Showing 23 ideas for tag "development"

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.

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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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 »

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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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?

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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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?

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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Goal 4: Develop Workforce and Resources

Implementation Research Workforce Addressing Health Inequities

What are the best strategies to develop a highly competent diverse Implementation Science research workforce to address health inequities?

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

Details on the impact of addressing this CQ or CC

• Enhance fundamental knowledge about new and trans-disciplinary D&I field.
• Improve understanding on ways to scale-up and deliver proven interventions to address health inequities.
• New knowledge generation regarding important adaptations of interventions implemented in the local context.
• Improve health outcomes, particularly in underserved populations in both the U.S. and abroad.
• Successful D&I research training programs will help ensure a competent diverse D&I research workforce.
• Identification of the most effective career timing and combination (balance) of discipline-specific and trans-disciplinary courses essential to develop a cadre of trans-disciplinary implementation science researchers.

Feasibility and challenges of addressing this CQ or CC

Feasibility:
• Dedicated NHLBI Center to promote, develop, implement, and disseminate research findings to address heart, lung, blood, sleep-related conditions and diseases.
• Identified new approaches to creating partnerships with trans-disciplinary research teams that expand beyond academia and increased understanding of the unique nature of mentorship needed for this discipline.
• Experience from several other ICs can be leveraged to improve or ability to be successful and decrease our launch time.
• Field is gaining momentum because of the realization of the unsustainable economic burden of health inequities (expected to increase in the future) in the U.S.

Challenges:

 

• Dedicated training mechanisms are needed to develop and meet our current/future T4 research workforce needs to address health inequities.

• Resources needed that provide unique training approaches (e.g., a trans-disciplinary scientific training environment, knowledge and experience with health disparity populations, unique training faculty (mentor) composition and opportunities to train mentees as future D&I mentors, innovative research tools and research experiences, and broad and diverse partnerships.

• Unique linkages with practice settings across disciplines needed.

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

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11 net votes
20 up votes
9 down votes
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Goal 4: Develop Workforce and Resources

Modernizing Research Training

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:

 

Since the focus of research has changed over the past decade, training programs need to be encouraged to use newer models of research in their training and mentoring of potential research faculty.

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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10 net votes
23 up votes
13 down votes
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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.

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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7 net votes
26 up votes
19 down votes
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Goal 3: Advance Translational Research

Genome Profiling

What structural changes need to be implemented in the health-care community in order to support the use of genomic information in clinical trials and drug development for hematologic diseases?

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

Details on the impact of addressing this CQ or CC

In various blood disorders, including hematologic malignancies, there are both inherited and somatic genetic alterations that contribute to predisposition, transformation, disease progression, responsiveness to therapy, and treatment complications. The presence of such genetic alterations underscore the need for the identification of rare but traceable mutations as well as the integration of such genomic information into clinical trials. By implementing a few structural changes in the healthcare sector, a clinical trial infrastructure can be established that accounts for proper application of sequencing technology. Some examples include the creation of genome diagnostic networks that address accrual of sufficient patients, procurement of suitable tumor/non-tumor material for sequencing, as well as pharmacodynamic and correlative biology studies in hematologic diseases.

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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11 net votes
22 up votes
11 down votes
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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 »

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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Goal 1: Promote Human Health

Critical Windows in Early Development to Maximize Lung Health

Is there a critical window of growth and development for maximizing lung function?

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

Details on the impact of addressing this CQ or CC

Low lung function during childhood tracks to early adulthood and contributes to early onset disease. Lung health promotion is needed, but we know little about what can enhance and protect human health during rapid phases of lung development in utero and growth postnatally to adulthood.

Feasibility and challenges of addressing this CQ or CC

Researchers could turn their attention on healthy and “maximally” health populations (human and model organisms) to understand genetic and environmental exposures that influence lung function at upper ends of the spectrum (>2 SD from the mean).
Recent findings suggest that there is an urban-rural continuum of lung function in specific ethnic groups; and interventions with maternal dietary supplements can enhance lung function in offspring. These set the stage for further study on developing knowledge of early life events that can inform lung health promotion.

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

Translating cardiac development/genetics knowledge into therapy

What is needed to translate our knowledge of cardiac development and congenital heart disease genetics into novel diagnostic and/or therapeutic strategies for congenital or acquired heart disease?

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 new therapies for congenital or acquired heart disease.

Feasibility and challenges of addressing this CQ or CC

We are poised to take advantage of the incredible advances in our understanding of cardiac development and genetics which have resulted from the development of high throughput technologies.

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

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10 net votes
21 up votes
11 down votes
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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 »

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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Goal 2: Reduce Human Disease

Fetal basis for Adult Disease

Maternal exposures during pregnancy have the potential to alter development and lead to lifelong susceptibility to disease. There is epidemiological evidence of this in the asthma field, where maternal smoking leads to increased asthma rates. However, the molecular mechanisms by which maternal exposures cause lung disease later in life are not known and the influence of in utero exposures on susceptibility to lung cancer,... more »

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 the impact of maternal exposures on fetal lung disease would make a major impact on human health by giving regulatory agencies the data they need to make educated decisions with respect to issues such as nicotine regulation, air pollution, water pollution, and other sources of maternal exposures.

Feasibility and challenges of addressing this CQ or CC

With the established animal and stem cell models of development, this research is immediately feasible.

Name of idea submitter and other team members who worked on this idea Diane Carlisle

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10 net votes
17 up votes
7 down votes
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