Journal of Health Economics and Outcomes Research最新文献

Background: People living with hemophilia A face challenges impacting their daily lives despite treatment innovations. Previous studies have explored perceptions and treatment experiences; however, there is a lack of an evidence-based, comprehensive model to identify concepts (clinical, physical, and psychological functioning) relevant for people with hemophilia A (PwHA). Objectives: The aim of this qualitative study was to address the question: What is the humanistic and symptomatic experience of adolescents, adults, and children living with hemophilia A and what is the impact of hemophilia A on their quality of life? Methods: Participants, identified through patient associations in the UK, were male PwHA and caregivers of male PwHA receiving prophylactic treatment. Qualitative research was conducted involving semistructured telephone interviews with PwHA and caregivers between April 2020 and September 2020 in the UK. Standard analytical techniques of conceptual model development were used. Results: Of 30 participants, 23 were PwHA and 7 were caregivers. A conceptual model was produced describing patient experience of symptoms, physical functioning, treatment experiences, and the impact of symptoms and treatment on daily lives. Participants reported hemophilia-related symptoms, including bleeding, pain, and joint stiffness, as well as difficulties engaging with social and leisure activities. They also reported protection from bleeds provided by their treatment, relief from symptoms, and the resultant sense of normality. Concepts were broadly relevant across all age groups; however, psychological impacts were reported only by adult PwHA, and caregivers reported impacts related to outdoor activities, play, and education. Participants indicated that their ideal treatment would be delivered orally. Discussion: This study highlights the range of symptoms experienced by PwHA across a broad range of age groups, thus enabling the evaluation of relevant concepts across different stages of life. The research supports development of a conceptual model documenting symptoms, impacts, and treatment experience relevant to PwHA. Conclusion: Insights gathered through the interviews and resulting conceptual model support development of new therapies to address the physical and social challenges identified by PwHA and highlight a need for novel hemophilia A treatments that can ease treatment administration, provide adequate level of protection, and enable life to be lived normally.

Background: Although increasing in prevalence, nonalcoholic steatohepatitis (NASH) is often undiagnosed in clinical practice. Objective: This study identified patients in the Veterans Affairs (VA) health system who likely had undiagnosed NASH using a machine learning algorithm. Methods: From a VA data set of 25 million adult enrollees, the study population was divided into NASH-positive, non-NASH, and at-risk cohorts. We performed a claims data analysis using a machine learning algorithm. To build our model, the study population was randomly divided into an 80% training subset and a 20% testing subset and tested and trained using a cross-validation technique. In addition to the baseline model, a gradient-boosted classification tree, naïve Bayes, and random forest model were created and compared using receiver operator characteristics, area under the curve, and accuracy. The best performing model was retrained on the full 80% training subset and applied to the 20% testing subset to calculate the performance metrics. Results: In total, 4 223 443 patients met the study inclusion criteria, of whom 4903 were positive for NASH and 35 528 were non-NASH patients. The remainder was in the at-risk patient cohort, of which 514 997 patients (12%) were identified as likely to have NASH. Age, obesity, and abnormal liver function tests were the top determinants in assigning NASH probability. Conclusions: Utilization of machine learning to predict NASH allows for wider recognition, timely intervention, and targeted treatments to improve or mitigate disease progression and could be used as an initial screening tool.

Background: Pompe disease is a rare lysosomal storage disorder, leading to accumulation of glycogen characterized by muscle weakness, fatigue, pain, and, in the longer term, a requirement for ventilatory and ambulatory support, and early mortality if untreated. Clinical evidence suggests that enzyme replacement therapy improves health outcomes for adults with late-onset Pompe disease (LOPD). PROPEL was a Phase 3, double-blind, randomized controlled trial, which evaluated cipaglucosidase alfa plus miglustat, vs alglucosidase alfa plus placebo in 123 adult patients with LOPD (clinicaltrials.gov: NCT03729362). Objectives: To analyze EQ-5D health-related quality of life (HRQoL) utility data from PROPEL. Methods: Multilevel modeling techniques (mixed regression methods) were used to analyze PROPEL EQ-5D-3L estimates and predict utility values for 7 health states previously identified in an economic evaluation for LOPD. In PROPEL, EQ-5D-5L values were assessed at screening and at weeks 12, 26, 38, and 52. EQ-5D-5L utility values were mapped to EQ-5D-3L values using the van Hout algorithm as recommended by the EuroQoL and the National Institute of Health and Care Excellence position statement at time of analysis. UK population tariffs were applied for all EQ-5D utility valuations. Utility values were predicted according to 6-minute walk distance (6MWD) and percent predicted sitting forced vital capacity. Results: The mixed model predicted that EQ-5D-3L utility values for patients who could walk >75 m with LOPD ranged between 0.55 and 0.67 according to patient 6MWD and respiratory function. In this analysis, patients with a 6MWD ≤75 m, consistent with a health state requiring wheelchair support in the economic analysis, had a predicted utility value of 0.49. There were few patients in PROPEL who could walk ≤75 m at any time point in the study, hence, these utility estimates should be interpreted with caution. EQ-5D-3L utility estimates from PROPEL were consistent with previously reported EQ-5D-3L values in LOPD. Conclusions: Overall, the results from our analysis indicate that important HRQoL losses are associated with reductions in mobility and respiratory function for patients with Pompe disease. The study provides important evidence of HRQoL utility values for patients with advanced LOPD, a population for whom published data are limited.

Background: Attention-deficit/hyperactivity disorder (ADHD) affects approximately 4.4% of US adults. ADHD is associated with high-risk driving behavior and costly motor vehicle accidents. DYANAVEL XR (DXR) (Tris Pharma, Inc.) is a once-daily fast-acting amphetamine developed for ADHD treatment. A randomized controlled trial showed that DXR patients were 43% less likely to crash during a driving simulation than individuals taking placebo. Study outcomes suggest a DXR crash rate similar to that of a driver without ADHD, while patients treated with the current standard of care (SOC) have a 52% higher crash risk than non-ADHD drivers. Objective: The aim was to evaluate the economic benefits attributable to improved driving abilities and avoided crashes in DXR patients compared with patients treated with the SOC or those who are untreated. Methods: A cost-impact model estimated 1-year crash-related cost outcomes for DXR-treated patients compared with SOC-treated and untreated ADHD patients. SOC was assumed to consist of a combination of short-, intermediate-, and long-acting ADHD stimulant and non-stimulant medications. DXR crash risk was assumed equivalent to the non-ADHD population risk, as supported by trial data. Crash risk for untreated and SOC-treated ADHD patients were assumed to be 99% and 52% higher than the general US population, respectively. Model outcomes included the cost impact (medication- and crash-related costs) and the number of crashes, injuries, and fatalities avoided with DXR. Results: Treatment with DXR would avoid 0.82 crashes, 0.016 injuries, and 0.036 fatalities per year compared with untreated patients, and 0.036 crashes, 0.007 injuries, and 0.0001 fatalities per year compared with SOC-treated patients. Compared with a population of 25% SOC-treated patients and 75% untreated patients, DXR use would save an average of $4581perpersonperyearacrossallagegroupswhenpricedat$ 80 per month, assuming all SOC-treated and untreated patients utilized DXR. When the value of quality-of-life improvement is considered, savings increase over 7-fold. Discussion: Outcomes suggest that DXR may be an economically beneficial treatment compared with SOC for ADHD patients. Conclusions: The economic model showed that DXR is cost-saving compared with no treatment and SOC by reducing the number of motor vehicle crashes in the ADHD population.

Background: Individuals with type 2 diabetes (T2D) show high risk of heart failure (HF). Left ventricular ejection fraction is a major factor for disease progression. In Germany, no recent longitudinal data are available. Objectives: To (1) measure the proportion of individuals with T2D who acquire HF over 2 years and (2) categorize ejection fraction using routine data and an algorithm, and (3) understand progression of HF in 5-year follow-up. Methods: This descriptive, retrospective study used longitudinal data from German statutory health insurance claims. A model using coded data classified the patients with HF into ejection fraction (EF) categories. Individuals were selected during 2013, with an inclusion period from 2014 to 2015 and a follow-up from 2016 to 2020. Baseline characteristics included demographic data, disease stage, comorbidities, and risk factors. Follow-up criteria included major adverse cardiac events (MACEs), EF category, and mortality. Disease progression was visualized by Sankey plots. Results: Among the 173 195 individuals with T2D identified in 2013, 6725 (median age, 74 years) developed HF in 2014 or 2015. 34.4% of individuals had MACEs, and 42.9% died over 5 years. Myocardial infarction (42%) was the most common event, followed by stroke (32%) and hospitalization (28%). A total of 5282 (78.54%) patients were classified into preserved EF and 1443 (21.46%) into reduced EF. Survival after 5 years was 71% in HF for preserved EF patients, and 29% in the HF for those with reduced EF. Conclusion: Heart failure is relevant in individuals with diabetes. A high number of patients may likely not survive a 5-year period. Validation of the model with German data is highly desirable. New ways of close monitoring could help improve outcomes.

Background: For patients with locally advanced or metastatic urothelial carcinoma (la/mUC), prognosis is poor and effective treatment options are limited. Erdafitinib is an oral fibroblast growth factor receptor (FGFR) kinase inhibitor approved by the FDA for the treatment of adults with la/mUC harboring FGFR alterations whose disease progressed following at least 1 prior line of therapy, including a PD-1 or PD-L(1) inhibitor, based on the phase 3, randomized THOR trial (NCT03390504, Cohort 1). Objective: To compare the efficacy and safety of erdafitinib vs enfortumab vedotin-ejfv (EV) in the absence of head-to-head comparison via an anchored matching-adjusted indirect comparison (MAIC). Methods: An anchored MAIC was conducted according to the National Institute for Health and Care Excellence Decision Support Unit guidance, with physician's choice of chemotherapy (docetaxel/paclitaxel and vinflunine) as the common comparator. Individual patient data from THOR were adjusted to match published key eligibility criteria and average baseline characteristics of EV-301, such as Bellmunt risk score, liver or visceral metastases, primary site, among others. Erdafitinib was then indirectly compared with EV using the relative treatment effects for the reweighted THOR population and those published for EV-301. Results: After matching, the effective sample size for THOR was 126 patients. The MAIC-recalculated hazard ratio (95% credible interval) for erdafitinib vs EV was 0.92 (0.54, 1.57) for overall survival and 0.93 (0.55, 1.56) for progression-free survival, yielding Bayesian probabilities of erdafitinib being better than EV of 62.1% and 60.5%, respectively. For response outcomes, the MAIC-recalculated risk ratio was 1.49 (0.56, 3.90) for confirmed objective response rate and 2.89 (0.27, 30.33) for confirmed complete response with probabilities of 72.6% and 81.3% for erdafitinib being better than EV, respectively. For safety, MAIC-yielded risk ratios of 1.09 (0.99, 1.21) for any treatment-related adverse events, 0.86 (0.57, 1.28) for grade 3+ TRAEs, and 1.02 (0.98, 1.06) for any treatment-emergent adverse events. Conclusion: The MAIC indicates comparable efficacy of erdafitinib vs EV for overall survival and progression-free survival, with erdafitinib showing a higher probability of achieving deep responses. While erdafitinib is associated with slightly more adverse events compared with EV, these events seem to be less severe.

Background: The use of androgen receptor signaling inhibitors, including apalutamide, in combination with androgen deprivation therapy is recommended for the treatment of metastatic castration-sensitive prostate cancer (mCSPC) and non-metastatic castration-resistant prostate cancer (nmCRPC). Objective: To describe real-world treatment patterns and clinical outcomes among patients with mCSPC or nmCRPC who initiated apalutamide in the United States. Methods: A retrospective cohort study of patients with mCSPC or nmCRPC who initiated apalutamide was conducted using electronic medical record data from US community-based urology practices (Feb. 1, 2017-April 1, 2022). Persistence with apalutamide was reported at 6-, 12-, and 18-months post treatment initiation. Clinical outcomes described up to 24 months after apalutamide initiation using Kaplan-Meier analyses included progression to castration resistance, castration resistance-free survival (CRFS), and metastasis-free survival (MFS). Outcomes were reported separately based on mCSPC or nmCRPC status and race (ie, Black or non-Black). Results: This study included 589 patients with mCSPC (mean age, 75.9 years) and 406 patients with nmCRPC (mean age, 78.8 years). Using a treatment gap of >90 days, persistence with apalutamide at 12 months remained high for both the mCSPC (94.9%) and nmCRPC (92.7%) cohorts, and results were descriptively similar among Black and non-Black patients, and when a treatment gap of >60 days was considered. In patients with mCSPC, overall progression to castration resistance rates at 12 and 24 months were 20.9% and 33.5%, and overall CRFS rates were 76.2% and 62.0%, respectively. In patients with nmCRPC, overall MFS rates at 12 and 24 months were 89.7% and 75.4%, respectively. Rates of these clinical outcomes were descriptively similar between Black and non-Black patients. Discussion: While clinical trials have demonstrated the efficacy and safety of apalutamide, there is limited real-world data describing treatment persistence and clinical outcomes among patients with mCSPC and nmCRPC who initiated apalutamide. Conclusions: In this real-world study of patients with mCSPC or nmCRPC initiated on apalutamide, treatment persistence was high and apalutamide demonstrated robust real-world effectiveness with respect to progression to castration resistance, CRFS, and MFS, overall and among Black and non-Black patients.

Background: Cataract surgery is an effective and commonly utilized procedure and can significantly improve quality of life and restore economic productivity. Certificate of need (CON) laws aim to regulate healthcare facility expansion and equipment acquisition to curtail costs, enhance quality, and ensure equitable access to care. However, little is known about the impact of CON laws on cataract surgery utilization and reimbursement. Objectives: To compare utilization and reimbursement for non-complex cataract surgery in CON and non-CON states. Methods: This retrospective database review analyzed publicly available data from the Centers for Medicare and Medicaid Services from 2017 to 2021 to identify the Medicare beneficiaries who underwent non-complex cataract surgery using Current Procedural Terminology code 66984 in Medicare outpatient hospitals. Utilization and reimbursement patterns were analyzed in states with and without CON laws using the compound annual growth rate, with reimbursement adjusted by the US Bureau of Labor Statistics Consumer Price Index. Results: The Centers for Medicare and Medicaid Services reported 893 682 non-complex cataract surgeries in the study period; of these, 609 237 were in CON and 280 215 in non-CON states. Inflation-adjusted reimbursement increased in both CON (1.17%) and non-CON (1.83%) states, while the reimbursement in non-CON states was greater than the national average adjusted reimbursement (1.67%). Utilization of non-complex cataract surgery declined during the study period in both CON and non-CON states. A larger decline in utilization was observed in CON states (-7.32%) than in non-CON states (-6.49%). Utilization was slightly higher in non-CON than in CON states for each year except 2019. Discussion: Utilization of non-complex cataract surgery by Medicare beneficiaries declined over the study period in both CON and non-CON states, possibly impacted by the COVID-19 pandemic. Inflation-adjusted reimbursement adjusted for Consumer Price Index increased more in non-CON than CON states, possibly reflecting shifts in market dynamics in CON-regulated states. Conclusions: Surgeons and policymakers should consider the implications of CON laws on the utilization and reimbursement of cataract surgery. Further study is necessary to ascertain whether these trends persist beyond 2021.