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 »

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 2: Reduce Human Disease

Lung Transplantation

Although the majority of lung recipients experience significant health improvement, they also frequently face serious symptom distress, impaired physical functioning and poor quality of life due to post-transplant morbidity, such as chronic rejection, infection and multiple side-effects of immunosuppression. a) Conduct clinical trials of interventions designed to maximize clinicians' support of patients' self-management ...more »

Submitted by (@nhlbiforumadministrator)

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

Feasibility and challenges of addressing this CQ or CC :

a) Conduct clinical trials of interventions designed to maximize clinicians' support of patients' self-management behaviors so that patients and clinicians working together can achieve optimal control of disease, reduce symptom distress and complications, and promote quality of life.

 

b) Evaluate the impact of integrating palliative care and transplant care for symptom management, goal setting and advanced care planning along the entire lung transplant illness trajectory (pre, post and end of life).

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

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Goal 2: Reduce Human Disease

Life-Course Approach to Science

We encourage the NHLBI to include research focused on children as a priority in their strategic vision. We encourage them to consider the implications of a life-course approach, where childhood presents a unique opportunity to set the trajectory for health risk as an adult. This also implies that interventions targeting children may have the greatest impact on the population as a whole in the long term.

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 Society of Pediatric Nephrology (ASPN)

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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 »

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 :

While we do not mean for a moment that we should drift away from mechanistic science, we believe that there should also be openness to addressing some questions that are simple and perhaps even slightly dull, but can be unequivocally answered with a trial. In this way, beliefs can be converted to facts.

Feasibility and challenges of addressing this CQ or CC :

It is difficult to seek funding for such trials because reviewers want to see more testing of mechanisms, more physiologic outcomes, more testing of hypothesized mediating variables, and more exciting scientific hypothesis tests.

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 2: Reduce Human Disease

Adequately powered clinical trials

Pragmatic and adequately powered clinical trials are an ongoing challenge.

­Develop new tools to facilitate identification and recruitment of subjects, remote/wireless data collection, and functional measures.

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 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

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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 »

Submitted by (@javed.butler)

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

Details on the impact of addressing this CQ or CC :

Timely conduct of US relevant data to translate basic science discoveries into either clinical practice or knowing that something is not useful or harmful.

Feasibility and challenges of addressing this CQ or CC :

This will be challenging because it will need public education and advocacy as well as institutional leadership developing incentives and making clinical trials a priority.

Name of idea submitter and other team members who worked on this idea : Javed Butler, MD MPH MBA

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Goal 2: Reduce Human Disease

Clinical Trials & Duplicative Regulatory Standards

The initiation of clinical trials remains difficult, time-consuming, and costly. Repetitive institutional review board oversight is one of several obstacles to efficient clinical trial initiation and completion. New strategies for addressing duplicative regulatory standards are necessary to ease the initiation and completion of trials.

Submitted by (@skrenrich)

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 : Cystic Fibrosis Foundation

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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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Goal 3: Advance Translational Research

Obstacles to the timely completion of clinical trials

There are numerous obstacles to the timely completion of trials, and there is a crisis in US enrollment rates. Overcoming barriers to timely completion of clinical trials would have a profound impact on accelerating research translation to improving health. Clinical investigations are necessary to advance the prevention, diagnosis, treatment and cures of human disease. The rate of basic scientific discovery has overwhelmed ...more »

Submitted by (@rebecca.lehotzky)

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

Feasibility and challenges of addressing this CQ or CC :

For comparative effectiveness trials, incentives are misaligned; it is often easier, financially advantageous and benefits practice/hospital metrics to perform tests/procedures clinically than in the context of a trial. A collaboration between NHLBI, FDA and CMS would markedly accelerate many trials by aligning incentives. There are a few small examples with some success eg, Carotid stenting. Regulatory burdens should be reduced.

 

Solutions to enrollment challenges need to address physician and participant related barriers. NHLBI and AHA could lead a public private partnership and with patient advocacy groups (eg, Research America) and the media to enhance the understanding and acceptance of participation in clinical research.

 

To advance the efficiency of clinical trials NHLBI should support investigation of methodologies such as event driven trials, adaptive design, Bayesian approaches, multiple randomized groups, group/cluster randomized trials, maximizing information from subgroup analyses.

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

Building a bridge - new approaches to dissemination

There is a need to fundamentally rethink the approach to translation and dissemination of clinical trial results. The current approach relegates dissemination as a side effect to the end of a trial and does not require sufficient funds be set aside to conduct adequate outreach. As a result, it takes up to 17 years for the results of trials to be adopted and used in everyday practice. It is time for a new model which re-prioritizes ...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 benefits of clinical research on the quality of health are compromised by the slow and ineffective process by which they find their way into clinical practice. Decades of theorizing on the dissemination of scientific knowledge has resulted in overreliance on outmoded theoretical approaches to dissemination. Much of the work to date has the appearance of “victim blaming” (e.g., the characteristics of “slow adopters” are identified and many physicians are thought to evidence “clinical inertia”). Much less attention is focused on: a) the outmoded and ineffective process of dissemination itself, disregarding the important (even critical) role of the digital age and social media; b) the ever changing organizational contexts in which dissemination is supposed to occur (not considering the important role of the consumer or patient in dissemination); and c) the demonstrated efficiency of the private sector in disseminating information to organizations, providers and patients (commonly known as direct to consumer advertising). Delays in dissemination result in delays in receipt of new therapies, effective new approaches, safe and more cost effective treatments and contributes to social injustices which perpetuate health disparities.

Feasibility and challenges of addressing this CQ or CC :

Many federally funded clinical trials are completed with fastidious attention to scientific quality a key challenge is the relegation of dissemination to an afterthought, often unfunded and neglected. The feasibility of improving dissemination has been repeatedly demonstrated by the private sector driven by a need to complete trials rapidly for immediate dissemination and adoption (time to market). The challenge is to re-prioritize dissemination to be part of the entire clinical trial process (proposal to study close), and to create a separate funding requirement within PAs and RFPs to ensure that there is both a requirement and resources dedicated to dissemination. Conducting a consensus conference between the federal and private sectors to learn the best practices to improve dissemination while maintaining research quality is essential.

Name of idea submitter and other team members who worked on this idea : Lisa Marceau. Team Member: John McKinlay

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Goal 1: Promote Human Health

THE RELEVANCE OF PREVENTION TRIALS

Prevention trials, implemented to reduce or delay progression to overt disease in a population at risk to the disease, are an important approach to health promotion. Therapies shown to reduce disease severity in patients with a specific disease are obvious, but not the only, candidates for a prevention trial in populations at high risk for prevalent diseases (such as heart failure, diabetes, COPD, asthma in children). ...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 of implementing such trials is considerable. They will clearly address an important component of NHLBI’s mission with respect to effectiveness of therapies and behavioral interventions, and it has minimal and clearly definable overlap with commercial trials of specific therapeutic products. It will also provide an important public health focus – preventing disease or reducing the impact of disease processes, thus potentially reducing chronic care costs and increasing years of useful life.

Feasibility and challenges of addressing this CQ or CC :

The biggest challenge in designing and implementing prevention trials is identifying the target, “at risk” population most likely to develop the clinical disease from known biomarkers or early signs/symptoms. Increasing availability of large, population-based registries or databases maintained for other purposes provides a very cost-efficient mechanism to electronically screen and identify “at risk” individuals. The same mechanism may also facilitate implementation of pragmatic, electronically managed, cost efficient trials.

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

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