Goal 3: Advance Translational Research

In-Vitro Assays to Predict Clinical Response

How can NHLBI support studies that produce in-vitro assays to predict clinical response and ways to translate those results into patient therapies through novel clinical trials, including those for small patient populations and rare diseases?

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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3 net votes
6 up votes
3 down votes
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Goal 3: Advance Translational Research

To Improve Clinical Practice Recommendations for Asthma

What are the strategies to improve the use of evidence-based clinical practice recommendations and thereby increase the quality of care and improve outcomes for people with asthma? • Lack of provider awareness, knowledge, agreement, and/or self-efficacy in using the guidelines • Inconsistent use of guidelines-based asthma care in clinical practice. • Scarce/limited resources and limited access to target audiences. • ...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 :

• Asthma is a chronic lung disease that affects 26 million people in the U.S., including more than 7 million children, at an estimated cost to the nation of $56 billion. Asthma accounts for 14.2 million missed work days, 10.5 million missed school days, 10.6 million physician office visits, and 1.8 million emergency department visits, and 439,000 hospitalizations each year.

• Despite widespread availability of evidence-based clinical practice guidelines for the diagnosis and management of asthma, only about half of individuals with asthma in the U.S. receive guidelines-based care.

• Identifying strategies to improve use of evidence-based clinical practice recommendations would 1) increase the number of people with asthma who receive evidence-based clinical care, 2) increase the number of health care providers who use (implement) evidence-based clinical practice recommendations, 3) increase the quality of care of people with asthma, and 4) improve outcomes and quality of life for people with asthma.

Feasibility and challenges of addressing this CQ or CC :

• Investigators could evaluate guidelines-based implementation strategies in implementation settings such as community and regional health systems, private medical practices, federally qualified health centers and other safety-net clinics, and hospitals throughout the U.S.

• Documented successful and sustainable implementation strategies could be shared more broadly and applied to future efforts to improve asthma care and control as well as reduce asthma disparities.

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

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1 net vote
6 up votes
5 down votes
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Goal 2: Reduce Human Disease

New models for clinical trials

There is a need to educate the scientific community and program staff about the use of metrics, results-based accountability, and other business models to improve the science, productivity, and efficiency of clinical trials and clinical trial networks.

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 :

Improve the efficiency of clinical trials and a return on research investments

Feasibility and challenges of addressing this CQ or CC :

Yes, this is feasible.

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

Voting

4 net votes
10 up votes
6 down votes
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Goal 4: Develop Workforce and Resources

Pipeline of clinician scientists

Maintaining the pipeline of clinician scientists via early and mid-career awards. Promote the development of collaborative 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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5 net votes
6 up votes
1 down votes
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Goal 1: Promote Human Health

Non-opioid management of pain

The use of opioid agents has increased dramatically in the United States. Prescription drug overdoses now account for more deaths than traumatic injuries in the 1-44 age group. There is a need for more high level evidence to target optimal pain management strategies in the acute (ED) and non-acute settings (office practice) especially for chronic disorders such as low back pain, headaches, fibromyalgia, acupuncture etc. ...more »

Submitted by (@dayam0)

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

Details on the impact of addressing this CQ or CC :

Huge problem in the US compared to other countries where opioid use is more restricted. Potential to enhance population health and reduce accidental death.

Feasibility and challenges of addressing this CQ or CC :

Complex since affecting by many factors and groups that would need to work together in conjunction with patients to change the way we approach pain.

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

Voting

6 net votes
6 up votes
0 down votes
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Goal 2: Reduce Human Disease

Testing PCSK9 Inhibitors

Several inhibitors of PCSK9 are in phase 3 development and show considerable promise for improving the lipid profile; they will be especially appropriate for patients with Familial Hypercholesterolemia and those with statin intolerance. The sponsoring pharmaceutical companies need to complete CVD endpoint trials with full safety testing. However, there may well be more than one drug approved for marketing. The sponsors ...more »

Submitted by (@stephen.fortmann)

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

Details on the impact of addressing this CQ or CC :

Admittedly, this idea is on the mid-range horizon at best, since it will be some years before we know which, if any, PCSK9 inhibitors make it to market. However, if the data produced in phase 3 trials is inadequate to select among marketed drugs, a comparative effectiveness trial would be in order. If the then existing data are compatible with equipoise between the agents, a pragmatic trial design could likely be justified.

Feasibility and challenges of addressing this CQ or CC :

Such a trial would be quite feasible, assuming it is justified by the data.

Name of idea submitter and other team members who worked on this idea : Stephen P. Fortmann

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1 net vote
4 up votes
3 down votes
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Goal 3: Advance Translational Research

Advancing the science of translating evidence into practice

What are the best ways for the NHLBI to advance the evolving science of translating robust evidence into clinical practice domestically and globally? How to personalize broad research evidence for individual patients? How to predict and evaluate the impact of evidence-based interventions? How to identify implementation methods available in industry and elsewhere that work best and are most translatable in healthcare? ...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 :

• Reduce mortality and morbidity

• Improved quality of life

• Higher proportion of people receiving evidence-based care and at goal for that care

• Reduced disparities in health and healthcare

Feasibility and challenges of addressing this CQ or CC :

Challenges:

• Lack of research methodology in this area – may need new scientific approaches

 

• Lack of current capacities and capabilities in this area

 

• Current silos that separate research enterprise from industry, as well as NHLBI from other ICs

 

• Divisions between performance of clinical trials and implementation research

 

• Lack of clarity which federal agencies and NIH Institutes are ‘in charge’ of implementation and/or prioritize this as part of their mission and budget

 

• Lack of wide sharing of best practices of other implementation models

 

• Improving the science in this area needs to include methods and metrics development

 

• The accumulated knowledge of clinical trialists and implementation researchers is often not shared

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

Voting

13 net votes
27 up votes
14 down votes
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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?

Submitted by (@wheeze)

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 : Mitchell Grayson on behalf of the American Academy of Allergy, Asthma, and Immunology

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2 net votes
15 up votes
13 down votes
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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 »

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 :

If successful this approach should enable the conduct of cheap pragmatic trials that are fueled by data from clinical care. The integration into clinical care helps assure efficiency and generalizability of results.

Feasibility and challenges of addressing this CQ or CC :

The advent of electronic medical records and the explosion of big data technology has made it possible to gain access to and analyze data in a manner that would have been unthinkable 10 years ago. This is already going on in other fields.

Health care systems are increasingly using "big data" approaches to track outcomes in the patients treated with different strategies and drugs, and apply the knowledge gained from outcomes in previous patients to inform decision making in subsequent patients ("learning"). This approach could be used to personalize treatment. A recent example from cancer is to genotype lung tumors, and tailor the treatment of a new patients to drugs producing good results in patients with similar tumor genotypes. When two or more treatments produce similar results, one could randomize. Cardiovascular disease presents a challenge in using genotyping information to personalize treatment, because the manifestations are the results of complex genetic and environmental risk factors.

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

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4 net votes
15 up votes
11 down votes
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Goal 2: Reduce Human Disease

Support for Cardiothoracic Surgery and Pediatric Heart Clinical Trial Networks

Continued and expanded support for the Cardiothoracic Surgical Trials Network (CTSN) and Pediatric Heart Network (PHN) is essential as both design, conduct, and analyze multiple, collaborative clinical trials that evaluate surgical interventions, and related management approaches for the treatment of cardiovascular disease. To date both networks have reported on and developed a portfolio of studies which need continued ...more »

Submitted by (@meaton)

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 work and support provided to date have allowed for the creation of an infrastructure for both the CTSN and PHN. Each network is now providing valuable results to the cardiothoracic surgery specialty which will allow an increase in quality patient care in the years and decades to come. The continued support is essential for the success of these networks as any reduction will limit the resources available for site participation and ultimately results. Due to the existing infrastructure for each network, the financial burden associated with de-funding and then restarting the networks in future years would be at least triple the financial commitment currently in place.

Feasibility and challenges of addressing this CQ or CC :

Conducting multi-center clinical trials is a substantial financial commitment but a vital part for the future of the cardiothoracic surgery specialty.

Name of idea submitter and other team members who worked on this idea : Matt E.

Voting

108 net votes
151 up votes
43 down votes
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Goal 3: Advance Translational Research

ASCVD Risk and Treatment Options

There is little evidence about the best ways in which to effectively communicate atherosclerotic cardiovascular disease (ASCVD) risk to patients so that they clearly understand the potential benefits and harms of treatments in order to make informed decisions about their care. A shift toward shared decision-making and tailored treatment makes it imperative that effective risk communication strategies be developed and ...more »

Submitted by (@nhlbiforumadministrator)

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

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-2 net votes
6 up votes
8 down votes
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Goal 2: Reduce Human Disease

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?

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 :

Addressing thecritical challenge would improve clinical trial design, clinical trial efficacy and ultimately completion and reporting of trial results. This approach would most likely save money.

Feasibility and challenges of addressing this CQ or CC :

The Other Transaction Authority already exists within the PHS and NIH and this method has been tested at NCI.

Competition is an important element of successful achievement in everything from sports rivalries to scientific innovation. Examples of this can be cited for the Larry Bird and Magic Johnson hoops competition, Muhammad Ali versus Joe Frazier boxing duels, and the contest between the Human Genome Project and Celera for completion of the mapping of the human genome. The US Defense Advanced Research Project Agency (DARPA http://www.darpa.mil) has used the concept of competition to advance the development of never before imagined scientific and technological innovations including development of the internet, the SIRI voice recognition system, UNIX and the cloud, and the global positioning system, among many products and innovations. Under this paradigm great advances are initiated through a public challenge which brings competitors together to achieve a goal set by the government. This is accomplished through the use of the Other Transaction Authority (OTA), which has been extended to the NIH. Subsequent management of the challenge involves the setting of milestones and utilization of “go/no go” decision making. The NCI has used a “DARPA-like” approach to instill competition for the design and conduct of clinical trials and as a result has substantially improved the performance of its clinical trials networks / cooperative groups.

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

Voting

-1 net votes
6 up votes
7 down votes
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