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 5 studies 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 | 5 |
Cost-effectiveness analyses
Analyses of Deramiocel (CAP-1002)
No evidence found.
Treatments for the same indication
Deflazacort versus prednisone in Duchenne muscular dystrophy, United States: ICER (2019a)
Relevance: Evaluates deflazacort against prednisone in Duchenne muscular dystrophy in the United States; corticosteroids were the background therapy of both arms of the HOPE-3 trial.
Ataluren and eteplirsen versus standard of care in Duchenne muscular dystrophy, Egypt: Shehata et al. (2023)
Relevance: Evaluates ataluren and eteplirsen against standard of care in Duchenne muscular dystrophy in Egypt; mutation-specific exon-skipping therapies are among the current treatment options listed in section 2.
“This study describes 2 cost-utility models comparing the drugs with the standard of care.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“We used a partition-survival model with 5 states based on the ambulatory status to model a cohort of ambulatory patients at the age of 5 years.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Prices were estimated based on other orphan drugs' prices.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“In the base case, ataluren 1000 mg and eteplirsen 50 mg/mL resulted in an incremental cost-effectiveness ratio of EGP 51 745 605 and EGP 69 652 533/quality-adjusted life-year, respectively, at their hypothetical prices of EGP 308 600 for ataluren 30-sachet pack and EGP 62 800 for eteplirsen 10 mL vial.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“At EGP 911 719/quality-adjusted life-year threshold, the value-based prices were EGP 4680 for ataluren 1000 mg and EGP 733 for eteplirsen 10 mL vial.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
The same disease in other populations or settings
Hypothetical treatment pausing progression versus standard of care in early ambulatory and early non-ambulatory Duchenne muscular dystrophy, United States: Klimchak et al. (2024)
Relevance: Models a hypothetical treatment that pauses progression from the early ambulatory or early non-ambulatory stage of Duchenne muscular dystrophy against standard of care in the United States; HOPE-3 enrolled late ambulatory and non-ambulatory participants.
Access: Not open access
Not quoted: The results for each pause duration are reported in the full text, which is closed access.
“A previously published, 5-state model, which analyzed treatments for early ambulatory (EA) DMD patients, was replicated, validated, and adapted to include early nonambulatory (ENA) DMD patients.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The model was used to assess a QALY-based threshold price (maximum cost-effective price) for a hypothetical treatment for 13-year-old ENA and 5-year-old EA patients (initial health states with lower and higher utility, respectively). All inputs were replicated including willingness-to-pay thresholds of $50 000 to $200 000/QALY.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“In contrast to EA patients, ENA patients had a 98% modeled decline in QALY-based threshold price at a willingness-to-pay of $150 000/QALY or higher, despite equal treatment benefit (delayed progression/death).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“At $100 000/QALY or lower, net nontreatment costs exceeded health benefits, implying any treatment for ENA patients would not be considered cost-effective, even at $0 price, including an indefinite pause in disease progression.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Hypothetical treatment versus standard of care in Duchenne muscular dystrophy, United Kingdom: Landfeldt et al. (2017)
Relevance: Sets out Markov frameworks based on functional score, stage of disease, and ventilation status that compare a hypothetical intervention with standard of care in Duchenne muscular dystrophy in the United Kingdom, the frameworks cited in section 4.1 of this report.
“We constructed three Markov cohort state-transition models to evaluate the cost-effectiveness of a hypothetical intervention for DMD versus standard of care in a UK setting. Model I was based on the DMDSAT, model II on stages of disease as defined in the DMD clinical care guidelines and model III on patients’ ventilation status.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“For the evaluation results presented in this report, a 12-month cycle was chosen to accommodate the disease progression data” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“All cohorts were followed from the age of 5 years until death (or an age of 100 years).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Our base-case scenario was executed from a healthcare perspective including direct medical costs, excluding informal care costs, as well as the impact of the disease on patient HR-QOL. In addition, we analysed a scenario from the societal perspective comprising all costs (i.e. direct medical and non-medical, including informal care costs, and indirect costs) as well as the impact on patient and primary caregiver HR-QOL.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“we specified a base-case scenario of a lifelong hypothetical intervention that reduced the probability of disease progression across all model states by a conservative (but realistic) 25%” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Costs and QALYs were discounted at 3.5%. Cost results are presented in 2015 Great British pounds (£), rounded to the nearest ten.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“ICER (ΔC/ΔE) Healthcare perspective 1,442,710 1,939,590 3,574,770 Societal perspective 1,266,510 1,760,650 3,121,890” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Across frameworks, lifetime direct medical costs with standard of care ranged between £217,510 and £284,640, total costs between £624,240 and £713,840, and total number of quality-adjusted life-years between 5.96 and 7.17.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Destination ventricular assist device therapy versus medical management in Duchenne muscular dystrophy with advanced heart failure, United States: Magnetta et al. (2018)
Relevance: Evaluates ventricular assist device destination therapy for advanced heart failure in Duchenne muscular dystrophy, the end stage of the cardiomyopathy that HOPE-3 assessed through a cardiac endpoint and a cardiomyopathy subgroup analysis.
“We created a Markov-state transition model (5-year horizon) to compare survival, costs, and quality of life (QOL) between medical management and DT-VAD in DMD with advanced HF. Model input parameters were derived from the literature.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“DT-VAD had higher costs ($435,602 vs. $125,696), survival (3.13 vs. 0.60 years), and quality-adjusted survival (1.99 vs. 0.26 years) than medical management. The incremental cost-effectiveness ratio (ICER) for DT-VAD was $179,086 per quality-adjusted life year (QALY).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Only when VAD implantation costs were <$113,142 did the DT-VAD strategy fall below the $100,000/QALY willingness-to-pay threshold commonly considered to be "cost-effective."” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Related diseases and analogous treatments
Home with a wearable cardioverter-defibrillator versus home without one or inpatient care in pediatric dilated cardiomyopathy, United States: Evers et al. (2020)
Relevance: Evaluates wearable cardioverter-defibrillators for children with cardiomyopathy in the United States, a pediatric cardiomyopathy model whose cardiac health states a model of Duchenne cardiomyopathy could adapt.
Access: Not open access
Not quoted: The result for inpatient care is reported in the full text, which is closed access.
“A Markov state transition model was constructed for the 3 clinical approaches to compare costs, clinical outcomes, and quality of life: (1) "Inpatient," (2) "Home-WCD," and (3) "Home-No WCD." Transitional probabilities, costs, and utility metrics were extracted from the existing literature.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Cost-effectiveness was assessed comparing each paradigm's incremental cost-effectiveness ratio against a societal willingness-to-pay threshold of $50,000 per quality-adjusted life year.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The cost-utility analysis illustrated that Home-WCD met the willingness-to-pay threshold with an incremental cost-effectiveness ratio of $20,103 per quality-adjusted life year and 4 mortalities prevented per 100 patients as compared with Home-No WCD.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“One-way sensitivity analyses demonstrated that Home-No WCD became the most cost-effective solution when the probability of SCA fell below 0.2% per week, the probability of SCA survival with a WCD fell below 9.8%, or the probability of SCA survival with Home-No WCD quadrupled from base-case assumptions.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Prophylactic implantable cardioverter-defibrillator versus typical management in childhood dilated cardiomyopathy, United States: Feingold et al. (2010)
Relevance: Evaluates prophylactic implantable cardioverter-defibrillators added to typical management in children with dilated cardiomyopathy and symptomatic heart failure in the United States from a societal perspective, a model of outpatient management for a pediatric cardiomyopathy population.
“We developed a Markov model to compare typical management of childhood dilated cardiomyopathy with symptomatic heart failure to prophylactic ICD implantation plus typical management.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“a hypothetical cohort (age 10–15 years) with dilated cardiomyopathy and symptomatic heart failure receiving outpatient medical management with or without an ICD” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The model adhered to the recommendations of the Panel on Cost-Effectiveness in Health and Medicine by examining costs from a societal perspective and applying a 3% annual discount to both costs and health benefits.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Time horizon was up to 20 years from model entry.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“All costs were adjusted using the gross domestic product deflator method and reported in 2006 US dollars.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Total per patient costs were $433,000 for the ICD strategy and $355,000 for typical management. Quality adjusted survival was greater for the ICD strategy relative to typical management (6.78 years vs. 6.43 years). Under base-case assumptions, the incremental cost utility ratio for the ICD strategy, as compared to typical management, was $281,622 per quality-adjusted life year (QALY) gained.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The ICD strategy cost less than $100,000/QALY only when the annual probability of sudden death as an outpatient exceeded 13% or when the ICD improved the patient’s quality of life by a utility of ≥ 0.11.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Extracorporeal membrane oxygenation bridging to transplant versus conventional intensive care in children with end-stage heart failure from dilated cardiomyopathy, United Kingdom: Brown et al. (2009)
Relevance: Evaluates extracorporeal membrane oxygenation as a bridge to heart transplantation for children with end-stage heart failure from dilated cardiomyopathy in the United Kingdom, an end-stage cardiac pathway a model of Duchenne cardiomyopathy could include.
“We performed a cost utility evaluation with a decision model approach, including 75 children with dilated cardiomyopathy. A cohort of patients with end stage heart failure who were offered ECMO bridging was compared with a similar cohort offered only conventional intensive care. Outcome was measured in cost per quality adjusted life year (QALY).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The reference incremental cost-effectiveness ratio (ICER) was pound65,645 per QALY and pound54,284 per life-year gained. Average life expectancy rose from 6.78 to 9.79 years and costs from pound146,398 to pound309,599 per patient with ECMO bridging.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The ICER was sensitive to ECMO cost, the long-term transplant survival rate, and quality of life in transplant recipients.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Nusinersen and onasemnogene abeparvovec versus best supportive care in spinal muscular atrophy, United States: ICER (2019b)
Relevance: Evaluates nusinersen and onasemnogene abeparvovec against best supportive care in spinal muscular atrophy from a U.S. health care sector perspective, a progressive pediatric neuromuscular disease treated with disease-modifying therapies.
Utility weights
Utility weights from analyses of Deramiocel (CAP-1002)
No evidence found.
Utility weights for the same indication
No evidence found.
Utility weights for the same disease in other populations or settings
Patient and caregiver utility weights by functional score, ambulatory status, and ventilation status, Duchenne muscular dystrophy: Landfeldt et al. (2017)
Relevance: Utilities by functional score, ambulatory status, and ventilation status in Duchenne muscular dystrophy, for people with the disease and their caregivers.
“Patient utilities (ranging from 0 = dead to 1 = perfect health) were obtained from the Health Utilities Index Questionnaire and caregiver utilities from the EuroQol EQ-5D-3L” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Patient and caregiver utility weights by ambulatory stage, Duchenne muscular dystrophy: ICER (2019a)
Relevance: Utilities by ambulatory stage of Duchenne muscular dystrophy for people with the disease and their caregivers, used in the U.S. assessment of deflazacort.
Utility weights by ambulatory stage, Duchenne muscular dystrophy: Klimchak et al. (2024)
Relevance: Utilities for the early and late ambulatory and non-ambulatory stages of Duchenne muscular dystrophy used in a U.S. threshold-price model; the late ambulatory and non-ambulatory stages describe the HOPE-3 population.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
“The model was used to assess a QALY-based threshold price (maximum cost-effective price) for a hypothetical treatment for 13-year-old ENA and 5-year-old EA patients (initial health states with lower and higher utility, respectively).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights by ambulatory stage, Duchenne muscular dystrophy: Shehata et al. (2023)
Relevance: Utilities for the early and late ambulatory and non-ambulatory stages of Duchenne muscular dystrophy used in the Egyptian analysis of ataluren and eteplirsen.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
“Costs and utilities were from a local study.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The incremental cost-effectiveness ratio was sensitive to health state utilities but not to state costs.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights for related diseases and analogous treatments
Utility weights for life with and without a wearable defibrillator, shocks, and sudden cardiac arrest, pediatric dilated cardiomyopathy: Evers et al. (2020)
Relevance: Utilities for children with dilated cardiomyopathy living with and without a wearable defibrillator, and for shocks and sudden cardiac arrest, for the cardiac states of a Duchenne cardiomyopathy model.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
Search strategy
| Dataset | Query | Results |
|---|---|---|
| Ratios | deramiocel OR "CAP-1002" OR cardiosphere | 0 |
| Ratios | "Duchenne" | 15 |
| Methods | "muscular dystrophy" | 5 |
| Ratios | "muscular dystrophy" | 15 |
| Weights | "Duchenne" OR "muscular dystrophy" | 34 |
| Ratios | eteplirsen OR golodirsen OR viltolarsen OR casimersen OR ataluren OR "exon skipping" | 4 |
| Ratios | delandistrogene OR Elevidys OR givinostat OR vamorolone OR deflazacort | 4 |
| Ratios | nusinersen OR risdiplam OR onasemnogene OR "spinal muscular atrophy" | 85 |
| Ratios | "cardiomyopathy" | 79 |
| Ratios | "ventricular assist" OR "heart transplant" OR "heart transplantation" | 93 |
| Ratios | "cell therapy" OR "stem cell" OR "mesenchymal" | 335 |
| Weights | "dilated cardiomyopathy" OR "pediatric cardiomyopathy" | 20 |
| Methods | deramiocel OR "CAP-1002" OR cardiosphere | 0 |
| Ratios | "ventricular assist" AND "Duchenne" | 0 |
| Weights | "ventricular assist" AND "Duchenne" | 0 |