Journal of Health Economics and Outcomes Research最新文献

Background: Glycogen storage disease type Ia (GSDIa) is a rare inherited disorder that can lead to renal and hepatic complications, brain damage, and death. There is a lack of qualitative research describing the impact of GSDIa on individuals and their caregivers. Objective: This study was designed to describe the individual experiences of those affected by GSDIa through qualitative methods. Methods: Individuals with GSDIa and caregivers were recruited through two patient associations (US, UK). Participants were asked to write narratives about their experiences with GSDIa in their own words, with no set questions or topics. The resulting narrative accounts were analyzed thematically using a primarily inductive approach. Themes describing the impact of GSDIa on individuals and caregivers were identified. Results: Eight caregivers, three of whom had lost a child to GSDIa, and 11 individuals living with GSDIa provided written summaries of their experiences of life with the condition. Participants described symptoms, the burden of managing frequent cornstarch feeds, and the broader impacts of GSDIa. These included effects on daily activities, family, emotional and social functioning, romantic relationships, and educational/professional impacts. Participants also described challenges related to obtaining diagnosis and support, and the lack of GSDIa knowledge from healthcare professionals. Discussion: This study provided novel insights into the impact of GSDIa on individuals living with the condition and their caregivers. Participants reported a wide range of issues in their narratives and openly shared sensitive information about their experiences, which may not have been captured if they had taken part in a semi-structured interview format. Conclusion: The narrative method yielded a rich data set that provided details of the varied and individual experiences of GSDIa not captured by traditional quantitative methods or structured interviews. The narratives highlighted the unmet needs of both caregivers of and patients with GSDIa around cornstarch management, obstacles to proper treatment from healthcare professionals, and the absence of effective treatments. The open-ended narrative approach had advantages over standard qualitative methods to capture greater insight into individual experiences directly from participants in their own words.

Background: The disease burden of heart failure is mainly driven by high hospital readmission rates. Remote monitoring devices can be used to assess the status of patients after discharge and identify early signs of worsening symptoms. Initial studies indicated that Heart Failure Management System (HFMS), a novel monitoring device, can prevent hospital readmission. Objective: To determine the cost effectiveness of HFMS compared with standard of care (SOC) in the United States. Methods: A Markov model was developed to follow patients after their discharge from index hospitalization for heart failure. The costs and outcomes were estimated for 5 years. The patient cohort was initially in "outpatient care," where they are at risk of an emergency room visit or hospital readmission. If hospitalized, patients returned to a second outpatient care health state. An "escalation of care" (eg, surgical intervention) may have removed patients from the intervention. The model took the payer perspective with costs in 2022 US dollars. The incremental cost-effectiveness ratio measured effectiveness through hospital readmissions. The willingness-to-pay threshold was set to the published cost of a heart failure rehospitalization ($10 737). Sensitivity and scenario analyses explored the robustness of the model to changes in inputs. Results: Compared with SOC, HFMS reduced the mean cost of care by $6723($ 155 122 vs $161 846) over the 5-year period. The mean number of hospital readmissions was reduced to 1.075 with HFMS from 1.201 with SOC (-0.126 events). The incremental cost-effectiveness ratio showed that HFMS was a dominant strategy compared with SOC, leading to reduced costs and hospital readmissions in 93.4% of the 1000 Monte Carlo simulations; 94.1% of the simulations fell below the willingness-topay threshold. Savings with HFMS emerged from the third month. Discussion: The results indicated the cost-effectiveness of HFMS compared with SOC. The sensitivity analyses supported this finding. Reducing costly hospital readmissions may help to alleviate the burden of heart failure. Longer-term data on HFMS are encouraged to confirm or contest the model outcomes. Conclusions: The use of HFMS is expected to save costs and reduce hospitalizations over a 5-year period compared with the current SOC.

Background: The 2022 US Supreme Court decision in Dobbs v. Jackson Women's Health Organization eliminated the constitutional right to abortion and activated trigger laws in 21 states, either banning or significantly restricting abortion access. This study estimated changes in postpartum depression (PPD) diagnoses after Dobbs in states with trigger laws vs those without. Methods: Medicaid data from Kythera Labs spanning December 2019 to June 2024 were utilized. Difference-in-difference models assessed changes in PPD diagnosis rates post-Dobbs (21 trigger states, 29 non-trigger states). Results: Women in trigger states were younger (mean, 26.53 vs 27.98 years), more likely to reside in low socioeconomic status areas (41.28% vs 24.42%) and less likely to have obstetrical complications (66.06% vs 77.36%), maternal complications (16.41% vs 18.9%), and lifestyle risk factors (13.58% vs 21.17%). Baseline PPD diagnosis rates were 8.51% in trigger states and 12.66% in non-trigger states. Post-Dobbs, PPD diagnosis rates were 10.20% in trigger states and 14.34% in non-trigger states. Conclusions: Overall, women in states with abortion trigger laws experienced a small positive but statistically insignificant increase in PPD diagnoses following Dobbs compared with those in non-trigger states.

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.