A roadmap for affordable genetic medicines.

Twenty genetic therapies have been approved by the US Food and Drug Administration to date, a number that now includes the first CRISPR genome-editing therapy for sickle cell disease-CASGEVY (exagamglogene autotemcel, Vertex Pharmaceuticals). This extraordinary milestone is widely celebrated owing to the promise for future genome-editing treatments of previously intractable genetic disorders and cancers. At the same time, such genetic therapies are the most expensive drugs on the market, with list prices exceeding US$4 million per patient. Although all approved cell and gene therapies trace their origins to academic or government research institutions, reliance on for-profit pharmaceutical companies for subsequent development and commercialization results in prices that prioritize recouping investments, paying for candidate product failures and meeting investor and shareholder expectations. To increase affordability and access, sustainable discovery-to-market alternatives are needed that address system-wide deficiencies. Here we present recommendations of a multidisciplinary task force assembled to chart such a path. We describe a pricing structure that, once implemented, could reduce per-patient cost tenfold and propose a business model that distributes responsibilities while leveraging diverse funding sources. We also outline how academic licensing provisions, manufacturing innovation and supportive regulations can reduce cost and enable broader patient treatment.

Forecasting the Economic Burden of Cardiovascular Disease and Stroke in the United States Through 2050: A Presidential Advisory From the American Heart Association.

BACKGROUND: Quantifying the economic burden of cardiovascular disease and stroke over the coming decades may inform policy, health system, and community-level interventions for prevention and treatment. METHODS: We used nationally representative health, economic, and demographic data to project health care costs attributable to key cardiovascular risk factors (hypertension, diabetes, hypercholesterolemia) and conditions (coronary heart disease, stroke, heart failure, atrial fibrillation) through 2050. The human capital approach was used to estimate productivity losses from morbidity and premature mortality due to cardiovascular conditions. RESULTS: One in 3 US adults received care for a cardiovascular risk factor or condition in 2020. Annual inflation-adjusted (2022 US dollars) health care costs of cardiovascular risk factors are projected to triple between 2020 and 2050, from $400 billion to $1344 billion. For cardiovascular conditions, annual health care costs are projected to almost quadruple, from $393 billion to $1490 billion, and productivity losses are projected to increase by 54%, from $234 billion to $361 billion. Stroke is projected to account for the largest absolute increase in costs. Large relative increases among the Asian American population (497%) and Hispanic American population (489%) reflect the projected increases in the size of these populations. CONCLUSIONS: The economic burden of cardiovascular risk factors and overt cardiovascular disease in the United States is projected to increase substantially in the coming decades. Development and deployment of cost-effective programs and policies to promote cardiovascular health are urgently needed to rein in costs and to equitably enhance population health.

Trends in direct health care costs among US adults with atherosclerotic cardiovascular disease with and without diabetes.

OBJECTIVE: Population-based national data on the trends in expenditures related to coexisting atherosclerotic cardiovascular diseases (ASCVD) and diabetes is scarce. We assessed the trends in direct health care expenditures for ASCVD among individuals with and without diabetes, which can help to better define the burden of the co-occurrence of diabetes and ASCVD. METHODS: We used 12-year data (2008-2019) from the US national Medical Expenditure Panel Survey including 28,144 U.S individuals aged ≥ 18 years. Using a two-part model (adjusting for demographics, comorbidities and time), we estimated mean and adjusted incremental medical expenditures by diabetes status among individuals with ASCVD. The costs were direct total health care expenditures (out-of-pocket payments and payments by private insurance, Medicaid, Medicare, and other sources) from various sources (office-based visits, hospital outpatient, emergency room, inpatient hospital, pharmacy, home health care, and other medical expenditures). RESULTS: The total direct expenditures for individuals with ASCVD increased continuously by 30% from $14,713 (95% confidence interval (CI): $13,808-$15,619) in 2008-2009 to $19,145 (95% CI: $17,988-$20,301) in 2008-2019. Individuals with diabetes had a 1.5-fold higher mean expenditure that those without diabetes. A key driver of the observed increase in direct costs was prescription drug costs, which increased by 37% among all individuals with ASCVD. The increase in prescription drug costs was more pronounced among individuals with ASCVD and diabetes, in whom a 45% increase in costs was observed, from $5184 (95% CI: $4721-$5646) in 2008-2009 to $7501 (95% CI: $6678-$8325) in 2018-2019. Individuals with ASCVD and diabetes had $5563 (95% CI: $4643-$6483) higher direct incremental expenditures compared with those without diabetes, after adjusting for demographics and comorbidities. Among US adults with ASCVD, the estimated adjusted total direct excess medical expenditures were $42 billion per year among those with diabetes vs. those without diabetes. CONCLUSIONS: In the setting of ASCVD, diabetes is associated with significantly increased health care costs, an increase that was driven by marked increase in medication costs.

Current and Future Cost Burden of Ischemic Stroke in Australia: Dynamic Model.

BACKGROUND: Stroke remains one of the leading causes of morbidity and mortality in Australia. The objective of this study was to estimate the current and future cost burden of ischemic stroke (IS) in Australia. METHOD: First, the annual chronic management cost per person following IS were derived for all people aged ≥30 years discharged from a public or private hospital in Victoria, Australia between July 2012 and June 2017 (with follow-up data until June 2018 [n = 34,471]). Then extrapolated the data from from Victoria to the whole Australian population aged between 30 years and 99 years to project the total healthcare costs following IS (combination of acute event and chronic management cost) over a 20-year period (2019-2038) using a dynamic multistate life table model. Data for the dynamic model were sourced from the Victorian Admitted Episodes Dataset (VAED) and supplemented with other published data. RESULT: The estimated annual total chronic management cost following IS was 13,525 Australian dollars (AUD) per person (95% CI: AUD 13,380, AUD 13,670) for cohorts in the VAED between July 2012 and June 2017. The annual chronic management cost was estimated to decline following IS. The highest cost was incurred in the first year of follow-up post-IS (AUD 14,309 per person) and declined to AUD 9,776 in the sixth year of follow-up post-IS. The total healthcare cost for people aged 30-99 years was projected to be AUD 47.7 billion (95% UI: AUD 44.6 billion, AUD 51.0 billion) over the 20-year period (2019-2038) Australia-wide, of which 91.3% (AUD 43.6 billion) was attributed to chronic management costs and the remaining 8.7% (AUD 4.2 billion) were due to acute IS events. CONCLUSION: IS has and will continue to have a considerable financial impact in the next 2 decades on the Australian healthcare system. Our estimated and projected cost burden following IS provides important information for decision making in relation to IS.

Trends in inpatient burden from pressure injuries in the United States: Cross-sectional study National Inpatient Sample 2009-2019.

Pressure injuries are a significant comorbidity and lead to increased overall healthcare costs. Several European and global studies have assessed the burden of pressure injuries; however, no comprehensive analysis has been completed in the United States. In this study, we investigated the trends in the burden of pressure injuries among hospitalised adults in the United States from 2009 to 2019, stratified by sociodemographic subgroups. The length of admission, total cost of hospitalisation, and sociodemographic data was extracted from the National Inpatient Sample provided by the Healthcare Cost and Utilisation Project, Agency for Healthcare Research and Quality. Overall, the annual prevalence of pressure injuries and annual mean hospitalisation cost increased ($69,499.29 to $102,939.14), while annual mean length of stay decreased (11.14-9.90 days). Among all races, minority groups had higher average cost and length of hospitalisation. Our findings suggest that while the length of hospitalisation is decreasing, hospital costs and prevalence are rising. In addition, differing trends among racial groups exist with decreasing prevalence in White patients. Further studies and targeted interventions are needed to address these differences, as well as discrepancies in racial groups.

High-cost users after sepsis: a population-based observational cohort study.

BACKGROUND: High-cost users (HCU) represent important targets for health policy interventions. Sepsis is a life-threatening syndrome that is associated with high morbidity, mortality, and economic costs to the healthcare system. We sought to estimate the effect of sepsis on being a subsequent HCU. METHODS: Using linked health-administrative databases, we conducted a population-based, propensity score-weighted cohort study of adults who survived a hospitalization in Ontario, Canada between January 2016 and December 2017. Sepsis was identified using a validated algorithm. The primary outcome was being a persistent HCU after hospital discharge (in the top 5% or 1% of total health care spending for 90 consecutive days), and the proportion of follow-up time since discharge as a HCU. RESULTS: We identified 927,057 hospitalized individuals, of whom 79,065 had sepsis. Individuals who had sepsis were more likely to be a top 5% HCU for 90 consecutive days at any time after discharge compared to those without sepsis (OR 2.24; 95% confidence interval [CI] 2.04-2.46) and spent on average 42.3% of their follow up time as a top 5% HCU compared to 28.9% of time among those without sepsis (RR 1.46; 95% CI 1.45-1.48). Individuals with sepsis were more likely to be a top 1% HCU for 90 consecutive days compared to those without sepsis (10% versus 5.1%, OR 2.05 [95% CI 1.99-2.11]), and spent more time as a top 1% HCU (18.5% of time versus 10.8% of time, RR 1.68 [95% CI 1.65-1.70]). CONCLUSIONS: The sequelae of sepsis result in higher healthcare costs with important economic implications. After discharge, individuals who experienced sepsis are more likely to be a HCU and spend more time as a HCU compared to individuals who did not experience sepsis during hospitalization.

Medical costs of Swedish nursing home residents at the end of life: a retrospective observational registry study.

BACKGROUND: There are many studies of medical costs in late life in general, but nursing home residents' needs and the costs of external medical services and interventions outside of nursing home services are less well described. METHODS: We examined the direct medical costs of nursing home residents in their last year of life, as well as limited to the period of stay in the nursing home, adjusted for age, sex, Hospital Frailty Risk Score (HFRS), and diagnosis of dementia or advanced cancer. This was an observational retrospective study of registry data from all diseased nursing home residents during the years 2015-2021 using healthcare consumption data from the Stockholm Regional Council, Sweden. T tests, Wilcoxon rank sum tests and chi-square tests were used for comparisons of groups, and generalized linear models (GLMs) were constructed for univariable and multivariable linear regressions of health cost expenditures to calculate risk ratios (RRs) with 95% confidence intervals (95% CIs). RESULTS: According to the adjusted (multivariable) models for the 38,805 studied nursing home decedents, when studying the actual period of stay in nursing homes, we found significantly greater medical costs associated with male sex (RR 1.29 (1.25-1.33), p < 0.0001) and younger age (65-79 years vs. ≥90 years: RR 1.92 (1.85-2.01), p < 0.0001). Costs were also greater for those at risk of frailty according to the Hospital Frailty Risk Score (HFRS) (intermediate risk: RR 3.63 (3.52-3.75), p < 0.0001; high risk: RR 7.84 (7.53-8.16), p < 0.0001); or with advanced cancer (RR 2.41 (2.26-2.57), p < 0.0001), while dementia was associated with lower medical costs (RR 0.54 (0.52-0.55), p < 0.0001). The figures were similar when calculating the costs for the entire last year of life (regardless of whether they were nursing home residents throughout the year). CONCLUSIONS: Despite any obvious explanatory factors, male and younger residents had higher medical costs at the end of life than women. Having a risk of frailty or a diagnosis of advanced cancer was strongly associated with higher costs, whereas a dementia diagnosis was associated with lower external, medical costs. These findings could lead us to consider reimbursement models that could be differentiated based on the observed differences.

Trends in heart failure costs for commercially insured patients in the United States (2006-2021).

BACKGROUND: Although prior research has estimated the overarching cost burden of heart failure (HF), a thorough analysis examining medical expense differences and trends, specifically among commercially insured patients with heart failure, is still lacking. Thus, the study aims to examine historical trends and differences in medical costs for commercially insured heart failure patients in the United States from 2006 to 2021. METHODS: A population-based, cross-sectional analysis of medical and pharmacy claims data (IQVIA PharMetrics® Plus for Academic) from 2006 to 2021 was conducted. The cohort included adult patients (age > = 18) who were enrolled in commercial insurance plans and had healthcare encounters with a primary diagnosis of HF. The primary outcome measures were the average total annual payment per patient and per cost categories encompassing hospitalization, surgery, emergency department (ED) visits, outpatient care, post-discharge care, and medications. The sub-group measures included systolic, diastolic, and systolic combined with diastolic, age, gender, comorbidity, regions, states, insurance payment, and self-payment. RESULTS: The study included 422,289 commercially insured heart failure (HF) patients in the U.S. evaluated from 2006 to 2021. The average total annual cost per patient decreased overall from $9,636.99 to $8,201.89, with an average annual percentage change (AAPC) of -1.11% (95% CI: -2% to -0.26%). Hospitalization and medication costs decreased with an AAPC of -1.99% (95% CI: -3.25% to -0.8%) and - 3.1% (95% CI: -6.86-0.69%). On the other hand, post-discharge, outpatient, ED visit, and surgery costs increased by an AAPC of 0.84% (95% CI: 0.12-1.49%), 4.31% (95% CI: 1.03-7.63%), 7.21% (95% CI: 6.44-8.12%), and 9.36% (95% CI: 8.61-10.19%). CONCLUSIONS: The study's findings reveal a rising trend in average total annual payments per patient from 2006 to 2015, followed by a subsequent decrease from 2016 to 2021. This decrease was attributed to the decline in average patient costs within the Medicare Cost insurance category after 2016, coinciding with the implementation of the Medicare Access and CHIP Reauthorization Act (MACRA) of 2015. Additionally, expenses related to surgical procedures, emergency department (ED) visits, and outpatient care have shown substantial growth over time. Moreover, significant differences across various variables have been identified.

Is It Getting More Expensive to Treat Patients Who Have a High Comorbidity Burden? Financial Trends in Total Knee Arthroplasty From 2013 to 2021.

BACKGROUND: Utilization of total knee arthroplasty (TKA) continues to rise among patients who have a high comorbidity burden (HCB). With changes in reimbursement models over the past decade, it is essential to assess the financial impact of HCB TKA on healthcare systems. This study aimed to examine trends in revenue and costs associated with TKA in HCB patients over time. METHODS: Of 14,978 TKA performed at a large, urban academic medical center between 2013 and 2021, we retrospectively analyzed HCB patients (Charlson comorbidity index ≥ 5 and American Society of Anesthesiology scores of 3 or 4). A total of 1,156 HCB TKA patients who had complete financial data were identified. Patient demographics, perioperative data, revenue, costs, and contribution margin were collected for each patient. Changes in these financial values over time, as a percentage of 2013 values, were analyzed. Linear regression was performed with a trend analysis to determine significance. RESULTS: From 2013 to 2021, the percentage of HCB TKAs per year increased from 4.2% in 2013 to 16.5% in 2021 (P < .001). The revenue of TKA in HCB patients remained steady (P = .093), while direct costs increased significantly (32.0%; P = .015), resulting in a decline of contribution margin to a low of 82.3% of 2013 margins. There was no significant change in rates of 90-day complications or home discharge following HCB TKA during the study period. CONCLUSIONS: The results of this study indicate a major rise in cost for TKA among HCB patients, without a corresponding rise in revenue. As more patients who have HCB become candidates for TKA, the negative financial impact on institutions should be considered, as payments to institutions do not adequately reflect patient complexity. A re-evaluation of institutional payments for medically complex TKA patients is warranted to maintain patient access among at-need populations.

Trends in Revenue, Cost, and Contribution Margin of Patients Who Have a High Comorbidity Burden Undergoing Total Hip Arthroplasty From 2013 to 2021.

BACKGROUND: With the increasing utilization of total hip arthroplasty (THA) in patients who have a high comorbidity burden (HCB), coinciding with modifications to reimbursement models over the past decade, an evaluation of the financial impact of HCB on THA over time is warranted. This study aimed to investigate trends in revenue and cost associated with THA in HCB patients. METHODS: Of 13,439 patients who had primary, elective THA between 2013 and 2021 at our institution, we retrospectively reviewed 978 patients considered to have HCB (Charlson comorbidity index ≥ 5 and American Society of Anesthesiology scores 3 or 4). We collected patient demographics, perioperative data, revenue, cost, and contribution margin (CM) of the inpatient episode. We analyzed changes as a percentage of 2013 values over time for these financial markers. Linear regression determined trend significance. The final analysis included 978 HCB patients who had complete financial data. RESULTS: Between 2013 and 2021, direct costs increased significantly (P = .002), along with a nonsignificant increase in total costs (P = .056). While revenue remained steady during the study period (P = .486), the CM decreased markedly to 38.0% of 2013 values, although not statistically significant (P = .222). Rates of 90-day complications and home discharge remained steady throughout the study period. CONCLUSIONS: Increasing costs for HCB patients undergoing THA were not matched by an equivalent increase in revenue, leading to dwindling CMs throughout the past decade. Re-evaluation of reimbursement models for THA that account for patients' HCB may be necessary to preserve broad access to care. LEVEL OF EVIDENCE: III.

Global costs, health benefits, and economic benefits of scaling up treatment and imaging modalities for survival of 11 cancers: a simulation-based analysis.

BACKGROUND: In addition to increased availability of treatment modalities, advanced imaging modalities are increasingly recommended to improve global cancer care. However, estimates of the costs and benefits of investments to improve cancer survival are scarce, especially for low-income and middle-income countries (LMICs). In this analysis, we aimed to estimate the costs and lifetime health and economic benefits of scaling up imaging and treatment modality packages on cancer survival, both globally and by country income group. METHODS: Using a previously developed model of global cancer survival, we estimated stage-specific cancer survival and life-years gained (accounting for competing mortality) in 200 countries and territories for patients diagnosed with one of 11 cancers (oesophagus, stomach, colon, rectum, anus, liver, pancreas, lung, breast, cervix uteri, and prostate) representing 60% of all cancer diagnoses between 2020 and 2030 (inclusive of full years). We evaluated the costs and health and economic benefits of scaling up packages of treatment (chemotherapy, surgery, radiotherapy, and targeted therapy), imaging modalities (ultrasound, x-ray, CT, MRI, PET, single-photon emission CT), and quality of care to the mean level of high-income countries, separately and in combination, compared with no scale-up. Costs and benefits are presented in 2018 US$ and discounted at 3% annually. FINDINGS: For the 11 cancers studied, we estimated that without scale-up (ie, with current availability of treatment, imaging, and quality of care) there will be 76·0 million cancer deaths (95% UI 73·9-78·6) globally for patients diagnosed between 2020 and 2030, with more than 70% of these deaths occurring in LMICs. Comprehensive scale-up of treatment, imaging, and quality of care could avert 12·5% (95% UI 9·0-16·3) of these deaths globally, ranging from 2·8% (1·8-4·3) in high-income countries to 38·2% (32·6-44·5) in low-income countries. Globally, we estimate that comprehensive scale-up would cost an additional $232·9 billion (95% UI 85·9-422·0) between 2020 and 2030 (representing a 6·9% increase in cancer treatment costs), but produce $2·9 trillion (1·8-4·0) in lifetime economic benefits, yielding a return of $12·43 (6·47-33·23) per dollar invested. Scaling up treatment and quality of care without imaging would yield a return of $6·15 (2·66-16·71) per dollar invested and avert 7·0% (3·9-10·3) of cancer deaths worldwide. INTERPRETATION: Simultaneous investment in cancer treatment, imaging, and quality of care could yield substantial health and economic benefits, especially in LMICs. These results provide a compelling rationale for the value of investing in the global scale-up of cancer care. FUNDING: Harvard TH Chan School of Public Health and National Cancer Institute.

Access to Mechanical Thrombectomy for Ischemic Stroke in the United States.

Background and Purpose: Mechanical thrombectomy helps prevent disability in patients with acute ischemic stroke involving occlusion of a large cerebral vessel. Thrombectomy requires procedural expertise and not all hospitals have the staff to perform this intervention. Few population-wide data exist regarding access to mechanical thrombectomy. Methods: We examined access to thrombectomy for ischemic stroke using discharge data from calendar years 2016 to 2018 from all nonfederal emergency departments and acute care hospitals across 11 US states encompassing 80 million residents. Facilities were classified as hubs if they performed mechanical thrombectomy, gateways if they transferred patients who ultimately underwent mechanical thrombectomy, and gaps otherwise. We used standard descriptive statistics and unadjusted logistic regression models in our primary analyses. Results: Among 205 681 patients with ischemic stroke, 100 139 (48.7% [95% CI, 48.5%­48.9%]) initially received care at a thrombectomy hub, 72 534 (35.3% [95% CI, 35.1%­35.5%]) at a thrombectomy gateway, and 33 008 (16.0% [95% CI, 15.9%­16.2%]) at a thrombectomy gap. Patients who initially received care at thrombectomy gateways were substantially less likely to ultimately undergo thrombectomy than patients who initially received care at thrombectomy hubs (odds ratio, 0.27 [95% CI, 0.25­0.28]). Rural patients had particularly limited access: 27.7% (95% CI, 26.9%­28.6%) of such patients initially received care at hubs versus 69.5% (95% CI, 69.1%­69.9%) of urban patients. For 93.8% (95% CI, 93.6%­94.0%) of patients with stroke at gateways, their initial facility was capable of delivering intravenous thrombolysis, compared with 76.3% (95% CI, 75.8%­76.7%) of patients at gaps. Our findings were unchanged in models adjusted for demographics and comorbidities and persisted across multiple sensitivity analyses, including analyses adjusting for estimated stroke severity. Conclusions: We found that a substantial proportion of patients with ischemic stroke across the United States lacked access to thrombectomy even after accounting for interhospital transfers. US systems of stroke care require further development to optimize thrombectomy access.

Ending AIDS as a public health threat by 2030: Time to reset targets for 2025.

Paul De Lay and co-authors introduce a Collection on the design of targets for ending the AIDS epidemic.

Out-of-pocket and private pay in clinical genetic testing: A scoping review.

Full coverage of the cost of clinical genetic testing is not always available through public or private insurance programs, or a public healthcare system. Consequently, some patients may be faced with the decision of whether to finance testing out-of-pocket (OOP), meet OOP expenses required by their insurer, or not proceed with testing. A scoping review was conducted to identify literature associated with patient OOP and private pay in clinical genetic testing. Seven databases (EMBASE, MEDLINE, CINAHL, PsychINFO, PAIS, the Cochrane Database of Systematic Reviews, and the JBI Evidence-Based Practice database) were searched, resulting in 83 unique publications included in the review. The presented evidence includes a descriptive analysis, followed by a narrative account of the extracted data. Results were divided into four groups according to clinical indication: (1) hereditary breast and ovarian cancer, (2) other hereditary cancers, (3) prenatal testing, (4) other clinical indications. The majority of studies focused on hereditary cancer and prenatal genetic testing. Overall trends indicated that OOP costs have fallen and payer coverage has improved, but OOP expenses continue to present a barrier to patients who do not qualify for full coverage.

Predicting Unplanned Health Care Utilization and Cost: Comparing Patient-reported Outcomes Measurement Information System and Claims.

OBJECTIVES: There is little literature describing if and how payers are utilizing patient-reported outcomes to predict future costs. This study assessed if Patient-reported Outcomes Measurement Information System (PROMIS) domain scores, collected in routine practice at neurology clinics, improved payer predictive models for unplanned care utilization and cost. STUDY DESIGN: Retrospective cohort analysis of private Health Plan-insured patients with visits at 18 Health Plan-affiliated neurology clinics. METHODS: PROMIS domains (Anxiety v1.0, Cognitive Function Abilities v2.0, Depression v1.0, Fatigue v1.0, Pain Interference v1.0, Physical Function v2.0, Sleep Disturbance v1.0, and Ability to Participate in Social Roles and Activities v2.0) are collected as part of routine care. Data from patients' first PROMIS measures between June 27, 2018 and April 16, 2019 were extracted and combined with claims data. Using (1) claims data alone and (2) PROMIS and claims data, we examined the association of covariates to utilization (using a logit model) and cost (using a generalized linear model). We evaluated model fit using area under the receiver operating characteristic curve (for unplanned care utilization), akaike information criterion (for unplanned care costs), and sensitivity and specificity in predicting top 15% of unplanned care costs. RESULTS: Area under the receiver operating curve values were slightly higher, and akaike information criterion values were similar, for PROMIS plus claims covariates compared with claims alone. The PROMIS plus claims model had slightly higher sensitivity and equivalent specificity compared with claims-only models. CONCLUSION: One-time PROMIS measure data combined with claims data slightly improved predictive model performance compared with claims alone, but likely not to an extent that indicates improved practical utility for payers.

Optimizing oropharyngeal cancer management by using proton beam therapy: trends of cost-effectiveness.

BACKGROUND: Proton beam therapy (PBT) is a new-emerging cancer treatment in China but its treatment costs are high and not yet covered by Chinese public medical insurance. The advanced form of PBT, intensity-modulated proton radiation therapy (IMPT), has been confirmed to reduce normal tissue complication probability (NTCP) as compared to conventional intensity-modulated photon-radiation therapy (IMRT) in patients with oropharyngeal cancer (OPC). Herein, we evaluated the cost-effectiveness and applicability of IMPT versus IMRT for OPC patients in China, aiming at guiding the proper use of PBT. METHODS: A 7-state Markov model was designed for analysis. Base-case evaluation was performed on a 56-year-old (median age of OPC in China) patient under the assumption that IMPT could provide a 25% NTCP-reduction in long-term symptomatic dysphagia and xerostomia. Model robustness was examined using probabilistic sensitivity analysis, cohort analysis, and tornado diagram. One-way sensitivity analyses were conducted to identify the cost-effective scenarios. IMPT was considered as cost-effective if the incremental cost-effectiveness ratio (ICER) was below the societal willingness-to-pay (WTP) threshold. RESULTS: Compared with IMRT, IMPT provided an extra 0.205 quality-adjusted life-year (QALY) at an additional cost of 34,926.6 US dollars ($), and had an ICER of $170,082.4/ QALY for the base case. At the current WTP of China ($33,558 / QALY) and a current IMPT treatment costs of $50,000, IMPT should provide a minimum NTCP-reduction of 47.5, 50.8, 55.6, 63.3 and 77.2% to be considered cost-effective for patient age levels of 10, 20, 30, 40 and 50-year-old, respectively. For patients at the median age level, reducing the current IMPT costs ($50,000) to a $30,000 level would make the minimum NTCP-reduction threshold for "cost-effective" decrease from 91.4 to 44.6%, at the current WTP of China (from 69.0 to 33.5%, at a WTP of $50,000 / QALY; and from 39.7 to 19.1%, at a WTP of $100,000 / QALY). CONCLUSIONS: Cost-effective scenarios of PBT exist in Chinese OPC patients at the current WTP of China. Considering a potential upcoming increase in PBT use in China, such cost-effective scenarios may further expand if a decrease of proton treatment costs occurs or an increase of WTP level.

First-line pharmacotherapy for incident type 2 diabetes: Prescription patterns, adherence and associated costs.

AIMS: To use real-world prescription data from Alberta, Canada to: (a) describe the prescribing patterns for initial pharmacotherapy for those with newly diagnosed uncomplicated type 2 diabetes; (b) describe medication-taking behaviours (adherence and persistence) in the first year after initiating pharmacotherapy; and (c) explore healthcare system costs associated with prescribing patterns. METHODS: We employed a retrospective cohort design using linked administrative datasets from 2012 to 2017 to define a cohort of those with uncomplicated incident diabetes. We summarized the initial prescription patterns, adherence and costs (healthcare and pharmaceutical) over the first year after initiation of pharmacotherapy. Using multivariable regression, we determined the association of these outcomes with various sociodemographic characteristics. RESULTS: The majority of individuals for whom metformin was indicated as first-line therapy received a prescription for metformin monotherapy (89%). Older individuals, those with higher baseline A1C and those with no comorbidities, were most likely to be started on non-metformin agents. Adherence with the initially prescribed regimen was suboptimal overall, with nearly half (48%) being non-adherent over the first year. One-third of those who started metformin discontinued it in the first 3 months. Those started on non-metformin agents had roughly twice the healthcare costs, and five to seven times higher medication costs, compared to those started on metformin, in the first year after starting therapy. CONCLUSIONS: With the addition of new classes of medications, healthcare providers who look after those with type 2 diabetes have more pharmaceutical options than ever. Most individuals continue to be prescribed metformin monotherapy. However, adherence is suboptimal, and drops off considerably within the first 3 months.

On All Accounts: Cost-Effectiveness Analysis of Limited Preoperative Optimization Efforts Before Colon Cancer Surgery.

BACKGROUND: Reports suggest that preoperative optimization of a patient's serious comorbidities is associated with a reduction in postoperative complications. OBJECTIVE: The purpose of this study was to assess the cost and benefits of preoperative optimization, accounting for total costs associated with postoperative morbidity. DESIGN: This study is a decision tree cost-effectiveness analysis with probabilistic sensitivity analysis (10,000 iterations). SETTING: This is a hypothetical scenario of stage II colon cancer surgery. PATIENT: The simulated 65-year-old patient has left-sided, stage II colon cancer. INTERVENTION: Focused preoperative optimization targets high-risk comorbidities. OUTCOMES: Total discounted (3%) economic costs (US $2018), effectiveness (quality-adjusted life-years), incremental cost-effectiveness ratio (incremental cost-effectiveness ratio, cost/quality-adjusted life-years gained), and net monetary benefit. RESULTS: We calculated the per individual expected health care sector total cost of preoperative optimization and sequelae to be $12,395 versus $15,638 in those not optimized (net monetary benefit: $1.04 million versus $1.05 million). A nonoptimized patient attained an average 0.02 quality-adjusted life-years less than one optimized. Thus, preoperative optimization was the dominant strategy (lower total costs; higher quality-adjusted life-years). Probabilistic sensitivity analysis demonstrated 100% of simulations favoring preoperative optimization. The breakeven cost of optimization to remain cost-effective was $6421 per patient. LIMITATIONS: Generalizability must account for the lack of standardization among existing preoperative optimization efforts, and decision analysis methodology provides guidance for the average patient or general population, and is not patient-specific. CONCLUSIONS: Although currently not comprehensively reimbursed, focused preoperative optimization may reduce total costs of care while also reducing complications from colon cancer surgery. See Video Abstract at http://links.lww.com/DCR/B494. EN TODO CASO ANLISIS DE RENTABILIDAD DE LOS ESFUERZOS LIMITADOS DE OPTIMIZACIN PREOPERATORIA ANTES DE LA CIRUGA DE CNCER DE COLON: ANTECEDENTES:Los informes sugieren que la optimización preoperatoria de las comorbilidades graves de un paciente se asocia con una reducción de las complicaciones postoperatorias.OBJETIVO:El propósito de este estudio fue evaluar el costo y los beneficios de la optimización preoperatoria, teniendo en cuenta los costos totales asociados con la morbilidad postoperatoria.DISEÑO:Análisis de costo-efectividad de árbol de decisión con análisis de sensibilidad probabilístico (10,000 iteraciones).AJUSTE ENTORNO CLINICO:Escenario hipotético Cirugía de cáncer de colon en estadio II.PACIENTE:Paciente simulado de 65 años con cáncer de colon en estadio II del lado izquierdo.INTERVENCIÓN:Optimización preoperatoria enfocada dirigida a comorbilidades de alto riesgo.RESULTADOS:Costos económicos totales descontados (3%) (US $ 2018), efectividad (años de vida ajustados por calidad [AVAC]), relación costo-efectividad incremental (ICER, costo / AVAC ganado) y beneficio monetario neto (NMB).RESULTADOS:Calculamos que el costo total esperado por sector de atención médica individual de la optimización preoperatoria y las secuelas es de $ 12,395 versus $ 15,638 en aquellos no optimizados (NMB: $ 1.04 millones versus $ 1.05 millones, respectivamente). Un paciente no optimizado alcanzó un promedio de 0.02 AVAC menos que uno optimizado. Por lo tanto, la optimización preoperatoria fue la estrategia dominante (menores costos totales; mayores AVAC). El análisis de sensibilidad probabilístico demostró que el 100% de las simulaciones favorecían la optimización preoperatoria. El costo de equilibrio de la optimización para seguir siendo rentable fue de $ 6,421 por paciente.LIMITACIONES:La generalización debe tener en cuenta la falta de estandarización entre los esfuerzos de optimización preoperatorios existentes y esa metodología de análisis de decisiones proporciona una guía para el paciente promedio o la población general, no específica del paciente.CONCLUSIONES:Si bien actualmente no se reembolsa de manera integral, la optimización preoperatoria enfocada puede reducir los costos totales de la atención y al mismo tiempo reducir las complicaciones de la cirugía de cáncer de colon. Consulte Video Resumen en http://links.lww.com/DCR/B494.

The Affordable Care Act at 10 Years: Evaluating the Evidence and Navigating an Uncertain Future.

The year 2020 marks the 10th anniversary of the signing of the Affordable Care Act (ACA). Perhaps the greatest overhaul of the US health care system in the past 50 y, the ACA sought to expand access to care, improve quality, and reduce health care costs. Over the past decade, there have been a number of challenges and changes to the law, which remains in evolution. While the ACA's policies were not intended to specifically target surgical care, surgical patients, surgeons, and the health systems within which they function have all been greatly affected. This article aims to provide a brief overview of the impact of the ACA on surgical patients in reference to its tripartite aim of improving access, improving quality, and reducing costs.