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Aim: Pulmonary hypertension (PH) is a rare, severe, and progressive pulmonary vascular disease, which includes five subgroups with similar presentation. Symptoms include dyspnea, and fatigue, and can significantly impact one's health-related quality of life (HRQL). Although treatments are mainly medical, PH group 4, chronic thromboembolic pulmonary hypertension (CTEPH), can be managed with procedures, ie pulmonary endarterectomy (PEA) and balloon pulmonary angioplasty (BPA). Currently, drugs in Europe are only approved for pulmonary arterial hypertension (PAH), and CTEPH therefore the aim of this study was to elicit novel societal health state utilities in the UK for PAH and CTEPH based on disease severity, functional class (FC), clinical events, and treatment procedures specifically for CTEPH.Material and Methods: Six health states were defined: World Health Organization (WHO)-FC II, WHO-FC III, and WHO-FC IV [defined by the New York Heart Association (NYHA)]; PH-related hospitalization; and "BPA procedure and recovery" and "PEA surgery and recovery". Health states were based on a targeted literature review and two rounds of interviews with clinical experts (N = 4) and patients (N = 6). Draft health states were validated in cognitive debriefing interviews with clinical experts (N = 3). Health states were valued by the UK general public (n = 200), using a visual analogue scale (VAS) and time trade-off (TTO) assessment with the lead time method.Results: The mean TTO values/utilities were 0.81 (FC II), 0.80 (BPA), 0.78 (PEA), 0.59 (FC III), 0.28 (FC IV), and 0.25 (PH-related hospitalization). Each progression in FC was associated with worse TTO scores.Conclusion: This study reports societal utility values for PAH and CTEPH in the UK. It provides first utility estimates for states such as BPA procedure and recovery, PEA surgery and recovery and PH-related hospitalization for this population. The results show important distinctions between FC, treatment procedures, and hospitalization, and the significant burden of disease on HRQL. [ABSTRACT FROM AUTHOR]

Quantitative data on health state utilities in primary hyperoxaluria type 1 (PH1) are lacking. This study was conducted to estimate utility values in PH1 using 3 standard valuation techniques. Health state vignettes were developed with input from expert clinicians to describe different stages of chronic kidney disease (CKD) within the setting of PH1, along with a post-combined liver and kidney transplant (CLKT) health state ≥12 months following transplantation. The utility associated with living in each PH1 health state, as described by the vignettes, was evaluated by members of the UK general public using standard utility assessment techniques, including EQ-5D-5L, Visual Analog Scale, and Time Trade-Off. A similar pattern across the three valuation methods was observed. Utility values were roughly constant from CKD stage 1–3b and then dropped sharply from these states to CKD stage 4. Decreases in utility in the later stages of CKD (stages 4–5) were followed by a recovery in quality of life in the post-CLKT health state. Vignettes may not fully capture the burden of living with PH1. This study serves as an informative source of data on how the burden of PH1 varies across disease stages of CKD and post-CLKT in patients with PH1. The study findings highlight the unique clinical features of PH1 compared with non-PH1-related CKD, such as the need for earlier and more intensive hemodialysis, the risk of systemic oxalosis, and the potential need for CLKT (as opposed to kidney-only transplant). The impact of PH1 on health-related quality of life, which worsens once hemodialysis is required and systemic disease manifestations occur, is captured in this study using quantitative health state utilities. These data provide an understanding of the impact of PH1 on health-related quality of life and will facilitate health economic evaluation of future treatments. Primary hyperoxaluria type 1 (PH1) is a rare genetic disease in which the body produces too much oxalate, leading to kidney damage. Over time it can affect other organs such as the bones, the heart, and the eyes. As kidney damage progresses, patients with PH1 eventually need to receive kidney dialysis. New and emerging treatments aim to reduce oxalate production. To assess the economic value of treatments, data are needed on the quality of life of patients in different stages of PH1. This study collected these data using standard valuation methods. First, we developed written descriptions of different stages of PH1 using expert clinical input and information from published studies. Then, members of the UK general public valued the descriptions in three ways: via a survey called EQ-5D-5L (which asks participants about their mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), via EQ-VAS (a visual scale, resembling a ruler, used to rate quality of life), and via time trade-off (in which participants were asked how many years of perfect health they would give up to avoid living in each disease stage described). The results show that PH1 is likely to have a considerable impact on patients' quality of life, especially in advanced stages, when dialysis is needed and multiple organs are impacted. The study provides measurable estimates of quality of life in people with PH1, which helps healthcare providers, policy makers, and payers understand the disease burden of PH1. The results can also be used in economic evaluation of new treatments. [ABSTRACT FROM AUTHOR]