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Zeleciment rostudirsen (z-rostudirsen)

Duchenne muscular dystrophy

Also known as DYNE-251
Manufacturer
Dyne Therapeutics
Last updated
78 sources

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.

8 cost-effectiveness analyses and 4 studies reporting utility weights · 8 sources

Overview

ItemDetail
Literature searchedTufts Medical Center Cost-Effectiveness Analysis Registry, searched October 21, 2026; the queries are listed in section 3.
Cost-effectiveness analyses8
Studies reporting utility weights4

Cost-effectiveness analyses

Analyses of Zeleciment rostudirsen (z-rostudirsen and DYNE-251)

No evidence found.

Treatments for the same indication

Ataluren and eteplirsen versus standard of care in Duchenne muscular dystrophy, Egypt: Shehata et al. (2023)

Relevance: Evaluates eteplirsen, the approved exon 51 skipping therapy that section 2 lists as the treatment alternative and that the PROMOVI study evaluated, and ataluren against standard of care in Duchenne muscular dystrophy in Egypt.

“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

Deflazacort versus prednisone in Duchenne muscular dystrophy, United States: ICER (2019a)

Relevance: Evaluates deflazacort against prednisone in Duchenne muscular dystrophy in the United States, within an assessment of deflazacort, eteplirsen, and golodirsen; DELIVER required a stable glucocorticoid dosage at enrollment.

“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.” (opens the source at this quote in a new tab)

“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.” (opens the source at this quote in a new tab)

“Costs and outcomes were discounted at 3% per year.” (opens the source at this quote in a new tab)

“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.” (opens the source at this quote in a new tab)

“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” (opens the source at this quote in a new tab)

“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).” (opens the source at this quote in a new tab)

“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.” (opens the source at this quote in a new tab)

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 the threshold price of 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; DELIVER enrolled ambulatory and recently 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, by functional score, ambulatory status, 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

Related diseases and analogous treatments

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 United States health care sector perspective, in symptomatic infantile-onset, symptomatic later-onset, and presymptomatic populations.

“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.” (opens the source at this quote in a new tab)

“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.” (opens the source at this quote in a new tab)

“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.” (opens the source at this quote in a new tab)

“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” (opens the source at this quote in a new tab)

“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” (opens the source at this quote in a new tab)

“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” (opens the source at this quote in a new tab)

“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).” (opens the source at this quote in a new tab)

“For Zolgensma, all simulations produced results that were cost-effective at and above a threshold of $300,000 per QALY.” (opens the source at this quote in a new tab)

Nusinersen versus best supportive care in infantile-onset spinal muscular atrophy, United States: Thokala et al. (2020)

Relevance: Evaluates nusinersen against best supportive care in infantile-onset spinal muscular atrophy from a United States health care sector perspective, based on the ENDEAR and SHINE studies.

“A de novo model was developed in Microsoft Office Excel 2016, to estimate the lifetime cost-effectiveness of nusinersen compared to best supportive care (BSC) for patients with infantile-onset SMA, from the US health care sector perspective. Costs, life years (LYs), and quality-adjusted life years (QALYs) were discounted at 3% per annum.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote

“Short-term data were sourced from the pivotal clinical trials and studies of nusinersen (ENDEAR and SHINE). Motor function milestones achieved at the end of follow-up in the clinical trials were assumed to be sustained until death.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote

“All costs were inflated to 2017 values.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote

“The total lifetime costs associated with nusinersen were approximately $3.9 million and were $790,000 for BSC (Table 5 ). Nusinersen produces greater QALYs and LYs (3.24 and 7.64, respectively) compared with BSC (0.46 QALYs and 2.40 LYs). This resulted in an incremental cost per QALY gained of approximately $1,112,000 and an incremental cost per LY gained of $590,000 for nusinersen compared with BSC.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote

“The incremental cost effectiveness ratios did not fall below $990,000 per QALY gained in scenario and sensitivity analyses.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote

Nusinersen versus standard of care in infantile-onset and later-onset spinal muscular atrophy, Sweden: Zuluaga-Sanchez et al. (2019)

Relevance: Evaluates nusinersen against standard of care in infantile-onset and later-onset spinal muscular atrophy in Sweden, including caregiver quality-adjusted life-years.

“One Markov cohort health-state transition model was developed for each population. The infantile-onset and later-onset models were based on the efficacy results from the ENDEAR phase III trial and the CHERISH phase III trial, respectively. The cost effectiveness of nusinersen in both models was compared with standard of care in Sweden.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

“For a time horizon of 40 years in the infantile-onset model and 80 years in the later-onset model, treatment with nusinersen resulted in 3.86 and 9.54 patient incremental quality-adjusted life-years and 0.02 and 2.39 caregiver incremental quality-adjusted life-years and an incremental cost of 21.9 and 38.0 million SEK (Swedish krona), respectively.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

“These results translated into incremental cost-effectiveness ratios (including caregiver quality-adjusted life-years) of 5.64 million SEK (€551,300) and 3.19 million SEK (€311,800) per quality-adjusted life-year gained in the infantile-onset model and later-onset model, respectively.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

“Nusinersen was not cost effective when using a willingness-to-pay threshold of 2 million SEK (€195,600), which has been considered in a recent discussion by the Dental and Pharmaceutical Benefits Agency as a reasonable threshold for rare disease.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote

Inotersen and patisiran versus best supportive care in hereditary transthyretin amyloidosis, United States: ICER (2018)

Relevance: Evaluates inotersen and patisiran against best supportive care in hereditary transthyretin amyloidosis from United States health care sector and societal perspectives, with health states defined by disease stage.

“We conducted a cost-effectiveness analysis using a de novo Markov model featuring FAP disease stages and a one-month cycle length over a lifetime time horizon.” (opens the source at this quote in a new tab)

“The model was developed with dual base cases (a health care sector perspective and a societal perspective) under the ultra-rare disease adaptation of ICER’s value framework, with costs and outcomes discounted at 3% annually.” (opens the source at this quote in a new tab)

“The target population for the first economic evaluation was adults with hATTR with an indication for treatment with inotersen (as reflected in the NEURO-TTR trial). The target population for the second economic evaluation was adults with hATTR with an indication for treatment with patisiran (as reflected in the APOLLO trial).” (opens the source at this quote in a new tab)

“When discounting both costs and outcomes at 3%, total costs were $1,507,450 for inotersen and $329,858 for BSC, with corresponding life years of 7.9 years and 7.3 years, respectively. Quality adjustment of these life years produced estimates of 4.54 QALYs for inotersen and 3.86 QALYs for BSC.” (opens the source at this quote in a new tab)

“Incremental Cost-Effectiveness Ratios (QALYs)* Health Care Sector Perspective $1,730,000 Modified Societal Perspective $1,730,000” (opens the source at this quote in a new tab)

“When discounting both costs and outcomes at 3%, total costs were $3,173,084 for patisiran and $312,062 for BSC, with corresponding life years of 9.7 years and 6.3 years, respectively. Quality adjustment of these life years produced estimates of 6.54 QALYs for patisiran and 3.11 QALYs for BSC.” (opens the source at this quote in a new tab)

“Incremental Cost-Effectiveness Ratios (QALYs)* Health Care Sector Perspective $835,000 Modified Societal Perspective $853,000” (opens the source at this quote in a new tab)

Utility weights

Utility weights from analyses of Zeleciment rostudirsen (z-rostudirsen and DYNE-251)

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

Health statePatientCaregiver
“Initial score”View highlighted excerpt for this quote“0.879 (0.037)”View highlighted excerpt for this quote“0.862 (0.016)”View highlighted excerpt for this quote
“Per lost score (multiplier)”View highlighted excerpt for this quote“0.905 (1.003)”View highlighted excerpt for this quote“0.995 (1.001)”View highlighted excerpt for this quote
“Early ambulatory”View highlighted excerpt for this quote“0.699 (0.036)”View highlighted excerpt for this quote“0.858 (0.017)”View highlighted excerpt for this quote
“Late ambulatory”View highlighted excerpt for this quote“0.607 (0.029)”View highlighted excerpt for this quote“0.839 (0.017)”View highlighted excerpt for this quote
“Early non-ambulatory”View highlighted excerpt for this quote“0.224 (0.014)”View highlighted excerpt for this quote“0.784 (0.021)”View highlighted excerpt for this quote
“Late non-ambulatory”View highlighted excerpt for this quote“0.146 (0.010)”View highlighted excerpt for this quote“0.810 (0.018)”View highlighted excerpt for this quote
“None”View highlighted excerpt for this quote“0.518 (0.027)”View highlighted excerpt for this quote“0.837 (0.014)”View highlighted excerpt for this quote
“Night-time”View highlighted excerpt for this quote“0.129 (0.017)”View highlighted excerpt for this quote“0.775 (0.030)”View highlighted excerpt for this quote
“Day- and night-time”View highlighted excerpt for this quote“0.051 (0.010)”View highlighted excerpt for this quote“0.774 (0.033)”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, eteplirsen, and golodirsen.

“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.” (opens the source at this quote in a new tab)

Health statePatientCaregiver
“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”

Utility weights by ambulatory stage, Duchenne muscular dystrophy: Klimchak et al. (2024)

Relevance: Utilities by ambulatory stage of Duchenne muscular dystrophy in a United States model of a hypothetical treatment, covering the ambulatory and non-ambulatory participants DELIVER enrolled.

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 by ambulatory stage of Duchenne muscular dystrophy, taken from a local study, 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

No evidence found.

Search strategy

DatasetQueryResults
Ratios"Duchenne"15
Methods"muscular dystrophy"5
Ratios"muscular dystrophy"15
Weights"Duchenne" OR "muscular dystrophy"34
Ratioseteplirsen OR golodirsen OR viltolarsen OR casimersen OR ataluren OR "exon skipping"4
Ratiosdelandistrogene OR Elevidys OR givinostat OR vamorolone OR deflazacort4
Ratiosnusinersen OR risdiplam OR onasemnogene OR "spinal muscular atrophy"85
Ratiosrostudirsen OR "DYNE-251" OR zeleciment0
Methodsrostudirsen OR "DYNE-251" OR zeleciment0
Ratios"antisense" OR oligonucleotide OR inotersen OR eplontersen OR tofersen4