Assessing regression to the mean effects in health care initiatives
BMC: September 28, 2013 Background Interventions targeting individuals classified as “high-risk” have become common-place in health care. High-risk may represent outlier values on utilization, cost, or clinical measures. Typically, such individuals are invited to participate in an intervention intended to reduce their level of risk, and after a period of time, a follow-up measurement is […]
Power calculation for overall hypothesis testing with high-dimensional commensurate outcomes
Wiley: SEP 30, 2013 he complexity of system biology means that any metabolic, genetic, or proteomic pathway typically includes so many components (e.g., molecules) that statistical methods specialized for overall testing of high-dimensional and commensurate outcomes are required. While many overall tests have been proposed, very few have power and sample size methods. We develop accurate […]
Patient-Reported Outcome Alerts Ethical and Logistical Considerations in Clinical Trials
JAMA: September 25, 2013 The assessment of patient-reported outcomes (PROs) in clinical trials poses a number of potential problems. What happens when a patient reports a severe symptom and no one is monitoring that information; for example, when questionnaires are not reviewed until the end of a study? Do hospitals or researchers face liability if a patient reports […]
Empowered by Fox Trial Finder, patients play a critical role in tackling the chronic challenge of recruitment
Phrma: 9/25/13 Guest Perspective: Elizabeth Joyce, Marketing & Communications Officer, The Michael J. Fox Foundation for Parkinson’s Research Recruiting volunteers to participate in clinical trials is a well-known and enduring obstacle to research progress, and the effect is staggering: 85 percent of trials across all diseases face delays and 30 percent never even get off the ground […]
An estimate of the science-wise false discovery rate and application to the top medical literature
Biostat: September 25, 2013 The accuracy of published medical research is critical for scientists, physicians and patients who rely on these results. However, the fundamental belief in the medical literature was called into serious question by a paper suggesting that most published medical research is false. Here we adapt estimation methods from the genomics community to […]
Subjective endpoints in clinical trials: the case for blinded independent central review
Dove press: 9/25/13 Abstract: Primary efficacy and safety endpoints in clinical trials are often subjective assessments made by site personnel. For international confirmatory trials conducted over broad geographic regions and different clinical practice settings, variability in these subjective assessments can be substantial. Centralized endpoint assessment committees (EACs) offer a mechanism through which to reduce assessment bias and […]
Large Simple Trials and Knowledge Generation in a Learning Health System – Workshop Summary
IOM: September 24, 2013 Despite a robust clinical research enterprise, a gap exists between the evidence needed to support care decisions and the evidence available. Streamlined approaches to clinical research provide options for progress on these challenges. Large simple trials (LSTs), for example, generally have simple randomization, broad eligibility criteria, enough participants to distinguish small […]
