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
| 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 | 4 |
Cost-effectiveness analyses
Analyses of Lonvoguran ziclumeran (lonvo-z and NTLA-2002)
No evidence found.
Treatments for the same indication
Lanadelumab versus standard care for long-term prophylaxis in hereditary angioedema in people aged 12 years or older, Brazil: Conitec (2021)
Relevance: A Brazilian health technology assessment that evaluates lanadelumab, a first-line long-term prophylaxis option listed in section 2 and the study drug of the HELP trial in section 3, against standard care in adolescents and adults with hereditary angioedema whose current prophylaxis did not reduce attacks or was not tolerated.
“Tipo de estudo Custo-utilidade” (opens the source at this quote in a new tab)
“Taxa de desconto 5%” (opens the source at this quote in a new tab)
“Perspectiva da análise Sistema Único de Saúde (SUS)” (opens the source at this quote in a new tab)
“Cuidado padrão R$ 422.563,00 10,627” (opens the source at this quote in a new tab)
On-demand recombinant and plasma-derived C1 inhibitor, icatibant, and ecallantide for hereditary angioedema attacks, United States: Bernstein et al. (2019)
Relevance: Compares on-demand recombinant and plasma-derived C1 inhibitor, icatibant, and ecallantide for hereditary angioedema attacks from a United States commercial payer perspective, with icatibant the bradykinin B2 receptor antagonist section 2 lists.
“To estimate the cost-effectiveness of on-demand treatment for HAE attacks, a decision-tree model from the U.S. commercial payer perspective was developed using TreeAge Pro 2018 R1 release” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Four on-demand HAE treatments were included: ecallantide, icatibant, plasma-derived (pd)C1-INH, and recombinant human (rh)C1-INH.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“All costs are dated to 2019, either directly or by inflation using the medical-care inflation subset of the Consumer Price Index for All Urban Consumers.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The base case model outputs show costs and calculated effectiveness per attack at $12,905 and 0.806 for rhC1-INH, $14,806 and 0.765 for icatibant, $14,668 and 0.769 for pdC1-INH, and $21,068 and 0.792 for ecallantide, respectively.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Cost per QALY was calculated using 26.9 attacks per person-year, leading to results of $420,941 for rhC1-INH, $488,349 for icatibant, $483,892 for pdC1-INH, and $689,773 for ecallantide.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Results from this model indicate that rhC1-INH is the dominant treatment option with lower expected costs and higher calculated effectiveness than comparators.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Conestat alfa and plasma-derived C1 esterase inhibitor versus placebo for acute hereditary angioedema attacks, Poland: Kawalec et al. (2013)
Relevance: Evaluates conestat alfa and plasma-derived C1 esterase inhibitor, each against placebo, for acute hereditary angioedema attacks from a Polish healthcare payer perspective, the C1 esterase inhibitor class listed in section 2.
“The cost-utility analysis from the Polish healthcare payer's perspective was performed for 1 year (2012).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The incremental costs of each intervention compared with placebo were listed together (direct or indirect comparisons between options were impossible due to limited clinical data available).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Discrete event simulation (DES) is one way of observing the time-dependent (or dynamic) behavior of a system” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Time horizon was set at one year, which seemed to be a reasonable compromise between the short clinical trials observation time and the median life expectancy horizon.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“In the base case analysis with a one year's perspective, patients with HAE suffering from life-threatening angioedema attacks gained on average 0.0261 quality adjusted life years (QALY) when treated with Ruconest® (conestat alfa) and 0.0262 QALY when treated with Berinert® P compared with placebo (Table 2).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The incremental costs of the therapy of a single acute angioedema attack with conestat alfa amounted to app. EUR 419, app. EUR 755 per patient for human C1-INH treatment in comparison with placebo, thus indicating that conestat alfa is the cheapest option (Table 2).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The median incremental cost-utility ratios for assessed interventions compared with placebo were as follows: EUR 15,226 per QALY (conestat alfa) and EUR 27,786 per QALY (human C1 esterase inhibitor)” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The cost-utility of the therapy was assumed when the ICUR was lower than EUR 24,279 (PLN 99,543) per QALY, which is set as threshold acceptability in Poland (tripled GDP per capita)” (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
No evidence found.
Related diseases and analogous treatments
AAV-mediated gene therapy versus on-demand and prophylactic factor IX in severe hemophilia B, United States: Bolous et al. (2021)
Relevance: Evaluates AAV-mediated factor IX gene therapy against on-demand factor IX replacement and primary factor IX prophylaxis in severe hemophilia B from a United States third-party payer perspective, a gene therapy compared with continuous prophylaxis.
Access: Not open access
Not quoted: The results are reported in the full text, which is closed access.
“This study compared the potential cost-effectiveness of AAV-mediated factor IX (FIX)-Padua gene therapy for patients with severe hemophilia B in the United States vs on-demand FIX replacement and primary FIX prophylaxis, using either standard or extended half-life FIX products.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A microsimulation Markov model was constructed, and transition probabilities between health states and utilities were informed by using published data.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“A time horizon from 18 years old until death, from the perspective of a third-party payer in the United States, was conducted.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Gene therapy was more cost-effective than both alternatives considering a $150 000/quality-adjusted life-year threshold.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“The price for gene therapy was assumed to be $2 000 000 in the base case scenario” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Probabilistic sensitivity analysis estimated gene therapy to be cost-effective at 92% of simulations considering a $150 000/quality-adjusted life-year threshold.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Etranacogene dezaparvovec versus factor IX prophylaxis in severe hemophilia B, United States: Sarker et al. (2025)
Relevance: Evaluates etranacogene dezaparvovec against factor IX prophylaxis in severe hemophilia B from a United States health system perspective over a lifetime horizon, a gene therapy compared with continuous prophylaxis.
“A semi-Markov model simulated a cohort of adult males with severe hemophilia B to assess the economic impact of EDZ versus FIX prophylaxis over a lifetime horizon from a health system perspective in the USA.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Scenario analyses incorporated frameworks suggested by the Institute for Clinical and Economic Review for single or short-term transformative therapies.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“Base-case analysis showed that at a cost of US$3.5 million, EDZ led to lifetime cost savings of US$11 million and an additional 0.64 quality-adjusted life years (QALYs) compared with FIX.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“When annual cost offsets attributed to EDZ were capped at US$150,000, EDZ was found to have a threshold price of US$3.1 million at a willingness-to-pay of US$150,000 per QALY.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
“EDZ proved to be a dominant strategy over FIX prophylaxis in the base-case scenario, providing large cost savings and slightly better outcomes.” (opens the original PubMed page at this quote in a new tab)View highlighted excerpt for this quote
Valoctocogene roxaparvovec versus factor VIII prophylaxis in severe hemophilia A, United States: Cook et al. (2020)
Relevance: Evaluates single-administration valoctocogene roxaparvovec against prophylactic factor VIII in severe hemophilia A from a United States health care system perspective over a lifetime horizon, a gene therapy compared with continuous prophylaxis.
Access: Free full text
Valoctocogene roxaparvovec versus factor VIII prophylaxis and emicizumab in severe hemophilia A, Netherlands: Ten Ham et al. (2022)
Relevance: Evaluates valoctocogene roxaparvovec against factor VIII prophylaxis and emicizumab in severe hemophilia A from a Dutch societal perspective, a gene therapy compared with continuous prophylaxis.
“We constructed a Markov model to model bleeds over time which were linked to costs and quality-of-life decrements.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“A Dutch societal perspective was applied with a 10-year time horizon.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Costs and (QA)LYs were discounted at 4.0% and 1.5% per annum, respectively, in line with Dutch guidance.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Drug costs were expressed in 2019 euros (€).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“As no list price is available for valrox we used a price of €2,125,000 in the base case based on previous research” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Total cost €2,839,210 (€487,449–€10,545,521) €3,284,690 (€282,686–€10,444,562) €4,252,167 (€408,737–€14,853,421)” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Total QALYs 7.03 (5.86−8.14) 6.38 (5.59−7.36) 6.90 (5.98−7.96)” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Valrox was found to dominate both FVIII and emicizumab” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The CEAC shows valrox has a 54.8% probability of being cost-effective when compared to FVIII prophylaxis” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“This means 54.8% of ICER-estimates in the PSA were lower than €80,000/QALY. In the comparison between valrox and emicizumab the estimated percentage was 38.9%.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Gene therapy versus common care in sickle cell disease, United States: Basu et al. (2024)
Relevance: Evaluates gene therapy against common care in sickle cell disease from United States healthcare sector and societal perspectives over a lifetime horizon.
“Comparative modeling analysis across two independently developed simulation models (UW-MEASURE and FH-HISCORE) using the same databases.).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Target Population: Gene therapy-eligible individuals. Time Horizon: Lifetime Perspectives: U.S. healthcare sector and societal. Intervention: Gene therapy versus Common Care.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The primary outcomes were lifetime costs in 2021 U.S. dollars and QALYs for each treatment arm. Future costs and outcomes were discounted at an annual rate of 3%.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“At an assumed $2M price for gene therapy, the UW-MEASURE and FH-HISCORE estimated ICERs of $193,000/QALY and $427,000/QALY under the healthcare sector perspective, respectively. Corresponding estimates from the societal perspective were $126,000/QALY and $281,000/QALY.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Incremental discounted quality-adjusted life years were 9.8 (95% CI: 6.9, 12.7) for patients and 11.9 (95% CI: 8.5, 15.3) when combined with family losses.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“The discounted incremental QALYs were 5.1 QALYs (95%CI: 4.7, 5.9) for patients, and 5.4 (95% CI: 4.9, 6.0) when combined with family QALY losses.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“From a societal perspective, acceptable (>90% confidence) value-based prices ranged from $1M to $2.5M depending on the use of alternative effective metrics or equity-informed threshold values.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights
Utility weights from analyses of Lonvoguran ziclumeran (lonvo-z and NTLA-2002)
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 with and without an acute attack, hereditary angioedema: Bernstein et al. (2019)
Relevance: Utilities for hereditary angioedema with and without attack symptoms, drawn from the literature for a United States model of on-demand attack treatment.
“Utility values were drawn from the literature” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“citing a utility of 0.51 when experiencing attack symptoms and 0.83 without symptoms.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Nonattack baseline utility and attack utility were implemented for effectiveness calculations; time to attack resolution was considered as well.” (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
Utility weights for on-demand treatment, prophylaxis, gene therapy, and bleeds, severe hemophilia B: Bolous et al. (2021)
Relevance: Utilities for severe hemophilia B on on-demand treatment, on prophylaxis, and after gene therapy, and for bleeds, from a United States model of gene therapy against factor IX replacement.
Access: Not open access
Not quoted: The utility weights are reported in the full text, which is closed access.
Utility weights with and without a bleed, severe hemophilia A: Ten Ham et al. (2022)
Relevance: Utilities for severe hemophilia A with and without a bleed, with baseline utilities in the target joint submodels derived from the SF-6D, from a model of valoctocogene roxaparvovec against prophylaxis.
“Baseline utility (eg, a standardized quality of life score with 0 reflecting death and 1 perfect health) was set at 0.82 for patients in the no-bleed health state in submodel i (no target joints) (see also Table 1).” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“In the 1 and 2+ target joint submodels, baseline utilities were related to PS derived from a study by Fischer et al43 based on the SF-6D (utility derived from the Short Form-36) questionnaire with higher PS reflecting lower quality of life.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Utility, no bleed submodel i 0.88” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Utility, bleed submodel i 0.66” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
“Patients experiencing a bleed were assigned an utility of 0.66 for 2 days across submodels.” (opens the original PMC page at this quote in a new tab)View highlighted excerpt for this quote
Utility weights by bleed status and for factor VIII infusion, severe hemophilia A: Cook et al. (2020)
Relevance: Utilities for severe hemophilia A by bleed status and for factor VIII infusion, from a United States model of valoctocogene roxaparvovec against prophylaxis.
Access: Free full text
Search strategy
| Dataset | Query | Results |
|---|---|---|
| Methods | NTLA-2002 OR lonvoguran OR "lonvo-z" OR ziclumeran | 221 |
| Ratios | "hereditary angioedema" OR "hereditary angio-oedema" | 7 |
| Methods | lonvoguran | 0 |
| Methods | "NTLA-2002" | 0 |
| Methods | ziclumeran | 0 |
| Ratios | angioedema | 8 |
| Methods | "hereditary angioedema" OR angioedema | 4 |
| Weights | angioedema | 4 |
| Methods | lanadelumab OR berotralstat OR garadacimab OR donidalorsen | 0 |
| Methods | "C1 esterase inhibitor" OR "C1 inhibitor" OR Cinryze OR Haegarda OR Berinert | 2 |
| Methods | icatibant OR ecallantide OR sebetralstat OR "conestat alfa" | 2 |
| Ratios | lanadelumab OR lanadelumabe OR berotralstat OR garadacimab OR donidalorsen | 1 |
| Ratios | CRISPR OR "gene editing" OR "genome editing" OR exagamglogene OR "exa-cel" | 0 |
| Ratios | CRISPR OR exagamglogene OR "gene editing" | 0 |
| Ratios | "gene therapy" AND (hemophilia OR haemophilia) | 12 |
| Ratios | "sickle cell" AND ("gene therapy" OR "gene-modified" OR lovotibeglogene OR "autologous") | 23 |
| Weights | "attack-free" OR "attack free" OR "swelling" | 15 |