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 7 studies reporting utility weights · 12 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 | 8 |
| Studies reporting utility weights | 7 |
Cost-effectiveness analyses
Analyses of Molgramostim Inhalation Solution
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
Nintedanib and pirfenidone versus symptom management in idiopathic pulmonary fibrosis, United States: Dempsey et al. (2022)
Relevance: Evaluates nintedanib and pirfenidone against symptom management in idiopathic pulmonary fibrosis from a United States health care payer perspective, with utilities assigned by percent-predicted forced vital capacity.
“We used a previously published Markov model (from the UK) that incorporates U.S.-specific data and project the lifetime costs and health benefits of treating IPF with: (1) symptom management; (2) pirfenidone; or (3) nintedanib” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“We assumed a health care payer’s perspective.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“All future costs and quality-adjusted life years (QALYs) were discounted at 3% per year” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Symptom management was associated with lifetime costs and benefits of $79,815 and 3.78 QALYs, respectively.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Nintedanib was associated with lifetime costs and benefits of $675,544 and 4.15 QALYs, respectively.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“This amounts to an incremental cost-effectiveness ratio of $1.6 million per QALY” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Pirfenidone had lifetime costs of $688,778 and benefits of 4.10 QALYs.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“In our model, pirfenidone was slightly more expensive than nintedanib and provided the same amount of benefit” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Nintedanib versus placebo, both with usual care, in progressive fibrosing interstitial lung disease, Netherlands: Westerink et al. (2022)
Relevance: Evaluates nintedanib against placebo in progressive fibrosing interstitial lung disease in the Netherlands, with lung transplantation among its modeled states; section 2 lists lung transplantation as an option for refractory autoimmune pulmonary alveolar proteinosis.
“An individual patient simulation model was built in Microsoft Excel.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The long-term clinical effects, costs and cost-effectiveness were forecasted for treatment with nintedanib versus placebo, both on top of UC in patients with PF-ILD, using a 10-year time horizon as the follow-up period.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“To account for future devaluations, a societal perspective with discounting rates of 4.0% for costs and 1.5% for effects and a time horizon of 10 years were applied, in line with Dutch guideline recommendations” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Overall, treatment with nintedanib was predicted to result in 2.87 discounted QALYs versus 2.00 discounted QALYs for placebo, both on top of UC.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The total costs per patient over 10 years of treatment were €91,301 for nintedanib versus €38,358 for placebo, both on top of UC.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“this produced an incremental cost-effectiveness ratio (ICER) of €40,418 per life year gained (LYG) and an ICER of €60,690 per QALY.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“When lung transplantation was excluded from the model, the ICER increased to €84,926 per QALY.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Augmentation therapy until FEV1 is below 35% predicted or for life versus no augmentation therapy in severe alpha1-antitrypsin deficiency, United States: Gildea et al. (2003)
Relevance: Evaluates augmentation therapy for life and until a lung function threshold against no augmentation in severe alpha1-antitrypsin deficiency in the United States, with health states defined by percent-predicted FEV1 and by lung transplantation.
“A Markov-based decision model was created to assess the cost-effectiveness of augmentation therapy (Aug) for severe alpha1-antitrypsin deficiency, comparing strategies of: (1) no Aug, (2) Aug for life, and (3) Aug until FEV1 is below 35% predicted.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A hypothetical cohort of 46-year-old patients with FEV1 49% predicted was followed over time using Monte Carlo simulation across five possible health states: (1) FEV1 50 to 79% predicted, (2) FEV1 35 to 49% predicted, (3) FEV1 below 35% predicted, (4) status-post-lung transplantation, and (5) dead.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Treatment for life yielded 7.19 quality-adjusted life-years (QALYs) and cost 895,243 dollars.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Treating until FEV1 is below 35% predicted cost 511,930 dollars and produced 6.64 QALYs.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“"No Aug" cost 92,091 dollars with 4.62 QALYs.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The incremental cost-effectiveness ratio was 207,841 dollars/QALY for Aug until FEV1 is below 35% predicted and 312,511 dollars/QALY for the "Aug for life" strategy.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“In all sensitivity analyses, the incremental cost-effectiveness ratio for Aug for life exceeded 100,000 dollars.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Tobramycin inhalation powder versus tobramycin inhalation solution in cystic fibrosis with chronic Pseudomonas aeruginosa infection, United States: Panguluri et al. (2017)
Relevance: Evaluates tobramycin inhalation powder against tobramycin inhalation solution for chronic Pseudomonas aeruginosa infection in cystic fibrosis from a United States payer perspective.
“A patient-level simulation model was developed to compare TIP with TIS.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Both costs and benefits were predicted over a 10-year time horizon from a payer’s perspective, and were discounted annually at 3%.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“All costs were presented in 2016 US dollars.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The model was developed from the US healthcare perspective over a time horizon of 10 years, with a cycle length of 6 months.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The use of TIP over TIS was associated with cost savings per patient over a time horizon of 10 years for nebuliser and exacerbation costs when high adherence to TIP and low adherence to TIS was assumed” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The incremental total costs were lower (by US$36,168) and QALYs gained were higher (by 0.27) with TIP as compared to TIS.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“This equates to lower costs with TIP per QALY gained (−US$133,195/QALY) for the base case (ICER).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“TIP was dominant over TIS in 100% of the simulations” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Aztreonam lysine for inhalation versus tobramycin solution for inhalation in cystic fibrosis with chronic Pseudomonas aeruginosa infection, United States: Schechter et al. (2015)
Relevance: Evaluates aztreonam lysine for inhalation against tobramycin solution for inhalation in cystic fibrosis from a United States third-party payer perspective, with health states defined by percent-predicted FEV1.
“A cost-effectiveness analysis with a 3-year time horizon was performed to simulate the economic consequences of either treatment from the perspective of a third party payer in the United States.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A Markov structure was used to consider transitions between health states, defined principally by levels of percent predicted of FEV1.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Use of aztreonam lysine for inhalation was associated with an average cost saving of $41,947 per patient over 3 years, as well as greater quality-adjusted life-years and total life-years.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Scenario analyses demonstrated that these findings were robust to changes in key assumptions.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Inhaled mannitol added to best supportive care versus best supportive care in cystic fibrosis, Australia: Warren et al. (2019)
Relevance: Evaluates inhaled mannitol added to best supportive care against best supportive care alone in cystic fibrosis from the perspective of the Australian government-funded national healthcare system.
Access: Not open access
Not quoted: The results by concomitant dornase alfa use are reported in the full text, which is closed access.
“The objective of this study was to assess the cost effectiveness of inhaled mannitol (in addition to best supportive care) in the Australian setting from the perspective of a government-funded national healthcare system.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A probabilistic patient-level simulation Markov model estimated life-time costs and outcomes of mannitol when added to best supportive care, compared with best supportive care alone in patients aged 6 years and older.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The utility value for the cystic fibrosis health state was as measured in the trials using the Health Utility Index, whereas the impact of pulmonary exacerbations and lung transplantation on utility was ascertained from the published literature.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The addition of inhaled mannitol to best supportive care resulted in a discounted cost per quality-adjusted life-year of AU$39,165.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The result was robust with 77% of probabilistic sensitivity analysis samples below a willingness-to-pay threshold of AU$45,000/quality-adjusted life-year.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“our model suggests inhaled mannitol provides a cost-effective addition to best supportive care in patients with cystic fibrosis, irrespective of concomitant dornase alfa use.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Lung transplantation versus no transplantation by type of end-stage pulmonary disease, Netherlands: Groen et al. (2004)
Relevance: Evaluates lung transplantation by type of end-stage pulmonary disease in the Netherlands; section 2 lists lung transplantation as an option for refractory autoimmune pulmonary alveolar proteinosis.
Access: Free full text
| Diagnosis | Mean cost-utility ratio (SE), 5% discount rate (×1000 US dollars) |
|---|---|
| “COPD” | “89.9 (0.77)” |
| “ATD” | “83.4 (0.55)” |
| “BE” | “77.0 (0.37)” |
| “PPH” | “79.9 (0.62)” |
| “SPH” | “88.7 (0.94)” |
| “CF” | “83.2 (0.59)” |
| “PF” | “86.9 (0.70)” |
Continuous and nocturnal long-term oxygen therapy versus no oxygen therapy in chronic obstructive pulmonary disease, United States: Oba (2009)
Relevance: Evaluates continuous and nocturnal long-term oxygen therapy in chronic obstructive pulmonary disease in the United States; section 2 lists oxygen supplementation as supportive care, and the IMPALA-2 protocol permitted it.
“A Markov model was developed to estimate the incremental cost-effectiveness ratios (ICERs) for continuous and nocturnal oxygen therapies.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The maximum time horizon was set to 5 years.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Cost variables were based on the current Medicare reimbursement rate and on appropriate sources.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The ICER for continuous oxygen therapy ($16,124 per quality-adjusted life-year [QALY]) was within bounds considered to be cost-effective, while that of nocturnal oxygen therapy was not ($306,356/QALY).” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The estimated ICER for continuous oxygen therapy was robust (95% confidence interval, $13,153-$24,658/QALY)” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The ICER for nocturnal oxygen therapy was sensitive to variation in the mortality rate; it could be as low as $18,267/QALY gained.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“At the other end, nocturnal oxygen therapy could be less effective than no oxygen therapy, despite additional costs.” (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 Molgramostim Inhalation Solution
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 percent-predicted forced vital capacity and after acute exacerbation, idiopathic pulmonary fibrosis: Dempsey et al. (2022)
Relevance: EQ-5D utilities by percent-predicted lung function band and post-exacerbation disutilities in idiopathic pulmonary fibrosis, from a United States model.
“used the EQ-5D to measure QOL by % forced vital capacity (FVC)” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“assumed that exacerbations decreased QOL by 0.140 in the initial cycle that it is experienced and decreased QOL by 0.078 in subsequent months” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights by percent predicted forced vital capacity and for acute exacerbations, idiopathic pulmonary fibrosis: Porte et al. (2018)
Relevance: Utilities by percent-predicted forced vital capacity and for acute exacerbations in idiopathic pulmonary fibrosis, from a lifetime model adapted to the French setting.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
“A previously developed lifetime Markov model was adapted to the French setting by simulating the progression of IPF patients in terms of lung function decline, incidence of acute exacerbations, and death.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Considering the effect of IPF on patients' quality-of-life, a CUA integrating quality adjusted life years (QALY) was chosen as the primary outcome measure in the main analysis.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights and disability weights for progression-free, progressed, and acute exacerbation states, idiopathic pulmonary fibrosis: Zhao et al. (2023)
Relevance: Utilities and disability weights for progression-free, progressed, and acute exacerbation states in idiopathic pulmonary fibrosis, with utilities abstracted preferentially as EQ-5D from the trials of a network meta-analysis.
“For estimating QALYs, data were collected from the enrolled RCTs in our NMA, which reported utility scores of quality of life in different health states related to the economic model, comprising EuroQol Five Dimensions Questionnaire (EQ-5D) and St. George's Respiratory Questionnaire (SGRQ) index.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“EQ-5D was preferentially abstracted, when reported.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Global DALYs were sourced from Global Burden of Disease (GBD) Study 2017.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights for the lung transplant waiting list, after lung transplantation, and end-stage pulmonary disease: Groen et al. (2004)
Relevance: Utilities for the lung transplant waiting list, after lung transplantation, and for end-stage pulmonary disease, for the lung transplantation that section 2 lists for refractory disease.
Access: Free full text
Utility weights after lung transplantation and for gastrointestinal adverse events, progressive fibrosing interstitial lung disease: Westerink et al. (2022)
Relevance: Utilities derived from EQ-5D-3L data, with different baseline utilities after lung transplantation and decrements for treatment-related gastrointestinal adverse events, in progressive fibrosing interstitial lung disease.
Access: Free full text
Not quoted: The utility and disutility values are reported in Appendix C of the Electronic Supplementary Material, which the article text does not include.
“A regression equation was used to determine patient-specific health state utilities based on EQ-5D-3L data” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“UK preference weights were used to derive the utilities because no Dutch patients were included in INBUILD” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The economic model assumed utility decrements associated with treatment-related adverse events, and in the case of lung transplantation, different baseline utilities were applied.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights before and after liquid oxygen and concentrator treatment, chronic hypoxaemia: Andersson et al. (1998)
Relevance: Utilities in chronic hypoxaemia before and after domiciliary oxygen treatment.
Access: Free full text
Not quoted: The utility weights are reported in the full text, a PDF at the publisher’s site that could not be retrieved for checking, so this page does not quote them.
“A prospective, randomized multicentre trial comparing C/C with L for LTOT was conducted during a six-month period.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Fifty-one patients (29 on L and 22 on C/C) with chronic hypoxaemia, regularly active outside the home, participated in the study initially.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Health-related quality of life was measured by the Sickness Impact Profile (SIP) and the EuroQol, instruments at the start and after 6 months.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights for long-term oxygen therapy without and with moderate or severe exacerbation, chronic obstructive pulmonary disease: Groessl et al. (2023)
Relevance: Utilities for chronic obstructive pulmonary disease requiring long-term oxygen therapy without exacerbation and with moderate or severe exacerbation, from a United States healthcare system model.
“With our analysis based on a US healthcare system perspective, we estimated the utility of our sample to be 0.630 when no exacerbations were present using estimates from the National Health and Wellness Survey (NHWS)” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“utility decrements of 15% for moderate exacerbations and 50% for severe exacerbations resulted in monthly utility values of 0.045 and 0.026” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“This translates to a monthly decrement of 0.008 and 0.027 for moderate and severe exacerbations, respectively.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Search strategy
| Dataset | Query | Results |
|---|---|---|
| Methods | "alveolar proteinosis" OR molgramostim OR sargramostim OR "whole lung lavage" | 0 |
| Ratios | "alveolar proteinosis" OR molgramostim OR sargramostim OR "lung lavage" OR "GM-CSF" | 0 |
| Weights | "alveolar proteinosis" OR "lung lavage" OR "GM-CSF" | 0 |
| Ratios | "granulocyte-macrophage" OR "granulocyte macrophage" OR "bronchoalveolar lavage" OR "pulmonary alveolar" | 6 |
| Ratios | "idiopathic pulmonary fibrosis" OR "interstitial lung disease" | 43 |
| Ratios | "alpha-1 antitrypsin" OR "alpha1-antitrypsin" OR "lung transplantation" OR "lung transplant" | 67 |
| Ratios | "nebulized" OR "nebulised" OR "nebulizer" | 11 |
| Weights | "diffusing capacity" OR "DLCO" OR "gas transfer" OR "St George" | 16 |
| Weights | "pulmonary fibrosis" OR "interstitial lung disease" | 98 |
| Weights | "long-term oxygen" OR "supplemental oxygen" OR "home oxygen" OR "oxygen therapy" | 78 |
| Ratios | "long-term oxygen" OR "home oxygen" OR "rare lung" OR "orphan lung" | 11 |
| Ratios | "antitrypsin" OR "augmentation therapy" | 12 |
| Ratios | "cystic fibrosis" AND (inhaled OR inhalation) | 9 |