Strategic Goal: Goal 4: Develop Workforce and Resources

Increase and Support Research Based Faculty

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: There has been a decline in research-based faculty in the past few years.  The challenge is two-fold.  First, increase the research faculty pipeline with increased focus on training and recruitment of research focused fellows ...more »

Submitted by (@wheeze)

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

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59 net votes
76 up votes
17 down votes
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Strategic Goal: Goal 2: Reduce Human Disease

Neurocognitive development and delays in sickle cell disease

Are neurocognitive developmental delays significantly present in children and adolescents living with sickle cell disease? What effect do these delays have on the overall morbidity associated with sickle cell disease?

Submitted by (@sicklecellwarrior)

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

Details on the impact of addressing this CQ or CC :

Conversations among the sickle cell population are increasingly focusing on mental health, neuropsycology as it relates to mental health, and the need to develop community life skills and personal development.

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
41 up votes
11 down votes
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Strategic Goal: 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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Strategic Goal: Goal 2: Reduce Human Disease

US-based Clinical Development of Innovative Medical Devices

Though innovative medical devices are often conceived of and developed in the US, US consumers are frequently the last to benefit. Innovators frequently go to market first in Europe and are now moving toward emerging countries, delaying the medical benefits available to the US population. Can the NHLBI and FDA’s CDRH, working together as sister agencies, develop strategies such as funding opportunities or collaborative ...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 :

Addressing this CC may empower the development of new regulatory paradigms within CDRH, enable the streamlined development of several NHLBI medical devices in the US, lead to a minimized delay in US availability for truly innovative technologies, and grow the pool of US clinicians experienced in working with device developers at the earliest stages of human/device interaction.

Feasibility and challenges of addressing this CQ or CC :

In the past 18 months

• NHLBI and CDRH have executed a structured working relationship, within the NIH Centers for Accelerated Innovations, where CDRH provides high-level feedback to early stage NHLBI medical device developers.

• CDRH has developed two new programs –one to enable US conduct of early feasibility studies/first-in-human (EFS/FIH) studies and a second to provide expanded access to senior agency reviewers for innovators developing high risk technologies.

Additionally, CDRH is focused on exploring and evaluating additional pilot programs to expand first-in-human trials within the US. NHLBI’s portfolio of awardees includes a number of medical device development projects that could qualify for the EFS/FIH program. Development of new collaboration or funding opportunities focused on this segment of device developers could attract additional innovators to the NHLBI family and encourage the US-based clinical development of their innovative technologies. The relationship that has been built between NHLBI and CDRH, in conjunction with CDRH’s more open approach to working with innovators, makes this the perfect time to expand NHLBI/CDRH innovator support beyond the NCAI program and into the overall NHLBI portfolio.

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

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

Role of vascular development in pulmonary hypertension

Does interrupted or aberrant pulmonary vascular development contribute to pulmonary hypertension?

Submitted by (@nhlbiforumadministrator)

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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16 net votes
28 up votes
12 down votes
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Strategic Goal: Goal 1: Promote Human Health

Intersecting Developmental Biology with Vascular Physiology and Biology

Although many think of the vasculature as a lump sum of vessels that all react in a similar fashion to a certain stimulus, e.g., alpha-adrenergic activation, this is not the situation. For example, coronary resistance vessels show little to no direct response to alpha-adrenergic activation while resistance vessels in most organs show marked constriction. Another example is the response of different vessels to angioplasty ...more »

Submitted by (@wchilian)

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

Details on the impact of addressing this CQ or CC :

A challenge facing many specialists in vascular medicine, vascular surgery, and cardiology is understanding the ramifications, and the basis, of the vascular pathology in the context of the organ system. Another way of re-stating this as a question is: Are the unique attributes of the vascular biology, pathology and physiology of a particular organ system connected to specific aspects of development. This question would help both the basic on clinical scientists understand the basis of why a blood vessel in the kidney may be different than one in the heart, or in the brain with the goal of devising more selective therapies to approach vascular disease in specific organs. Scientists in the area of vascular development have long appreciated that vascular cells in different organs arise from different embryological origins; yet how this information translates into the intricacies of vascular control, or responses to pathology is not resolved. Understanding the basic biological mechanisms of how the embryological source of the vasculature affects pathology and physiology could engender treatment of vascular disorders.

Feasibility and challenges of addressing this CQ or CC :

This idea could be implemented by encouraging multi-PI efforts from vascular developmental biologists, and investigators engaged in studies of microvascular control mechanisms and/or vascular biologists interested in vascular pathologies such as restenosis and vascular lesions. Advances in fate mapping techniques have enabled developmental biologists to track embryological origins of cells into specific organ systems into adulthood. With such a multi-faceted approach a better understanding of vascular physiology and pathophysiology will be obtained that hopefully will be translated into more effective treatments.

Name of idea submitter and other team members who worked on this idea : William M. Chilian

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15 net votes
26 up votes
11 down votes
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Strategic Goal: Goal 3: Advance Translational Research

Incentivizing Earlier Investment in NHLBI-Funded Technologies

How might NHLBI assist its awardees to attract private sector funding or partnerships earlier in the product development process to help bridge the gap between academic discoveries and product commercialization?

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 :

Attracting private sector support earlier in the development pipeline would help fill an important funding gap between academic discoveries and product commercialization, enabling products to reach patients more quickly, and improving the return on NHLBI’s investments in basic research.

Feasibility and challenges of addressing this CQ or CC :

Some existing initiatives such as the SBIR Phase IIB Bridge and Small Market Awards encourage non-federal investors to invest earlier in NHLBI-funded technologies. In addition, the NCAI is designed to support critical feasibility studies and business case development to de-risk earlier investment by the private sector. These efforts are showing early signs of success, but impact only a small proportion of NHLBI-funded basic research discoveries.

Estimates for the cost of developing a new drug or device range from the hundreds of millions to billions of dollars and 10-15 years to get from the lab to the patient. The NHLBI cannot fully support that development, so private sector support is critical for biomedical technologies to be commercialized. Overall private capital investment in the life sciences is increasing, but it is not being targeted at heart, lung, blood, and sleep technologies or at the seed stage of development. Venture capital investment in heart, lung, blood, and sleep technologies has declined or remained stagnant since 2008 (http://graphics.wsj.com/venture-capital-and-the-human-body/) and seed stage investment from the private sector for early stage high-risk projects is in short supply (PWC Moneytree: https://www.pwcmoneytree.com/HistoricTrends/CustomQueryHistoricTrend).

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

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15 net votes
23 up votes
8 down votes
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Strategic Goal: Goal 1: Promote Human Health

Lung Development Reactivation

What stimuli cause developmental processes to be reactivated in the lung?

Submitted by (@nhlbiforumadministrator1)

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
13 up votes
8 down votes
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Strategic Goal: Goal 3: Advance Translational Research

Substituting scientific-medical insight before profit in drug development

Since the Pure Food and Drug Act of 1906 and the rise of the FDA, the US federal government has directly inserted itself into medical research, primarily from a business perspective. The Orphan Drug Act of 1983 monetarily incentivized the process for rare diseases, but for ultra-rare diseases of < 1,000 patients, it does not work. Successful drugs for rare diseases have enormous price tags to compensate their development ...more »

Submitted by (@mtothbsf)

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

Details on the impact of addressing this CQ or CC :

By harnessing the vast resources of US biomedical research and biomedical researchers, many of whom were trained with NIH funding, the US can more effectively translate scientific discoveries into clinical benefit--many treatments for many diseases using many PIs. While the monetary profit incentive has been a vital aspect of pharmaceutical development over the last 50 years, in this era of personalized medicine it may not be as relevant. The individual PI with a good idea and sound scientific background may hold the key to a cure for a rare or personal disease, but is prevented from translating this because of ignorance, lack of institutional support, or the need for substantial capital investment to perform a proper clinical study. The FDA could recalibrate its requirements for clinical studies to allow even a single PI to test their idea economically, and perhaps offer the funding to do it properly. It should not go unnoticed that by understanding and treating a specific rare disease, the medical world may use this knowledge to better understand and treat diseases that affect larger numbers of people. For example, understanding or treating heart failure in a specific rare disease, may offer benefits to the larger group of heart failure patients.

Feasibility and challenges of addressing this CQ or CC :

Meeting this challenge will require a major rethinking of the traditional FDA approach of drug development and testing in humans at least for the non-profit or single PI scenario. For ultra-rare diseases of < 1,000 individuals there seems little hope that the for-profit sector would choose to be involved. For that reason and to take advantage of the enormous biomedical research resources the the US federal government has sponsored though the NIH, the US could advance translational research and usher in an era of true "personalized medicine'.

Name of idea submitter and other team members who worked on this idea : Matthew J. Toth, PhD, Science Director, Barth Syndrome Foundation Inc.

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6 net votes
11 up votes
5 down votes
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Strategic Goal: Goal 3: Advance Translational Research

Screening for SDB and Sleep Disorders in School-Aged Chidren by School Nurses

Can school nurses effectively screen for SDB and Sleep Disorders in school aged children? Who else in the school setting could provide such screening? Should such screening be limited to "at risk" children who display identified markers, or be open to all children? What is the role of teachers to "identify" children in need of such screening? What role will such screening serve to mitigate learning, behavioral, developmental ...more »

Submitted by (@nancyh.rothstein)

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 impact of screening at risk children for sleep disorders and sleep disordered breathing, including subsequent referrals and professional treatment, may serve to mitigate future health, learning, developmental and behavioral risks/issues for children by addressing these issues in early childhood. Research based protocols will be accessed and used for screening.

Feasibility and challenges of addressing this CQ or CC :

Additional considerations:

Does the nurse refer an at risk child to the Pediatrician? Dentist? ENT?

What questionnaires or other identifiers would be used for screening? Is there a bio test to assess risk for a SD or SDB?

What should the target age level be for children undergoing proposed screening?

 

How can sleep education and training be integrated with this screening process to promote good sleeping habits/hygiene at home, for all children, parents and caretakers, as well as teachers. Who creates and provides the educational material? Who does the teaching?

 

Parental involvement- KEY!

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

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5 net votes
6 up votes
1 down votes
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Strategic Goal: Goal 3: Advance Translational Research

Rx for HFpEF

HLBI should make it a priority to develop therapeutic options for the treatment of heart failure with preserved ejection fraction.

Submitted by (@johnrobinson)

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

Details on the impact of addressing this CQ or CC :

Heart failure with reduced ejection fraction (HFrEF), formerly called systolic heart failure, is the classical form of heart failure that is characterized by defective ventricular contraction. The most common variant of heart failure is heart failure with preserved ejection fraction (HFpEF), formerly called diastolic heart failure, characterized by resistance to ventricular filling. The prevalence of HFpEF has been rising steadily over the past two decades at a rate of increase of 1% per year, while the prevalence of HFrEF which has remained stationary. The most common causes of HFpEF are ischemia, obesity, hypertension, diabetes and ageing. Since the population is increasingly obese, hypertensive, diabetic and ageing, the incidence of HFpEF will be the dominant heart failure phenotype over the next decade. The clinical management of HFpEF is complicated by lack of therapeutic options that provide survival benefit. Therapies of proven benefit in HFrEF have repeatedly been shown to add little if any benefit in HFpEF. The prognosis of HFpEF is about the same as HFrEF, with 5-year mortality ranging from 54% to 65%.

Feasibility and challenges of addressing this CQ or CC :

Recent developments in our understanding of the molecular mechanisms of myofilament regulation and assays can be used to develop lead compounds for treating HFpEF.

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

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1 net vote
13 up votes
12 down votes
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Strategic Goal: Goal 4: Develop Workforce and Resources

Career Development in "Group Based" Science

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:

 

NHLBI should be challenged on how best to provide career development grants to junior faculty involved in “group based” clinical and bench science.

Submitted by (@wheeze)

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

Voting

7 net votes
26 up votes
19 down votes
Active