Showing 6 ideas for tag "design"

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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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
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Goal 3: Advance Translational Research

Novel Mechanism for Clinical Trials of New Pro-Hemostatic Agents in Hemophilia

There are new exciting novel pro-hemostatic therapeutics in early phase clinical trials for hemophilia and hemophilia inhibitor patients. Yet, it is difficult to design randomized trials to compare these agents, or compare them with standard treatment, given the small sample size and competing studies for such patients. It is critical to develop novel approaches to compare new agents in rare populations. For example,... 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

Should rare disease-specific strategies for clinical trials be developed, it would revolutionize the approach to study of not only rare disease populations, but all disease groups, no matter their size. If validated, rare disease-specific clinical trial strategies, would potentially reduce the cost, time, patient burden, and research effort to conduct clinical trials. If validated, consideration could be given to drug licensure earlier in the trial process, with a requirement for all such trials to initiate and continue ongoing data collection post-licensure for safety and efficacy.

Feasibility and challenges of addressing this CQ or CC

Novel statistical methodologies are greatly needed to help with rare disease research. NHLBI might consider a grant mechanism RFA to encourage development of novel clinical trial strategies utilizing smaller sample size. The proof would be to develop the methodology as part of a feasibility study, and then, if feasible, adapt the novel approach to development and conduct of a clinical trial in a rare disease clinical tria,l to test the concept.

Name of idea submitter and other team members who worked on this idea Margaret V. Ragni, MD, MPH (aspects discussed with Don Brambilla PhD.

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

Data from regulatory studies a barrier to evidence-based medicine

Alignment of regulatory, healthcare, and research arms of the government is poor. There is a need to improve the design, quality and usefulness of data from regulatory studies to address major clinical questions and also to facilitate scientific inquiry. This is a barrier to evidence based medicine and improved treatments.

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 Society for Vascular Surgery

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Goal 3: Advance Translational Research

Spinal Cord Injury: hype, unmet promises, and misery which does not need to be

Research to "fix" spinal cord injury in humans, has been insanely hyped, rare in reality, and very disappointing in its clinical applicability to human patients.

After a parade of rat models, mouse models, cat models, dog models, African green monkey models, pig models, guinea pig models, hamster models, rabbit models, gerbil models, etc. one wonders whether most researchers or funders will ever have any interest in... 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

So, how about we put a moratorium on FDA approval of all research related to a cure for spinal cord injury when done by any persons or organizations who have not published every single one of their past experiments in the time required, and for any research which involves other species than humans.

Also, how many different ways of creating stem-like cells do we need? Let's stop creating stem-like cells and start applying the ones we have to human patients.

How about spending the animal model money instead on improving the quality of life for people living with disabilities. Let us start with actually enforcing the ADA on all new enough buildings.

When we make technology for doctors to use, we consult doctors. When we make technology for teachers to use, we consult teachers. When we make technology for disabled people to use… we consult insurance companies, and medical professionals who are not and have never been disabled. We fail to apply the most basic usability testing to the tools which disabled people must use. For example, has any wheelchair designer tried to propel a manual wheelchair uphill on wet grass? How about across a cobblestone street? or down a normally bumpy sidewalk? Now imagine that your butt has atrophied and you are sitting on your hip bones. How painful would that be? Now remember that pressure sores resulting from this bad design can be fatal, and then tell me why we do not take this more seriously.

Feasibility and challenges of addressing this CQ or CC

The misery which does not need to be is not a new idea: http://badcripple.blogspot.com/2015/01/obsession-with-walking.html

Let's get the price of tools for disabled people down to the point where most patients can really afford them, or where the insurance can actually cover them. Let's get exoskeletons price-competitive with wheelchairs, for example, instead of using them to make soldiers able to carry heavier packs in war.

Name of idea submitter and other team members who worked on this idea Mary-Anne Wolf (inspired by the Bad Cripple blog of William Peace and by the Wheelchair Driver website forum)

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12 down votes
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