---
title: "Deramiocel (CAP-1002) in Duchenne muscular dystrophy: cost-effectiveness analyses and utility weights"
description: "Published cost-effectiveness analyses and health-state utility weights for Deramiocel (CAP-1002) in Duchenne muscular dystrophy, each value quoted from the article: 9 cost-effectiveness analyses and 5 studies reporting utility weights. Analyses: Deflazacort versus prednisone in Duchenne muscular dystrophy, United States: ICER (2019a); Ataluren and eteplirsen versus standard of care in Duchenne muscular dystrophy, Egypt: Shehata et al. (2023); Hypothetical treatment pausing progression versus standard of care in early ambulatory and early non-ambulatory Duchenne muscular dystrophy, United States: Klimchak et al. (2024); Hypothetical treatment versus standard of care in Duchenne muscular dystrophy, United Kingdom: Landfeldt et al. (2017); 5 further articles. Utility weights: Patient and caregiver utility weights by functional score, ambulatory status, and ventilation status, Duchenne muscular dystrophy: Landfeldt et al. (2017); Patient and caregiver utility weights by ambulatory stage, Duchenne muscular dystrophy: ICER (2019a); Utility weights by ambulatory stage, Duchenne muscular dystrophy: Klimchak et al. (2024); Utility weights by ambulatory stage, Duchenne muscular dystrophy: Shehata et al. (2023); 1 further article. Every value is a verbatim quote from the primary article it is shown with."
canonical: "https://www.evicenter.com/drugs/deramiocel--duchenne-muscular-dystrophy/cost-effectiveness-analyses"
published: "2026-10-22T07:49:06-04:00"
modified: "2026-10-22T07:49:06-04:00"
---

# Deramiocel (CAP-1002) in Duchenne muscular dystrophy: 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.

<a id="cost-effectiveness-analyses-overview"></a>

## Overview

<a id="cost-effectiveness-analyses-topic-overview-1"></a>

| 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 |

<a id="cost-effectiveness-analyses-cost-effectiveness-analyses"></a>

## 1 Cost-effectiveness analyses

<a id="cost-effectiveness-analyses-heading-1.1"></a>

### 1.1 Analyses of Deramiocel (CAP-1002)

No evidence found.

<a id="cost-effectiveness-analyses-heading-1.2"></a>

### 1.2 Treatments for the same indication

<a id="cost-effectiveness-analyses-topic-1.2-1"></a>

#### 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.

> “A five-state partitioned survival model was developed for this evaluation, informed by key clinical trials, cohort studies, and prior relevant studies related to economic modeling in DMD.24,36-40 The five health states were early ambulatory, late ambulatory, early non-ambulatory, late non- ambulatory, and death. Children entered the model in the early ambulatory health state at age five.”
> “The modeled population consisted of children diagnosed with DMD, with diagnosis and treatment commencing at age five. All comparative treatments were assumed to be given over a lifetime in accordance with the ICER Reference Case.”
> “Costs and outcomes were discounted at 3% per year.”
> “In this corrected version posted on April 22, 2022, we updated the cost-effectiveness analysis to correct an error in how treatment costs were estimated for prednisone and deflazacort.”
> “Deflazacort Compared to Prednisone Health Sector Perspective $663,000 $632,000 $458,000 Deflazacort Compared to Prednisone Modified Societal Perspective $692,000 $659,000 $478,000”
> “The base-case results for incremental cost-effectiveness ratios comparing deflazacort to prednisone represent cost-utility results that are well beyond the range of commonly accepted thresholds of $50,000 to $150,000 per QALY despite highly favorable assumptions about treatment effects being incorporated in the model (see Table ES5).”
> “Results of the probabilistic sensitivity analysis showed that at a $150,000 per QALY threshold, deflazacort had less than a 0.01% probability of being cost-effective. When increasing the threshold to $500,000 per QALY, deflazacort had roughly a 13% probability of being cost-effective.”

Source: [ICER (2019a)](https://icer.org/wp-content/uploads/2020/10/Corrected_ICER_DMD-Final-Report_042222.pdf)

<a id="cost-effectiveness-analyses-topic-1.2-2"></a>

#### 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.”
> “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.”
> “Prices were estimated based on other orphan drugs' prices.”
> “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.”
> “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.”

Source: [Shehata et al. (2023)](https://pubmed.ncbi.nlm.nih.gov/37862859/)

<a id="cost-effectiveness-analyses-heading-1.3"></a>

### 1.3 The same disease in other populations or settings

<a id="cost-effectiveness-analyses-topic-1.3-1"></a>

#### 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.”
> “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.”
> “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).”
> “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.”

Source: [Klimchak et al. (2024)](https://pubmed.ncbi.nlm.nih.gov/39094692/)

<a id="cost-effectiveness-analyses-topic-1.3-2"></a>

#### 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.”
> “For the evaluation results presented in this report, a 12-month cycle was chosen to accommodate the disease progression data”
> “All cohorts were followed from the age of 5 years until death (or an age of 100 years).”
> “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.”
> “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%”
> “Costs and QALYs were discounted at 3.5%. Cost results are presented in 2015 Great British pounds (£), rounded to the nearest ten.”
> “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”
> “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.”

Source: [Landfeldt et al. (2017)](https://pmc.ncbi.nlm.nih.gov/articles/PMC5253157/)

<a id="cost-effectiveness-analyses-topic-1.3-3"></a>

#### 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.”
> “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).”
> “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."”

Source: [Magnetta et al. (2018)](https://pubmed.ncbi.nlm.nih.gov/29774392/)

<a id="cost-effectiveness-analyses-heading-1.4"></a>

### 1.4 Related diseases and analogous treatments

<a id="cost-effectiveness-analyses-topic-1.4-1"></a>

#### 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.”
> “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.”
> “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.”
> “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.”

Source: [Evers et al. (2020)](https://pubmed.ncbi.nlm.nih.gov/31476408/)

<a id="cost-effectiveness-analyses-topic-1.4-2"></a>

#### 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.”
> “a hypothetical cohort (age 10–15 years) with dilated cardiomyopathy and symptomatic heart failure receiving outpatient medical management with or without an ICD”
> “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.”
> “Time horizon was up to 20 years from model entry.”
> “All costs were adjusted using the gross domestic product deflator method and reported in 2006 US dollars.”
> “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.”
> “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.”

Source: [Feingold et al. (2010)](https://pmc.ncbi.nlm.nih.gov/articles/PMC2929392/)

<a id="cost-effectiveness-analyses-topic-1.4-3"></a>

#### 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).”
> “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.”
> “The ICER was sensitive to ECMO cost, the long-term transplant survival rate, and quality of life in transplant recipients.”

Source: [Brown et al. (2009)](https://pubmed.ncbi.nlm.nih.gov/19134528/)

<a id="cost-effectiveness-analyses-topic-1.4-4"></a>

#### 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.

> “We developed three de novo economic models that evaluated the cost-effectiveness of Spinraza and Zolgensma, each compared to best supportive care (BSC), from a US health care sector perspective for patients with SMA, in alignment with ICER’s Value Assessment Framework for Ultra Rare Diseases. The models included 1) one for symptomatic patients with infantile-onset (Type I) SMA; 2) one for symptomatic patients with later-onset (Type II/III) SMA; and 3) one for presymptomatic SMA patients.”
> “For each population, we estimated the lifetime costs, life years gained, and quality adjusted life years (QALYs) gained, discounted at 3% per annum, for Spinraza and BSC.”
> “Results for Zolgensma were derived using a placeholder price of $2,000,000. Both interventions resulted in more QALYs and life years gained relative to BSC, resulting in incremental cost effectiveness ratios of approximately $1.1 million per QALY for Spinraza and approximately $243,000 per QALY for Zolgensma.”
> “Spinraza $2,231,000 $1,653,000 $3,884,000 3.24 7.64 $1,112,000 $590,000 BSC $0 $789,000 $789,000 0.46 2.40”
> “Zolgensma $2,000,000* $1,657,000 $3,657,000 12.23 18.17 $243,000 $182,000 BSC $0 $789,000 $789,000 0.46 2.40”
> “Spinraza $7,634,000 $1,514,000 $9,148,000 12.28 18.90 $8,156,000 Dominated BSC $0 $1,442,000 $1,442,000 11.34 18.90”
> “From the health care sector perspective, the cost per QALY and cost per LY gained were approximately $709,000 and $652,000 respectively (Table ES15).”
> “For Zolgensma, all simulations produced results that were cost-effective at and above a threshold of $300,000 per QALY.”

Source: [ICER (2019b)](https://icer.org/wp-content/uploads/2020/10/ICER_SMA_Final_Evidence_Report_110220.pdf)

<a id="cost-effectiveness-analyses-utility-weights"></a>

## 2 Utility weights

<a id="cost-effectiveness-analyses-heading-2.1"></a>

### 2.1 Utility weights from analyses of Deramiocel (CAP-1002)

No evidence found.

<a id="cost-effectiveness-analyses-heading-2.2"></a>

### 2.2 Utility weights for the same indication

No evidence found.

<a id="cost-effectiveness-analyses-heading-2.3"></a>

### 2.3 Utility weights for the same disease in other populations or settings

<a id="cost-effectiveness-analyses-topic-2.3-1"></a>

#### 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”

Source: [Landfeldt et al. (2017)](https://pmc.ncbi.nlm.nih.gov/articles/PMC5253157/)

| Health state | Patient | Caregiver |
| --- | --- | --- |
| “Initial score” | “0.879 (0.037)” | “0.862 (0.016)” |
| “Per lost score (multiplier)” | “0.905 (1.003)” | “0.995 (1.001)” |
| “Early ambulatory” | “0.699 (0.036)” | “0.858 (0.017)” |
| “Late ambulatory” | “0.607 (0.029)” | “0.839 (0.017)” |
| “Early non-ambulatory” | “0.224 (0.014)” | “0.784 (0.021)” |
| “Late non-ambulatory” | “0.146 (0.010)” | “0.810 (0.018)” |
| “None” | “0.518 (0.027)” | “0.837 (0.014)” |
| “Night-time” | “0.129 (0.017)” | “0.775 (0.030)” |
| “Day- and night-time” | “0.051 (0.010)” | “0.774 (0.033)” |

Source: [Landfeldt et al. (2017)](https://pmc.ncbi.nlm.nih.gov/articles/PMC5253157/)

<a id="cost-effectiveness-analyses-topic-2.3-2"></a>

#### 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 scores based on the Health Utility Index (HUI; proxy assessed by the primary caregivers) were included and separated into early and late ambulatory and early and late non- ambulatory in this study. Caregiver utilities were elicited from the EuroQol EQ-5D-3L.”

Source: [ICER (2019a)](https://icer.org/wp-content/uploads/2020/10/Corrected_ICER_DMD-Final-Report_042222.pdf)

| Health state | Patient | Caregiver |
| --- | --- | --- |
| “Early Ambulatory” | “0.730” | “0.845” |
| “Late Ambulatory” | “0.640” | “0.839” |
| “Early Non-Ambulatory” | “0.210” | “0.784” |
| “Late Non-Ambulatory” | “0.180” | “0.810” |

Source: [ICER (2019a)](https://icer.org/wp-content/uploads/2020/10/Corrected_ICER_DMD-Final-Report_042222.pdf)

<a id="cost-effectiveness-analyses-topic-2.3-3"></a>

#### 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).”

Source: [Klimchak et al. (2024)](https://pubmed.ncbi.nlm.nih.gov/39094692/)

<a id="cost-effectiveness-analyses-topic-2.3-4"></a>

#### 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.”
> “The incremental cost-effectiveness ratio was sensitive to health state utilities but not to state costs.”

Source: [Shehata et al. (2023)](https://pubmed.ncbi.nlm.nih.gov/37862859/)

<a id="cost-effectiveness-analyses-heading-2.4"></a>

### 2.4 Utility weights for related diseases and analogous treatments

<a id="cost-effectiveness-analyses-topic-2.4-1"></a>

#### 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.

> “Transitional probabilities, costs, and utility metrics were extracted from the existing literature.”

Source: [Evers et al. (2020)](https://pubmed.ncbi.nlm.nih.gov/31476408/)

<a id="cost-effectiveness-analyses-search-strategy"></a>

## 3 Search strategy

<a id="cost-effectiveness-analyses-topic-3-1"></a>

| 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 |

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