(@katherinek)

Goal 2: Reduce Human Disease

How can we increase the pharmaceutical clinical research of targeted therapies in pediatric PAH patients, including encouraging

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 »

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(@nhlbiforumadministrator)

Goal 2: Reduce Human Disease

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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(@nhlbiforumadministrator)

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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(@nhlbiforumadministrator1)

Goal 2: Reduce Human Disease

Risk-benefit of oral hypoglycemic medication in type 2 diabetes

Is an increase in macrovascular endpoints outweighed by the benefit in microvascular end points in new oral type 2 diabetes drugs?

 

It would go against the current regulatory paradigm in type 2 diabetes. Although drug companies do not like the current paradigm, they would prefer to go back to the pre-rosiglitazone state of affairs, in which new drugs had only to prove that they lowered blood glucose and HbA1c.

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(@javed.butler)

Goal 3: Advance Translational Research

Improving the conduct of cardiovascular clinical trial

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 »

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(@nhlbiforumadministrator1)

Goal 3: Advance Translational Research

Embedding Clinical Trials in Learning Health Systems

What are the best methods for using genotype information and other EMR data to randomize heart, lung, blood, sleep patients to different treatment strategies? One big challenge is how to consent patients for this sort of trial. Must patients be consented separately for every such trial or could there be blanket consent for participating in the learning health care model? This would also require a paradigm shift in how ...more »

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(@wheeze)

Goal 2: Reduce Human Disease

Heterogeneity in Asthma Phenotypes

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: Asthma appears to be due to heterogenous etiologies.  To better characterize the various phenotypes and potential etiologies, it would be important to create more epidemiologic and biomarker focused databases, which could ...more »

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