BackgroundWe explore whether the number of null results in large National Heart Lung, and Blood Institute (NHLBI) funded trials has increased over time.MethodsWe identified all large NHLBI supported RCTs between 1970 and 2012 evaluating drugs or dietary supplements for the treatment or prevention of cardiovascular disease. Trials were included if direct costs >$500,000/year, participants were adult humans, and the primary outcome was cardiovascular risk, disease or death. The 55 trials meeting these criteria were coded for whether they were published prior to or after the year 2000, whether they registered in clinicaltrials.gov prior to publication, used active or placebo comparator, and whether or not the trial had industry co-sponsorship. We tabulated whether the study reported a positive, negative, or null result on the primary outcome variable and for total mortality.Results17 of 30 studies (57%) published prior to 2000 showed a significant benefit of intervention on the primary outcome in comparison to only 2 among the 25 (8%) trials published after 2000 (χ2=12.2,df= 1, p=0.0005). There has been no change in the proportion of trials that compared treatment to placebo versus active comparator. Industry co-sponsorship was unrelated to the probability of reporting a significant benefit. Pre-registration in clinical trials.gov was strongly associated with the trend toward null findings.ConclusionsThe number NHLBI trials reporting positive results declined after the year 2000. Prospective declaration of outcomes in RCTs, and the adoption of transparent reporting standards, as required by clinicaltrials.gov, may have contributed to the trend toward null findings.

BackgroundTwenty-five to 30 million Americans live with a rare disease (RD) and share challenges unique to RD. The majority of research on RDs has focused on etiology, treatment and care, while the limited health-related quality of life (HRQL) research has been restricted to single RDs, small samples, or non-validated measures. This study reports HRQL among adults with diverse RDs, and compares their scores to those of the U.S. population and people with common chronic health conditions.MethodsWe conducted a cross-sectional survey of adults living in the U.S. diagnosed with any RD. Participants were recruited through RD organizations and completed the online survey between December 2016 and May 2017 (n = 1218). HRQL was assessed using the standardized Patient-Reported Outcomes Measurement Information System (PROMIS). RDs were classified into categories defined by Orphanet. Means and 95% confidence intervals were calculated for the main sample and for RD categories and were compared to published U.S. population norms and common chronic disease norms. Intercorrelations were conducted between HRQL, demographics, and RD experiences.ResultsWhen compared to the norms for the U.S. population and for those with common chronic diseases, mean HRQL scores were significantly poorer across all six PROMIS domains for the main sample, and were usually poorer when analyzed by sub-sets of specific RD classifications. People with rare systemic and rheumatologic, neurological, and immune diseases had the poorest HRQL. Participants had poorer HRQL if they had multiple RDs, lower income, were female, or older. Having symptoms longer was associated with worse HRQL, however, having a formal diagnosis longer was associated with better HRQL.ConclusionsThis study is the first to examine HRQL in a large, heterogeneous sample of RDs using validated measures. There is a significant disparity in HRQL among people with RD compared to the general population and people with common chronic diseases. Poor HRQL could be attributed to challenges accessing diagnoses, medical information, treatment, psychosocial support, and coping with stigma and uncertainty. As most individuals with RDs will not be cured in their lifetimes, identifying ways to improve HRQL is crucial to patient-centered care and should be a funding priority.

Key Points Question What is the level of investment by the National Institutes of Health (NIH) to fund clinical research focused on Asian American, Native Hawaiian, and Pacific Islander populations? Findings This cross-sectional study found 529 clinical research projects focused on Asian American, Native Hawaiian, and Pacific Islander participants funded by the NIH between 1992 and 2018, composing 0.17% of the total NIH budget. This proportion of the total NIH budget has only increased from 0.12% before 2000 to 0.18% after 2000. Meaning These findings suggest that without overt direction from federal entities, dedicated funds for health disparities research, and parallel efforts to increase diversity in the biomedical workforce, investments may continue to languish for Asian American, Native Hawaiian, and Pacific Islander populations.

This study aimed to determine whether self-assessed puberty is sufficiently reliable and valid to substitute for physician examination when feasibility of physician examination is low (e.g., behavioral research). Adolescents (convenience sample N = 178 endocrinology patients and N = 125 from educational trial; mean age 12.7 and 11.3 years, respectively) participated. Self-assessments were validated against physician Tanner ratings and by associations with bone density, gender, and age. Highest exact agreement between physicians and adolescents was 54% for females' and 55% for males' ratings of pubic hair. More than 85% agreement within one stage was obtained for most measures. Significant associations were found for age and bone between adolescents in earlier and later stages of development and for developmental stage between genders for each age group. Results suggest predictive and discriminate validity of self-assessments. Selfassessments may be useful when estimates of pubertal development are sufficient and for studies barred from physician examination.Pubertal stage and time of puberty are associated with risk behaviors and consequential morbidity. Early pubertal development, as measured by the onset of menarche or secondary sexual characteristics (both male and female),

This study presents population estimates of cigarette use among adults of Korean descent residing in California. Data were drawn from telephone interviews with adults (N=2,830) developed from a random sampling of listed persons in California with Korean surnames. A total of 86% of attempted interviews were completed, and 85% of the interviews were conducted in Korean. Less acculturated men and more acculturated women reported higher present and predicted future rates of smoking after multivariate statistical controls were applied. Sharply divergent rates of cigarette use were found between the genders. Although men did not smoke their first cigarette or smoke cigarettes regularly earlier than women, both groups began smoking later than adults born in the United States. To be effective, tobacco intervention efforts must be tailored specifically to cultures of each minority. Results suggest that acculturation processes influence tobacco use differentially by gender, and future research is needed to identify the implications of processes in tobacco initiation and cessation.