Showing 9 ideas for tag "trial"

Goal 2: Reduce Human Disease

Diet and prevention of cardiovascular events

In the US, what kind of diet(s) is/are best for preventing hard cardiovascular events?

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

Details on the impact of addressing this CQ or CC

Provide an evidence base for public policy on diet.

Feasibility and challenges of addressing this CQ or CC

The Spaniards have shown that this kind of trial is possible. We have tools and interest in place for pragmatic trials.

The PREDIMED trial (done in Spain) randomized ~7000 adults and found that a diet supplemented with olive oil or nuts reduced cardiovascular events compared to a "low-fat" diet. However, in Spain the Mediterranean diet is arguably the norm.

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

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20 up votes
12 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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19 up votes
15 down votes
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Goal 3: Advance Translational Research

T4 Translation Research Informing Early Stage Translational Research

There is a need to utilize insights gained from T4 translation research and implementation science to inform the design and execution of early-stage translational research and 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

• Assure that early stage translation research will be suitable for implementation in real world setting
• Aligns the research interventions from T1-T3 research to those appropriate to T4 research
• Potential to focus early stage research in key high burden areas
• Provides research community an understanding of the connections from early stage to late stage translation research which will potentially refine research strategies and directions at all levels

Feasibility and challenges of addressing this CQ or CC

• Promote the importance of translation to population of heart, lung, blood, and sleep researcher to broader research community
• Potential for more T4 research contributions for guiding investment into translation research from T1-T3
• Provide avenues for T1-T3 investigators to translate their ideas into positive outcomes for population health
• Successful T4 research will stimulate feedback loop and identify opportunities for early translation research

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

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3 net votes
13 up votes
10 down votes
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Goal 2: Reduce Human Disease

Should clinical primary prevention of ASCVD be guided by subclincal disease or estimated risk?

Current approaches to guiding use of clinical primary prevention interventions, e.g., statins and aspirin, are based on treating patients who exceed a specific risk threshold. The performance of risk estimation is good, but not outstanding, and results from clinical and population studies continue to support the value of new biomarkers. Given the widespread use of preventive therapies, the lack of untreated cohorts is... 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

The size of the US and global population qualifying for treatment with a statin or aspirin for primary prevention of ASCVD is immense. Given the performance of risk estimation, even if risk estimation were universally implemented, patients would be misclassified with the consequence of being under or over treated. If treatment based on presence of subclinical disease is more cost-effective, the benefits of preventive therapies can be enjoyed by larger proportions of our population and more ASCVD can be averted. Given the ionizing radiation, albeit low intensity, associated with CT scanning, it is incumbent on the biomedical research community to document the advantages, if any, of a subclinical disease guided approach to provision of clinical primary prevention services for ASCVD.

Feasibility and challenges of addressing this CQ or CC

Many people will be concordant for the two methods of guiding provision of therapy, about 65% of middle aged and older adults. That is, many people will be high risk and have subclinical disease and many people will below risk and not have subclinical disease. It is only the discordant people, i.e., high risk people without subclinical disease and low risk people with subclinical disease, who will be informative study participants. Hence, many people will need to be screened to identify the roughly 35% who are discordant, and would be treated differently by the two approaches.

People may be unwilling to accept randomization once they know the information about their estimated risk and presence or absence of subclinical disease. If a low participation rate among eligible persons is observed, an even larger population of screenees would be needed.

A vanguard phase could provide information about these potential challenges.

Name of idea submitter and other team members who worked on this idea David Goff, Donald Lloyd-Jones, Phil Greeland.....

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-3 net votes
6 up votes
9 down votes
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Goal 2: Reduce Human Disease

The registry randomized trial

Conventional trials are becoming too expensive and complex, and have low recruitment rates and are not generalizable.

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 challenge is to conduct efficient, streamlined, inexpensive and generalizable trials. The RRCT can address this challenge.

Feasibility and challenges of addressing this CQ or CC

High feasibility. Concept and platform are in place for example in Sweden and are extendable to e.g. the VA and Medicare.

Name of idea submitter and other team members who worked on this idea Lars H. Lund

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11 up votes
10 down votes
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Goal 2: Reduce Human Disease

Testing PCSK9 Inhibitors

Several inhibitors of PCSK9 are in phase 3 development and show considerable promise for improving the lipid profile; they will be especially appropriate for patients with Familial Hypercholesterolemia and those with statin intolerance. The sponsoring pharmaceutical companies need to complete CVD endpoint trials with full safety testing. However, there may well be more than one drug approved for marketing. The sponsors... 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

Admittedly, this idea is on the mid-range horizon at best, since it will be some years before we know which, if any, PCSK9 inhibitors make it to market. However, if the data produced in phase 3 trials is inadequate to select among marketed drugs, a comparative effectiveness trial would be in order. If the then existing data are compatible with equipoise between the agents, a pragmatic trial design could likely be justified.

Feasibility and challenges of addressing this CQ or CC

Such a trial would be quite feasible, assuming it is justified by the data.

Name of idea submitter and other team members who worked on this idea Stephen P. Fortmann

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4 up votes
3 down votes
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Goal 2: Reduce Human Disease

Bariatric surgery and CV outcome

Does bariatric surgery reduce the risk of MI, stroke, heart failure, and premature death in moderately obese adults?

Does the type of surgery matter?

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

Details on the impact of addressing this CQ or CC

Should bariatric surgery be offered to moderately obese adults as a way to prevent CV events.

Feasibility and challenges of addressing this CQ or CC

A number of smaller trials have been done and have shown that bariatric surgery can induce remission of diabetes. The bariatric surgery community is primed to conduct trials. Through the HCS collaboratory and PCORI, we believe it is possible to conduct pragmatic trials at reasonable cost by leveraging existing resources.
Bring in parties to aid in data collection and procedure payments (insurance companies, CMS, professional society registries)

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

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-9 net votes
21 up votes
30 down votes
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