The Rising Burden of Preeclampsia in the United States Impacts Both Maternal and Child Health

Preeclampsia is one of the top six causes of maternal mortality in the United States (US) and is associated with considerable perinatal morbidity and mortality. Evidence suggests the US incidence of preeclampsia has increased dramatically over the past two decades. This study aims to compile, summarize, and critique the literature on the health and economic burden of preeclampsia and early-onset preeclampsia. We reviewed the literature for estimates of burden of preeclampsia and early-onset preeclampsia to both mother and child, summarized the evidence on economic and social burden, and highlighted current gaps in the literature. No recent studies comprehensively assess the costs and health consequences of preeclampsia or early-onset preeclampsia for both mother and child. Where it exists, the literature suggests preeclampsia and early-onset preeclampsia cause numerous adverse health consequences, but these conditions currently lack effective treatment. The need for preterm delivery from early-onset preeclampsia suggests its costs are substantial: very (28-31 weeks) and extremely (<28 weeks) preterm birth cost approximately 40 and 100 times a term pregnancy, respectively. Given the severity of outcomes from preeclampsia, further research on its health and economic consequences is essential to inform policy and resource allocation decisions in health care

Additional file 1: of Estimating the future burden of cardiovascular disease and the value of lipid and blood pressure control therapies in China

Future burden of CVD in China Appendix. (DOCX 3274 kb

Additional file 2: of Estimating the future burden of cardiovascular disease and the value of lipid and blood pressure control therapies in China

Future burden of CVD in China Additional Data Dictionary. (DOCX 22 kb

The value of surrogate endpoints for predicting real-world survival across five cancer types

<p><b>Objective</b> It is unclear how well different outcome measures in randomized controlled trials (RCTs) perform in predicting real-world cancer survival. We assess the ability of RCT overall survival (OS) and surrogate endpoints – progression-free survival (PFS) and time to progression (TTP) – to predict real-world OS across five cancers.</p> <p><b>Methods</b> We identified 20 treatments and 31 indications for breast, colorectal, lung, ovarian, and pancreatic cancer that had a phase III RCT reporting median OS and median PFS or TTP. Median real-world OS was determined using a Kaplan–Meier estimator applied to patients in the Surveillance and Epidemiology End Results (SEER)-Medicare database (1991–2010). Performance of RCT OS and PFS/TTP in predicting real-world OS was measured using <i>t</i>-tests, median absolute prediction error, and <i>R</i>² from linear regressions.</p> <p><b>Results</b> Among 72,600 SEER-Medicare patients similar to RCT participants, median survival was 5.9 months for trial surrogates, 14.1 months for trial OS, and 13.4 months for real-world OS. For this sample, regression models using clinical trial OS and trial surrogates as independent variables predicted real-world OS significantly better than models using surrogates alone (<i>P</i> = 0.026). Among all real-world patients using sample treatments (<i>N</i> = 309,182), however, adding trial OS did not improve predictive power over predictions based on surrogates alone (<i>P</i> = 0.194). Results were qualitatively similar using median absolute prediction error and <i>R</i>² metrics.</p> <p><b>Conclusions</b> Among the five tumor types investigated, trial OS and surrogates were each independently valuable in predicting real-world OS outcomes for patients similar to trial participants. In broader real-world populations, however, trial OS added little incremental value over surrogates alone.</p

Cost-effectiveness of sequenced treatment of rheumatoid arthritis with targeted immune modulators

<p><b>Aims:</b> To determine the cost-effectiveness of treatment sequences of biologic disease-modifying anti-rheumatic drugs or Janus kinase/STAT pathway inhibitors (collectively referred to as bDMARDs) vs conventional DMARDs (cDMARDs) from the US societal perspective for treatment of patients with moderately to severely active rheumatoid arthritis (RA) with inadequate responses to cDMARDs.</p> <p><b>Materials and methods:</b> An individual patient simulation model was developed that assesses the impact of treatments on disease based on clinical trial data and real-world evidence. Treatment strategies included sequences starting with etanercept, adalimumab, certolizumab, or abatacept. Each of these treatment strategies was compared with cDMARDs. Incremental cost, incremental quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) were calculated for each treatment sequence relative to cDMARDs. The cost-effectiveness of each strategy was determined using a US willingness-to-pay (WTP) threshold of $150,000/QALY.</p> <p><b>Results:</b> For the base-case scenario, bDMARD treatment sequences were associated with greater treatment benefit (i.e. more QALYs), lower lost productivity costs, and greater treatment-related costs than cDMARDs. The expected ICERs for bDMARD sequences ranged from ∼$126,000 to $140,000 per QALY gained, which is below the US-specific WTP. Alternative scenarios examining the effects of homogeneous patients, dose increases, increased costs of hospitalization for severely physically impaired patients, and a lower baseline Health Assessment Questionnaire (HAQ) Disability Index score resulted in similar ICERs.</p> <p><b>Conclusions:</b> bDMARD treatment sequences are cost-effective from a US societal perspective.</p