Goal 3: Advance Translational Research

NHLBI Cohort Populations for T4 Implementation Research

How best can NHLBI observational cohorts be utilized to study observational T4 Implementation Research among both general and vulnerable US populations?

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 :

• Would help identify key factor associated with successful implementation that could be studied in interventional T4 implementation research

• Result would refine implementation strategies and health and social policy aimed to reduce heart, lung, blood, sleep diseases and conditions

• Builds on excellent established platform of research with high quality outcomes in well characterized study populations over long term follow-up.

Feasibility and challenges of addressing this CQ or CC :

• Big data is developing methods to link large data sets from national, state, and community level surveys – surveys that can define exposures to various policies and interventions in place, time, and population.

• A family of high quality cohorts are available for ancillary observation studies

• Collection of community level and more broad policy level exposures is feasible through data already collected and through potentially new data collection.

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

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11 up votes
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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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1 up votes
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Goal 3: Advance Translational Research

Culturally Specific Preventative research

There are ample research evidence related factors contributing to obesity,type 2 diabetes, cardiovascular disease.The research grant money is diverted heavily on "novel" topic such as genes.The preventative efforts are the key to tackle the issues.Often times,researchers on cardiovascular prevention find it difficult to add novel ideas to convince the grant reviewers in their application.There are many communities here ...more »

Submitted by (@athomas4)

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

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7 up votes
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Goal 4: Develop Workforce and Resources

Suipport new research using the R21 mechanism

The decision by NHLBI to not support the R21 mechanism may be stifling new and innovative research, partcularly by young investigators who do not have a track record of R01 funding. The critical challenge is to keep funding new ideas from younger investigators to keep their careers viable while they obtain the data and publications necessary for further R01-level funding.

Submitted by (@georgeporter)

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 state of NHLBI funding rates for R01s are so low that we are undoubtedly loosing many young researchers as the fail to obtain adequate support for their research. Funding R21 grants will allow new, innovative, and perhps risky projects to proceed, while keeping less established researchers in the field. Re-establishing R21 funding may prevent the impression that NHLBI is more interested in supporting established labs and not advocating for and supporting new investigators.

Feasibility and challenges of addressing this CQ or CC :

Given the limited budget of R21s, they will not have as large an impact on the overall budget of NHLBI as the equivalent number of funded R01 grants. Therefore, this change is feasible from a financial standpoint. Obviously, funding R21s will decrease funding for other mechanisms. Finally, it is possible that many of these grants will not lead to advances in the field, but it is my understanding that studies show the same thing about R01s.

Name of idea submitter and other team members who worked on this idea : George Porter

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87 net votes
101 up votes
14 down votes
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Goal 3: Advance Translational Research

Improving heart, lung, blood, sleep Health Outcomes for Minority and Underserved Men

What are the best strategies to improve implementation of evidence-based practices (EBP) to enhance effective health risk communication strategies among racial and ethnic minority males and underserved men? Examples of several issues that need to be addressed are: • Need for better definition of the role of families/communities in EBP (as co-therapists). • Requires less system fragmentation • Need for improved measurement, ...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 :

Our improved ability to develop, implement and disseminate EBPs tailored specifically for men in health disparity populations may help us move beyond current obstacles in addressing health inequities and improve health outcomes.

Some current challenges:

• High blood pressure affects more than 40 percent of African Americans.

• The odds for stroke, the third leading cause of death in the United States, are especially high for African American men at 70%.

• African Americans are about 50% more likely to experience stroke than Caucasians.

• Sleep apnea is seen more frequent among men than among women, particularly among African-American and Hispanic men.

• Life expectancy for African American men is 4.7 years less than for white men (2010).

• Native American men have an average life expectancy of 71 years old compared to white men who have an average life expectancy of 76.5 year.

Feasibility and challenges of addressing this CQ or CC :

• Shifting demographics of race as well as ageing of the population in this country will have a major impact on the utilization, organization and delivery of health care.

• Country acknowledges significant economic burden of health inequities in the U.S. in the near future.

• Hospitals and health systems are working hard to align quality improvement goals with disparities solutions. Opportunity to leverage these efforts for the development and implementation of targeted health disparities initiatives is timely.

• HL has a number of large population-based studies (such as JHS, Strong Heart, Hispanic Community Health) that could be leveraged to specifically identify EBP for wider implementation and dissemination to underserved areas.

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

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14 net votes
32 up votes
18 down votes
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Goal 3: Advance Translational Research

When will NIH truly support translational research?

There is a huge gap between basic science discoveries (some of which have no clinical application) and clinic practice (even when the basic science is related). Rarely do physicians, physician scientists, and basic scientists get together to answer a question that has public health or patient health impact. Without such an effort, a lot of taxpayer money is wasted on 'science for the sake of science' as well as in applied ...more »

Submitted by (@sanjivkaul)

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

Details on the impact of addressing this CQ or CC :

We will start solving relevant clinical issues using the support of basic science

Feasibility and challenges of addressing this CQ or CC :

infrastructure within NIH

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71 up votes
32 down votes
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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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2 up votes
1 down votes
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Goal 3: Advance Translational Research

Animal Models for Translational Research and Drug Development

There is a need to identify and develop suitable animal models (e.g. larger, non-primate animal models) that faithfully predict the outcomes of new medicines and treatments in heart, lung, blood, and sleep (HLBS) disorders prior to human 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 :

If animal models can faithfully predict the outcomes in human clinical trials of new medicines and treatments, it will reduce the economic burden for the failure of drug development.

Feasibility and challenges of addressing this CQ or CC :

Identification of current available animal models;

Development of new animal models with recent advances in mammalian genome projects and gene targeting technologies could be done over the next 5-10 years

Medical research, especially in basic discovery, has benefited significantly from the use of various animal models, such as gene-targeted and transgenic mouse models. However, many discoveries from animal models (e.g. mouse models) failed to translate into human applications.

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

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73 net votes
92 up votes
19 down votes
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Goal 3: Advance Translational Research

Incentivizing Translational Research

Support for scientific research depends on making a compelling case that we contribute to the health of Americans and the health of the US economy. This idea is to address the critical challenge of making basic research relevant to the lives of Americans by incentivizing NHBLI researchers to engage meaningfully in translational research.

Submitted by (@tomtherramus)

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 specific proposal is to give a 5 to 10 percentile bump (similar to that given to junior investigator) to researchers whose NIH funding has led to translational outcomes that are of tangible benefit to the health of Americans and/or the US economy.

 

Categories that would meet the translational bump might include:

1. A clinical trial based on their basic or clinical research;

2. Generation of a device, drug or other therapy that has entered cllinical testing;

3. Granting of a patent that has been licensed by a company,

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

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16 net votes
25 up votes
9 down votes
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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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3 up votes
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Goal 4: Develop Workforce and Resources

Medical student research training in LMIC settings

What are the strategies for heart, lung, blood, sleep workforce to gain first-hand international experience in clinical research/implementation research training in low- and middle-income country (LMIC) settings?

Submitted by (@nhlbiforumadministrator1)

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

Details on the impact of addressing this CQ or CC :

• Among the students who earn medical degrees in the United States, very few of them have been sufficiently trained to address the health needs of the most vulnerable populations.

• An international exposure in an LMIC setting would have enormous impact on clinical practice and research

• Any medical student interested in broadening their training may have opportunities for clinical and non-clinical hands-on experience and in turn this would increase number of physicians and researchers in global health.

Feasibility and challenges of addressing this CQ or CC :

Feasibility: • The NHLBI has a wealth of experience in training and career development programs in general and has supported and worked with global centers of excellence in this area.

• This experience can be used to leverage international experience in clinical practice and research outcomes in LMIC settings.

 

Challenges: • NHLBI would have to develop additional training mechanism(s) to foster clinical practice and research in an LMIC area.

 

• Resources for this unique training may be a challenge.

 

• Helping patients from vulnerable populations would involve confronting cultural differences and language barriers.

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

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5 up votes
35 down votes
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Goal 3: Advance Translational Research

Improve patient compliance through community health workers

What are effective strategies for community-based participatory dissemination and implementation research initiatives in rural settings? • Limited number of studies that adapt evidence- or practice-based interventions to rural settings • Needs innovative approaches to engaging the rural community • New intervention approaches needs testing in rural communities • Building a team would have to involve of local community ...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 :

• Nearly 60 million Americans living in rural communities face additional barriers to receiving effective prevention and treatment services.

• Rural residents often enter care later in the course of their illness than their urban peers, enter care with more serious symptoms, and require more intensive and expensive treatment.

• The rural communities in the US are closely connected and can be leveraged to disseminate heart, lung, blood, and sleep relevant preventive and/or treatment options within those communities.

• Enhance understanding of community resilience factors, cultural, and individual factors that may enhance the provision and utilization of prevention and treatment services in these communities.

• Generate knowledge to improve the organization, efficiency, effectiveness, quality, and services for rural and frontier populations.

• Support movement of evidence-based effective health care and prevention and the public health knowledge base in rural communities in the US.

• Stimulate implementation science in rural and frontier communities

• High return on investment.

Feasibility and challenges of addressing this CQ or CC :

• NHLBI investigators could initiate controlled trials that would maximize the opportunities available in rural communities.

• Documented sustainable interventions and scientific research could serve to reduce morbidity and mortality in high-risk rural populations.

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

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7 net votes
15 up votes
8 down votes
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