1) Phase III efficacy trials of new tuberculosis drugs (e.g., bedaquiline, delamanid, PA-824) that have shown promise in early phase studies for multidrug-resistant tuberculosis. 2) Phase III efficacy trials of new and existing tuberculosis drugs to development very short course regimens (3-4 months). 3) Phase III efficacy trials of new and existing drugs for treatment of latent tuberculosis infection in contacts of ...more »

Although the majority of lung recipients experience significant health improvement, they also frequently face serious symptom distress, impaired physical functioning and poor quality of life due to post-transplant morbidity, such as chronic rejection, infection and multiple side-effects of immunosuppression. a) Conduct clinical trials of interventions designed to maximize clinicians' support of patients' self-management ...more »

We encourage the NHLBI to include research focused on children as a priority in their strategic vision. We encourage them to consider the implications of a life-course approach, where childhood presents a unique opportunity to set the trajectory for health risk as an adult. This also implies that interventions targeting children may have the greatest impact on the population as a whole in the long term.

We believe there should be greater openness to large, simple trials that answer clear questions of interest (e.g. does giving children more fruits and vegetables while changing nothing else lead to weight loss?; does eating breakfast regularly lead to weight loss?; etc.). The conduct of such trials may sometimes be expensive but can sometimes be only modestly costly if they are kept simple. However, it is difficult to ...more »

Pragmatic and adequately powered clinical trials are an ongoing challenge.

­Develop new tools to facilitate identification and recruitment of subjects, remote/wireless data collection, and functional measures.

The use of opioid agents has increased dramatically in the United States. Prescription drug overdoses now account for more deaths than traumatic injuries in the 1-44 age group. There is a need for more high level evidence to target optimal pain management strategies in the acute (ED) and non-acute settings (office practice) especially for chronic disorders such as low back pain, headaches, fibromyalgia, acupuncture etc. ...more »

There are increasing demands on clinicians for clinical productivity and increasing both clinical and research regulatory requirement. A paralle trend is an increase in clinical trials being conducting outside of US, which is a significant concern in terms of US participation and data applicability to US patients, and the quality of trials data from other regions. These trends have multiple roots spanning from patients ...more »

The initiation of clinical trials remains difficult, time-consuming, and costly. Repetitive institutional review board oversight is one of several obstacles to efficient clinical trial initiation and completion. New strategies for addressing duplicative regulatory standards are necessary to ease the initiation and completion of trials.

How can NHLBI support studies that produce in-vitro assays to predict clinical response and ways to translate those results into patient therapies through novel clinical trials, including those for small patient populations and rare diseases?

There are numerous obstacles to the timely completion of trials, and there is a crisis in US enrollment rates. Overcoming barriers to timely completion of clinical trials would have a profound impact on accelerating research translation to improving health. Clinical investigations are necessary to advance the prevention, diagnosis, treatment and cures of human disease. The rate of basic scientific discovery has overwhelmed ...more »

There is a need to fundamentally rethink the approach to translation and dissemination of clinical trial results. The current approach relegates dissemination as a side effect to the end of a trial and does not require sufficient funds be set aside to conduct adequate outreach. As a result, it takes up to 17 years for the results of trials to be adopted and used in everyday practice. It is time for a new model which re-prioritizes ...more »

Prevention trials, implemented to reduce or delay progression to overt disease in a population at risk to the disease, are an important approach to health promotion. Therapies shown to reduce disease severity in patients with a specific disease are obvious, but not the only, candidates for a prevention trial in populations at high risk for prevalent diseases (such as heart failure, diabetes, COPD, asthma in children). ...more »

Clinical research, especially randomized pharmaceutical clinical trials, poses many unique challenges compared to research in adult subjects. In pulmonary arterial hypertension, a disease characterized by high blood pressure of the lungs with increased pulmonary vascular resistance leading to right ventricular failure, there are 12 FDA-approved PAH-targeted therapies for adults. None of these medications are currently ...more »

In 2010, the IOM issued a report stating that waste accounted for 30% of health-care spending, or some $750 billion dollars annually, approximately 25 times the annual NIH budget. How can we address and avoid waste and low-value care? Like any complex problem, there are myriad causes and no simple solutions. Defensive medicine, financial incentives, and physician knowledge deficits all contribute and represent potential ...more »

What structural changes need to be implemented in the health-care community in order to support the use of genomic information in clinical trials and drug development for hematologic diseases?