Estimating fetal weight in gastroschisis: A 10 year audit of outcomes at the National Maternity Hospital.

OBJECTIVE: To identify whether conventional methods of estimating fetal growth (Hadlock's formula), which relies heavily on abdominal circumference measurements, are accurate in fetuses with gastroschisis. METHODS: A retrospective cohort study was performed between the period January 1, 2011 and December 31, 2021 in a tertiary referral maternity hospital identifying all pregnancies with a diagnosis of gastroschisis. Projected fetal weight was obtained using the formula (EFW [Hadlock's formula] + 185 g × [X/7]) where X was the number of days to delivery. RESULTS: During the study period 41 cases were identified. The median maternal age was 25. The median BMI was 25 and 63% were primiparous women (n = 26). Median gestation at diagnosis was 21 weeks. Median gestation at delivery was 36 weeks. A total of 4.8% of mothers had a history of drug use (n = 2). The rate of maternal tobacco use was 21.9% (n = 9). A total of 4.8% of fetuses had additional congenital anomalies including amniotic band syndrome and myelomeningocele (n = 2). Estimated fetal weight (EFW) and birth weight data were available for 34 cases. A Wilcoxon signed-rank test showed projected EFW using Hadlock's formula did not result in a statistically significant different birth weight (Z = -1.3, P = 0.169). Median projected weight and actual birth weight were 2241.35 and 2415 g respectively. Median difference was 0.64 g (95% CI: -148 to -28.5). CONCLUSION: Our data showed accuracy using standard formulae for EFW in fetuses with gastroschisis.

Risk preferences over health: Empirical estimates and implications for medical decision-making.

Mainstream health economic theory implies that an expected gain in health-related quality of life (HRQoL) produces the same value for consumers, regardless of baseline health. Several strands of recent research call this implication into question. Generalized Risk-Adjusted Cost-Effectiveness (GRACE) demonstrates theoretically that baseline health status influences value, so long as consumers are not risk-neutral over health. Prior empirical literature casts doubt on risk-neutral expected utility-maximization in the health domain. We estimate utility over HRQoL in a nationally representative U.S. population and use our estimates to measure risk preferences over health. We find that individuals are risk-seeking at low levels of health, become risk-averse at health equal to 0.485 (measured on a 0-1 scale), and are most risk-averse at perfect health (coefficient of relative risk aversion = 4.51). We develop the resulting implications for medical decision making, cost-effectiveness analyses, and the proper theory of health-related decision making under uncertainty.

Multidisciplinary Surgical Approach to Increase Survival for Advanced Ovarian Cancer in a Tertiary Gynaecological Oncology Centre.

PURPOSE: The purpose of this paper is to report on changes in overall survival, progression-free survival, and complete cytoreduction rates in the 5-year period after the implementation of a multidisciplinary surgical team (MDT). METHODS: Two cohorts were used. Cohort A was a retrospectively collated cohort from 2006 to 2015. Cohort B was a prospectively collated cohort of patients from January 2017 to September 2021. RESULTS: This study included 146 patients in cohort A (2006-2015) and 174 patients in cohort B (2017-2021) with FIGO stage III/IV ovarian cancer. Median follow-up in cohort A was 60 months and 48 months in cohort B. The rate of primary cytoreductive surgery increased from 38% (55/146) in cohort A to 46.5% (81/174) in cohort B. Complete macroscopic resection increased from 58.9% (86/146) in cohort A to 78.7% (137/174) in cohort B (p < 0.001). At 3 years, 75% (109/144) patients had disease progression in cohort A compared with 48.8% (85/174) in cohort B (log-rank, p < 0.001). Also at 3 years, 64.5% (93/144) of patients had died in cohort A compared with 24% (42/174) of cohort B (log-rank, p < 0.001). Cox multivariate analysis demonstrated that MDT input, residual disease, and age were independent predictors of overall (hazard ratio [HR] 0.29, 95% confidence interval [CI] 0.203-0.437, p < 0.001) and progression-free survival (HR 0.31, 95% CI 0.21-0.43, p < 0.001). Major morbidity remained stable throughout both study periods (2006-2021). CONCLUSIONS: Our data demonstrate that the implementation of multidisciplinary-team, intraoperative approach allowed for a change in surgical philosophy and has resulted in a significant improvement in overall survival, progression-free survival, and complete resection rates.

The Broader Economic Value of Treatment for Diabetic Macular Edema.

OBJECTIVE: To simulate economic outcomes for individuals with diabetic macular edema (DME) and estimate the economic value of direct and indirect benefits associated with DME treatment. RESEARCH DESIGN AND METHODS: Our study pairs individual and cohort analyses to demonstrate the value of treatment for DME. We used a microsimulation model to simulate self-reported vision (SRV) and economic outcomes for individuals with DME. Four scenarios derived from clinical trial data were simulated and compared for a lifetime horizon: untreated, anti-VEGF therapy, laser, and steroid. To quantify the relative magnitude of costs and benefits of DME treatment in the U.S., we used a cohort-level analysis based on real-world treatment parameters derived from published data. RESULTS: In the model, excellent/good SRV roughly corresponded to 20/40 or better visual acuity. A representative 51-year-old treated for DME would spend 30-35% additional years with excellent/good SRV and 29-32% fewer years with fair/poor SRV relative to being untreated. A treated individual would experience 4-5% greater life expectancy and 9-13% more quality-adjusted life-years. Indirect benefits from treatment included 6-9% more years working, 12-19% greater lifetime earnings, and 8-16% fewer years with disability. For the U.S. DME cohort (1.1. million people), total direct benefit was $63.0 billion over 20 years, and total indirect benefit was $4.8 billion. Net value (benefit - cost) of treatment ranged from $28.1 billion to $52.8 billion. CONCLUSIONS: Treatment for DME provides economic value to patients and society through improved vision, life expectancy, and quality of life and indirectly through improved employment and disability outcomes.

Management of Hemorrhage During Cesarean Delivery in a Patient With Vascular Ehlers-Danlos Syndrome: A Case Report.

Parturients with vascular Ehlers-Danlos syndrome are at particular risk of hemorrhage, and there is little evidence to guide prevention or management of hemorrhage in these patients. We present the case of a patient with vascular Ehlers-Danlos syndrome who had a cesarean delivery complicated by an intraoperative hemorrhage. Administration of desmopressin and tranexamic acid appeared to be of marked benefit in achieving hemostasis. To the best of our knowledge, this is the first report of the use of desmopressin during major obstetric hemorrhage in vascular Ehlers-Danlos syndrome.

Cesarean scar niche: An evolving concern in clinical practice.

Increasing cesarean section rates have led to an increased awareness of associated complications such as the formation of cesarean scar niche, defined as an indentation at the site of the cesarean scar with a depth of at least 2 mm, diagnosed by ultrasound or magnetic resonance imaging. The precise prevalence of cesarean scar niche is unclear. The cause of a cesarean scar niche appears to be multifactorial and likely a combination of technical factors (low incision location), anatomical factors (uterine retroflexion), and patient factors, which might impair healing (body mass index, smoking, maternal age). Most patients with cesarean scar niche are asymptomatic; however, women can present with postmenstrual bleeding, pelvic pain, and subfertility. In pregnancy, cesarean scar niches have been associated with placenta accreta spectrum disorder and uterine rupture. Treatment should be reserved for symptomatic women. Hormonal treatment using either the combined oral contraceptive pill or a progesterone-containing intrauterine device may address irregular vaginal bleeding. Surgical management should be reserved for those in whom hormonal manipulation has failed or is contraindicated. The aim of this review was to summarize current literature pertaining to the cause, prevalence, diagnosis, and symptoms of cesarean scar niche and to make recommendations for managing this relatively new condition.

Placenta accreta spectrum ultrasound stage and fetal growth.

OBJECTIVE: to evaluate fetal growth in pregnancies complicated by placenta accreta spectrum (PAS) and to compare fetal growth between cases stratified by ultrasound stage of PAS. METHODS: This was a prospective multicenter cohort study of women diagnosed with PAS between January 2018 and December 2021. We grouped participants into cases by ultrasound stage (PAS stage 1-3) and controls (PAS0). Fetal growth centiles at three timepoints with median gestational ages of 21 ± 1 weeks (interquartile range [IQR], 20 ± 1-22 ± 0 weeks), 28 ± 0 weeks (IQR, 27 ± 0-28 ± 5 weeks), and 33 ± 0 weeks (IQR, 32 ± 1-34 ± 0 weeks) and birth weight centiles were compared between cases and controls and between those with PAS stratified by ultrasound stage. RESULTS: A total of 53 women met inclusion criteria, with a mean age of 37 years (standard deviation, ±4.0 years) and body mass index of 27 kg/m2 (standard deviation, ±5.8 kg/m2 ). Median (IQR) fetal weight centiles were around the 50th centile at each timepoint, with no difference between groups. The incidence of small for gestational age (birth weight ≤ 10th percentile) and large for gestational age (birth weight ≥ 90th percentile) was 11.3% (n = 6) and 15.1% (n = 8), respectively, with no differences by ultrasound stage. The median birth weight centile was 64 (IQR, 26-85), with no differences between cases and controls or by ultrasound stage. CONCLUSIONS: In our cohort, a diagnosis of PAS was not associated with fetal growth restriction.

Manufacturer Signal-to-Cutoff Threshold Underestimates Cumulative Incidence of SARS-CoV-2 Infection: Evidence from the Los Angeles Firefighters Study.

BACKGROUND: The objective of this analysis was to compare the performance sensitivity and specificity of manufacturer-recommended signal-to-cutoff (S/Co) thresholds with modified S/Co values to estimate the prevalence of SARS-CoV-2-specific antibodies in a cohort of firefighters with a known infection history. METHODS: Plasma venipuncture samples were used for serologic analysis of firefighters in Los Angeles, CA, USA, in October 2020. Seropositivity was assessed using the manufacturer's recommended S/Co (≥1.4 IgG) and modified S/Co thresholds based on measured antibody levels in 178 negative control patients who had blood drawn prior to the emergence of COVID-19. Optimal S/Co threshold was determined by receiver operating characteristic (ROC) curve analysis. RESULTS: Of 585 firefighters included in the study, 52 (8.9%) reported having a PCR-positive test history prior to antibody testing. Thirty-five (67.3%) firefighters with a previous PCR-positive test were seropositive based on the manufacturer S/Co thresholds, consistent with an estimated 67.3% sensitivity and 100% specificity. After evaluating multiple modified S/Co thresholds based on pre-pandemic negative samples, a modified S/Co of 0.36 was found to yield optimal sensitivity (88.5%) and specificity (99.4%) by ROC curve analysis. This modified threshold improved serostatus classification accuracy by 21.2%. CONCLUSIONS: S/Co thresholds based on known negative samples significantly increase seropositivity and more accurately estimate cumulative incidence of disease compared to manufacturer-based thresholds.

What do nonprofit hospitals reward? An examination of CEO compensation in nonprofit hospitals.

Nonprofit hospital chief executive officer (CEO) compensation has received considerable attention in light of nonprofits' tax-favored status as well as the high costs of hospital care. Past studies have found that hospital financial performance is a significant determinant of CEO pay but nonprofit performance, including quality and charity care, are not. Using post-ACA data, we re-examine whether a variety of hospital performance measures are important determinants of nonprofit hospital CEO compensation. We found mixed evidence with respect to the significance of the association between financial performance and uncompensated care and CEO compensation. Among the other nonprofit performance measures, patient satisfaction was significantly associated with CEO compensation, but other measures were not significant determinants of CEO compensation. Our results suggest nonprofit hospitals balance their financial health against their mission when setting CEO incentives. Additional policy targeting transparency in hospital CEO compensation may be warranted to help policymakers understand the specific factors used by hospital boards to incentivize CEOs.

SARS-CoV-2 seroprevalence among firefighters in Los Angeles, California.

OBJECTIVE: We estimate the seroprevalence of SARS-CoV-2 antibodies among a sample of firefighters in the Los Angeles (LA), California fire department in October 2020 and compare demographic and contextual factors for seropositivity. METHODS: We conducted a serological survey of firefighters in LA, California, USA, in October 2020. Individuals were classified as seropositive for SARS-CoV-2 if they tested positive for IgG, IgM or both. We compared demographic and contextual factors for seropositivity. RESULTS: All firefighters in LA, California, USA were invited to participate in our study, but only roughly 21% participated. Of 713 participants with valid serological data, 8.8% tested positive for SARS-CoV-2 antibodies, and among the 686 with complete survey data 8.9% tested positive for antibodies. Seropositivity was not associated with gender, age or race/ethnicity. Seropositivity was highest among firefighters who reported working in the vicinity of LA International Airport, which had a known outbreak in July 2020. CONCLUSIONS: Seroprevalence among firefighters in our sample was 8.8%, however, we lack a full workplace seroprevalence estimate to compare the relative magnitude against general population seroprevalence (15%). Workplace safety protocols, such as access to personal protective equipment and testing, can mitigate increased risk of infection at work, and may have eliminated differences in disease burden by geography and race/ethnicity in our sample.

Comparing three-dimensional models of placenta accreta spectrum with surgical findings.

OBJECTIVE: Placenta accreta spectrum (PAS) is associated with significant maternal morbidity mainly related to blood loss. Pre-operative planning is aided by antenatal ultrasound and magnetic resonance imaging. We sought to assess whether three-dimensional (3D) models from MR images were accurate when compared with surgical and pathological findings. METHODS: Digital Imaging and Communications in Medicine files containing MR images with varying severity of PAS (n = 4) were modeled using 3D Slicer. Placenta, bladder, and myometrial defects were modeled. Myometrial defects at three different uterine locations were included-anterior, lateral and inferior. 3D models were used to identify the relationship between the myometrial defect and the internal cervical os. Findings were validated in a larger series of PAS cases (n = 14) where patterns of invasion were compared with estimated blood loss and distance from defect to the internal os. RESULTS: The defect illustrated in the four 3D models correlates to both surgical and pathological findings in terms of depth and pattern of invasion, location of defect, bladder involvement. Blood loss and topography of the defect from 3D modeling were examined in 14 further cases. Inferior defects were associated with increased blood loss compared with anterior defects. Increased distance from cervix was associated with reduced blood loss (R2  = 0.352, P = 0.01). CONCLUSION: Three-dimensional models of PAS provide an accurate preoperative description of placental invasion and should be investigated as a tool for selecting patients for uterine-conserving surgery. Accurate 3D models of placenta accreta spectrum are achievable and may provide additional information, such as distance of the defect from the internal os.

The addition of sodium thiosulphate to hyperthermic intraperitoneal chemotherapy with cisplatin in ovarian cancer.

Cisplatin based hyperthermic intraperitoneal chemotherapy (HIPEC) has been shown to prolong recurrence free and overall survival of women with ovarian cancer who have responded to neoadjuvant chemotherapy. The aim of this study was to assess the impact of cytoreductive surgery with or without the addition of HIPEC on renal function. METHOD: This is a retrospective case-controlled study at a tertiary teaching hospital in Dublin, Ireland. All patients who had interval cytoreductive surgery (CRS) and HIPEC from October 2017 to October 2020 were included. A cohort of patients who had interval CRS without HIPEC were included as a control. Sodium thiosulphate (ST) was added to the HIPEC protocol in 2019. In order to assess the impact of ST as a renal protectant, renal function and post-operative outcomes were compared between the groups. RESULTS: Sixty patients who had interval CRS were included, thirty of whom received cisplatin-based HIPEC. Seven received cisplatin 50 mg/m2 without the addition of ST. Twenty three patients received cisplatin 100 mg/m2 and ST. There were no statistically differences in age, body mass index BMI, American society of anaesthesia score, estimated blood loss or peritoneal cancer index between the cohorts (p > 0.05). The only episode of acute kidney injury (AKI) was within the HIPEC cohort, after cisplatin 50 mg/m2 (without ST) and this was sustained at three months. In contrast, no patients within the CRS cohort or cisplatin 100 mg/m2 that received the addition of ST, sustained a renal injury and all had a creatinine within the normal range at three days post operatively. CONCLUSION: The renal toxicity associated with cisplatin HIPEC and major abdominal surgery can be minimised with careful preoperative optimisation, intra operative fluid management and attention to renal function. The addition of sodium thiosulphate is a safe and effective method to minimise toxicity and should be added to any cisplatin HIPEC protocol.

Access to the 340B Drug Pricing Program: is there evidence of strategic hospital behavior?

OBJECTIVE: The 340B Drug Pricing Program allows hospitals to purchase covered drugs at a discount and potentially generate profit if they are reimbursed at rates that exceed 340B acquisition prices. Disproportionate share hospitals (DSH) are eligible to participate in 340B if their DSH adjustment-a measure that identifies hospitals that treat a disproportionate share of low income Medicare or Medicaid patients-is above 11.75%. To assess whether hospitals behave strategically to gain access to the program, we examined data on the number of hospitals just above versus below the DSH adjustment threshold for 340B eligibility and conducted McCrary density tests to assess statistical significance. RESULTS: In 2014-2016, the number of hospitals increases by 41% just above the 340B eligibility threshold. McCrary density tests found this increase to be statistically significant across a range of bandwidths in 2014-2016 (p < 0.01). From 2011-2013, the findings are sensitive to the bandwidth around the threshold, but insignificant in 2008-2010. We found no comparable change among hospitals ineligible for the 340B program. These data are consistent with the hypothesis that some hospitals adjust their DSH to gain 340B eligibility. Our findings support recent calls from the Government Accountability Office to improve oversight of the 340B program.

Maternal morbidity in placenta accreta spectrum following introduction of a multi-disciplinary service compared to standard care: an Irish perspective.

AIM: The purpose of this study is to compare maternal outcomes in patients with placenta accreta spectrum (PAS) when managed as part of a multi-disciplinary team (MDT) compared to standard care. METHODS: Patients in the standard care group were retrospectively identified from pathology records, with patients in the MDT group prospectively collected on an electronic database. Data on maternal demographics, delivery, estimated blood loss (EBL), transfusion requirements, and morbidity were recorded. RESULTS: Sixty patients were diagnosed with PAS between 2006 and 2019, of whom 32 were part of the standard care group and 28 in the MDT group. Compared to standard care, MDT care was associated with an increase in antenatal diagnosis from 56.3 to 92.9% (p < 0.0001), a significant reduction in EBL (4150 mL (800-19500) vs 1975 (495-8500), p < 0.0001), and transfusion requirements (median 7 (0-30) units of RCC vs 1 (0-13), p < 0.0001). CONCLUSION: PAS is associated with significant maternal morbidity and warrants management in an MDT setting with specialist input, which is associated with improved outcomes.

Multidisciplinary Surgical Approach to Increase Complete Cytoreduction Rates for Advanced Ovarian Cancer in a Tertiary Gynecologic Oncology Center.

BACKGROUND: Surgical resection remains the cornerstone of ovarian cancer management. In 2017, the authors implemented a multi-disciplinary surgical team comprising gynecologic oncologists as well as colorectal, hepatobiliary, and upper gastrointestinal (GI) surgeons to increase gross macroscopic resection rates. This report aims to describe changes in complete cytoreduction rates and morbidity after the implementation of a multi-disciplinary surgical team comprising gynecologic oncologists as well as colorectal, hepatobiliary, and upper GI surgeons in a tertiary gynecologic oncology unit. METHODS: The study used two cohorts. Cohort A was a retrospectively collated cohort from 2006 to 2015. Cohort B was a prospectively collated cohort of patients initiated in 2017. A multidisciplinary approach to preoperative medical optimization, intraoperative management, and postoperative care was implemented in 2017. The patients in cohort B with upper abdominal disease were offered primary cytoreduction with or without hyperthermic intraperitoneal chemotherapy (HIPEC). Before 2017, the patients with upper abdominal disease received neoadjuvant chemotherapy (cohort A). RESULTS: This study included 146 patients in cohort A (2006-2015) and 93 patients in cohort B (2017-2019) with stages 3 or 4 ovarian cancer. The overall complete macroscopic resection rate (CC0) increased from 58.9 in cohort A to 67.7% in cohort B. The rate of primary cytoreductive surgery (CRS) increased from 38 (55/146) in cohort A to 42% (39/93) in cohort B. The CC0 rate for the patients who underwent primary CRS increased from 49 in cohort A to 77% in cohort B. Major morbidity remained stable throughout both study periods (2006-2019). CONCLUSIONS: The study data demonstrate that implementation of a multidisciplinary team intraoperative approach and a meticulous approach to preoperative optimization resulted in significantly improved complete resection rates, particularly for women offered primary CRS.

Do companies in the pharmaceutical supply chain earn excess returns?

Rising drug spending has led to increased calls to curtail drug costs. However, it is unclear where to target policy solutions. We estimated excess returns (the extent to which a firm's profits are higher than expected given the risk associated with their investments) for manufacturers and middlemen in the pharmaceutical supply chain to determine who is making excessive profits. Excess returns were calculated as the difference between return on invested capital and the expected returns given risk, which is known as the weighted average cost of capital. We compared excess returns for manufacturers and middlemen to the average for S&P 500 companies. We find that both manufacturers and middlemen have higher excess returns in 2013-2018 compared with the S&P 500. However, if we treat research and development (R&D) as an investment rather than an expense, we find that excess returns for pharmaceutical manufacturers are lower than the S&P 500 (1.7% vs. 3.6%), but biotech manufacturers (9.6%), wholesalers (8.1%), and insurers/PBM/retailers (5.9%) continue to have significantly higher excess returns compared to the S&P 500. Our findings suggest public policies that promote competition in all areas of the pharmaceutical supply chain are important avenues for curtailing drug spending.

A life changing experience: An interpretative phenomenological analysis of women's experiences of placenta accreta spectrum.

BACKGROUND: The incidence of Placenta Accreta Spectrum (PAS) is increasing. This study explores women's personal experience of a pregnancy, birth and early postpartum period complicated by PAS. METHODS: A qualitative study involving seven women (age range: 33-44 years) following a pregnancy in the previous two years complicated by PAS was conducted. Using an Interpretative Phenomenological Analysis approach data were collected and analysed in 2019 and 2020 through face-to-face interviews. Interviews were transcribed verbatim. RESULTS: Women described their experience from diagnosis up to two years following the birth and focussed on the strategies they used to cope with an unexpected diagnosis of PAS. Six main themes emerged. In pregnancy, women were focussed on the diagnosis and managing family life from hospital "it's not until you are told", "sad but safe", "relying on others can be unbearable". In terms of the birth a 'fear of dying' related to self and baby was central. Postnatally key themes related to the ongoing consequences of the condition as "no one believed I could be feeling so much pain" and "it's not all over when you go home". DISCUSSION: Women who have experienced a pregnancy complicated by PAS experience significant challenges associated with long term hospitalization, post-operative pain and many express feelings of being generally worried lasting many months after their pregnancy. This study makes an important contribution to the growing body of research in PAS, namely that of the woman's own voice.

New Evidence on the Compensation of Chief Executive Officers at Nonprofit U.S. Hospitals.

Chief executive officer (CEO) compensation is highly scrutinized, with nonprofit organizations often receiving additional attention due to their tax-exempt status. Understanding hospital CEO compensation is of increasing importance as health care costs remain high and strong leadership is required to implement new health policies. This study documents CEO compensation at nonprofit hospitals in the United States for 2010 and 2015. We compare hospital CEO compensation with CEO compensation in other institution types, including nonhospital health care. We also explore changes in hospital CEO compensation over time and differences across states. We find CEOs at hospitals earn substantially less than CEOs of publicly traded companies though more than presidents of nonprofit institutions of higher education. Additionally, we find that the relationship between CEO compensation and hospital size was weaker in 2015 than in 2010, and substantial variation in CEO compensation exists across states.

Economic Value of Anti-Vascular Endothelial Growth Factor Treatment for Patients With Wet Age-Related Macular Degeneration in the United States.

Importance: Anti-vascular endothelial growth factor (anti-VEGF) is a breakthrough treatment for wet age-related macular degeneration (wAMD), the most common cause of blindness in western countries. Anti-VEGF treatment prevents vision loss and has been shown to produce vision gains lasting as long as 5 years. Although this treatment is costly, the benefits associated with vision gains are large. Objective: To estimate the economic value of benefits, costs for patients with wAMD, and societal value in the United States generated from vision improvement associated with anti-VEGF treatment. Design, Setting, and Participants: This economic evaluation study used data from the published literature to simulate vision outcomes for a cohort of 168 820 patients with wAMD aged 65 years or older and to translate them into economic variables. Data were collected and analyzed from March 2018 to November 2018. Main Outcomes and Measures: Main outcomes included patient benefits, costs, and societal value. Each outcome was estimated for a newly diagnosed cohort and the full population across 5 years, with a focus on year 3 as the primary outcome because data beyond that point may be less representative of the general population. Drug costs were the weighted mean across anti-VEGF therapies. Two current treatment scenarios were considered: less frequent injections (mean [SD], 8.2 [1.6] injections annually) and more frequent injections (mean [range], 10.5 [6.8-13.1] injections annually). The 2 treatment innovation scenarios, improved adherence and best case, had the same vision outcomes as the current treatment scenarios had but included more patients treated from higher initiation and lower discontinuation. Results: The study population included 168 820 patients aged 65 years at the time of diagnosis with wAMD. The underlying clinical trials that were used to parameterize the model did not stratify visual acuity outcomes or treatment frequency by sex; therefore, the model parameters could not be stratified by sex. The current treatment scenario of less frequent injections generated $1.1 billion for the full population in year 1 and $5.1 billion in year 3, whereas the scenario of more frequent injections generated $1.6 billion (year 1) and $8.2 billion (year 3). Three-year benefits ranged from $7.3 billion to $11.4 billion in the improved adherence scenario and from $9.7 billion to $15.0 billion if 100% of the patients initiated anti-VEGF treatment and the discontinuation rates were 6% per year or equivalent to clinical trial discontinuation (best-case scenario). Societal value (patient benefits net of treatment cost) ranged from $0.9 billion to $3.0 billion across 3 years in the current treatment scenarios and from $0.9 billion to $4.3 billion in the treatment innovation scenarios. Conclusions and Relevance: This study's findings suggest that improved vision associated with anti-VEGF treatment may provide economic value to patients and society if the outcomes match published outcomes data used in these analyses; however, future innovations that increase treatment utilization may result in added economic benefit.

The Value of Progression-Free Survival in Metastatic Breast Cancer: Results From a Survey of Patients and Providers.

Background. Value assessments and treatment decision making typically focus on clinical endpoints, especially overall survival (OS). However, OS data are not always available, and surrogate markers may also have some value to patients. This study sought to estimate preferences for progression-free survival (PFS) relative to OS in metastatic breast cancer (mBC) among a diverse set of stakeholders-patients, oncologists, and oncology nurses-and estimate the value patients and providers place on other attributes of treatment. Methods. Utilizing a combined conjoint analysis and discrete choice experiment approach, we conducted an online prospective survey of mBC patients and oncology care providers who treat mBC patients across the United States. Results. A total of 299 mBC patients, 100 oncologists, and 99 oncology nurses completed the survey. Virtually all patients preferred health state sequences with contiguous periods of PFS, compared with approximately 85% and 75% of nurses and oncologists, respectively. On average, longer OS was significantly (P < 0.01) preferred by the majority (75%) patients, but only 15% of nurses preferred longer OS, and OS did not significantly affect oncologists' preferred health state. However, in the context of a treatment decision, whether a treatment offered continuous periods of stable disease holding OS constant significantly affected nurses' treatment choices. Patients and providers alike valued reductions in adverse event risk and evidence from high-quality randomized controlled clinical trials. Conclusions. The strong preference for observed PFS suggests more research is warranted to better understand the reasons for PFS having positive value to patients. The results also suggest a range of endpoints in clinical trials may have importance to patients.