Journal of Health Economics and Outcomes Research最新文献

Background: The second most common hematologic cancer worldwide is multiple myeloma (MM), with incidence and mortality rates that have more than doubled over the past 30 years. The safety and efficacy of daratumumab regimens in the treatment of newly diagnosed MM (NDMM) is demonstrated in clinical trials. Objective: To assess the financial effects of the adoption of subcutaneous daratumumab (dara-SC) rather than intravenous daratumumab (dara-IV) for the treatment of NDMM in three Gulf countries (Qatar, Oman and the United Arab Emirates; UAE), a cost-minimization model was constructed. Methods: We performed static cost minimization analyses from a societal perspective to evaluate the costs and possible reductions in resource utilization associated with a shift from dara-IV infusion to dara-SC injection for NDMM patients over a 5-year time horizon. The model included 2 scenarios: the current scenario in which 100% of patients with NDMM are treated with dara-IV infusion and a future scenario in which dara-SC injection is gradually adopted over the modeled time horizon. The model differentiated precisely between autologous stem cell transplantation (ASCT)-eligible and ASCT-ineligible NDMM patients in terms of their number in each group and the associated therapeutic regimens. One-way sensitivity analyses were also conducted. Results: The model showed that the use of dara-SC in NDMM patients who were eligible or ineligible for ASCT resulted in lower non-drug costs, including premedication drug costs, adverse-effect costs, administration costs, medical staff costs, and indirect costs. The resulting total savings over the 5-year time horizon of the model for Hamad Medical Corporation, Sultan Qaboos University Hospital/Royal Hospital, Sheikh Shakhbout Medical City (SSMC), and Tawam Hospital were QAR -2 522 686, OMR -143 214, AED -30 010 627, and AED -5 003 471, respectively. Conclusion: The introduction of dara-SC as a front-line treatment for NDMM patients in Qatar (Hamad Medical Corporation), Oman (Sultan Qaboos University Hospital, Royal Hospital-MOH), and the UAE (SSMC and Tawam Hospital) can help save resources and minimize constraints on the healthcare system.

Background: Understanding how population density affected the transmission of COVID-19 is vitally important, since crowded cities were the epicenters for the disease. Since human contact was the main cause of the spread, population-weighted densities have been shown to be a better measure than conventional densities, since the variation in density across subareas matters more than the density in the total area. Objectives: This study investigates the impact of population-weighted density and other demographics on the rate of COVID-19 spread in the United States. Methods: The study considers population-weighted density and many other demographics. The population-weighted density index is the weighted average of density across the tracts, where tracts are weighted by population. Multivariate analysis has been used to determine the elasticity of the spread. Results: Using U.S. county-level data, we calculated the elasticity of COVID-19 spread with respect to population-weighted density to be 0.085 after controlling for other factors. In addition to the density, the proportion of people over 65 years of age, the number of total healthcare workers, and average temperature in each county positively contributed to the case numbers, while education level and income per capita had a negative effect. Discussion: For the spread, understanding the population characteristics and dynamics is as important as understanding the infectious disease itself. This will help policy makers to utilize and reallocate the resources more effectively. If the spread is successfully contained early, there will be less stress placed upon the healthcare system, resulting in better healthcare access for those who are sick. Conclusions: Our analysis suggests that population-weighted density can be a useful tool to control and manage outbreaks, especially within the early stage of the spread. We presented the early dynamics of the spread and recommended a policy measure on how to transfer healthcare workers from low-spread-risk areas to high-spread-risk areas to utilize resources better.

Background: Spinal cord stimulation (SCS) is a treatment for chronic intractable pain powered by an implantable pulse generator that may be rechargeable or not rechargeable (NR). It is performed in 2 stages (a trialing phase followed by permanent device implantation) and necessitates 2 hospitalizations, which may increase infection risk. Objective: This analysis explores the cost impact of improvements in battery longevity and the adoption of 1-step (direct-to-implant [DTI]) SCS implantation. Methods: Since 2019, 3 leading NR-SCS devices have been launched: Device A (2019), Device B (2020), and Device C (2021). The battery longevity of the newest Device C was estimated at comparable stimulation settings for Devices A and B. A Markov model simulated individual patient pathways across 2 scenarios: Device A vs Device C and Device B vs Device C (both with the DTI approach and 2-step approach). Costs considered were the initial device implantation procedure, device replacements, and serious adverse event (SAE) management. Italian diagnosis-related group (DRG) tariffs were applied for costs, and a 15-year time horizon was used. Results: Over 15 years, using a DTI approach, the undiscounted total costs for Device A vs Device C were €26 860 and €22 633, respectively, and €25 111 and €22 399 for Device B vs Device C, respectively. Compared with Devices A and B, Device C offered savings of €4227 and €2712, respectively; similar savings were predicted with a 2-step implant approach. Discussion: The battery longevity of NR-SCS devices directly impacts long-term costs to a payer. The longer the device lasts, the lower mean total cumulative costs the patient will have, especially with regard to device replacement costs. With novel devices and specific programming settings, the lifetime cost per patient to a payer can be decreased without compromising the patient's safety and positive clinical outcome. Conclusions: Extended SCS battery longevity can translate into tangible cost savings for payers. The DTI approach for SCS supports National Healthcare System cost efficiencies and offers the additional benefits of optimizing operating room time while having only one recovery period for the patient.

Background: One in 7 US adults has knee osteoarthritis (OA) and almost two-thirds of them suffer from low back pain. OA is the third most rapidly rising condition associated with disability and leads to a significant burden on the healthcare system and society. Objective: This study looked at the healthcare resource utilization (HCRU) in patients with knee OA and low back pain before and after the utilization of a new, home-based, noninvasive, biomechanical intervention. Methods: This was a retrospective claims analysis of 585 patients treated with a personalized, noninvasive, home-based, biomechanical treatment that aims to alleviate knee pain and improve function (AposHealth®). The date of the first AposHealth claim was the index date. Data prior to the index date and post-index date were used to monitor changes in HCRU while in treatment. Descriptive statistics, including frequencies, means and standard deviations, were used to present patient characteristics. To standardize the results, an average monthly claims data rate was calculated and an expected annual rate was extrapolated. Annual HCRU rate per 1000 members was calculated. Results: HCRU decreased after utilizing the new intervention including a decrease of 79% in diagnostic claims, a 70% decrease in outpatient services, a 22% decrease in non-operative treatments, a 61% decrease in pain medications including an 85% drop in opioids use, and a 44% decrease in intra-articular injections. The pre-index estimated rate for total knee replacement (TKR), which is based on existing literature, was 15.1%, whereas the post-index rate of TKR was 0.9%. Conclusions: Patients with knee OA treated with a home-based, noninvasive, biomechanical intervention incurred fewer healthcare resources, leading to an overall reduction in the cost of care.

Background: Hemophilia is a congenital disorder characterized by deficiency or absence of clotting factor VIII in hemophilia A (HA) or clotting factor IX in hemophilia B (HB), resulting in frequent, repeated, and prolonged spontaneous or traumatic bleeding into joints or soft tissue. Severity is classified by the patient's baseline level of clotting factor activity as mild (>5%-40%), moderate (1%-5%), or severe (<1%). In Spain, there is limited information on the societal economic burden of disease. Objective: To estimate the economic and humanistic burden of disease in adult patients with non-inhibitor moderate and severe HA and HB in Spain. Methods: Spanish data from the CHESS II study (2018-2020) on patients' clinical characteristics, health-related quality of life (HRQoL) and hemophilia-related healthcare resource utilization were analyzed. Economic burden was determined by estimating condition-related annual per-patient direct (medical and nonmedical) and indirect costs, stratified according to hemophilia type and severity and presented as 2022 Euros. HRQoL was assessed via the EQ-5D-5L. Results: Of 341 patients in the Spanish CHESS II cohort, 288 patients met the inclusion criteria: 181 had HA (37% [n = 66] moderate and 63% [n=115] severe) and 107 had HB (26% [n = 28] moderate and 74% [n = 79] severe). Mean annual direct cost was higher in HB than in HA, and higher in severe than in moderate patients, resulting in an annual cost/patient of €17 251 (moderate HA), €17 796 (moderate HB), €116 767 (severe HA) and €206 996 (severe HB). The main direct cost component in all groups except moderate HA was factor replacement therapy. Mean per-patient indirect cost was €4089 (moderate HA), €797 (moderate HB), €8633 (severe HA) and €8049 (severe HB). Finally, the mean total cost (direct and indirect) for moderate and severe patients were €91 017 (HA) and €163 924 (HB). EQ-5D-5L [SD] scores were lower in patients with severe HA (0.77 [0.18]) and severe HB (0.70 [0.22]) compared with patients with moderate HA (0.81 [0.15]) and moderate HB (0.86 [0.17]). Conclusions: Independently of the type of hemophilia, greater condition severity was associated with increased costs and a decrease in HRQoL.

Background: The economic burden associated with type 2 diabetes mellitus (T2DM) and concurrent cardiovascular disease (CVD) among patients with COVID-19 is unclear. Objective: We compared healthcare resource utilization (HCRU) and costs in patients with COVID-19 and T2DM and CVD (T2DM + CVD), T2DM only, or neither T2DM nor CVD (T2DM/CVD). Methods: A retrospective observational study in COVID-19 patients using data from the Healthcare Integrated Research Database (HIRD®) was conducted. Patients with COVID-19 were identified between March 1, 2020, and May 31, 2021, and followed from first diagnosis or positive lab test to the end of health plan enrollment, end of study period, or death. Patients were assigned one of 3 cohorts: pre-existing T2DM+CVD, T2DM only, or neither T2DM/CVD. Propensity score matching and multivariable analyses were performed to control for differences in baseline characteristics. Study outcomes included all-cause and COVID-19-related HCRU and costs. Results: In all, 321 232 COVID-19 patients were identified (21 651 with T2DM + CVD, 28 184 with T2DM only, and 271 397 with neither T2DM/CVD). After matching, 6967 patients were in each group. Before matching, 46.0% of patients in the T2DM + CVD cohort were hospitalized for any cause, compared with 18.0% in the T2DM-only cohort and 6.3% in the neither T2DM/CVD cohort; the corresponding values after matching were 34.2%, 26.0%, and 21.2%. The proportion of patients with emergency department visits, telehealth visits, or use of skilled nursing facilities was higher in patients with COVID-19 and T2DM + CVD compared with the other cohorts. Average all-cause costs during follow-up were $12 324,$7882, and $7277 per-patient-per-month after matching for patients with T2DM + CVD, T2DM-only, and neither T2DM/CVD, respectively. COVID-19-related costs contributed to 78%, 75%, and 64% of the overall costs, respectively. The multivariable model showed that per-patient-per-month all-cause costs for T2DM + CVD and T2DM-only were 54% and 21% higher, respectively, than those with neither T2DM/CVD after adjusting for residual confounding. Conclusion: HCRU and costs in patients were incrementally higher with COVID-19 and pre-existing T2DM + CVD compared with those with T2DM-only and neither T2DM/CVD, even after accounting for baseline differences between groups, confirming that pre-existing T2DM + CVD is associated with increased HCRU and costs in COVID-19 patients, highlighting the importance of proactive management.

Background: The association of neovascular age-related macular degeneration (nAMD), diabetic macular edema (DME), and retinal vein occlusion (RVO) with functional status in the general Medicare population are not well established. Objectives: This study examined patient-reported survey data linked with Medicare claims to describe the burden of these vision-threatening retinal diseases (VTRDs) among Medicare beneficiaries. Methods: Medicare Current Beneficiary Survey data linked with Medicare Fee-for-Service claims data from 2006 to 2018 were used in a nationally representative retrospective pooled cross-sectional population-based comparison study. Outcomes between community-dwelling beneficiaries with nAMD (n = 1228), DME (n = 101), or RVO (n = 251) were compared with community-dwelling beneficiaries without any VTRDs (n = 104 088), controlling for baseline demographic and clinical differences. Beneficiaries with a diagnosis of nAMD, DME, or RVO during the data year were included; those with other VTRDs were excluded. Outcomes included vision function and loss, overall functioning as assessed by difficulties with activities of daily living (ADLs) and instrumental ADLs (iADLs), anxiety/depression, falls, and fractures. Results: In patient cohorts with nAMD, DME, and RVO, approximately one-third (34.2%-38.3%) reported "a little trouble seeing" (vs 28.3% for controls), and 26%, 17%, and 9%, respectively, reported "a lot of trouble seeing/blindness" (vs 5% of controls). Difficulty walking and doing heavy housework were the most reported ADLs and iADLs, respectively. Compared with those without VTRDs, beneficiaries with nAMD had higher odds of diagnosed vision loss (odds ratio [OR], 5.39; 95% confidence interval, 4.06-7.16; P < .001) and difficulties with iADLs (odds ratio, 1.41; 95% confidence interval, 1.11-1.80; P = .005); no differences were observed for DME or RVO vs control. After adjusting for age, sex, race/ethnicity, poverty status, comorbidities, and other relevant covariates, nAMD, DME, and RVO were not significantly associated with anxiety/depression, falls, or fractures. Discussion: Patients with nAMD or DME were more likely to report severe visual impairment than those without VTRDs, although only those with nAMD were more likely to be diagnosed with vision loss. Conclusions: Patients with nAMD continue to experience more vision impairment and worse functional status compared with a similar population of Medicare beneficiaries despite availability of therapies like antivascular endothelial growth factor to treat retinal disease.

Background: Medication formularies, initially designed to promote the use of cost-effective generic drugs, are now designed to maximize financial benefits for the pharmacy benefit management companies that negotiate purchase prices. In the second-largest pharmacy benefit management formulary that is publicly available, 55% of mandated substitutions are not for generic or biosimilar versions of the same active ingredient and/or formulation and may not be medically or financially beneficial to patients. Methods: We modeled the effect of excluding novel agents for atrial fibrillation/venous thromboembolism, migraine prevention, and psoriasis, which all would require substitution with a different active ingredient. Using population data, market share of the 2 largest US formularies, and 2021 prescription data, we calculated how many people could be affected by such exclusions. Using data from the published literature, we calculated how many of those individuals are likely to discontinue treatment and/or have adverse events due to a formulary exclusion. Results: The number of people likely to have adverse events due to the exclusion could be as high as 1 million for atrial fibrillation/venous thromboembolism, 900 000 for migraine prevention, and 500 000 for psoriasis. The numbers likely to discontinue treatment for their condition are as high as 924 000 for atrial fibrillation/venous thromboembolism, 646 000 for migraine, and 138 000 for psoriasis. Conclusion: Substitution with a nonequivalent treatment is common in formularies currently in use and is not without substantial consequences for hundreds of thousands of patients. Forced medication substitution results in costly increases in morbidity and mortality and should be part of the cost-benefit analysis of any formulary exclusion.

Background: Atherosclerotic cardiovascular disease (ASCVD) remains the leading cause of mortality and disability in the United States and worldwide. Objective: To assess the multimorbidity burden and its associations with adverse cardiovascular events (ACE) and healthcare costs among patients with ASCVD. Methods: This is a retrospective observational cohort study using Aetna claims database. Patients with ASCVD were identified during the study period (1/1/2018-10/31/2021). The earliest ASCVD diagnosis date was identified as the index date. Qualified patients were ≥18 years of age and had ≥12 months of health plan enrollment before and after the index date. Comorbid conditions were assessed using all data available within 12 months prior to and including the index date. Association rule mining was applied to identify comorbid condition combinations. ACEs and healthcare costs were assessed using all data within 12 months after the index date. Multivariable generalized linear models were performed to examine the associations between multimorbidity and ACEs and healthcare costs. Results: Of 223 923 patients with ASCVD (mean [SD] age, 73.6 [10.7] years; 42.2% female), 98.5% had ≥2, and 80.2% had ≥5 comorbid conditions. The most common comorbid condition dyad was hypertension-hyperlipidemia (78.7%). The most common triad was hypertension-hyperlipidemia-pain disorders (61.1%). The most common quartet was hypertension-hyperlipidemia-pain disorders-diabetes (30.2%). The most common quintet was hypertension-hyperlipidemia-pain disorders-diabetes-obesity (16%). The most common sextet was hypertension-hyperlipidemia-pain disorders-diabetes-obesity-osteoarthritis (7.6%). The mean [SD] number of comorbid conditions was 7.1 [3.2]. The multimorbidity burden tended to increase in older age groups and was comparatively higher in females and in those with higher social vulnerability. The increased number of comorbid conditions was significantly associated with increased ACEs and increased healthcare costs. Discussion: Extremely prevalent multimorbidity should be considered in the context of clinical decision-making to optimize secondary prevention of ASCVD. Conclusions: Multimorbidity was extremely prevalent among patients with ASCVD. Multimorbidity patterns varied considerably across ASCVD patients and by age, gender, and social vulnerability status. Multimorbidity was strongly associated with ACEs and healthcare costs.

Objectives: Regulatory bodies, health technology assessment agencies, payers, physicians, and other decision-makers increasingly recognize the importance of real-world evidence (RWE) to provide important and relevant insights on treatment patterns, burden/cost of illness, product safety, and long-term and comparative effectiveness. However, RWE generation requires a careful approach to ensure rigorous analysis and interpretation. There are limited examples of comprehensive methodology for the generation of RWE on patients who have undergone neuromodulation for drug-resistant epilepsy (DRE). This is likely due, at least in part, to the many challenges inherent in using real-world data to define DRE, neuromodulation (including type implanted), and related outcomes of interest. We sought to provide recommendations to enable generation of robust RWE that can increase knowledge of "real-world" patients with DRE and help inform the difficult decisions regarding treatment choices and reimbursement for this particularly vulnerable population. Methods: We drew upon our collective decades of experience in RWE generation and relevant disciplines (epidemiology, health economics, and biostatistics) to describe challenges inherent to this therapeutic area and to provide potential solutions thereto within healthcare claims databases. Several examples were provided from our experiences in DRE to further illustrate our recommendations for generation of robust RWE in this therapeutic area. Results: Our recommendations focus on considerations for the selection of an appropriate data source, development of a study timeline, exposure allotment (specifically, neuromodulation implantation for patients with DRE), and ascertainment of relevant outcomes. Conclusions: The need for RWE to inform healthcare decisions has never been greater and continues to grow in importance to regulators, payers, physicians, and other key stakeholders. However, as real-world data sources used to generate RWE are typically generated for reasons other than research, rigorous methodology is required to minimize bias and fully unlock their value.