Measuring maternal mortality: An overview of opportunities and options for developing countries

Background  

There is currently an unprecedented expressed need and demand for estimates of maternal mortality in developing countries. This has been stimulated in part by the creation of a Millennium Development Goal that will be judged partly on the basis of reductions in maternal mortality by 2015.     

Epidemiology of achondroplasia: A population‐based study in Europe

Achondroplasia is a rare genetic disorder resulting in short‐limb skeletal dysplasia. We present the largest European population‐based epidemiological study to date using data provided by the European Surveillance of Congenital Anomalies (EUROCAT) network. All cases of achondroplasia notified to 28 EUROCAT registries (1991–2015) were included in the study. Prevalence, birth outcomes, prenatal diagnosis, associated anomalies, and the impact of paternal and maternal age on de novo achondroplasia were presented. The study population consisted of 434 achondroplasia cases with a prevalence of 3.72 per 100,000 births (95%CIs: 3.14–4.39). There were 350 live births, 82 terminations of pregnancy after prenatal diagnosis, and two fetal deaths. The prenatal detection rate was significantly higher in recent years (71% in 2011–2015 vs. 36% in 1991–1995). Major associated congenital anomalies were present in 10% of cases. About 20% of cases were familial. After adjusting for maternal age, fathers >34 years had a significantly higher risk of having infants with de novo achondroplasia than younger fathers. Prevalence was stable over time, but regional differences were observed. All pregnancy outcomes were included in the prevalence estimate with 80.6% being live born. The study confirmed the increased risk for older fathers of having infants with de novo achondroplasia.     

Impacts of Medicaid Expansion on Health Insurance and Coverage Transitions among Women with or at Risk for HIV in the United States

BackgroundAs employment, financial status, and residential location change, people can gain, lose, or switch health insurance coverage, which may affect care access and health. Among Women's Interagency HIV Study participants with HIV and participants at risk for HIV attending semiannual visits at 10 U.S. sites, we examined whether the prevalence of coverage types and rates of coverage changes differed by HIV status and Medicaid expansion in their states of residence.MethodsGeocoded addresses were merged with dates of Medicaid expansion to indicate, at each visit, whether women lived in Medicaid expansion states. Age-adjusted rate ratios (RRs) and rate differences of self-reported insurance changes were estimated by Poisson regression.ResultsFrom 2008 to 2018, 3,341 women (67% Black, 71% with HIV) contributed 43,329 visits at aged less than 65 years (27% under Medicaid expansion). Women with and women without HIV differed in their proportions of visits at which no coverage (14% vs. 19%; p < .001) and Medicaid enrollment (61% vs. 51%; p < .001) were reported. Women in Medicaid expansion states reported no coverage and Medicaid enrollment at 4% and 69% of visits, respectively, compared with 20% and 53% of visits for those in nonexpansion states. Women with HIV had a lower rate of losing coverage than those without HIV (RR, 0.81; 95% confidence interval [CI], 0.70 to 0.95). Compared with nonexpansion, Medicaid expansion was associated with lower coverage loss (RR, 0.62; 95% CI, 0.53 to 0.72) and greater coverage gain (RR, 2.32; 95% CI, 2.02 to 2.67), with no differences by HIV status.ConclusionsBoth women with HIV and women at high risk for HIV in Medicaid expansion states had lower coverage loss and greater coverage gain; therefore, Medicaid expansion throughout the United States should be expected to stabilize insurance for women and improve downstream health outcomes.     

Exploring Differences in Work Disability Duration by Size of Firm in Canada and Australia

Journal of Occupational Rehabilitation - Purpose To identify whether there were differences in work disability duration between injured workers employed by small, medium, large, and self-insured...     

Comparison of the PROMIS Preference Score (PROPr) and EQ-5D-5L Index Value in General Population Samples in the United Kingdom,France, and Germany

ObjectivesThe Patient-Reported Outcome Measurement Information System (PROMIS) Preference score (PROPr) can be used to assess health state utility (HSU) and estimate quality-adjusted life-years in cost-effectiveness analyses. It is based on item response theory and promises to overcome limitations of existing HSU scores such as ceiling effects. The PROPr contains 7 PROMIS domains: cognitive abilities, depression, fatigue, pain, physical function, sleep disturbance, and ability to participate in social roles and activities. We aimed to compare the PROPr with the 5-level EQ-5D (EQ-5D-5L) in terms of psychometric properties using data from 3 countries.MethodsWe collected PROMIS-29 profile and EQ-5D-5L data from 3 general population samples (United Kingdom = 1509, France = 1501, Germany = 1502). Given that cognition is not assessed by the PROMIS-29, it was predicted by the recommended linear regression model. We compared the convergent validity, known-groups construct validity, and ceiling and floor effects of the PROPr and EQ-5D-5L.ResultsThe mean PROPr (0.48, 0.53, 0.48; P<.01) and EQ-5D-5L scores (0.82, 0.85, 0.83; P<.01) showed significant differences of similar magnitudes (d = 0.34; d = 0.32; d = 0.35; P<.01) across all samples. The differences were invariant to sex, income, occupation, education, and most conditions but not for age. The Pearson correlation coefficients between both scores were r = 0.74, r = 0.69, and r = 0.72. PROPr’s ceiling and floor effects both were minor to moderate. The EQ-5D-5L’s ceiling (floor) effects were major (negligible).ConclusionsBoth the EQ-5D-5L and the PROPr assessed by the PROMIS-29 show high validity. The PROPr yields considerably lower HSU values than the EQ-5D-5L. Consequences for quality-adjusted life-year measurements should be investigated in future research.     

US Food and Drug Administration Analysis of Patient-Reported Diarrhea and Its Impact on Function and Quality of Life in Patients Receiving Treatment for Breast Cancer

ObjectivesMany trials conclude “no clinically meaningful detriment” to health-related quality of life (HRQL) or function between arms, even when notable differential toxicity is observed. Mean change from baseline analyses of function or HRQL can possibly obscure important change in subgroups experiencing symptomatic toxicity. We evaluate the impact of diarrhea, a key treatment arm toxicity, on patient-reported HRQL and functioning in clinical trials submitted to US Food and Drug Administration.MethodsThis study used 4 randomized, breast cancer trials (adjuvant to late-line metastatic) as case examples. Diarrhea, physical functioning (PF), and global health status and quality of life (GHS/QoL) from the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-C30 were analyzed at baseline and approximately 3 and 6 months.ResultsGenerally, patients reporting very much diarrhea at months 3 and 6 had worse PF (9-19 points lower) and GHS/QoL (16-19 points lower) than patients reporting no diarrhea regardless of treatment arm. In the change from baseline analysis, patients reporting very much diarrhea also experienced a greater decrease in PF (6-13 points) and GHS/QoL (6-16 points) versus patients reporting no diarrhea in both arms.ConclusionsIn trials with moderate to large differences in symptomatic toxicity by arm, reporting “no meaningful difference in functioning and HRQL between arms” based on mean change from baseline analysis is insufficient and may obscure important impacts on subgroups experiencing symptomatic adverse events. Additional exploratory analyses with simple data visualizations evaluating functioning or HRQL in patient subgroups experiencing expected symptomatic toxicities can further inform the safety and tolerability of an investigational agent.     

The History and Future of the “ISPOR Value Flower”: Addressing Limitations of Conventional Cost-Effectiveness Analysis

ObjectivesSince its publication as part of the 2018 ISPOR Special Task Force (STF) on US Value Assessments, the “ISPOR value flower,” with its petals highlighting elements that may be overlooked or underappreciated in conventional drug value assessments, has been discussed and debated. We review the history of the value flower, describe recent developments, and consider implications for future value assessments.MethodsWe discuss various antecedents to the value flower, as well as conceptual and empirical articles published in the past 4 years.ResultsSince the publication of the ISPOR STF report, researchers have provided more rigorous theoretical and mathematical foundations for certain novel value elements (eg, severity of illness, value of insurance, value of hope) through “generalized risk-adjusted cost-effectiveness analysis,” which incorporates risk aversion in people’s preferences and uncertainty in treatment outcomes. Empirical estimates are also emerging to support key elements, such as insurance value, real option value, value of hope, and value of knowing. Although health technology assessment bodies have applied or are considering certain elements (eg, severity modifiers to cost-effectiveness thresholds), other elements have yet to gain traction.ConclusionsFive years after the STF began its work, the development of novel value measures continues to evolve. Although it is encouraging to see supporting empirical studies emerging, more are needed. Additional efforts are also needed to illustrate how the estimates can be used in the deliberative processes that are integral to health technology assessments.