Goal 2: Reduce Human Disease

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.

Submitted by (@nhlbiforumadministrator)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Developing tools to facilitate and streamline the day-to-day performance of clinical trials would help investigators and NHLBI alike. Trials would recruit more quickly, at less cost. We could, therefore, potentially fund more trials. In addition, if we have common templates, this would facilitate a number of things ranging from peer review to IRB submissions, and would also be beneficial to new investigators by providing a framework for them to use.

Feasibility and challenges of addressing this CQ or CC :

Some of it will involve 'just doing it' - for example, we have the capability now to require common protocol and informed consent formats. We have sufficient data to develop performance milestones and implement them. The hardest part will be figuring out how to develop pools of patients, or with which organizations to collaborate in order to achieve this. However, 5-10 years should be ample for this and related activities.

For example, we need to emulate and collaborate with other organizations in figuring out how to identify large numbers of patients willing to participate in our trials. We need performance milestones and metrics, and we need tools like common templates for protocols and informed consent forms that all NHLBI-funded trials would use.

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Goal 2: Reduce Human Disease

Reducing injury following ischemia and reperfusion

What therapeutic strategies can reduce tissue and organ injury caused by total body ischemia and reperfusion?

Submitted by (@rebecca.lehotzky)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Name of idea submitter and other team members who worked on this idea : AHA Staff & Volunteers

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Goal 3: Advance Translational Research

Risk Mitigation of Implantable Cardiovascular Devices

There is a serious need to improve implantable cardiovascular devices to eliminate, or at least substantially reduce, the risk of serious adverse events from occurring to more effectively treat patients.

Submitted by (@nhlbiforumadministrator1)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Cardiovascular devices would be more widely used to treat such cardiovascular diseases as heart failure, coronary artery disease, valvular disease and, therefore, have a greater public health benefit. Furthermore, costs associated with treating the serious adverse events (e.g. hospitalizations, therapeutics, etc.) would decrease.

Feasibility and challenges of addressing this CQ or CC :

Technologies such as surface coatings and topographies as well as evolved design tools are under development already or are being used to mitigate the risks of devices currently being used or under development. A more concerted effort to do this would almost certainly help to substantially reduce the risks further, especially over the next 5-10 years.

Despite the widespread and growing use of implantable cardiovascular devices such as ventricular assist devices, stents, and prosthetic heart valves, substantial serious adverse events such as infections, neurological events, and bleeding limits the efficacy and use of the devices. Scientific and technological breakthroughs in such areas as biomaterials, design tools, coatings, and concomitant pharmacologic therapy are needed to address this challenge.

Name of idea submitter and other team members who worked on this idea : NHLBI Staff

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9 up votes
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Goal 3: Advance Translational Research

Translation Research Dissemination & Implementation Frameworks

We need to identify and test the proven effective dissemination and implementation frameworks that are relevant to heart, lung, and blood disorders in order to scale up evidence-based interventions in real world settings, ultimately improving health equity among minority populations, including low income minority residents living in public housing.

Submitted by (@nhlbiforumadministrator1)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

• Ability to determine how much of an evidence based intervention can be sustained in real world settings.

• Add utilization of D&I frameworks to researcher’s core competency training skills.

• Promote long term sustainability of evidence based interventions.

Feasibility and challenges of addressing this CQ or CC :

• Researchers from the 2014 NIH’s Annual Conference on the Science of Dissemination and Implementation: Transforming Health Systems to Optimize Individual and Population Health presented compelling evidence that dissemination and implementation frameworks are an effective means to scaling up evidence based interventions.

Name of idea submitter and other team members who worked on this idea : NHLBI Staff

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Goal 3: Advance Translational Research

Heart rate regulation as a therapeutic goal

Heart rate regulation is emerging as an effective treatment for heart failure and angina. However a critical challenge in the development of new therapeutics is the paucity of information about molecular and cellular determinants of heart rate.

Submitted by (@catherine.proenza)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

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Goal 3: Advance Translational Research

Community Collaborative Research Targeting Populations with CVD

In what ways can researchers better collaborate with community representatives from populations with high prevalence / morbidity / mortality of cardiovascular disease (CVD) to enhance and sustain interventions and achieve improved health outcomes? How can a combination of health behaviors and risk factors be used to conduct community-engaged research to prevent and treat CVD, chronic obstructive pulmonary disease (COPD) ...more »

Submitted by (@nhlbiforumadministrator)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Details on the impact of addressing this CQ or CC :

Studies designed to engage target populations at high risk for diseases such as CVD, COPD and stroke would help prevent and effectively treat such diseases. Comprehensive interventions addressing health behaviors and risk factors especially in co-morbid conditions will promote the administration of suitable therapies and adherence to medication regimens. Community consultation would generate more effective interventions and accelerate the translation of research results into practice.

Feasibility and challenges of addressing this CQ or CC :

The NHLBI formed COPD working group could be enhanced to engage additional stakeholders like community representatives and community-engaged researchers. Research could be conducted to implement the AHA 2020 impact goals to reduce CVD morbidity and mortality. Cultural adaptations of proven modalities are needed to reach populations most at risk to reduce health disparities. These populations include African Americans, Hispanics (including their subpopulations), and American Indians.

Name of idea submitter and other team members who worked on this idea : NHLBI Staff

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25 up votes
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Goal 3: Advance Translational Research

Better disease models

Many diseases of the heart, lung and blood systems are studied using animal models, often with genetically engineered mice. However, while mice get models, humans get diseases. Too many grants are devoted to curing models, a practice encouraged by many high profile journals who want to see “proof” in a standard model of disease. Much less time, effort and money will be wasted on developing ineffective therapies if focus ...more »

Submitted by (@nhlbiforumadministrator)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Morphologic appearance is necessary but insufficient as a criterion for validating a model. Comparative “omics” should be strongly encouraged. It is a waste of resources to develop therapies based on the effects of yet another gene knock out in something as unphysiological as atherosclerotic lesions in an ApoE knock out mouse or asthma in a mouse with eosinophilic airspace disease that largely ignores the bronchial tree or the infusion “stem cells” on cardiac function in a mouse with an experimental myocardial infarct. These are simply prominent examples of widely used standard models with little real utility. Therapies based on such approaches are likely to fail. Better models that more realistically capture phenotypic features of real diseases could lead to more promising therapies.

Feasibility and challenges of addressing this CQ or CC :

Model development is not very sexy and stands a better chance of acquiring support outside the standard study section review mechanism where proposals are judged by investigators with a vested interest in (and academic career based on) using existing models, despite their poor track records.

Name of idea submitter and other team members who worked on this idea : Jordan S. Pober

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126 up votes
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Goal 1: Promote Human Health

INVESTIGATE DIFFERENTIAL VULNERABILITY TO SLEEP DEFICIENCY AND CIRCADIAN DISRUPTION

Studies in different subjects have shown that there are major individual differences in response to sleep loss and circadian disruption. Twin studies have shown that this is heritable. There needs to be an intensive effort to assess basis of these individual differences. This could include in-depth phenotyping studies, e.g., neuroimaging, genetic studies, “-omic” studies, epigenetic changes, etc.

Submitted by (@jnoel0)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Details on the impact of addressing this CQ or CC :

There are major individual differences in response to sleep loss (both acute and chronic) and to circadian disruption. This has major impact both in terms of health consequences and in safety. Some individuals are particularly vulnerable to sleep loss and hence are more likely to have adverse consequences of losing sleep—increased risk of crashes, errors by physicians, etc. They are also more likely to be affected by metabolic and other consequences if they have chronic insufficient sleep.

 

Identifying the basis of these individual differences will have several impacts:

 

1. It will provide likely targets for development of biomarkers to assess effect of sleep loss and circadian disruption.

2. It will provide tools to risk stratify individuals and to employ preventative strategies to reduce risk of major adverse consequences.

3. It will identify novel pathways that could be the target for future intervention studies.

Feasibility and challenges of addressing this CQ or CC :

These studies are highly feasible. Phenotyping and recruitment strategies to study this question have been established in many laboratories. Moreover, more laboratories are utilizing genetic, -omic approaches and epigenetic approaches that could be applied to this question. There is also a developing repertoire of neuroimaging studies that can be applied to address this question.

Name of idea submitter and other team members who worked on this idea : Sleep Research Society

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155 net votes
213 up votes
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Goal 4: Develop Workforce and Resources

NIH trans-IC office of critical care research

Would an NIH trans-IC office of critical care research improve coordination and strategic planning across?

Submitted by (@nhlbiforumadministrator)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Name of idea submitter and other team members who worked on this idea : American Thoracic Society member

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Goal 3: Advance Translational Research

ESTABLISHMENT OF APHERESIS MEDICINE CONSORTIUM TO ADVANCE DEVELOPMENT OF EVIDENCE BASED THERAPIES

The apheresis medicine encompasses treatment of numerous diseases many of which are directly related to blood, lung and heart. There is a need to establish consortia for Apheresis Medicine to facilitate networking, information exchange and research collaboration among investigators, including junior investigators. These consortia would perform basic science as well as translational research and investigate the best pathways ...more »

Submitted by (@zbigniew.m.szczepiorkowski)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

The creation of one or more Research Consortia devoted to research in Apheresis Medicine would allow for the establishment and support of a core group of investigators and institutions representing key specialty areas across the spectrum of Apheresis Medicine. An initial focus would be translational research priorities. In addition, we believe that a strong U.S. based consortium would facilitate participation of international investigators and societies, which would improve patient accrual on studies, especially those patients with rare disorders or who have rare indications for apheresis therapy. Such a group would significantly enhance the likelihood of completing high quality studies.

 

There is increasing national interest in developing new registries, bio-repositories and data-repositories. Very often such efforts do not include information regarding apheresis nor do they consider apheresis information as being important data points. A centralized, well organized and sustainable registry, either established and/or new for Apheresis Medicine, would be of great value to study the outcomes of therapeutic apheresis for different disease conditions. This need is particularly relevant for rare disorders and rare indications, for which a pilot effort is already being undertaken and sponsored by ASFA- the American Society for Apheresis.

Feasibility and challenges of addressing this CQ or CC :

Feasibility: There are hundreds of thousands apheresis procedures performed each year in the US. Many of these procedures are performed in tertiary and quaternary academic medical centers. These centers have experience in education, basic science, translational research and clinical trials. Despite the low frequency of some diseases there is a real possibility of performing clinical trials in the context of multicenter consortium. Such consortium could also assist in development of new projects related to Apheresis Medicine (both basic science and clinical) which then can proceed to clinical trials quickly as the appropriate infrastructure will be created within the consortium. This infrastructure would also serve education of the new investigators.

Challenges: Standardization of approaches between different medical centers might be initially difficult, but as it has been shown many centers follow in their clinical practice ASFA designated indications. Setting up research priorities for the consortium might be challenging as well as accrual patients to clinical trials within the consortium. Though standardization of the apheresis devices is not feasible, this could be mitigated by appropriate study designs. The number of investigators in apheresis medicine is limited but the consortium may serve as a great platform to expand its numbers through collaborative efforts of involved centers.

Name of idea submitter and other team members who worked on this idea : Zbigniew M. Szczepiorkowski, Yanyun Wu on behalf of ASFA

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Goal 4: Develop Workforce and Resources

Need to train and nurture more "translators"!

One of the major challenges in translating from bench-to-bedside and back is communication: the ability of basic and clinical scientists to understand each other's scientific language to be able to appreciate the importance of the other’s research questions and findings.

Submitted by (@nhlbiforumadministrator1)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Having an increased number of researchers able to connect dots across the continuum of translational research should increase overall success of translation of ideas into health.

Feasibility and challenges of addressing this CQ or CC :

This requires "rearranging" of already existing elements. Within 5-10 years of running specifically designed re/cross -training programs, the effects might be widely visible.

Basic scientists usually do not keep up with the latest outcomes of important clinical studies, and thus might miss important starting points for new basic research (e.g., negative trials that suggest the need for new hypotheses). The great majority of clinical scientists do not attend basic scientific sessions because are turned off by the specialized (dense/obscure) scientific terms used. Those who are interested in being translators have a hard time integrating and surviving in the "opposite camp" (i.e., at many medical schools, basic scientists are expected to bring in all their salary in a clinical department, and clinicians get little protected time for basic research)

Name of idea submitter and other team members who worked on this idea : NHLBI Staff

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22 net votes
39 up votes
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Goal 3: Advance Translational Research

Telemonitoring and COPD Exacerbations

What is the effectiveness of the telemonitoring system to detect and trigger treatment for COPD exacerbations?

Submitted by (@abryan)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

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8 up votes
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