Goal 3: Advance Translational Research

Development of integrated research teams

There needs to be development of integrated research teams that include clinicians, scientists, engineers, etc.

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 : Research Advocacy Committee, American Thoracic Society

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Goal 3: Advance Translational Research

Combining Research Efforts to Find Answers Faster

Combining Research Efforts to Find Answers Faster - collaboration across organs should be encouraged and practiced.

Submitted by (@nhlbiforumadministrator)

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

Feasibility and challenges of addressing this CQ or CC :

The ATS convened the first Fibrosis Across Organs (FAO) Effort in 2012 with the belief and understanding that FAO would move the science forward faster in fibrotic diseases of all kinds including the lungs, heart, liver, kidney, and skin as well as other areas. Since that time, progress is being made in research centers where collaboration across organs is being encouraged and practiced.

Name of idea submitter and other team members who worked on this idea : Research Advocacy Committee, American Thoracic Society

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Goal 3: Advance Translational Research

Move toward more probative research

As some of us described in a recent publication, in the fields of nutrition and obesity, and perhaps in other fields as well, there is often a great deal of research which uses up resources, investigator time, journal pages, and attention span for questions that do not advance the field. We call for scientists, reviewers, and funding decision makers to collectively ask much more rigorously, "How will this proposed study ...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 :

A quintessential example of this state of affairs is the large number of studies involving breakfast consumption and obesity. On the order of 100 studies have been done testing the association of breakfast eating vs. breakfast skipping with obesity. This seemed to lead to the belief and widely stated public health message that breakfast should be consumed to prevent obesity or promote weight loss. While the first several such epidemiologic studies were reasonable to conduct, what was needed thereafter were RCTs to test for causal effect. Instead the scientific community provided itself with a large body of observational studies to the point where an association was established far beyond any reasonable doubt (P≈10-42). Only recently have a handful of investigators conducted the RCTs needed to advance knowledge further, i.e. the "probative" studies. These have failed to support the hypothesis that breakfast consumption vs skipping leads to better weight control.

Feasibility and challenges of addressing this CQ or CC :

Similarly, we seem to be faced with a large stream of studies of varying quality testing the hypothesis that modest increases in physical activity in school settings for children will lead to important differences in weight outcomes. Yet, considerable research has already demonstrated to a reasonable degree of certainty that school-based programs with modest increases of physical activity do not have major effects on children's BMIs. This does not mean that physical activity is not important for other outcomes, or that there is not some way of inducing physical activity that would lead to major changes in BMI, but repetitively trying one minor variation on school-based programs after another is not the best use of our resources. These are just examples.

Name of idea submitter and other team members who worked on this idea : David B. Allison, Ph.D.; Kevin Fontaine, Ph.D.; Kathryn A. Kaiser, Ph.D.; Andrew W. Brown, Ph.D.; Edward C. Archer, Ph.D.

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Goal 3: Advance Translational Research

Accelerating Translational Research

NHLBI should define a strategy to promote collaborative research between clinician-scientists who perform patient-oriented research, and basic scientists who focus on the preclinical realm. There is not enough cross-talk between these two groups, and yet much to be gained from increasing interactions between the two (e.g. accelerating the translation of bench science findings into the clinic). In particular, funding strategies ...more »

Submitted by (@golan0)

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

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Goal 1: Promote Human Health

Hierarchical control of cardiac excitability

Imbalances in membrane excitability underlie a broad range of cardiac arrhythmias and conduction defects. Although we now know the genes encoding almost all ion channels, we have little understanding of how the macromolecular composition and relative numbers of different channel types is achieved to exert exquisite control over membrane potential changes in time. Even minor changes in this balance can lead to sudden ...more »

Submitted by (@garobert)

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

Details on the impact of addressing this CQ or CC :

The mechanisms involved reside at every conceivable level – genomic and epigenetic, transcriptional, translational and posttranslational. The tools required include structural biology, super resolution microscopy, single-molecule measurements, advanced molecular biology approaches, and bioinformatics and systems biology approaches. Fulfilling this unmet need will inform any system in which stoichiometry of macromolecular complexes critically determines normal function, and will therefore have a broad, transformative reach. The work will reveal novel mechanisms that will serve as targets for disease and therapeutic approaches.

Feasibility and challenges of addressing this CQ or CC :

Addressing this problem requires coordinated efforts by multidisciplinary investigators using diverse approaches as described above. Above all it requires a commitment of resources to basic science advances without which translation is impossible.

Name of idea submitter and other team members who worked on this idea : Gail Robertson

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Goal 3: Advance Translational Research

Research teams and interdisciplinary science

Breakdown silos to encourage team/interdisciplinary science, cross-institutional research teams.

Submitted by (@societyforvascularsurgery)

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 : Society for Vascular Surgery

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Goal 4: Develop Workforce and Resources

Leveraging big data for T1 translational research

How best to train T1 investigators in using big health care data to test their basic science hypotheses related to heart, lung, blood, and sleep disorders and thus generate sufficient confirmation to justify clinical interventions.

Submitted by (@collerb)

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

Details on the impact of addressing this CQ or CC :

• Hypotheses that emerge from small studies of patients focused on mechanistic questions often are not able to be tested at the population level and thus remain unconfirmed. The increasing availability of big data from EHRs permits corroboration at the population level, but requires skills in framing queries and minimizing bias and confounders.

• The emergence of large clinical data sets such as PCORNet and the NIH Collaboratory make this particularly timely.

• By sampling existing data sets rather than having to conduct new randomized studies, this type of research can be performed at relatively low cost and in a much more timely way.

 

T1 investigators are not usually trained in the techniques of using large clinical data sets and so require targeted training.

Feasibility and challenges of addressing this CQ or CC :

Developing educational programs for T1 investigators should be straightforward. Resources will be required to support the queries by T1 investigators required as part of the training, but this should be modest compared to other forms of research.

Name of idea submitter and other team members who worked on this idea : Barry Coller

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Goal 3: Advance Translational Research

Core funding for successful PBRNSs

Practiced-Based Research Networks (PBRNs) are critical for the conduct of pragmatic real world studies. Most PBRNs struggle to support their infrastructure. Funding is needed to support core administration and IT, i.e. electronic interfaces and data extraction, transfer and loading from multiple EHRs and PBRN registries. Continuous if not core funding is needed whether though Center (P) awards, through through NHLBI ...more »

Submitted by (@kevinfiscella)

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

Details on the impact of addressing this CQ or CC :

Pragmatic research designed to answer real world questions is best conducted in the real world of office-based practices. This type of research is critical to informing health care innovations. Dynamic registries are needed to answer questions quickly. Once these community laboratories are lost, they are expensive to replace or revive.

Feasibility and challenges of addressing this CQ or CC :

Competing priorities during an era of flat funding

Name of idea submitter and other team members who worked on this idea : Kevin Fiscella, MD, MPH

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Goal 2: Reduce Human Disease

Basic Research & Precision Medicine

How can NHLBI best encourage basic research areas that are critical to the development of precision medicine approaches for lung disease?

Submitted by (@skrenrich)

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 : Cystic Fibrosis Foundation

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Goal 4: Develop Workforce and Resources

Training for radiologist researchers for effective translational research

Critical Challenge

Submitted by (@str0001)

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

Details on the impact of addressing this CQ or CC :

As targeted therapy and molecular mechanisms of disease are emerging, a mechanism to improve the ability of radiologists to perform translational research is crucial. Such knowledge is essential for collaborative multidisciplinary research that ultimately leads to imaging as disease-specific diagnostic and therapeutic tools to combat pulmonary and cardiovascular disease.

Feasibility and challenges of addressing this CQ or CC :

Knowledge in the molecular mechanisms of disease and the potential for imaging technology to advance via targeted imaging agents, positron emission tomography (PET), functional MR methods, PET/computer tomography, and PET/MR is increasing. The radiologist has in depth expertise within imaging technology, performance of studies, and diagnostic abilities of imaging techniques. A program directed towards developing imagers towards translational imaging research will include in-depth education and training in lung physiology, pulmonary disease mechanisms, multimodality imaging bridging CT, PET/CT, MR and PET/MR, and the molecular techniques. With such knowledge and training, radiologists will be prepared to serve as principal investigators and collaborators in multidisciplinary teams. An understanding of imaging technologies and their capabilities, the clinical challenges, and molecular techniques will enable imagers to provide innovative solutions to diagnostic dilemmas in pulmonary and cardiovascular disease.

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

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Goal 4: Develop Workforce and Resources

Funding & Review Mechanisms

Funding and review mechanisms are essential resources that can facilitate or hinder innovative research to meet the NHLBI goals. These resources must be refocused to result in identification and funding of the type of innovative research being sought by NHLBI. The opportunities for leveraging available NHLBI funds with other sources should be considered formally as a component of overall budget planning, not just on ...more »

Submitted by (@media0)

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 impact should be more equitable access to funds, as perceived by potential recipients; provision of funds for high-priority topics; and revitalized, refocused review groups that are willing and able to consider recommending high risk, innovative approaches.

Feasibility and challenges of addressing this CQ or CC :

This is a very specific challenge that may require incremental implementation with some experimentation/pretesting and solicitation of ideas from a range of stakeholders identified during this goal-setting exercise. While the Institute may be able to make changes in how funding mechanisms are employed, making changes to review group membership may be more challenging, especially in the current context of recruiting members. Incentives for this service may also need to be reviewed, innovatively, under current budget constraints.

Name of idea submitter and other team members who worked on this idea : Sonja McKinlay. Team Members: Susan Assmann and Paul Stark

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Goal 2: Reduce Human Disease

Pick Me, Pick Me! The Challenges of Being at the Mercy of a Few

One significant challenge if you're a patient or a patient advocate is "who is going to find a life-saving treatment or cure for my disease. Who is going to Pick me and my disease?". The answer is complex but lies within the research community's priorities and interests. Think for a moment like a patient. You are at the mercy of the research community at large, government agencies and companies willing to make your ...more »

Submitted by (@teresabarnes)

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

Details on the impact of addressing this CQ or CC :

So how do we address the issues with the "Pick Me Approach"? Perhaps there are some clues.. The current approach that a few major things need to happen in order for patients to get treatments is: 1. The NIH has to identify a disease as a priority and provide intramural or extramural research funding for it 2. researchers need to be interested in the disease and seek/receive funding for their research and 2. The research that is done results in a device, tool or treatment that one or more companies is willing to invest in in the private sector to bring to market. NIH and FDA (as government agencies run by public dollars) have perhaps an obligation to identify gaps and needs/opportunities and fill them or enable them to be filled. Not to just stand by as guidance for researchers and companies doing the work they are interested in, but as facilitators of progress. Perhaps all stakeholders including NIH, researchers, clinical practitioners, patients/patient advocates and industry can come together to identify and address the areas of need and plot the path forward. By addressing the gaps and opportunities as the for-profit sector does when planning for engagement in a market, many more areas of research will benefit in the years to come. And ultimately patients will benefit.

Feasibility and challenges of addressing this CQ or CC :

The Office of Rare Diseases at FDA has made an impact with a similar strategy. Rather than acting as a traditional guidance agency, this office analyzes, reviews and identifies gaps and opportunities and develops strategies for them. It puts out RFPs for the areas identified as priority (and multiple stakeholders are provided opportunities for input) and fills the areas with potential solutions.

 

The NIH could consider reviewing its portfolio and evaluating its successes and failures over the last few decades. It could publish a record of the outcomes of all studies it has funded perhaps stratified by organ, system or disease areas. It could identify areas of overlap or duplication and find ways to help multiple disease areas benefit from the research in each.

Name of idea submitter and other team members who worked on this idea : Teresa Barnes and Dolly Kervitsky

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