Journal of Health Economics and Outcomes Research最新文献

Background: Glycogen storage disease type Ia (GSDIa) is a rare inherited disorder resulting in potentially life-threatening hypoglycemia, metabolic abnormalities, and complications often requiring hospitalization. Objective: This retrospective database analysis assessed the complications, resource utilization, and costs in a large cohort of patients with GSDIa. Methods: We conducted a retrospective cohort study of GSDIa patients and matched non-GSDIa comparators utilizing the PharMetrics® Plus database. International Classification of Diseases, Tenth Revision (ICD-10) diagnosis codes in any billing position for inpatient and outpatient claims (January 2016-February 2020) were identified for complications related to GSDIa. Healthcare use and costs were assessed by setting of care (inpatient, outpatient, physician office, emergency department, and pharmacy). Results: Overall, 557 patients with GSDIa and 5570 matched comparators (male, 63%; adults, 67%) were identified. The most frequent complications in patients with GSDIa vs comparators included anemia due to enzyme disorders (odds ratio, 4.0 × 103; 95% confidence interval, 555.9-2.8 × 104), hepatocellular adenoma (305.9; 41.6-2.2 × 104), liver transplantation (164.6; 21.8-1.2 × 103), and gastrostomy (152.2; 61.1-379.2), as well as acidosis (45.5; 29.4-70.3), hepatomegaly (43.6; 29.1-65.3), hyperuricemia (23.6; 11.9-46.9), and hypoglycemia (20.2; 14.3-28.7). Chronic complications (eg, gout, osteoarthritis, chronic kidney disease, and neoplasms) were more common in adults with GSDIa, whereas acute complications (eg, poor growth, gastrostomy, seizure, and hypoglycemia) were more common in children with GSDIa. Patients with GSDIa more often required hospitalization (0.53 vs 0.06 hospitalizations per patient per year) vs comparators, including 2 or more hospitalizations (26.6% vs 2.3%), longer length of stay (3.1 vs 0.4 days), and more annual visits in all care settings, including 4.3 times more visits in the emergency department. Mean annual total healthcare costs were higher for GSDIa patients vs comparators ( $33\hspace{0.17em}910vs$ 4410). Discussion: In this large, retrospective database analysis, complications observed among patients with GSDIa were consistent with prior reports and demonstrate the chronic and progressive nature of the disease. Resource utilization was substantial in GSDIa patients, and mean annual total healthcare costs were almost 8 times higher than those of comparators. Conclusions: GSDIa is associated with numerous potentially serious and sometimes fatal complications, extensive resource utilization, and high management costs.

Background: Late-onset Pompe disease (LOPD) is a rare, autosomal recessive metabolic disorder that is heterogeneous in disease presentation and progression. People with LOPD report a significantly lower physical, psychological, and social quality of life (QoL) than the general population. Objectives: This study investigated how individuals' self-reported LOPD status (improving, stable, declining) relates to their QoL. Participant experiences such as use of mobility or ventilation aids, caregivers, symptomology, and daily life impacts were also characterized. Methods: A 2-part observational study was conducted online between October and December 2023 using the 36-item short-form tool (SF-36) and a survey. Adults with LOPD (N=41) from Australia, France, Italy, and the Netherlands were recruited. Results: Participants reporting "declining" LOPD status (56%) had lower physical functioning SF-36 scores than those reporting as "stable" or "improving." Those self-reporting as stable or improving often described an acceptance of declining health in their responses. Physical functioning scores were generally stable in respondents who had been receiving enzyme replacement therapy (ERT) for 1-15 years, but those who had received ERT for >15 years had lower scores. Requiring ventilation and mobility aids had additive negative impacts on physical functioning. Difficulty swallowing, speaking, and scoliosis were the most burdensome symptoms reported by those on ERT for >15-25 years. Discussion: These results demonstrate the humanistic burden of LOPD; through declining physical functioning SF-36 scores over increasing time and increased use of aids, and also through factors related to self-reported LOPD status (where declining status was associated with lower scores) and symptomology variances. Taken holistically, these areas are valuable to explore when informing optimized care. Among a largely declining cohort, even those not self-reporting decline often assumed future deterioration, highlighting the need for improved therapies and the potential to initiate or switch ERT based on evolving symptomology and daily life impacts. Conclusion: Our results indicate that progressing LOPD leads to loss of QoL in ways that relate to time, use of aids, evolving symptomology, and the patient's own perspective. A holistic approach to assessing the individual can help ensure relevant factors are investigated and held in balance, supporting optimized care.

Background: Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) are severe, treatment-refractory, epileptic encephalopathies that often develop in infancy or early childhood. Since December 1, 2022, plant-derived highly purified cannabidiol (CBD) medicine (Epidyolex®; 100 mg/mL oral solution) has been reimbursed in the Netherlands for the adjunctive treatment of seizures associated with LGS or DS. Objective: To estimate the cost-effectiveness of CBD plus usual care vs usual care alone in patients with LGS or DS in the Netherlands. Methods: A cohort-based Markov model from a Dutch societal perspective, based on seizure frequency and seizure-free days, was developed for patients receiving CBD plus usual care (antiseizure medications, including clobazam) or usual care alone. Population characteristics, clinical inputs, and utility values were sourced from CBD clinical trials and quality-of-life studies. Drug acquisition, disease management, adverse events, and societal costs from published literature were included. A 2019/2020 price year in euros was used. The model used a mean dosage of 12 mg/kg/day, a lifetime (90-year) horizon, and a 3-month cycle length. Discount rates of 4.0% and 1.5% per annum were applied to costs and outcomes, respectively. Uncertainty was explored through deterministic and probabilistic sensitivity analyses. Results: In patients with LGS, CBD plus usual care led to additional costs of €28 338 and increased quality-adjusted life-years (QALYs) of 1.318 compared with usual care alone. The incremental cost-effectiveness ratio of €21 493/QALY in LGS is below the willingness-to-pay threshold of €80 000/QALY in the Netherlands. In patients with DS, CBD plus usual care dominated usual care alone, with cost savings of €23 642 and increased QALYs of 0.868. The probability that CBD plus usual care is cost-effective in the Netherlands compared with usual care alone is 96% and 99% in patients with LGS and DS, respectively. Discussion: Elicitation methods were used to address data gaps in model inputs (eg, healthcare resource utilization and utilities); Dutch clinical experts, sensitivity, and scenario analyses validated this approach. Conclusions: Based on a willingness-to-pay threshold of €80 000, the base case cost-utility analysis demonstrated the cost-effectiveness of CBD plus usual care in patients with treatment-refractory LGS or DS aged 2 years or older in the Netherlands.

Background: The Inflation Reduction Act's Medicare Drug Price Negotiation Program allows the federal government to negotiate caps for select medications. These price caps may reduce revenue for the pharmacy benefit managers (PBMs) that negotiate the actual price paid for medicines in the U.S. To offset the resulting pressure on their profit margins, it is possible that PBMs would, in turn, increase patients' out-of-pocket costs for medicines with capped prices. The model presented here evaluates how increased out-of-pocket costs for the anticoagulants apixaban (Eliquis) and rivaroxaban (Xarelto) could impact patients financially and clinically. Methods: Copay distributions for all 2023 prescription fills for apixaban and rivaroxaban managed by the 3 largest PBMs, CVS Caremark, Express Scripts International, and Optum Rx, were used to approximate current copay costs. Increased out-of-pocket costs were modeled as a shift of all apixaban and rivaroxaban prescriptions to the highest copay tier. The known linear relationship between copay costs and treatment abandonment was used to calculate the potential resulting increase in treatment abandonment. Known rates of morbidity and mortality due to abandoning anticoagulants were used to estimate resulting increases in morbidity and mortality. Results: If the 3 largest PBMs all shifted costs onto patients by moving all apixaban and rivaroxaban prescriptions to the highest formulary tier, Tier 6, patients' copay amount would increase by $235to$ 482 million for apixaban and $105to$ 206 million for rivaroxaban. Such an increase could lead to 169 000 to 228 000 patients abandoning apixaban and 71 000 to 93 000 abandoning rivaroxaban. The resulting morbidity and mortality could include up to an additional 145 000 major cardiovascular events and up to 97 000 more deaths. Conclusion: The Medicare Price Negotiation Program could impact patients negatively if it causes PBMs to increase patients' out-of-pocket costs for medicines. Policymakers should closely monitor changes in overall affordability, including all patient out-of-pocket expenditures, for medications in the program. Preemptive measures should be considered to ensure that the most vulnerable citizens are not placed in precarious situations, leading to poorer health outcomes.

Background: Two million Americans have type 1 diabetes (T1DM). Innovative treatments have standardized insulin delivery and improved outcomes for patients, but patients' access to such technologies depends on social determinants of health, including insurance coverage, proper diagnosis, and appropriate patient supports. Prior estimates of US prevalence, incidence, and patient characteristics have relied on data from select regions and younger ages and miss important determinants. Objectives: This study sought to use large, nationally representative healthcare claims data sets to holistically estimate the size of the current US population with T1DM and investigate geographic nuances in prevalence and incidence, patient demographics, insurance coverage, and device use. This work also aimed to project T1DM population growth over the next 10 years. Methods: We used administrative claims from 4 sources to identify prevalent and incident T1DM patients in the US, as well as various demographic and insurance characteristics of the patient population. We combined this data with information from national population growth projections and literature to construct an actuarial model to project growth of the T1DM population based on current trends and scenarios for 2024, 2029, and 2033. Results: We estimated 2.07 million T1DM patients nationally across all insurance coverages in our 2024 baseline model year: 1.79 million adults (≥20 years) and 0.28 million children. This represents a US T1DM prevalence rate of 617 per 100 000 and an incidence rate of 0.016%. By 2033, we project the US population with T1DM will grow by about 10%, reaching approximately 2.29 million patients. Discussion: Our results showed important differences in T1DM prevalence and incidence across regions, payers, and ethnic groups. This suggests studies based on more geographically concentrated data may miss important variation in prevalence and incidence across regions. It also indicates T1DM prevalence tends to vary by income, consistent with several international studies. Conclusions: Accurate projections of T1DM population growth are critical to ensure appropriate healthcare coverage and reimbursement for treatments. Our work supports future policy and research efforts with 2024, 2029, and 2033 projections of demographics and insurance coverage for people with T1DM.

Background: Infections attributable to respiratory syncytial virus (RSV) are a major cause of hospitalization among young children worldwide. Despite substantial clinical and economic burden, real-world data associated with RSV infections in the United Arab Emirates (UAE) are limited. Objectives: This study aimed to assess among children (<18 years) diagnosed with RSV the epidemiology, seasonality, comorbidities, treatment patterns, length of hospital stay, healthcare resource utilization (HCRU), and costs associated with pediatric infection in Dubai, UAE. Methods: This 10-year retrospective cohort study (Jan. 1, 2014-Sept. 30, 2023) utilized Dubai Real-World Database, a private insurance claims database. Patients aged <18 years with a first-episode diagnosis claim (primary or secondary, or a hospital admission) for RSV any time during the index period (Jan. 1, 2014-June 30, 2023) were included. Outcomes were analyzed during a 3-month follow-up. Patients were stratified into 3 cohorts: Cohort 1 (<2 years), Cohort 2 (2 to <6 years), and Cohort 3 (6 to <18 years). Results: Of 28 011 patients identified, 25 729 were aged <18 years with RSV infection. An increasing trend in reported cases was observed from 2014 to 2022, with an average annual increase of 55%. Half of study patients (49.3%) belonged to Cohort 1, with a mean age of 0.6 years, while less than 2% had known risk factors and 22% of the patients in cohort 1 were hospitalized. In Cohort 1, 32.0% had upper respiratory tract infections, 39.4% had lower respiratory tract infections, and 44.4% of patients had an "other respiratory disease." The average length of hospitalization was about 4 days across all cohorts. The total hospitalization cost was highest in patients <2 years, amounting to US $9\hspace{0.17em}798\hspace{0.17em}174(median,US$ 2241.30). Conclusion: Among the RSV patients, 49.3% were <2 years of age and few had recognized risk factors. Among patients <2 years, 22% were hospitalized, with an average hospital stay of 4 days; the cost of hospitalization totaled US $9 798 174. These findings can inform healthcare stakeholders about future policy measures and the need for effective preventive strategies.

Background: Medical management of patients with type 2 diabetes mellitus (T2DM) is complex because of the chronic nature of the disease and its associated comorbidities. Injectable once-weekly semaglutide for diabetes (OW sema T2D) is a type of glucagon-like peptide-1 receptor agonist approved for the treatment of patients with T2DM. Objectives: To describe patient characteristics and HbA1c changes for patients prescribed 1.0 mg maintenance dose OW sema T2D. Methods: This retrospective study included adult patients with T2DM with a pre-index glycated hemoglobin (HbA1c) of at least 7%, initiating treatment with OW sema T2D between January 1, 2018, and December 31, 2019, and prescribed a 1.0 mg maintenance dose. Patients were identified in the Optum Research Database and were included if they had continuous health plan enrollment for at least 12 months prior to (pre-index) and at least 12 months following (post-index) the date of the first OW sema T2D claim (index). Dose at initiation and prescriber specialty were captured. Change in HbA1c between the latest post-index and pre-index HbA1c measurement was calculated among all patients and among those with at least 90 days of continuous treatment (persistent patients). Results: A total of 2168 patients were included in this study. On average, patients were taking 13.5 different classes of medications. The majority of patients had lipid metabolism disorder (90.8%), hypertension (86.6%), diabetes with complications (86.8%), or other nutritional/endocrine/metabolic disorders (72.5%). The mean HbA1c reduction was 1.2% (P < .001). Patients persistent with OW sema T2D (n =1280) had a mean HbA1c reduction of 1.4% (P < .001). The mean (SD) days covered with a 1.0 mg maintenance dose was 236.1 (94.1) days. Discussion: Despite being medically complex, the patients in this real-world study experienced significant reductions in HbA1c following initiation of OW sema T2D. Conclusions: A 1.0 mg maintenance dose of OW sema T2D is an effective treatment for T2DM in the real world.

Background: Agitation in Alzheimer dementia is common, but the associated healthcare burden remains unclear. Objective: This retrospective analysis evaluated baseline characteristics, healthcare resource utilization, and costs among patients with agitation in Alzheimer dementia and those without agitation in Alzheimer dementia. Methods: Medicare beneficiaries from 100% of the Medicare Fee-for-Service claims database (2009-2016) with 2 or more claims 30 or more days apart for both Alzheimer's disease and dementia and continuous enrollment with medical/pharmacy coverage for 6 months before and 12 months after the index diagnosis were included. Patients with agitation in Alzheimer dementia were identified by 2 or more claims 14 or more days apart using International Classification of Diseases-9-CM/-10-CM codes based on the provisional International Psychogeriatric Association agitation definition. Patients with severe psychiatric disorders were excluded. Two cohorts of patients (with and without agitation) were then defined, and patient characteristics, healthcare resource utilization, and costs were compared in a descriptive exploratory analysis. Results: Of 2 684 704 Fee-for-Service patients with Alzheimer dementia, 769 141 met all inclusion criteria; among these, 281 042 (36.5%) had agitation. The mean age in patients with and without agitation in Alzheimer dementia was 83 years. Most patients in both groups were female, but the proportion of males was slightly higher in the agitation in Alzheimer dementia group (30.3% vs 28.2%, respectively). Patients with agitation in Alzheimer dementia were more likely than those without agitation in Alzheimer dementia to have lower socioeconomic status (dual eligibility for Medicaid, 45.0% vs 41.7%, respectively) or be disabled (10.5% vs 9.4%). Overall, healthcare costs were higher in the agitation in Alzheimer dementia population compared with those without agitation in Alzheimer dementia (mean cost PPPY, $32\hspace{0.17em}322and$ 30 121, respectively), with the largest differences observed in inpatient and post-acute care costs. Conclusions: These exploratory findings underscore the substantial economic burden of agitation in Alzheimer dementia and highlight the need for treatment options for the agitation in Alzheimer dementia population to improve associated health outcomes.