New Clinical Research Methodologies for Rare Diseases
What innovative methodologies applicable to small cohorts and rare outcomes can better ensure the success of clinical and implementation studies in the rare diseases affecting heart, lung, blood, and sleep?
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Life-Course Approach to Science
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.
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Tools for Clinical Research
Clinical trials are at a crossroads. They are too expensive, take too long, and often can't recruit adequately. A critical challenge is to develop tools that can change the cost, time, and recruiting practices for clinical trials.
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Utilizing Competition to Enhance NHLBI’s Clinical Trial Enterprise
During this period of diminishing Federal resources and funding, what novel methods can be employed to effectively conduct clinical trials that address existing and emerging medical and public health issues?
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Assessing current methodologies for clinical trials
Are the current methodologies for clinical trials still the best practices for conducting efficient clinical trials?
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Goal 3: Advance Translational Research
Increased receptivity to probative programmatic trials
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 »
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Goal 3: Advance Translational Research
Clinical Trial Methodology
As the current chair of the Research and Training Division, I would like to convey that the AAAAI membership would like the NHLBI to consider the following in the development of its strategic plan:
Are the current methodologies for clinical trials still the best practices for conducting efficient clinical trials?
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Goal 3: Advance Translational Research
Genome Profiling
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?
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What is the optimal treatment goal for hypertension?
In adults without diastolic hypertension (DBP ≥ 90 mm Hg), what is the best way to determine at what systolic blood pressure should treatment be initiated?
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Goal 3: Advance Translational Research
T4 Translation Research Informing Early Stage Translational Research
There is a need to utilize insights gained from T4 translation research and implementation science to inform the design and execution of early-stage translational research and clinical trials.
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THE RELEVANCE OF PREVENTION TRIALS
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 »
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Goal 3: Advance Translational Research
Improving Drug Safety through Precompetitive Research
The lack of transparency in Pharma clinical studies and the incomplete knowledge of the effect of genetic profiles and pharmacological factors on drug toxicities are challenges in decreasing drug development costs and increasing drug safety.
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Phase III efficacy trials of tuberculosis drugs
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 »
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Cardiometabolic Disease Risks Associated with Sleep Deficiency
How does insufficient sleep duration, irregular timed sleep schedules, and poor sleep quality contribute to the pathophysiology of lung, heart and blood diseases?