY gained.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The ICER decreased to $157,288 per QALY gained in the fixed-duration strategy, driven by large reductions in treatment costs.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“With 40% of patients discontinuing treatment at 6 months (test-and-discontinue strategy), the ICER was $125,631 per QALY gained.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Hypothetical disease-modifying and symptomatic therapies versus current care in amyotrophic lateral sclerosis, United Kingdom: Tappenden et al. (2024)
Relevance: Evaluates hypothetical disease-modifying and symptomatic therapies against current care in amyotrophic lateral sclerosis from a United Kingdom National Health Service and Personal Social Services perspective, starting from King's stage 2.
Access: Not open access
Not quoted: The results for the hypothetical symptomatic therapy are reported in the full text, which is closed access.
“We developed a health economic model to evaluate the cost effectiveness of future ALS treatments from a UK National Health Service and Personal Social Services perspective over a lifetime horizon using data from the ALS-CarE study.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Costs were valued at 2021/22 prices.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Two hypothetical interventions were evaluated: a DMT which delays progression and mortality, and a symptomatic therapy which improves utility only.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Starting from King's stage 2, patients receiving current care accrue an estimated 2.27 life-years, 0.75 QALYs and lifetime costs of £68,047.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Assuming a 50% reduction in progression rates and a UK-converted estimate of the price of edaravone, the incremental cost-effectiveness ratio for a new DMT versus current care is likely to exceed £735,000 per QALY gained.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Symptomatic therapies may be more likely to achieve acceptable levels of cost effectiveness.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Riluzole versus no treatment in amyotrophic lateral sclerosis, United States: Thakore et al. (2020)
Relevance: Evaluates riluzole against no treatment in amyotrophic lateral sclerosis in the United States from healthcare sector and societal perspectives, with stage-specific EQ-5D utilities.
Access: Free full text
Risdiplam with standard of care versus standard of care in spinal muscular atrophy types 1 and 2–3, Thailand: Khuntha et al. (2025)
Relevance: Evaluates risdiplam added to standard of care against standard of care alone in symptomatic spinal muscular atrophy, by type, in Thailand from provider and societal perspectives.
“A Markov model was used to analyze the lifetime costs and outcomes of these treatments compared with standard of care for symptomatic SMA types 1 and 2–3.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“a societal perspective over a lifetime horizon with a 3% annual discount rate for both costs and outcomes was used.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The societal perspective included both direct medical and direct non-medical care costs, and indirect costs incurred by caregivers, whereas the provider perspective included only direct medical costs.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“All costs were adjusted to 2023 values using the consumer price index (CPI)” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“whereas risdiplam produced 1.05 million USD of lifetime costs and 6.56 QALYs gained. For SOC, the lifetime cost was 294,000 USD with 1.82 QALYs gained.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“For SMA types 2–3 patients treated with risdiplam, the calculated ICER was 496,704 USD per QALY gained (Table 2).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Valbenazine and deutetrabenazine versus placebo in tardive dyskinesia, United States: ICER (2017)
Relevance: Evaluates valbenazine and deutetrabenazine against placebo in tardive dyskinesia from a United States health care system perspective, the vesicular monoamine transporter 2 inhibitors section 2 lists as treatment of Huntington's disease chorea.
Valbenazine versus deutetrabenazine in tardive dyskinesia, United States: Ganz et al. (2021)
Relevance: Evaluates valbenazine against deutetrabenazine in tardive dyskinesia in people receiving antipsychotic medication in the United States, vesicular monoamine transporter 2 inhibitors that section 2 lists as treatment of Huntington's disease chorea.
Access: Free full text
Utility weights
Utility weights from analyses of SKY-0515
No evidence found.
Utility weights for the same indication
No evidence found.
Utility weights for the same disease in other populations or settings
Utility weights by functional stage, Huntington disease: Guzauskas et al. (2024)
Relevance: EQ-5D utilities for premanifest Huntington's disease and for each Shoulson and Fahn stage defined by total functional capacity.
“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 for related diseases and analogous treatments
No evidence found.
Search strategy
| Dataset | Query | Results |
|---|---|---|
| Methods | "SKY-0515" OR Skyhawk | 0 |
| Methods | "Huntington's disease" OR Huntington OR "Huntington disease" | 19 |
| Ratios | "Huntington's disease" OR "Huntington disease" OR Huntington | 34 |
| Weights | "Huntington's disease" OR "Huntington disease" OR Huntington | 77 |
| Ratios | tominersen OR "AMT-130" OR tetrabenazine OR deutetrabenazine OR valbenazine | 3 |
| Ratios | risdiplam OR branaplam | 19 |
| Ratios | omaveloxolone OR "Friedreich ataxia" OR tofersen OR "amyotrophic lateral sclerosis" | 15 |
| Ratios | "disease-modifying" AND (neurodegenerative OR "hypothetical") | 58 |
| Ratios | lecanemab OR donanemab | 30 |
| Weights | chorea OR "total functional capacity" OR "Shoulson" | 5 |
| Ratios | splicing OR "splice modulator" OR "huntingtin" | 0 |