Showing 4 ideas for tag "methodology"

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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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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16 up votes
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Goal 2: Reduce Human Disease

Utilizing Competition to Enhance NHLBI’s Clinical Trial Enterprise

During this period of diminishing Federal resources and funding, what novel methods can be employed to effectively conduct clinical trials that address existing and emerging medical and public health issues?

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 thecritical challenge would improve clinical trial design, clinical trial efficacy and ultimately completion and reporting of trial results. This approach would most likely save money.

Feasibility and challenges of addressing this CQ or CC

The Other Transaction Authority already exists within the PHS and NIH and this method has been tested at NCI.
Competition is an important element of successful achievement in everything from sports rivalries to scientific innovation. Examples of this can be cited for the Larry Bird and Magic Johnson hoops competition, Muhammad Ali versus Joe Frazier boxing duels, and the contest between the Human Genome Project and Celera for completion of the mapping of the human genome. The US Defense Advanced Research Project Agency (DARPA http://www.darpa.mil)) has used the concept of competition to advance the development of never before imagined scientific and technological innovations including development of the internet, the SIRI voice recognition system, UNIX and the cloud, and the global positioning system, among many products and innovations. Under this paradigm great advances are initiated through a public challenge which brings competitors together to achieve a goal set by the government. This is accomplished through the use of the Other Transaction Authority (OTA), which has been extended to the NIH. Subsequent management of the challenge involves the setting of milestones and utilization of “go/no go” decision making. The NCI has used a “DARPA-like” approach to instill competition for the design and conduct of clinical trials and as a result has substantially improved the performance of its clinical trials networks / cooperative groups.

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

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Goal 2: Reduce Human Disease

Optimizing Utilization of Composite and Repeat Endpoints in RCT

The use of win-ratio and other new statistical methods to analyze endpoints in new and existing 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

It could enable trials to be powered with smaller sample sizes. It can also be used to combine diverse information in a way to better guide clinical decisions.

Feasibility and challenges of addressing this CQ or CC

The methodology exists and the need is growing as we try to sort out smaller treatment effects and our focus shifts from MI (which is fairly easy to define) to heart failure (which is more nebulous).
Traditional time-to-first endpoint analyses of clinical trials fail to capture the full impact of treatment in diseases with recurring endpoints (like heart failure hospitalization) or to capture the net benefit/risk of treatments with significant opposing effects (like the anti-thrombotic pro-bleeding effects of drugs like warfarin and dabigatran in atrial fibrillation or dual anti-platelet therapy after cardiovascular stenting), New statistical methods like the (win-ratio) when used together with patient- and clinician-based rankings of the importance of possible outcomes might provide a quantitative way to optimize the relevance of trials with multiple diverse endpoints to practical clinical decision making. I propose to encourage the use of these and other similar methodologies to analyze endpoints in new and existing trials. It would require a paradigm shift in how trialists look at endpoint data and in how regulators interpret trials.

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

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