Goal 2: Reduce Human Disease

Behavioral Science in Asthma Clinical Research

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:

 

Will integration of behavior science in clinical research improve effectiveness of interventions for asthma associated with behavioral risk factors? 

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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8 up votes
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Goal 2: Reduce Human Disease

Clinical Research for HIV/AIDS and HLB Health and Diseases

What HIV/AIDS-related clinical research can NHLBI support in the next 5-10 years on the prevention, diagnosis, and treatment of HIV-related heart, lung, and/or blood (HLB) diseases in adults and children to improve heart, lung, and blood health outcomes in HIV infections?

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 :

Remarkable progress in anti-retroviral therapy (ART) has increased survival in the HIV population and significantly reduced mother-to-child transmission of HIV both in resource-rich and resource-limited settings. However, this enhanced longevity is now associated with an increasing burden of chronic diseases, such as coronary artery disease (CAD), chronic obstructive pulmonary disease (COPD), and chronic anemia. In patients with HIV, there may be a complex interplay between the processes of inflammation, traditional risk factors, and the adverse effects of ART. Additionally, the long-term effects of in utero exposure to HIV and antiretroviral drugs in HIV-exposed but uninfected children on HLB diseases is unknown.

Furthermore, HIV control or possibly even HIV cure could result from developing novel cell therapies, especially hematopoietic stem cell (HSC) transplants, and might also result from early use of antiretroviral therapy in acutely HIV-infected individuals. Finally, other unique NHLBI research activities and resources could be leveraged for HIV/AIDS-related research.

Feasibility and challenges of addressing this CQ or CC :

• Collaborations between HIV and HLB investigators that have been facilitated by the NHLBI investments, including three RFAs.

 

• The Berlin patient has provided the proof of concept that HIV infection can be eradicated, that is, sterilizing cure can be achieved, through HSC transplantation in combination with other therapies;

 

• Recent studies have shown that early identification of HIV infection and treatment of infected individuals with anti-retroviral therapy as soon as possible can significantly limit the size of the HIV reservoirs even if such early treatment may not be able to completely prevent the establishment of HIV reservoirs; routine blood donor screening for both anti-HIV antibodies and HIV RNA among blood donors offers unique opportunities to identify acute HIV infections.

 

• For other NHLBI research activities and resources that could be leveraged: data and specimens are available in the NHLBI repositories as well as repositories of NHLBI investigators.

 

 

For HLB comorbidities of HIV infection, the challenges include:

 

• Closer collaboration and leverage of resources available

 

For HIV cure, the challenges include:

 

• Generation of HIV-resistant HSCs in adequate quantity for transplantation;

 

• Efficiency of homing and expansion of HIV-resistant HSC transplants, replacing HIV-infected cells, and immune reconstitution by HSC transplants

 

• Safety of HSC transplantation with needed GVHD to eliminate HIV-infected resting T cells

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

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Goal 3: Advance Translational Research

Validating surrogate/intermediate clinical outcome endpoints for ST segment elevated myocardial infarction (STEMI)

The introduction of troponin assays has changed the criteria for myocardial infarction (MI), with much smaller myocardial damage now classified as an MI. Since to date, the FDA has not approved new agents based on their decreasing size of an MI, regulatory approval has required endpoints like death or heart failure that require large numbers of patients and long periods of time. Thus, it is difficult to attract industry ...more »

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 :

Identifying a validated surrogate endpoint FDA approval of novel intervention for STEMI would potentially attract broad industry support to address this crucial health need.

Feasibility and challenges of addressing this CQ or CC :

NHLBI could potentially bring together all of the data in NHLBI-sponsored trials along with those sponsored by industry to assess the validity of infarct size measured by different methods as a surrogate indicator of death and clinical MI complications, including heart failure. By working with the FDA on this project, the impact on regulatory review would be immediate. The major challenges will be integrating data from multiple studies and choosing statistical methodology that is acceptable to the FDA for validating a surrogate endpoint.

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

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1 net vote
2 up votes
1 down votes
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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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4 net votes
5 up votes
1 down votes
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Goal 4: Develop Workforce and Resources

Number of clinical scientists

How can we insure that there are sufficient numbers of clinical scientists over the next 20 years?

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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2 net votes
2 up votes
0 down votes
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Goal 3: Advance Translational Research

Definitive Evidence of the Effectiveness of Pulmonary Rehabilitation

What is the clinical effectiveness of pulmonary rehabilitation in reducing hospital admissions and readmissions, improving health outcomes such as exercise tolerance and dyspnea, and positively impacting patient centered outcomes. Does this effectiveness vary based on the types of settings rehab is conducted in, urban vs rural environments, the components to the program, the timing of the program and the overall support ...more »

Submitted by (@gacdk0)

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

Details on the impact of addressing this CQ or CC :

Pulmonary rehabilitation is a critical component in the treatment of COPD patients but several barriers persist that have resulted in very limited access to rehab, low referral rates for eligible patients and limited standardization of best practices within the rehab facilities that do exist. Large, definitive studies accounting for patient subgroups, site characteristics and program components can generate the level of evidence needed to expand access, educate providers and improve referral systems and create quality programs. This level of evidence is necessary to change policy to properly value the role of pulmonary rehabilitation and to convince integrated health systems in a value based market that pulmonary rehabilitation is beyond a doubt, a requirement of providing quality COPD care.

Name of idea submitter and other team members who worked on this idea : Grace Anne Dorney Koppel, COPD Foundation Board of Directors, COPD Patient Advocate

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9 net votes
12 up votes
3 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 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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2 net votes
4 up votes
2 down votes
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Goal 2: Reduce Human Disease

New Clinical Research Methodologies for Rare Diseases

What innovative methodologies applicable to small cohorts and rare outcomes can better ensure the success of clinical and implementation studies in the rare diseases affecting heart, lung, blood, and sleep?

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 :

Efficient translation of clinical observation into discovery science, and, particularly, the timely translation of potential therapeutic target identification into the treatment of rare diseases has been impeded by the reality that rare disease populations are often too small to be studied using “classical” epidemiology, clinical trial, and implementation science methodology. Overcoming these barriers will require both the adaptation of current clinical research methods and the development of novel methodologies. The requirement for better methods in rare disease clinical science will become even more urgent with time as systems biology more specifically defines and sub-characterizes ‘common’ heart, lung, blood, and sleep disease populations

Feasibility and challenges of addressing this CQ or CC :

This problem has been recently addressed through special initiatives in the application of small trial methodology into the planning and design of clinical trials in rare hemostatic disorders and sickle cell disease. Furthermore, small clinical trial methodologists have begun to populate the CTSAs and Regulatory Agencies. Their expertise in clinical trial design and biostatistical methods, and their creative ideas can be brought to bear in the clinical trials required to advance NHLBI scientific priorities.

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

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24 net votes
37 up votes
13 down votes
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Goal 3: Advance Translational Research

Leveraging Networks of Federally Qualified Healthcare Centers

How best do we leverage the existing Federally Qualified Healthcare Center’s (FQHC) infrastructure to study T4 Implementation Research for heart, lung, blood, sleep diseases and conditions among high risk and vulnerable 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 :

• Develop strategies to reduced Health Inequities

• Potentially be scaled up across an entire health system with huge population impact

• Studies would be done in the environment and context where the findings with be implemented leading to better uptake and sustainability.

Feasibility and challenges of addressing this CQ or CC :

• Formative FQHC groups are already being organized but do not have strong leadership and support

• FQHCs have ready access to the high risk and vulnerable populations that would benefit most from the research

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

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-14 net votes
7 up votes
21 down votes
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Goal 3: Advance Translational Research

Behavioral and Clinical Researcher Interactions

A critical challenge is the need to bring together basic behavioral scientists interested in understanding human behavior at a fundamental level, including how and why people become motivated and capable of undertaking behavioral changes, with clinical researchers interested in developing and testing new strategies for tackling resistant behavioral problems such as obesity and non-adherence.

Submitted by (@nhlbiforumadministrator1)

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

Feasibility and challenges of addressing this CQ or CC :

Obesity and non-adherence are two of the most important yet challenging behavioral problems contributing to cardiovascular morbidity and mortality. One way to address this challenge is to fund a network of “behavior change labs,” each of which bring together teams of basic behavioral scientists who are investigating the bases of behavior and behavior change with clinical researchers interested in designing, optimizing and testing novel ideas for tackling the difficult behavior problems of obesity and non-adherence to cardiac medications.

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

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20 net votes
52 up votes
32 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.

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108 net votes
151 up votes
43 down votes
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