A Health State Utility Study to Elicit Societal Values Associated with Pulmonary Hypertension.

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.

Estimation of Health Utility Scores for Glycogen Storage Disease Type Ia.

BACKGROUND: Glycogen Storage Disease Type Ia (GSDIa) is a rare genetic metabolic disorder characterized by hypoglycemia, hepatomegaly, growth failure, and possible seizures/death. Patients frequently consume cornstarch to maintain blood glucose. Evidence demonstrating the impact of GSDIa on health-related quality of life (HRQoL) is lacking. This study aims to understand the burden of GSDIa by obtaining utility values for economic appraisals. METHODS: A targeted literature review and interviews with experts (n = 4) and caregivers (n = 4) informed the development of health state vignettes describing different GSDIa severities by age and level of hypothetical treatment (i.e., gene therapy) response. Health states reflecting caregivers' experiences were also developed. A convenience sample of the UK general public completed a time trade-off (TTO) exercise. Scores conceptually varied from 0 (dead) to 1 (full health). States were also rated using a visual analog scale (VAS) and the EQ-5D-5L. Data were descriptively summarized. RESULTS: One hundred participants completed the exercise (male: 48%; mean age: 42 years). Scores were lowest for the adolescent pre-treatment state (TTO = 0.38). Large increments in HRQoL were observed for the response (adult: TTO = 0.86; child: TTO = 0.84) and complete response (adult and child: TTO = 0.94) hypothetical treatment response states. Caregiver values were lowest for the pre-treatment state (TTO = 0.57) and highest for the complete response state (TTO = 0.95). VAS and EQ-5D-5L scores followed a similar pattern. CONCLUSION: This study found an HRQoL burden on GSDIa patients and caregivers, with potential large improvement from a hypothetical treatment. These findings may be useful for families, clinicians, regulatory agencies, and in therapy economic evaluations.

Estimating health state utilities in primary hyperoxaluria type 1: a valuation study.

AIMS: 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. MATERIALS AND METHODS: 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. RESULTS: 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. LIMITATIONS: Vignettes may not fully capture the burden of living with PH1. CONCLUSIONS: 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.

Patient Preferences for Metastatic Hormone-Sensitive Prostate Cancer Treatments: A Discrete Choice Experiment Among Men in Three European Countries.

INTRODUCTION: Various treatment options are available for metastatic hormone-sensitive prostate cancer. This study aimed to quantify how men with prostate cancer in the United Kingdom (UK), Germany, and Spain perceive the risks and benefits of hypothetical abiraterone acetate plus prednisone treatment and docetaxel-based chemotherapy treatment options. METHODS: A targeted literature review, exploratory interviews with prostate cancer patients and oncologists, and pre-test interviews were used to develop a discrete choice experiments (DCE). The final DCE included 32 choice sets, selected using a main-effects orthogonal design, divided into two survey blocks. Paired profiles presented hypothetical treatments for prostate cancer through six attributes that could be presented at two or four levels each. Preference estimates were estimated using a conditional logit regression model. Preference results were stratified by cancer stage. RESULTS: A total of 152 participants (mean age 69 years) completed the DCE in the UK, Germany, and Spain. Treatment effectiveness was the main concern for the patients (difference in preference estimates between 8 and 32 months 1.443). Participants wanted to avoid pain that was not well controlled (preference dummy coding estimate - 1.157). Participants valued a change from an oral medication to an intravenous treatment (change in preference estimate - 0.416) more negatively than a change from a 1% to a 5% risk of infection (change in preference estimate - 0.313). CONCLUSIONS: This study shows that treatment effectiveness and pain control were the most important attributes for patients with prostate cancer. These two attributes influenced more than 50% of their decision-making in this study. The risk of fatigue and mode of administration were least prioritised by patients. This study highlights the relative importance that Spanish, German, and British patients place on various aspects of treatment options for prostate cancer. Understanding patient preference and taking them into consideration shall help physicians when developing their treatment strategies for their patients. FUNDING: Janssen.

Patients' Preferences for the Treatment of Metastatic Castrate-resistant Prostate Cancer: A Discrete Choice Experiment.

PURPOSE: Patient treatment preferences are increasingly being used to inform health care decision making. This discrete choice experiment assessed how men perceive the risks and benefits of hypothetical treatment options for metastatic castrate-resistant prostate cancer (mCRPC). METHODS: Treatment attributes for inclusion were identified through a review of the literature and product labels. Expert interviews confirmed clinical appropriateness and patient relevance of the attributes, which included effectiveness (delay in months before chemotherapy), steroid use, possible drug interactions (additional hospital visits for monitoring), fogginess (effects on cognition and memory), fatigue (extreme tiredness), food restrictions, and bone pain. Following a pilot, the final discrete choice experiment included 18 choice sets presenting treatments for mCRPC and was completed by men with mCRPC in France, Germany, and the United Kingdom. Data were analyzed using a conditional logit model, with odds ratios (ORs) used to indicate preference for attributes, and tradeoff measures (TOM) were estimated using the ratio of coefficients. FINDINGS: Within each attribute category and with all other factors being equal, participants (N = 285) indicated a strong preference for treatments that fully control bone pain (OR = 12.069 [95% CI, 10.555-13.800]) and for treatments that delay chemotherapy (OR, 1.727 [95% CI, 1.548-1.927]). They also preferred treatments that were associated with the lowest risk of fogginess (OR, 2.115 [95% CI, 1.849-2.420]), a lower risk of fatigue (OR, 1.365 [95% CI 1.219-1.528]), and fewer additional hospital visits (OR, 1.245 [95% CI 1.111-1.397]) than the respective reference categories. Participants preferred to use steroids under advice from a physician (OR, 1.275 [95% CI 1.132-1.437]). Food restrictions related to taking medication were not a significant concern for participants. TOM results indicated that large tradeoffs in effectiveness, fogginess, and fatigue are required for patients to prefer a treatment with uncontrolled bone pain that is very difficult to live with. IMPLICATIONS: Men with mCRPC consider a wide range of factors when making decisions regarding their treatment. They showed a strong preference for treatment associated with better control of bone pain. They also placed value on treatments that could delay the need for chemotherapy, and they preferred to avoid side effects such as cognition and memory loss, and extreme tiredness. TOMs highlighted the importance of symptom control, even compared with potential side effects. An understanding of the degree to which patients value the attributes associated with various treatment options will assist clinicians and health care professionals when making decisions regarding the management of men with mCRPC.

Health state utility valuation in radioactive iodine-refractory differentiated thyroid cancer.

PURPOSE: The aim of this study was to elicit utilities for radioactive iodine-refractory differentiated thyroid cancer (RR-DTC) and evaluate the impact of treatment response and toxicities on quality of life. PATIENTS AND METHODS: RR-DTC health states were developed based on data from a previous qualitative study and iterative review by clinical experts. Following piloting, health states underwent valuation by 100 members of the UK public during time trade-off interviews. Mean utilities and descriptive distribution statistics were calculated, and a logistic regression analysis was conducted. RESULTS: The demographic characteristics of the study sample were generally reflective of the UK population. Clear differentiation in valuation between health states was observed. No response/stable disease had an adjusted utility value of 0.87, with a corresponding gain of +0.04 following a treatment response and a decline of -0.35 for disease progression. Adverse events were associated with utility decrements between -0.47 (grade III diarrhea) and -0.05 (grade I/II alopecia). CONCLUSION: The trade-off interviews derived utility weights show clear differentiation between RR-DTC health states in response to treatment. The values reported in this study are suitable for cost-effectiveness evaluations for new treatments in RR-DTC.