Elicitation of health state utilities associated with the mode of administration of drugs acting on the prostacyclin pathway in pulmonary arterial hypertension

Davies, Evan; Llewellyn, Samuel; Beaudet, Amélie; Kosmas, Charlotte E; Gin-Sing, Wendy; Doll, Helen

doi:10.2147/ppa.s160662

Cited by 10 publications

(10 citation statements)

References 41 publications

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“…Sixty-one patients underwent echocardiographic assessment at baseline and follow-up to assess the effects of selexipag on right heart size and function (Table 2). The RV diameter decreased in treated patients [38 (31, 47) vs. 35 (29,45) mm; p = 0.001], and the RA area showed a decreasing trend from 25 (19,34) to 21 (15, 33) cm 2 although this difference was not statistically significant (p = 0.113). TAPSE increased from 16 (14, 18) to 18 (15,20) mm (p = 0.002).…”

Section: Efficacymentioning

confidence: 94%

Selexipag-based triple combination therapy improves prognosis in Chinese pulmonary arterial hypertension patients

Cui

Lu²,

Zhang³

et al. 2022

Front. Cardiovasc. Med.

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AimSelexipag is an oral selective prostacyclin receptor agonist approved for treatment of patients with pulmonary arterial hypertension (PAH). In the present study, we aim to assess the safety and efficacy of selexipag in triple combination therapy with endothelial receptor antagonists (ERAs) and PDE5is for Chinese PAH patients.Methods and resultsA single center retrospective study was performed on group 1 PAH patients (n = 68) initiating triple combination therapy with selexipag from 1 February 2020 to 31 August 2021 in Qilu Hospital of Shandong University (Shandong, China). Adolescents, children, and PAH patients with unrepaired congenital heart disease were excluded. The French pulmonary hypertension network (FPHN) non-invasive risk assessment, echocardiogram parameters, and clinical data, including tolerability, safety, and death/hospitalization events associated with PAH, were collected. Of the 68 patients, 31 (45.6%) patients had tolerable side effects while only a single patient discontinued selexipag due to severe diarrhea. In the analysis of the efficacy set of 62 patients, the median selexipag treatment time from selexipag initiation to last risk assessment was 27 (21, 33) weeks. Compared to baseline parameters, the percentage of WHO FC III/IV decreased from 77.4% (48) to 24.2% (15) (p = 0.000), median 6-min walk distance (6MWD) increased 82 m [from 398 (318, 450) to 480 (420, 506) m; p = 0.000], and NT-proBNP levels decreased from 1,216 (329, 2,159) to 455 (134, 1,678) pg/mL (p = 0.007). Patients who improved to three low-risk criteria increased from 9.7 to 38.7%. Right ventricular diameter (RV) diameter also decreased and was accompanied by an improved tricuspid annular plane systolic excursion (TAPSE). Patients transitioning from subcutaneous treprostinil to selexipag continued to show improvements in WHO FC, 6MWD (404 ± 94 vs. 383 ± 127 m) and NT-proBNP levels (2,319 ± 2,448 vs. 2,987 ± 3,770 pg/mL). Finally, the 1-year event free survival rate was 96.7% for patients initiating the triple combination therapy within 3 years of PAH diagnosis.ConclusionTriple combination therapy with selexipag was safe and effective in Chinese PAH patients, which was confirmed by acceptable tolerability, and improved exercise capacity, right heart function, risk assessment, and prognosis.

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Section: Efficacymentioning

confidence: 94%

Selexipag-based triple combination therapy improves prognosis in Chinese pulmonary arterial hypertension patients

Cui

Lu²,

Zhang³

et al. 2022

Front. Cardiovasc. Med.

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“…11,26,40 Inhaled iloprost requires 6 to 9 doses daily due to its short half-life, 34 and each dosing takes up to 15 minutes. 41,42 Device setup, cleaning, and packing require additional time. 24 As a result of their side-effect profile and administration burden, these agents are typically reserved for high-risk patients.…”

Section: Discussionmentioning

confidence: 99%

Comparison of Healthcare Encounters and Drug Persistence in Patients With Pulmonary Arterial Hypertension Receiving Oral Selexipag, Inhaled Iloprost, or Parenteral Treprostinil: A Retrospective Database Analysis

Song

Kunovszki

Beaudet

2022

Journal of Health Economics and Outcomes Research

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Background: Agents targeting the prostacyclin (PGI2) pathway are important in managing pulmonary arterial hypertension (PAH). No head-to-head clinical trials have compared outcomes between the 3 different PGI2-pathway drugs most commonly available in countries with advanced healthcare: oral selexipag, inhaled iloprost, and parenteral (subcutaneous or intravenous) treprostinil. Objectives: To conduct retrospective database analyses to describe characteristics of patients with PAH initiating therapy with these agents and compare the rate and risk of healthcare facility encounters and drug persistence. Methods: Data were obtained from the Optum™ Clinformatics® Data Mart and Truven™ Health Analytics® MarketScan® Commercial Claims and Encounters databases from July 1, 2008, to September 30, 2020 (Optum™), or October 31, 2020 (Truven™). Patients were categorized into index-drug cohorts based on first pharmacy claims for selexipag, inhaled iloprost, or parenteral treprostinil. Eligible patients were ≥18 years of age with ≥1 ICD-9-CM or ICD-10-CM diagnosis code indicating pulmonary hypertension and no diagnosis code suggesting Group 3–5 pulmonary hypertension. Rates of hospitalization (inpatient admissions), emergency room visits, or outpatient visits per person-year were calculated. Drug persistence was measured as time to discontinuation of index drug. Multivariable analyses were performed to compare outcomes with selexipag vs inhaled iloprost and parenteral treprostinil, adjusting for baseline characteristics using inverse probability of treatment weighting. Results: Overall, 583 patients were included in the Optum™ sample and 482 in the Truven™ sample. Mean (SD) age was 61.7 (14.5) and 49.3 (11.3) years, respectively; 74.4% and 75.7% of patients, respectively, were women. In the pooled samples, after adjustment for baseline characteristics, selexipag had a lower risk than inhaled iloprost or parenteral treprostinil for hospitalization (relative rate ratio [95% CI], 0.40 [0.22, 0.75], and 0.26 [0.17, 0.39]) and outpatient visits (0.66 [0.56, 0.78] and 0.76 [0.66, 0.88]). Trends toward lower risk of emergency room visits did not attain statistical significance. Drug discontinuation risk was 16% and 36% lower with selexipag vs parenteral treprostinil and inhaled iloprost, respectively. Conclusions: In real-world use, selexipag appears to be associated with lower rates of hospitalization and outpatient visits than inhaled iloprost or parenteral treprostinil. Further research is required to identify factors underlying these differences.

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“…Boye et al 49 Davies et al 50 Krassioukov et al 53 Lloyd et al 54 Matza et al 56 Matza et al 17 Matza et al 16 Steen Carlsson et al 23 4. Acute and temporary health states Some medical conditions involve flares or exacerbations.…”

Section: Treatment Process Utilitiesmentioning

confidence: 99%

“…[45][46][47][48] These "process utilities" quantify the impact of attributes such as mode of administration, dose frequency, medical device attributes, and aspects of treatment convenience that may have an impact on patients' quality of life. 16,17,23,[49][50][51][52][53][54][55][56][57] Treatment process usually has less impact on utility than efficacy, safety, or symptom severity, 43 but it is often important to patients. Furthermore, treatment process could affect treatment adherence, which can influence outcomes.…”

Section: Treatment Process Utilitiesmentioning

confidence: 99%

Vignette-Based Utilities: Usefulness, Limitations, and Methodological Recommendations

et al. 2021

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Health technology assessment agencies often prefer that utilities used to calculate quality-adjusted life years in cost-utility analyses (CUAs) are derived using standardized methods, such as generic preference-based measures completed by patients in clinical trials. However, there are situations when no standardized approach is feasible or appropriate for a specific medical condition or treatment that must be represented in a CUA. When this occurs, vignette-based methods are often used to estimate utilities. A vignette (sometimes called a "scenario," "health state description," "health state vignette," or "health state") is a description of a health state that is valued in a preference elicitation task to obtain a utility estimate. This method is sometimes the only feasible way to estimate utilities representing a concept that is important for a CUA. Consequently, vignette-based studies continue to be conducted and published, with the resulting utilities used in economic models to inform decision making about healthcare resource allocation. Despite the potential impact of vignette-based utilities on medical decision making, there is no published guidance or review of this methodology. This article provides recommendations for researchers, health technology assessment reviewers, and policymakers who may be deciding whether to use vignette-based methods, designing a vignette study, using vignette-based utilities in a CUA, or evaluating a CUA that includes vignette-based utilities. Recommendations are provided on: (A) when to use vignette-based utilities, (B) methods for developing vignettes, (C) valuing vignettes, (D) use of vignette-based utilities in models, and (E) limitations of vignette methods.

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Elicitation of health state utilities associated with the mode of administration of drugs acting on the prostacyclin pathway in pulmonary arterial hypertension

Cited by 10 publications

References 41 publications

Selexipag-based triple combination therapy improves prognosis in Chinese pulmonary arterial hypertension patients

Selexipag-based triple combination therapy improves prognosis in Chinese pulmonary arterial hypertension patients

Comparison of Healthcare Encounters and Drug Persistence in Patients With Pulmonary Arterial Hypertension Receiving Oral Selexipag, Inhaled Iloprost, or Parenteral Treprostinil: A Retrospective Database Analysis

Vignette-Based Utilities: Usefulness, Limitations, and Methodological Recommendations

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