Showing 26 ideas for tag "clinical"

Goal 2: Reduce Human Disease

Cardiometabolic Disease Risks Associated with Sleep Deficiency

How does insufficient sleep duration, irregular timed sleep schedules, and poor sleep quality contribute to the pathophysiology of lung, heart and blood 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

Sleep deficiency and untreated sleep disorders threaten the health of 20-30 percent of US adults through an increased risk of stroke, hypertension, diabetes, inflammatory disease, and all-cause mortality. Developing the scientific evidence-base of validated interventions will enhance the management of cardiometabolic and pulmonary risks to health, present new opportunities for secondary prevention, and reduce associated burden on health care systems.

Feasibility and challenges of addressing this CQ or CC

Improving sleep health through informed public recognition of decision-relevant science, and relatively low cost therapies for management of sleep disorders are available for immediate assessment of impact in appropriate clinical trials to demonstrate efficacy and effectiveness.
Discovery research advances implicate an array of cellular sleep and circadian mechanisms in pathophysiological pathways leading to cardiometabolic and pulmonary disease.

Irregular and disturbed sleep impairs cellular biological rhythm in all tissues and organs leading to oxidative stress, unfolded protein responses, and impaired cell function. The pathophysiological findings juxtaposed with epidemiological evidence of disease risk indicate that sleep deficiency contributes to an erosion of health across the lifespan over and above the effects of aging.

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

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94 net votes
122 up votes
28 down votes
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Goal 2: Reduce Human Disease

Biomarkers of Pulmonary Hypertension

What are informative and clinically relevant biomarkers of pulmonary hypertension (PH)?

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

Details on the impact of addressing this CQ or CC

This research emphasis would help identify novel pulmonary hypertension biomarkers of disease risk and progression that can be used for early detection or as outcome measures in prevention trials or treatment of PH, which is a disease currently still not curable with high mortality rate.

Feasibility and challenges of addressing this CQ or CC

NHLBI Division of Lung Diseases just launched the multi-center PVDOMICS research program last September that will enroll ~1,500 patients in the next 5 years for deep phenotyping PH. PVDOMICS will provide a perfect foundation and platform for this proposed featured study about informative and clinically relevant biomarkers of PH, and make answering this proposed question more feasible in the next 5-10 years.
Although significant advances in the treatment of pulmonary hypertension have been made in the past two decades, currently pulmonary hypertension remains a devastating disease without many clinically relevant and specific biomarkers available. Novel new informative and clinically relevant pulmonary hypertension biomarkers would greatly help advance the subtype-specific early diagnosis and precision treatment of this disease that could potentially decrease the mortality of PH.

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

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75 net votes
87 up votes
12 down votes
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Goal 2: Reduce Human Disease

Can Psychological Science Improve Weight Loss?

Will sensitivity to the psychological aspects of obesity, including lifestyle priorities and motivations, improve the efficacy of long-term effectiveness of weight loss and obesity prevention interventions?

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 primary focus on principles of psychology may result in significantly improved control of the obesity epidemic. Effective interventions could reduce the risk of diabetes, sleep apnea, and hypertension. This research could also affect clinical practice guidelines for weight loss and obesity treatment.

Feasibility and challenges of addressing this CQ or CC

Psychological science has been successful in developing effective treatments for a number of conditions, including sleep disorders, depressive symptoms, anxiety and phobias. Many of the behavioral principles employed in such interventions (e.g., cognitive restructuring, motivational methods) could be translated for the prevention and treatment of obesity within a reasonable time frame. Additional attention should be directed to the needs of population subgroups in which obesity is most prevalent.
In their Viewpoint article on weight loss intervention research, Pagoto and Appelhans (JAMA, 2013, see attachment) question whether a continued focus on dietary factors in research on weight loss and obesity is warranted. Their commentary raises the importance of attention to the individual psychological characteristics that influence adherence to weight loss interventions rather than dietary composition.

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

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51 net votes
104 up votes
53 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 »

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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Goal 2: Reduce Human Disease

How can we increase the pharmaceutical clinical research of targeted therapies in pediatric PAH patients, including encouraging

Clinical research, especially randomized pharmaceutical clinical trials, poses many unique challenges compared to research in adult subjects. In pulmonary arterial hypertension, a disease characterized by high blood pressure of the lungs with increased pulmonary vascular resistance leading to right ventricular failure, there are 12 FDA-approved PAH-targeted therapies for adults. None of these medications are currently... 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

Pulmonary arterial hypertension is a heterogeneous condition generally characterized by high blood pressure in the lungs and increased pulmonary vascular resistance that leads to right heart failure if left untreated. Though some causes of PAH are seen in both adult and pediatric populations, some etiologies are seen exclusively in pediatric populations, including persistent pulmonary hypertension of the newborn, bronchopulmonary dysplasia, lung hypoplasia, and alveolar capillary dysplasia. Despite these differences in disease etiology, and known physiologic differences in pediatric populations, inhaled nitric oxide (iNO) in the acute setting is the only approved medication for PAH treatment in children. A number of issues have decreased pediatric PAH pharmaceutical research, including protection of the pediatric population as vulnerable subjects, principle of scientific necessity, balance of risk and potential benefit, parental consent/child assent, and feasibility of pediatric clinical trial design and implementation. Encouraging clinical trials of existing adult medications and potentially emerging, novel agents specifically for pediatrics—either through direct sponsorship or regulatory incentives—would not only lead to better outcomes for pediatric PAH patients, but potentially to a better and more comprehensive characterization of the developing pulmonary vascular system and right ventricle.

Feasibility and challenges of addressing this CQ or CC

Several challenges exist for addressing this critical challenge. First, there are a number of differences between conducting clinical research in pediatric populations compared to adult populations. This not only includes the broad items referenced above, but items as noted by Rose and colleagues related to clinical trial design and analysis including (1) accepted age-matched normal ranges for laboratory values; (2) requirements for the validation of clinical endpoints for the assessment of efficacy and safety; and (3) standards for long-term safety monitoring and pharmacovigilance (Rose K, et al. NEJM 2005). Sponsorship of this type of clinical research is a second concern, which could either be mitigated by direct support from the National Institutes of Health of pediatric PAH clinical trials or in regulatory changes incentivizing pediatric clinical research in rare diseases.

Name of idea submitter and other team members who worked on this idea Katherine Kroner, Michael Patrick Gray, PHA

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66 net votes
76 up votes
10 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?

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 2: Reduce Human Disease

Tools for Clinical Research

Clinical trials are at a crossroads. They are too expensive, take too long, and often can't recruit adequately. A critical challenge is to develop tools that can change the cost, time, and recruiting practices for 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

Developing tools to facilitate and streamline the day-to-day performance of clinical trials would help investigators and NHLBI alike. Trials would recruit more quickly, at less cost. We could, therefore, potentially fund more trials. In addition, if we have common templates, this would facilitate a number of things ranging from peer review to IRB submissions, and would also be beneficial to new investigators by providing a framework for them to use.

Feasibility and challenges of addressing this CQ or CC

Some of it will involve 'just doing it' - for example, we have the capability now to require common protocol and informed consent formats. We have sufficient data to develop performance milestones and implement them. The hardest part will be figuring out how to develop pools of patients, or with which organizations to collaborate in order to achieve this. However, 5-10 years should be ample for this and related activities.
For example, we need to emulate and collaborate with other organizations in figuring out how to identify large numbers of patients willing to participate in our trials. We need performance milestones and metrics, and we need tools like common templates for protocols and informed consent forms that all NHLBI-funded trials would use.

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31 net votes
44 up votes
13 down votes
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Goal 2: Reduce Human Disease

Improving Representation of the Elderly in Clinical Research

There is a need to optimize long-term cognitive and functional outcomes in the aging population during and after cardiothorasic surgery, including the development of simple, objective tools to enable risk stratification for vulnerability to neurocognitive deficit. First, cardiothoracic surgical trials and clinical studies should be more "age-representative" and reflect the increasing proportion of the aging population.... 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

Many answers would result from including the elderly in ongoing and planned clinical research and trials.

Feasibility and challenges of addressing this CQ or CC

This population is growing rapidly and available to be studied.
Elderly patients as a group face higher acute risks for complications and death following cardiothoracic surgery or coronary interventions, yet may also gain more long-term benefits from surgery due to their more severe disease. The balance between risks and benefits of surgery may be different in the older patient than in their younger counterparts. Major randomized trials that defined the “standard” for the field (e.g., CASS, VA cooperative, RITA, BARI, STICH) often failed to enroll many, if any, patients aged 75 years or older. The Joint NHLBI-AATS Working Group (http://aats.org/CME/2011-AATS-NHLBI-Symposium.cgi) identified several areas of high priority for cardiothoracic surgery research in the elderly.

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 2: Reduce Human Disease

Assessing current methodologies for clinical trials

Are the current methodologies for clinical trials still the best practices for conducting efficient clinical trials?

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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10 net votes
16 up votes
6 down votes
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Goal 2: Reduce Human Disease

Metrics to Predict Success of Clinical Trials

What are the metrics that can predict success of clinical trials?

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 ability to identify factors, both ahead of time, and during the trial, that will predict the success of the trial would permit considerable efficiencies for NHLBI. We should be able to select a group of trials that will be able to recruit on time and within budget. For others where there may be less chance of success, we may want to invest time and resources in mentoring the investigators (e.g., in the case of new investigators), or in helping to redesign the trial. This latter function could be especially helpful during the pre-application process.

Feasibility and challenges of addressing this CQ or CC

NHLBI has developed a critical mass of expertise in portfolio analysis, and is working on developing the IT tools to match. This area was identified as a high priority by the internal IMPACT Task Forces at their recent retreat, so there is also widespread interest in this approach as well.

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

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9 net votes
15 up votes
6 down votes
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Goal 2: Reduce Human Disease

Research in new methods for large simple trials

We need new ideas in how to make clinical trials more efficient and cost effective. (Randomized) comparison of different methods for accrual might be of interest; blinded versus unblinded trials; behavioral controls versus placebo controls or usual care controls might be explored.

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

Details on the impact of addressing this CQ or CC

This will aid our discussions of appropriate trials to fund.

Feasibility and challenges of addressing this CQ or CC

We need some answers in order to continue to fund the best possible trials with our limited resources.

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

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7 net votes
16 up votes
9 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

New models for clinical trials

There is a need to educate the scientific community and program staff about the use of metrics, results-based accountability, and other business models to improve the science, productivity, and efficiency of clinical trials and clinical trial networks.

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

Details on the impact of addressing this CQ or CC

Improve the efficiency of clinical trials and a return on research investments

Feasibility and challenges of addressing this CQ or CC

Yes, this is feasible.

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

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4 net votes
10 up votes
6 down votes
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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.

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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5 net votes
6 up votes
1 down votes
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Goal 2: Reduce Human Disease

Towards Collaborative Funding of Clinical Trials

A way for clinical trial investigators to submit ONE application with ONE review and ONE funding decision, and the application would ask for funding from multiple funders (e.g. NHLBI and another IC, NHLBI and PCORI, NHLBI and AHA, NHLBI and CIHR, NHLBI and MRC).

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 be much easier for investigators from multiple sites/countries to secure funding for large-scale trials from multiple sponsors. They would only have to submit ONE application, respond to ONE review, and anticipate ONE funding decision.

Feasibility and challenges of addressing this CQ or CC

Clinical trials have become increasingly difficult to afford, yet the need for them has never been greater. Many other sponsors (CIHR, PCORI, AHA, MRC, European Union) are eager to work with NHLBI to enable user-friendly multi-sponsor funding. Some similar type arrangements are already happening with other IC's (e.g. NINDS is working with CIHR and the UK MRC).
Large-scale clinical trials often require involvement of multiple sites, often located in > 1 country. Furthermore, the expense of trials often raises questions as to whether funders could collaborate, all contributing a certain amount. However, there is no simple user-friendly way for applicants to bring secure multiple sources of funding. Ideally, the division of funds would be agreed upon prior to application. In case of foreign funders, no monies would cross borders -- i.e. for NHLBI and UK MRC applications, the NHLBI would fund American sites while the UK MRC would fund UK sites, but all funding goes to ONE trial with ONE protocol and ONE data set.

One challenge would be politics. Who will do the review? NIH has traditionally acted as if it is the only agency capable to doing a valid review. Would NIH be willing to accept a review conducted by another sponsor? Would other sponsors be willing to accept a review fully run by NIH?

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

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8 up votes
9 down votes
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