Goal 3: Advance Translational Research

What strategies improve implementation of COPD therapy, palliation, and dyspnea management

Chronic obstructive pulmonary disease (COPD) affects over 12-24 million individuals in the U.S. where it is responsible for ~ 800,000 hospitalizations per year, and recently became America’s 3rd leading cause of death. The lag between clinical practice and treatment options described by efficacy studies to improve the quality of life, functional status, and survival in patients with COPD make it ideal for efforts in patient-centered... more »

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 Richard Mularski MD MSHS MCR

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20 net votes
29 up votes
9 down votes
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Goal 3: Advance Translational Research

Stem Cell Biology

There is a need to develop “designer platelets” and “designer red cells,” as well as facilitate large-scale production of these products for therapeutic and diagnostic use.

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 reprogramming of adult stem cells has resulted in the generation of induced pluripotent stem cells (iPSCs) that can develop into any tissue of the body. These iPSCs ultimately may be used as a transplantable source of stem cells for a variety of hematologic diseases. Although this technology has enabled the generation of patient-specific or disease-specific stem cells that are also amenable to genetic manipulation, the major scientific hurdle has been the ability to create clinically meaningful functional blood products, including transplantable HSCs from differentiating iPSCs. The production of clinically functional blood products -- i.e. red blood cells derived from autologous iPSCs --could replace allogeneic products in highly immunized patients and the generation of megakaryocytes for patient-specific platelet production from iPSCs could drive significant progress in this area. Furthermore, disease-specific iPSCs could serve as targets for both drug development and drug screening in patients with rare hematologic disorders. In addition, support for scale-up and GMP processes, which are difficult to fund via the R01 mechanism will require specific grant opportunities tailored to infrastructure and process development.

Name of idea submitter and other team members who worked on this idea Alice Kuaban on behalf of the American Society of Hematology (ASH)

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25 net votes
53 up votes
28 down votes
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Goal 3: Advance Translational Research

High Quality COPD care coordination

What factors are associated with high quality care coordination in patients with COPD?

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

Details on the impact of addressing this CQ or CC

Patients would benefit from high quality care coordination; costs to insurance companies would probably decrease as would hospitalizations; would result in better quality of life for patients.

Feasibility and challenges of addressing this CQ or CC

All providers must be willing to share information; PCP's must be willing to screen for COPD and make referrals when indicated; would be a challenge in very rural areas with very few resources.

Name of idea submitter and other team members who worked on this idea Karen Deitemeyer, COPD Foundation State Captain Program

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16 net votes
18 up votes
2 down votes
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Goal 3: Advance Translational Research

Overcoming barriers to translational regenerative medicine

Current stem cell based approaches to translational medicine predominantly show modest efficacy. Most research rest on accepting existing limitations and focusing upon "tweaks" to the experimental model rather than taking on important barriers head on. The efficacy of stem cell-based regenerative medicine will never be fully realized unless we stop trying overly simplistic approaches such as"more is better" and start... more »

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 field of regenerative medicine holds great potential but we risk losing the public trust by hyperbolic promises, modest efficacy, and incremental research steps. Truly innovative research will transform the landscape and offer truly novel therapeutic approaches to many current incurable conditions. The result is a dramatic shift in the practice of medicine, new options for treatment, enhanced engagement of the public in biomedical research and new growth opportunities for the NIH and biotech sectors.

Feasibility and challenges of addressing this CQ or CC

The future is here for regenerative medicine, but for the most part the potential and practice has been unrealized or poorly executed. The challenge is to identify the limiting factors and sweep them aside. There is broad and surprisingly consistent consensus on what the barriers are to successful regenerative therapy, but it seems most researchers are complacent and accept these limitations as inherent in the system rather than try to find truly combative approaches to overcome the barriers to enhancing regenerative processes. So it is essential to change the current mindset and push for a full frontal attack on the barriers that impede successful regeneration rather than minor modifications or uninspired brute force approaches that ignore the underlying mechanistic issues. Also, a major challenge is the hyperbole and overselling of research findings and impact by researchers and their institutions looking to capitalize upon the "discovery de jour." Such overly optimistic and unrealistic promises undermine our position in the public eye and compromise our future ability to earn the public trust.

Name of idea submitter and other team members who worked on this idea M Sussman

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-6 net votes
14 up votes
20 down votes
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Goal 3: Advance Translational Research

Develop relevant large animal models for various disease conditions

What is the possibility of investing funds primarily in clinically-relevant models where the findings could be translated in to human diseases?

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

Details on the impact of addressing this CQ or CC

Strong emphasis on the use of a clinically-relevant large animal model would hopefully be more productive in developing better therapeutic approaches and management of patients.

Feasibility and challenges of addressing this CQ or CC

In view of the lack of facility at many institutions and the cost involved, and the rules and regulations by the USDA and other regularity bodies, special emphasis will be required to build the animal facility at an institution. Where will the funds come from? Similar to many other core facilities set up by the NIH at various institutions, what is the possibility of developing specialized centers for testing a new idea in a clinically-relevant large animal facility?

Name of idea submitter and other team members who worked on this idea Devendra K. Agrawal, PhD

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-5 net votes
11 up votes
16 down votes
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Goal 3: Advance Translational Research

Create a National Action Plan for COPD

Lead a coordinated effort of government, patient advocacy organizations, professional organizations, payers and others to plan and implement a coordinated plan to improve COPD awareness, education for patients and healthcare professionals, treatment strategies, research and data collection, policies and public health infrastructure and programs.

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

Details on the impact of addressing this CQ or CC

Unlike most leading causes of death and disability, there is no coordinated effort to lower disease burden associated with COPD. Coordinated plans provide a forum for identifying the most pressing issues that must be tackled, for setting goals and convening partners from different disciplines and are a framework upon which policy change can be achieved. In order to make meaningful progress in the impact that COPD is having on patients, health systems and payers, coordinated planning and action is needed and NHLBI can lead the way but time is of the essence.

Feasibility and challenges of addressing this CQ or CC

There are proven models of multi-stakeholder, public and private partnerships to tackle disease burden and create national plans. There are also multiple national and regional organizations standing ready to assist in these efforts.

Name of idea submitter and other team members who worked on this idea COPD Foundation Board of Directors, COPDF MASAC, COPDF State Advocacy Captains

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

Engineering Challenges for Next Generation VADs

Critical challenges to overcome include the development of non-platelet activating impellers and hypothrombogenic surfaces in modern ventricular assist devices (VADs). Percutaneous and transcutaneous electric drive systems will need to evolve, and clinical research is needed to reduce thromboembolism and bleeding (acquired von Willebrand syndrome). Adverse events may also be further reduced if smaller pumps, for partial... more »

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

Details on the impact of addressing this CQ or CC

It would improve quality of life for VAD recipients by reducing or eliminating their need to lifelong anticoagulant medication.

Feasibility and challenges of addressing this CQ or CC

There have been steady incremental improvements in all the challenge areas, and there are already many patients who do not encounter problems. A convergence and focusing of research efforts is needed.
Use of modern ventricular assist devices (VADs) has been associated with improvements in survival. However, INTERMACS has revealed that many of the adverse events associated with VADs stem from bio-incompatibilities between the device and patient.

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

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

Quality Improvement in the ICU

How best can we engage all hospitals to participate in quality improvement initiatives to increase evidence-based practices in the ICU and other environments across diseases?

 

Is there a role for regionalization of care?

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 NHLBI Staff

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-5 net votes
7 up votes
12 down votes
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Goal 3: Advance Translational Research

Develop biomarker panel to predict CVD risk in -omics era

There is a need to utilize the vast data generated in -omics research to develop biomarker panels for better prediction of cardiovascular disease (CVD) risks.

•Cardiovascular diseases develop over decades and different panels of markers may be required for different stages

•Lead molecules as potential biomarkers need to be selected by a panel of experts

•Standard procedures about sample preparation, data acquisition,... more »

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

Details on the impact of addressing this CQ or CC

•Develop specific and sensitive markers for early prevention with more predictive power. Biomarkers that can detect specific perturbations in the system, such as metabolic status and vascular integrity prior to the occurrence of the diseases can be used for early preventive treatment of cardiovascular diseases.

•Identify vulnerable population who cannot be identified by the current LDL-HDL profiling

•Allow for more personalized treatment

Feasibility and challenges of addressing this CQ or CC

•An increase in system biology studies using –omic approaches have provided huge data to mine through and find potential biomarkers, such as microRNA, DNA, lipids, proteins, and other metabolites, which can be used to assess changes proceeding cardiovascular diseases occurrence.

•The NIH-wide Big Data to Knowledge (BD2K) initiative launched in 2012 may have laid out some framework.

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

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

Preventing Stroke from Atrial Fibrillation

How can health systems develop and implement validated measurement and feedback tools to identify patients with atrial fibrillation, categorize their risk factors for stroke, capture reasons for non-treatment, and develop interventions customized to those reasons to substantially improve the proportion of patients receiving effective oral anticoagulant stroke prevention treatment?

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

Details on the impact of addressing this CQ or CC

Registries show that only about half of patients with atrial fibrillation and risk for stroke are taking oral anticoagulants. Given 4 million Americans with atrial fibrillation, half of whom (2 million) are not treated, with 5% stroke rate per year, 67% of which can be prevented, there are 67000 strokes occuring in this untreated population per year in the US. Assuming half of these could be treated if programs were develped that were proven effective, this would result in 33,000 strokes prevented per year.

Feasibility and challenges of addressing this CQ or CC

NIH funds are needed to address the complex set of health system, psychosocial, and health IT issues to answer this question. Atrial fibrillation is a common condition with patients presenting and being treated accross various parts of the health care system. Small programs have shown promise for the use of the electronic health record to systematically identify patients with atrial fibrillation, but health system leadership needs evidence of success and guidance before this will be possible on a broad scale. With evidence of feasibility and impact, performance measures may be developed that would substantially enhance adaptation.

Name of idea submitter and other team members who worked on this idea Chris Granger

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

Point of care detection of rare cells in blood

Laboratory analyses at the bedside or in the hinterlands can reduce the cost and increase the capture of disease states in underserved populations. The injection of a blood draw directly into a portable device that requires no further operator interventions is a Critical Challenge. With today’s automated chemistry and a miniaturized flow cytometer this challenge could be met.

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

Details on the impact of addressing this CQ or CC

Although portable flow cytometers exist, they are not equipped with chemical automation and rare-cell enrichment capability. An integrated system could be applied to multiple diagnostic goals. A sufficiently versatile design should be able to detect and enumerate specific cell populations at any level (not only rare cells) in the circulation — T-cell subsets in AIDS, for example.

Feasibility and challenges of addressing this CQ or CC

Automated mixing and labeling, magnetic bioseparations, microfluidics, high-intensity LEDs and sensitive avalanche photodiodes can be combined to address this CC.

Name of idea submitter and other team members who worked on this idea Paul Todd

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-6 net votes
4 up votes
10 down votes
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Goal 3: Advance Translational Research

Improving Community-Based Care for Sickle Cell Disease

Sickle cell treatment centers are located throughout the United States, primarily in urban areas, and play an invaluable role. However, there is a critical need to identify and educate primary care providers who can provide routine and preventive care, but will also know when to consult with/refer to hematologists and other appropriate providers when necessary.

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

Details on the impact of addressing this CQ or CC

For the first time, comprehensive guidelines addressing the management of sickle cell disease were issued in 2014 by the NHLBI. (Previous guidelines were not comprehensive.) The 2014 guidelines, which address issues such as health maintenance and the treatment of acute and chronic complications, are based on a systematic review of available evidence; consensus of an expert panel; and published, vetted guidelines by other organizations when evidence was unavailable or insufficient. These guidelines can provide a solid overview of the knowledge essential for the care of people with sickle cell disease.

Identifying primary care providers who can provide routine and preventive care but at the same time are knowledgeable about sickle cell disease, should be a more efficient, less costly method of provide important health services to people with sickle cell disease and should ultimately improve the health and well being of this population, particularly for people who do not live near a sickle cell center.

Feasibility and challenges of addressing this CQ or CC

Addressing this question is very feasible. However, for a variety of reasons, including misconceptions about patients with the condition, it may be challenging to recruit large numbers of primary care providers.

Name of idea submitter and other team members who worked on this idea The Sickle Cell Association of New Jersey

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

What is the comparative effectiveness of short-term vs. chronic (e.g., 12 mo) pulmonary rehabilitation?

What is the comparative effectiveness of short-term vs. chronic (e.g. 12 mos) pulmonary rehabilitation on survival, patient-reported outcomes (symptom frequency, activities of daily living, quality of life, sleep quality, exacerbations), healthcare utilization, and costs from a societal and healthcare system perspective?

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

Details on the impact of addressing this CQ or CC

Modest sized efficacy trials demonstrate substantial health benefits, that wane as Pulmonary rehabilitation is discontinued. We need to test the health and resource implications of "chronic" (e.g., 12 or 24 mos) pulmonary rehabilitation. Such information will benefit health systems seeking to implement care models for high-risk, costly, patients - patients with COPD are of increasing interest to health systems. Such a comparative effectiveness trial should also involve behavioral interventions to promote self-management and involve caregivers.

Name of idea submitter and other team members who worked on this idea Jerry Krishnan

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

Translation of scientific information into clinical and public health asthma and allergies practices and programs

More evidence-based, scientifically proven interventions to ensure that scientific information is translated into clinical and public health practices and programs to reduce the burden of asthma and allergies on individuals, families and society

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 Asthma and Allergy Foundation of America (AAFA)

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

Sickle Cell Trait complications

What are the factors leading to severe health consequences in the sickle cell trait population?

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

Details on the impact of addressing this CQ or CC

It has been reported for many decades that the sickle cell trait population are an asymptomatic group; however, there have been an increasing number of reports in the media and the medical literature suggesting the opposite.

Name of idea submitter and other team members who worked on this idea Sickle Cell Warriors, Inc. community members

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30 net votes
38 up votes
8 down votes
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