Journal of Health Economics and Outcomes Research最新文献

Background: Alpha-1 antitrypsin deficiency (AATD) testing rates and associated clinical and economic outcomes data in the US Medicare population are limited. Objective: To characterize individuals with AATD, describe clinical outcomes/healthcare research utilization (HCRU) among individuals with chronic obstructive pulmonary disease (COPD) with or without AATD, and identify AATD testing rates among individuals newly diagnosed with COPD. Methods: This retrospective, observational analysis of claims data included individuals from the Humana Research Database (aged 18-89 years) enrolled in Medicare Advantage Prescription Drug plans. Three cohorts included individuals with evidence of AATD; individuals with COPD + AATD matched to individuals with COPD; and individuals with newly diagnosed COPD. AATD health-related outcomes, such as pulmonary and extrapulmonary conditions or events, and economic outcomes, including inpatient admissions, emergency department visits, and physician visits, were examined independently during the pre-index and post-index periods and compared between those with ATTD and without AATD. Results: We identified 1103 individuals with AATD (aged 67.2 ± 10.0 years, 56.3% women, 94.5% White); overall, 22.2% had exacerbations, respiratory distress, and respiratory failure. Individuals with COPD and AATD (n = 742) were matched to individuals with COPD (n = 7420), based on age (68 ± 9 years), sex (55.0% women), and race (97.2% White). The AATD group had a higher proportion of emphysema (47.4% vs 18.7%), COPD exacerbations (40.6% vs 24.7%), and cirrhosis (4.0% vs 1.3%) than the non-AATD group. All-cause inpatient admissions (31.7% vs 27.3%), COPD-specific inpatient admissions (7.4% vs 4.3%), and COPD-specific emergency department visits (19.5% vs 10.8%) were higher in individuals who had ATTD than in those without AATD. AATD testing rates among individuals with newly diagnosed COPD increased slightly over time (2015: 1.07%; 2020: 1.49%). Individuals with COPD and AATD had more comorbidities and higher HCRU. Testing rates increased slightly but remained low. Discussion: Further research is needed to assess the impact of improved AATD testing on those with COPD. Conclusion: Increased awareness, earlier testing, and treatment may reduce the healthcare burden of AATD in the US Medicare population.

Background: Chronic graft-versus-host disease (cGvHD) - a potentially debilitating complication of allogeneic hematopoietic stem cell transplantation - is a rare condition. Objectives: This vignette-based study aimed to generate utility values to inform an economic model via an online survey wherein cGvHD health state (HS) vignettes were valued by the general UK population using the EQ-5D-5L and the EQ-5D-visual analog scale (EQ-5D VAS). Methods: This non-interventional health-related quality of life (HRQoL) study was conducted in 3 stages across the UK: the development, validation, and valuation of HS vignettes to generate utility values for cGvHD. Four HS for cGvHD were defined based on an economic model partitioning different treatment level responses in patients with cGvHD receiving third-line (3L) therapy (HS1: complete response, HS2: partial response, HS3: lack of response, and HS4: recurrent cGvHD). Draft vignettes were developed for each HS based on 4 previously published GvHD vignettes. The contents of the draft vignettes were reviewed for all aspects of cGvHD symptoms and functional impact and validated through semistructured interviews with 5 clinical experts. The 4 finalized HS vignettes were valued by 300 participants from the UK general population using EQ-5D-5L and EQ-5D VAS. Results: Previously published vignettes were used to develop the vignettes for the current study that described GvHD in the context of blood cancer and other rare blood disorders (n = 2 each) and included symptoms, functioning, and quality of life for a patient in the HS. The highest and lowest mean EQ-5D-5L utility scores were observed for HS1 (mean [95% CI]: 0.577 [0.558-0.595]) and HS4 (0.061 [0.034-0.088]), respectively. The EQ-5D-VAS showed the highest and lowest mean utility scores for HS1 (46.8 [44.9-48.6]) and HS4 (25.6 [23.4-27.7]), respectively. Conclusion: This study generated utility values for HS vignettes describing symptoms, functioning, and HRQoL for patients with cGvHD receiving 3L therapy. The utility values highlighted a substantial burden of cGvHD and HRQoL impact associated with the treatment response level. However, assessing concordance between utility estimates derived from the vignette-based method in a general population and those from patients with cGvHD is further warranted.

Background: Prescription drug insurance in Canada is constituted of a patchwork of public and private insurance plans. The type of drug insurance may have a negative impact on access to treatment for patients covered by public plans compared with private plans. Objectives: In patients with psoriasis treated with advanced therapy in public vs private drug insurance groups, we compared: (1) psoriasis severity scores when an advanced therapy was prescribed, (2) psoriasis severity scores at follow-up, (3) treatment response, and (4) delay between prescription and first dose of advanced therapy. Methods: This unicentric, retrospective cohort study included patients suffering from psoriasis treated by advanced therapy, dermatologist-prescribed between September 2015 and August 2019, in a tertiary academic care center in Québec City, Canada. Data were collected from medical records. Results: Patients treated with an advanced therapy for psoriasis covered under the provincial public drug insurance plan (n = 78) and under a private drug plan (n = 93) did not differ regarding the studied outcomes. Patients' characteristics differed between groups. Patients in the public group were older (P < .0001), more socioeconomically deprived (P < .05), and more likely to benefit from compassion from the industry to access a prescribed medication free of charge (P < .0001) compared with patients from the privately insured group. Discussion: The high prevalence of compassionate programs from the industry in the public insurance group (42% vs 14%), and the high prevalence of psoriasis on difficult-to-treat areas (face, genitalia, and/or palmoplantar areas) in our cohort (85.4%) may mask differences in access to advanced therapy between the two groups. Conclusions: Prescribers of advanced therapy can be reassured, as we found no inequality in access or care based on patients' drug insurance coverage.

Background: Modeling techniques in the field of pediatrics present unique challenges beyond traditional model limitations, and sometimes difficulties in faithfully simulating the condition's evolution over time. Objective: This study aimed to identify whether economic modeling approaches in diabetes in pediatric patients align with the recommendations of clinical practice guidelines and the latest scientific evidence. Methods: A literature review was performed in March 2023 to identify modeling-based economic evaluations in diabetes in pediatric patients. Data were extracted and synthesized from eligible studies. Clinical practice guidelines for diabetes were gathered to compare their alignment with modeling strategies. Two endocrinology specialists provided insights on the latest findings in diabetes that are not yet included in the guidelines. A multidisciplinary group of experts agreed on the relevant themes to conduct the comparative analysis: parameter informing on glycemic control, diabetic ketoacidosis/hypoglycemia, C-peptide as prognostic biomarker, metabolic memory, age at diagnosis, socioeconomic status, pediatric-specific sources of risk equations, and pediatric-specific sources of utilities/disutilities. Results: Nineteen modeling-based studies (7 de novo, 12 predesigned models) and 34 guidelines were selected. Hemoglobin A1c was the main parameter to model the glycemic control; however, guidelines recommend the usage of complementary measures (eg, time in range) which are not included in economic models. Eight models included diabetic ketoacidosis (42.1%), 16 included hypoglycemia (84.2%), 2 included C-peptide (1 of those as prognostic factor) (10.5%) and 1 included legacy effect (5.3%). Neither guidelines nor models included recent findings, such as age at diagnosis or socioeconomic status, as prognostic factors. The lack of pediatric-specific sources for risk equations and utility/disutility values were additional limitations. Discussion: Economic models designed for assessing interventions in diabetes in pediatric patients should be based on pediatric-specific data and include novel adjuvant glucose-monitoring metrics and latest evidence on prognostic factors (C-peptide, legacy effect, age at diagnosis, socioeconomic status) to provide a more faithful reflection of the disease. Conclusions: Economic models represent useful tools to inform decision making. However, further research assessing the gaps is needed to enhance evidence-based health economic modeling that best represents reality.

Background: Cytomegalovirus prophylaxis in kidney transplant recipients (KTRs) is limited by post-transplant neutropenia and leukopenia (PTN/PTL). Despite its clinical significance, the healthcare resource utilization (HCRU) related to PTN/PTL remains poorly characterized. Objective: To evaluate HCRU among KTRs taking valganciclovir during their first year post-transplant. Methods: Using TriNetX Dataworks-USA, a federated, de-identified electronic medical record database, we identified adult KTRs who underwent their first kidney transplant from January 2012 to September 2020. All eligible patients were followed for 1 year. PTN/PTL was defined as absolute neutrophil count less than 1000/μL or white blood cell count less than 3500/μL. Multivariable logistic/Poisson regression models were used to assess the association between PTN/PTL and various HCRU types. Results: A total of 8791 KTRs were identified, of whom 6219 (70.7%) developed PTN/PTL at a mean of 5.7 months post-transplantation. Hospitalizations, rehospitalizations, emergency room visits, outpatient appointments, packed red blood cell transfusions, and granulocyte-colony stimulating factor administration were more prevalent among KTRs with PTN/PTL (61.1% vs 49.5%, 24.5% vs 14.1%, 35.2% vs 28.9%, 30.4 vs 26.2 visits, 22.3% vs 17.6%, 23.4% vs 2.2%, respectively; P < .001). Adjusted analyses confirmed that PTN/PTL correlated with increased HCRU across all categories. Conclusions: KTRs who developed PTN/PTL had significantly higher HCRU. Further studies are needed to evaluate strategies addressing PTN/PTL for KTRs.

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.