Journal of Health Economics and Outcomes Research最新文献

Background: The 2022 European Society of Cardiology/European Respiratory Society (ESC/ERS) pulmonary hypertension guidelines recommend initial combination of endothelin receptor antagonist (ERA) and phosphodiesterase type-5 inhibitor (PDE5i) in patients with pulmonary arterial hypertension (PAH) at low to intermediate risk without cardiopulmonary comorbidities. Objective: To examine US treatment patterns for newly diagnosed patients, including frequency of cardiopulmonary comorbidities. Methods: Treatment-naïve adults (≥18 years) initiating treatment, identified using claims data (IQVIA PharMetrics® Plus; April 2013-June 2023), were assigned dual therapy if initiating ERA/PDE5i within a treatment-determination period (3 months), or monotherapy if initiating ERA or PDE5i. Descriptive statistics captured 25th/75th percentiles, means (SD), and medians. Results: Of 2868 patients, 824 (28.7%) initiated dual therapy and 2044 (71.3%) monotherapy. In dual therapy, 461 (56.0%) initiated ERA first, 250 (30.3%) PDE5i first, and 113 (13.7%) both the same day. In monotherapy, 153 (7.5%) received ERA and 1891 (92.5%) PDE5i. For escalation to dual therapy, 330 (16.1%) monotherapy users initiated ERA (10.7%) or PDE5i (5.5%) during follow-up. Most had cardiopulmonary comorbidities (monotherapy: 86.8%; dual: 79.6%). Of the 824 on dual therapy, 20.4% started triple therapy during follow-up. Compared with monotherapy, dual therapy users were younger (54.9 vs 59.6 years) and mostly female (72.9% vs 60.9%). Discussion: This study found that in the United States, among newly diagnosed PAH patients, 71.3% initiated monotherapy and 28.7% dual therapy, with 16.1% of monotherapy patients eventually escalating to dual therapy. High rates of initial monotherapy may reflect the high proportion of patients with comorbidities and their possible intolerance of initial dual therapy. As these data mostly precede the 2022 guidelines, future research should include treatment post-guidelines, rationales behind decision making, differences between initial monotherapy and dual therapy users, and monotherapy overreliance and effects on morbidity and mortality. Conclusions: This analysis of real-world US treatment patterns for newly initiating PAH patients found low rates of upfront dual-therapy use with high rates of cardiopulmonary comorbidities.

Background: Glycogen storage disease type Ia (GSDIa) is a rare, inherited metabolic disorder characterized by a deficiency in glucose 6-phosphatase. People living with GSDIa are at high risk for clinical manifestations (including hypoglycemia and hepatomegaly) and clinical complications (including hyperlipidemia, stunted growth, liver adenomas, and renal failure). Evaluating symptom management and secondary care burdens is vital to understanding the patient experience and optimizing care pathways. Objective: We sought to quantify the number of patients with GSDIa within secondary care settings across England and to evaluate the burden of disease associated with living with GSDIa. Methods: This study utilized the United Kingdom Hospital Episode Statistics (HES) database across a 69-month time period (April 2015-December 2020) to investigate National Health Service (NHS) resource use and GSDIa mortality. Results: Patients (N = 943) with GSDIa were identified. Frequent manifestations included anemia (n = 421; 45%), hypoglycemia (n = 185; 20%), and hepatomegaly (n = 152; 16%). On average, patients had a total of 8 events/year, including 2 elective events, 2 nonelective emergencies, 1 outpatient visit, and 3 daycase visits. In the entire HES population, there was approximately 1 (~60% elective, ~40% nonelective) event/year. The highest total number of events across the entire patient journey tracked within the HES occurred with adolescents (12-17 years) who had an average of 28.5 events. Average length of stay was greatest in the pediatric infantile (0-2 years) population with 4.6 days and 3.4 days for nonelective and elective events, respectively. When benchmarked against the general population, patients with GSDIa had a mortality rate of 4.3%, compared with 0.9% for the entire HES population. The average age at mortality was 14.3 years lower for patients with GSDIa vs the entire HES population (63.7 years vs 78.0 years). Discussion: This study demonstrates high burden associated with GSDIa. Complications are a key driver of NHS resource use. Mortality associated with GSDIa in hospitalized patients is higher than the general population. Conclusions: GSDIa imposes a large burden on the healthcare system. There is a clear unmet need for patients with GSDIa, and complications are a substantial driver of resource use and burden of disease.

Background: Colorectal cancer (CRC) is the fourth most frequently diagnosed cancer and the second leading cause of cancer-related deaths in the United States. Screening can prevent CRC by detecting advanced precancerous lesions. Adherence to screening is crucial in reducing CRC disease burden; however, there is limited research on the impact of digital outreach screening uptake and adherence. Objective: This study evaluated the impact of different digital outreach channels on patient adherence to CRC screening with a multi-target stool DNA (mt-sDNA) test in a real-world setting. Methods: Patients were individuals aged 45 to 85 years with a valid mt-sDNA test order from Exact Sciences Laboratories, LLC (Jan. 1, 2023-Sept. 23, 2023). All patients received letters and phone calls; some received short message service (SMS), email, or both. Adherence and time to test return were compared across digital outreach categories stratified by patient characteristics. Multivariable regression evaluated the association of digital outreach methods with adherence and time to test return. Results: Among 2 425 308 patients (43.5% between 50 and 64 years, 58.2% female), digital SMS only (62.7%) was the most common outreach method. Overall adherence was 70.1%, with highest adherence in the digital SMS-plus-email group (72.9%). Mean time to test return from shipment of mt-sDNA kit to receipt of valid test was 25.8 days. In adjusted analyses, patients receiving digital SMS plus email had the highest odds of test return (odds ratio, 1.75; 95% confidence interval [CI], 1.73-1.78; P<.001) and had return times 8.7% shorter than the no-digital-outreach group (95% CI, 8.2-9.2; P<.001). Discussion: Among nationally insured individuals within the recommended age range for CRC screening, overall adherence to the mt-sDNA test was in the 70s, with the highest rates in the digital (SMS and email) outreach group and the lowest in the no-digital-outreach group. Conclusions: These findings highlight the importance of multichannel navigation in facilitating completion of CRC screening with the mt-sDNA test.

Background: Chronic kidney disease (CKD) affects 13% of the global population, is predicted to be the fifth leading cause of premature death by 2040, and is associated with increased risk of cardiovascular disease and acute cardiovascular events. With an aging population and rising diabetes rates, the prevalence of CKD is expected to escalate in the United Kingdom, leading to substantial healthcare costs. When patients reach end-stage kidney disease, interventions such as dialysis and transplantation are required. Dialysis is not only extremely costly but is also associated with a diminished quality of life and significantly elevated mortality. Objectives: This study assesses the cost-effectiveness of several population-level interventions designed to manage CKD, including its progression to end-stage kidney disease. Methods: A population-level Markov model was developed to evaluate the cost-effectiveness and population health impacts of 4 key interventions, individually and combined: (1) early/improved diagnosis, (2) enhanced CKD management, (3) increased use of SGLT-2 inhibitors, and (4) higher rates of pre-emptive live donor transplantation. The model incorporates both NHS direct costs and broader economic impacts, with a 10-year horizon and quarterly cycles. Two scenarios were analyzed: a base case (based on disease progression probabilities) and a constrained case (where growth in the number of patients receiving dialysis and transplantation is limited to historical rates observed in the UK National Health Service). Results: All interventions demonstrated cost-effectiveness, with the combined approach preventing 10 351 deaths and yielding 48 381 quality-adjusted life-years (QALYs) at a cost of £7675 per QALY in the base case scenario. In the constrained scenario, the combined interventions demonstrated cost-effectiveness, preventing 10 026 deaths and yielding 47 514 QALYs at a cost of £22 767 per QALY. Conclusions: The results demonstrate the cost-effectiveness of population level interventions for management of CKD, and the significant burden of dialysis, with avoidance of progression to dialysis a key driver of QALY gains and cost offsets.

Background: The association between falls or fall-related fractures and hypoglycemia in people with type 2 diabetes is well established. Insulin treatment is associated with an increased risk of hypoglycemia, which is compounded in people of older age, but the risk is lower with longer-acting vs intermediate- or long-acting basal insulin analogs. Objective: To examine healthcare resource utilization and costs related to falls/fractures in people with type 2 diabetes treated with the longer-acting basal insulin Gla-300 (insulin glargine 300 U/mL) vs long-acting basal insulins (insulin glargine 100 U/mL or insulin detemir)/neutral protamine Hagedorn (NPH). Methods: This retrospective study of Optum's de-identified Clinformatics® Data Mart Database compared data for people aged 50 years or older with at least 1 prescription claim for basal insulin (excluding insulin degludec) between April 1, 2015, and April 30, 2021, who initiated Gla-300 insulin (basal insulin-naive) or transitioned to Gla-300 from a different basal insulin (basal insulin-switch). Cohorts were propensity score-matched. The primary outcome was fall/fracture-related hospitalization and emergency department visit events (per 100 person-years of follow-up [P100PYFU]). The association between fall/fracture events and hypoglycemia and costs were secondary outcomes. Outcomes were compared using 95% confidence intervals of rate and other ratios; no statistical inference was performed. Results: Fall/fracture-related hospitalization (2.88 vs 3.33 P100PYFU) and emergency department visit events (5.28 vs 5.95 P100PYFU) were numerically lower in people who initiated basal insulin with Gla-300 vs long-acting basal insulins/NPH, and in those who switched to Gla-300 vs long-acting basal insulins/NPH (2.54 vs 3.38 and 4.48 vs 5.21 P100PYFU, respectively). People with vs without hypoglycemia experienced more falls/fractures, regardless of whether initiating basal insulin or switching basal insulin treatment. Costs tended to be lower for people who switched to Gla-300; however, low event rates caused variability. Conclusions: The results of this study suggest that there is a positive correlation between fall/fracture events and hypoglycemia in people with type 2 diabetes and also, that fall/fracture-related healthcare resource utilization was numerically lower in people who initiated basal insulin with Gla-300 vs long-acting basal insulins/NPH, and in those who switched to Gla-300 vs long-acting basal insulins/NPH.

Introduction: X-linked hypophosphatemia (XLH) is a rare, genetic disorder that severely impacts the health-related quality of life (HRQoL) of people living with the condition. This impact can also extend to carers and family members, described as a "spillover effect." Measurement of spillover effects can lead to greater understanding of disease burden and potentially to more equitable decision-making regarding adoption of treatments by health technology assessment (HTA) bodies. This study aimed to explore spillover effects among carers and family members of adults with XLH. Methods: This cross-sectional, mixed-methods study included carers and family members of adults diagnosed with XLH in the United Kingdom. Quantitative data included a background questionnaire, the EQ-5D-5L, and the Work Productivity and Activity Impairment Questionnaire (WPAI). Qualitative data were gathered through semi-structured interviews. Results: Twenty carers or family members of adults with XLH completed the study. Four (20%) had an XLH diagnosis themselves. Mean (SD) EQ-5D utility was 0.66 (0.33) for the total sample, and 0.20 (0.31) and 0.77 (0.21) for participants with and without a personal diagnosis of XLH, respectively. The WPAI activity impairment was 42% and overall work impairment was 28%. Interviews revealed 6 areas of participants' lives impacted by XLH: emotional wellbeing, daily activities, work, finances, social lives and relationships, and physical impacts. Conclusion: Findings of this study indicate a substantial health-related quality-of-life and productivity burden for carers and family members of adults with XLH. The magnitude of this burden highlights the importance of incorporating such spillover effects in HTAs and broader rare disease policies.

Background: Pulmonary vein isolation (PVI) is the main ablation strategy for the treatment of paroxysmal atrial fibrillation. Different technologies are available for PVI, including various cryoballoon catheters (CB-A). Compared with the Arctic Front Advance Pro™, the novel POLARx FIT™ CB-A might reduce costs for atrial fibrillation ablation. Objective: The aim of this study is to perform a health economic evaluation of two cryoballoon systems for PVI procedures. Methods: All patients undergoing their first PVI procedure with POLARx FIT™ CB-A or the Arctic Front Advance Pro™ CB-A were prospectively enrolled. The health economic analysis was performed on the index hospitalization and procedure. The primary safety endpoint included procedure-related adverse events within the index hospitalization. A decision tree model was built to estimate downstream costs. Results: A total of 80 patients with paroxysmal atrial fibrillation undergoing PVI were analyzed, with 40 patients in each arm. Compared with the Arctic Front Advance Pro™ CB-A, POLARx FIT™ CB-A showed a lower procedure time, left-atrium dwell time, and fluoroscopy time. The complication rate was low (6.3%) and included 3 reversible phrenic nerve palsies in the POLARx FIT™ CB-A group vs 2 in the Arctic Front Advance Pro™ CB-A group. Compared with the Arctic Front Advance Pro, the POLARx FIT™ CB-A was associated with lower procedural costs (€2069.7 ± €165.2 vs €2239.5 ±  €366.0; P =.009). Conclusion: The POLARx FIT™ CB-A was associated with a shorter procedure time, translating into lower procedural costs, compared with the Arctic Front Advance Pro. Complications were rare and comparable between the two technologies.

[This corrects the article DOI: 10.36469/jheor.2024.124604.].