We need new ideas in how to make clinical trials more efficient and cost effective. (Randomized) comparison of different methods for accrual might be of interest; blinded versus unblinded trials; behavioral controls versus placebo controls or usual care controls might be explored.

Since the Pure Food and Drug Act of 1906 and the rise of the FDA, the US federal government has directly inserted itself into medical research, primarily from a business perspective. The Orphan Drug Act of 1983 monetarily incentivized the process for rare diseases, but for ultra-rare diseases of < 1,000 patients, it does not work. Successful drugs for rare diseases have enormous price tags to compensate their development ...more »

To foster the IOM recommendation that every healthcare encounter contribute to a learning healthcare system, would clinical practice structured as N-of-1 trials and documented via EHR/EMR provide strong practice-based evidence?

There is a need for a randomized primary prevention trial comparing the effectiveness of cholesterol treatment strategies based on a high CAC score versus the AHA/ACC 10-year cardiovascular disease risk tool. Include cost-effectiveness as well as clinical effectiveness as endpoints.

Conventional trials are becoming too expensive and complex, and have low recruitment rates and are not generalizable.

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 »

A critical challenge is the need to bring together basic behavioral scientists interested in understanding human behavior at a fundamental level, including how and why people become motivated and capable of undertaking behavioral changes, with clinical researchers interested in developing and testing new strategies for tackling resistant behavioral problems such as obesity and non-adherence.

What are informative and clinically relevant biomarkers of pulmonary hypertension (PH)?

What are the best ways for the NHLBI to advance the evolving science of translating robust evidence into clinical practice domestically and globally? How to personalize broad research evidence for individual patients? How to predict and evaluate the impact of evidence-based interventions? How to identify implementation methods available in industry and elsewhere that work best and are most translatable in healthcare? ...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?

How can NHLBI harness the power of novel technologies involving nucleic acid delivery and gene editing for clinical therapeutics?

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 »

What HIV/AIDS-related clinical research can NHLBI support in the next 5-10 years on the prevention, diagnosis, and treatment of HIV-related heart, lung, and/or blood (HLB) diseases in adults and children to improve heart, lung, and blood health outcomes in HIV infections?

What are the metrics that can predict success of clinical trials?

Will sensitivity to the psychological aspects of obesity, including lifestyle priorities and motivations, improve the efficacy of long-term effectiveness of weight loss and obesity prevention interventions?