Future Directions for Cardiovascular Disease Comparative Effectiveness Research Report of a Workshop Sponsored by the National Heart, Lung, and Blood Institute

Comparative effectiveness research (CER) aims to provide decision makers with the evidence needed to evaluate the benefits and harms of alternative clinical management strategies. CER has become a national priority, with considerable new research funding allocated. Cardiovascular disease is a priority area for CER. This workshop report provides an overview of CER methods, with an emphasis on practical clinical trials and observational treatment comparisons. The report also details recommendations to the National Heart, Lung, and Blood Institute for a new framework for evidence development to foster cardiovascular CER, and specific studies to address 8 clinical issues identified by the Institute of Medicine as high priorities for cardiovascular CER

The Testosterone Trials: Seven coordinated trials of testosterone treatment in elderly men

Background The prevalence of low testosterone levels in men increases with age, as does the prevalence of decreased mobility, sexual function, self-perceived vitality, cognitive abilities, bone mineral density, and glucose tolerance, and of increased anemia and coronary artery disease. Similar changes occur in men who have low serum testosterone concentrations due to known pituitary or testicular disease, and testosterone treatment improves the abnormalities. Prior studies of the effect of testosterone treatment in elderly men, however, have produced equivocal results. Purpose To describe a coordinated set of clinical trials designed to avoid the pitfalls of prior studies and to determine definitively whether testosterone treatment of elderly men with low testosterone is efficacious in improving symptoms and objective measures of age-associated conditions. Methods We present the scientific and clinical rationale for the decisions made in the design of this set of trials. Results We designed The Testosterone Trials as a coordinated set of seven trials to determine if testosterone treatment of elderly men with low serum testosterone concentrations and symptoms and objective evidence of impaired mobility and/or diminished libido and/or reduced vitality would be efficacious in improving mobility (Physical Function Trial), sexual function (Sexual Function Trial), fatigue (Vitality Trial), cognitive function (Cognitive Function Trial), hemoglobin (Anemia Trial), bone density (Bone Trial), and coronary artery plaque volume (Cardiovascular Trial). The scientific advantages of this coordination were common eligibility criteria, common approaches to treatment and monitoring, and the ability to pool safety data. The logistical advantages were a single steering committee, data coordinating center and data and safety monitoring board, the same clinical trial sites, and the possibility of men participating in multiple trials. The major consideration in participant selection was setting the eligibility criterion for serum testosterone low enough to ensure that the men were unequivocally testosterone deficient, but not so low as to preclude sufficient enrollment or eventual generalizability of the results. The major considerations in choosing primary outcomes for each trial were identifying those of the highest clinical importance and identifying the minimum clinically important differences between treatment arms for sample size estimation. Potential limitations Setting the serum testosterone concentration sufficiently low to ensure that most men would be unequivocally testosterone deficient, as well as many other entry criteria, resulted in screening approximately 30 men in person to randomize one participant. Conclusion Designing The Testosterone Trials as a coordinated set of seven trials afforded many important scientific and logistical advantages but required an intensive recruitment and screening effort

American Society of Echocardiography Cardiovascular Technology and Research Summit : a roadmap for 2020

To realize the great potential of ultrasound for improving patient outcomes and offering novel therapy, closer partnerships among scientists, engineers, and those involved with research funding are needed. Improvements in technology, developed with these partnerships, will be necessary to advance 3D echocardiography, deformation imaging, and the quantitative assessment of VHD. Stepwise research studies will advance the development of sonothrombolysis and drug and gene delivery. The development of intelligent software offers the potential to improve the quality and efficiency of echocardiographic examinations.With demonstration of the value, reproducibility, and feasibility of quantitative echocardiographic biomarkers, users can then make the strong argument that echocardiography is a superior technology as a research tool for clinical trials in which large numbers of subjects must be imaged efficiently, cost-effectively, and safely. The leadership of the ASE will discuss the recommendations put forth in this document and formulate an action plan. Research remains an important mission of ASE, and the society must find a way to continue to expand and advance this endeavor to meet the needs of our patients