Showing 23 ideas for tag "clinical"

Goal 3: Advance Translational Research

Multicenter trials of therapies for rare diseases.

Infrastructure for performing research in rare diseases should be enhanced to allow efficient accrual to multicenter 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

Many of the malignant and non-malignant blood diseases that fall under the purview of the NHLBI are uncommon though, in aggregate, important contributors to the burden of disease in the US population. Although in some areas, like blood and marrow transplantation (BMT), there exists an infrastructure for multicenter trials, in many areas there does not. This makes testing potentially effective therapies very difficult. The large increase in the number of national BMT trials following the implementation of that network indicates the effectiveness of the approach, which could be expanded to include cellular therapies and other novel approaches.

Feasibility and challenges of addressing this CQ or CC

The current R01 process does not lend itself to efficient and rapid implementation of trials to test new approaches or to the time needed to complete trials that focus on long-term survival and quality of life endpoints. Even within the existing transplant network, efficiencies could be gained by infrastructural enhancements like a common IRB or government-assisted contracting (i.e., CRADAs), a streamlined process for protocol review (e.g. a one- versus two-step process), etc. Additionally, enhanced ability to collaborate with other organizations or institutes, without undue bureaucratic burden, would allow better use of NIH funds so that more trials could be done.

Name of idea submitter and other team members who worked on this idea Mary Horowitz

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80 net votes
114 up votes
34 down votes
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Goal 3: Advance Translational Research

Behavioral and Clinical Researcher Interactions

A critical challenge is the need to bring together basic behavioral scientists interested in understanding human behavior at a fundamental level, including how and why people become motivated and capable of undertaking behavioral changes, with clinical researchers interested in developing and testing new strategies for tackling resistant behavioral problems such as obesity and non-adherence.

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

Feasibility and challenges of addressing this CQ or CC

Obesity and non-adherence are two of the most important yet challenging behavioral problems contributing to cardiovascular morbidity and mortality. One way to address this challenge is to fund a network of “behavior change labs,” each of which bring together teams of basic behavioral scientists who are investigating the bases of behavior and behavior change with clinical researchers interested in designing, optimizing and testing novel ideas for tackling the difficult behavior problems of obesity and non-adherence to cardiac medications.

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

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20 net votes
52 up votes
32 down votes
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Goal 3: Advance Translational Research

Advancing the science of translating evidence into practice

What are the best ways for the NHLBI to advance the evolving science of translating robust evidence into clinical practice domestically and globally? How to personalize broad research evidence for individual patients?
How to predict and evaluate the impact of evidence-based interventions?
How to identify implementation methods available in industry and elsewhere that work best and are most translatable in healthcare?... 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

• Reduce mortality and morbidity
• Improved quality of life
• Higher proportion of people receiving evidence-based care and at goal for that care
• Reduced disparities in health and healthcare

Feasibility and challenges of addressing this CQ or CC

Challenges:
• Lack of research methodology in this area – may need new scientific approaches

• Lack of current capacities and capabilities in this area

• Current silos that separate research enterprise from industry, as well as NHLBI from other ICs

• Divisions between performance of clinical trials and implementation research

• Lack of clarity which federal agencies and NIH Institutes are ‘in charge’ of implementation and/or prioritize this as part of their mission and budget

• Lack of wide sharing of best practices of other implementation models

• Improving the science in this area needs to include methods and metrics development

• The accumulated knowledge of clinical trialists and implementation researchers is often not shared

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

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13 net votes
27 up votes
14 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 3: Advance Translational Research

Halt the Epidemic of Atrial Fibrillation

Effective approaches are needed to halt the epidemic of atrial fibrillation (AF) and its associated morbidity.

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

Details on the impact of addressing this CQ or CC

Reduce the almost epidemic increase in the occurrence of AF in the aging U.S. population. This is a critical need for which basic science and clinical tools presently exist to address and resolve into a means to reduce the clinical consequences of AF.

Feasibility and challenges of addressing this CQ or CC

To accomplish this goal the following approaches could be taken:
GENETIC: Investigate genetic factors that drive susceptibility to atrial fibrillation in various disease states and “lone” AF.
BASIC: Investigate the principles underlying electrical and structural remodeling which facilitate and perpetuate atrial fibrillation. Use a systems approach to aid the understanding of the role of neurohormonal and other organ system influences on human cardiac electromechanical activity. Develop new imaging modalities to better characterize conduction abnormalities in three dimensions. Investigate the nature of chamber-specific channels as potential targets for AF therapies. Encourage the development of new thrombin inhibitors and other potent but safe anticoagulants.
TRANSLATIONAL: Investigate promising pharmacologic or other interventions designed to reduce the incidence of atrial fibrillation in animal models with spontaneously occurring atrial fibrillation. Create the infrastructure for a “dynamic repository” of clinically obtained fresh human cardiac tissue for the study of AF.
CLINICAL: Improve the collection of atrial fibrillation as an endpoint in large phenotyped cohorts. Evaluate the safety and efficacy of ablative procedures relative to appropriate pharmacologic therapies. Evaluate interventions which may prevent first development and recurrence of AF (statins, ACE-I/ARB, beta blockers). Investigate the use of new antiplatelet thienopyridines for stroke prevention in AF.

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 3: Advance Translational Research

Definitive Evidence of the Effectiveness of Pulmonary Rehabilitation

What is the clinical effectiveness of pulmonary rehabilitation in reducing hospital admissions and readmissions, improving health outcomes such as exercise tolerance and dyspnea, and positively impacting patient centered outcomes. Does this effectiveness vary based on the types of settings rehab is conducted in, urban vs rural environments, the components to the program, the timing of the program and the overall support... 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

Pulmonary rehabilitation is a critical component in the treatment of COPD patients but several barriers persist that have resulted in very limited access to rehab, low referral rates for eligible patients and limited standardization of best practices within the rehab facilities that do exist. Large, definitive studies accounting for patient subgroups, site characteristics and program components can generate the level of evidence needed to expand access, educate providers and improve referral systems and create quality programs. This level of evidence is necessary to change policy to properly value the role of pulmonary rehabilitation and to convince integrated health systems in a value based market that pulmonary rehabilitation is beyond a doubt, a requirement of providing quality COPD care.

Name of idea submitter and other team members who worked on this idea Grace Anne Dorney Koppel, COPD Foundation Board of Directors, COPD Patient Advocate

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9 net votes
12 up votes
3 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 3: Advance Translational Research

Embedding Clinical Trials in Learning Health Systems

What are the best methods for using genotype information and other EMR data to randomize heart, lung, blood, sleep patients to different treatment strategies? One big challenge is how to consent patients for this sort of trial. Must patients be consented separately for every such trial or could there be blanket consent for participating in the learning health care model? This would also require a paradigm shift in how... 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

If successful this approach should enable the conduct of cheap pragmatic trials that are fueled by data from clinical care. The integration into clinical care helps assure efficiency and generalizability of results.

Feasibility and challenges of addressing this CQ or CC

The advent of electronic medical records and the explosion of big data technology has made it possible to gain access to and analyze data in a manner that would have been unthinkable 10 years ago. This is already going on in other fields.
Health care systems are increasingly using "big data" approaches to track outcomes in the patients treated with different strategies and drugs, and apply the knowledge gained from outcomes in previous patients to inform decision making in subsequent patients ("learning"). This approach could be used to personalize treatment. A recent example from cancer is to genotype lung tumors, and tailor the treatment of a new patients to drugs producing good results in patients with similar tumor genotypes. When two or more treatments produce similar results, one could randomize. Cardiovascular disease presents a challenge in using genotyping information to personalize treatment, because the manifestations are the results of complex genetic and environmental risk factors.

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

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4 net votes
15 up votes
11 down votes
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Goal 3: Advance Translational Research

Interventions to Reduce Low-Value Care

In 2010, the IOM issued a report stating that waste accounted for 30% of health-care spending, or some $750 billion dollars annually, approximately 25 times the annual NIH budget. How can we address and avoid waste and low-value care? Like any complex problem, there are myriad causes and no simple solutions. Defensive medicine, financial incentives, and physician knowledge deficits all contribute and represent potential... more »

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

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3 net votes
12 up votes
9 down votes
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Goal 3: Advance Translational Research

Substituting scientific-medical insight before profit in drug development

Since the Pure Food and Drug Act of 1906 and the rise of the FDA, the US federal government has directly inserted itself into medical research, primarily from a business perspective. The Orphan Drug Act of 1983 monetarily incentivized the process for rare diseases, but for ultra-rare diseases of < 1,000 patients, it does not work. Successful drugs for rare diseases have enormous price tags to compensate their development... 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

By harnessing the vast resources of US biomedical research and biomedical researchers, many of whom were trained with NIH funding, the US can more effectively translate scientific discoveries into clinical benefit--many treatments for many diseases using many PIs. While the monetary profit incentive has been a vital aspect of pharmaceutical development over the last 50 years, in this era of personalized medicine it may not be as relevant. The individual PI with a good idea and sound scientific background may hold the key to a cure for a rare or personal disease, but is prevented from translating this because of ignorance, lack of institutional support, or the need for substantial capital investment to perform a proper clinical study. The FDA could recalibrate its requirements for clinical studies to allow even a single PI to test their idea economically, and perhaps offer the funding to do it properly. It should not go unnoticed that by understanding and treating a specific rare disease, the medical world may use this knowledge to better understand and treat diseases that affect larger numbers of people. For example, understanding or treating heart failure in a specific rare disease, may offer benefits to the larger group of heart failure patients.

Feasibility and challenges of addressing this CQ or CC

Meeting this challenge will require a major rethinking of the traditional FDA approach of drug development and testing in humans at least for the non-profit or single PI scenario. For ultra-rare diseases of < 1,000 individuals there seems little hope that the for-profit sector would choose to be involved. For that reason and to take advantage of the enormous biomedical research resources the the US federal government has sponsored though the NIH, the US could advance translational research and usher in an era of true "personalized medicine'.

Name of idea submitter and other team members who worked on this idea Matthew J. Toth, PhD, Science Director, Barth Syndrome Foundation Inc.

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6 net votes
11 up votes
5 down votes
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Goal 3: Advance Translational Research

Obstacles to the timely completion of clinical trials

There are numerous obstacles to the timely completion of trials, and there is a crisis in US enrollment rates. Overcoming barriers to timely completion of clinical trials would have a profound impact on accelerating research translation to improving health. Clinical investigations are necessary to advance the prevention, diagnosis, treatment and cures of human disease. The rate of basic scientific discovery has overwhelmed... more »

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

Feasibility and challenges of addressing this CQ or CC

For comparative effectiveness trials, incentives are misaligned; it is often easier, financially advantageous and benefits practice/hospital metrics to perform tests/procedures clinically than in the context of a trial. A collaboration between NHLBI, FDA and CMS would markedly accelerate many trials by aligning incentives. There are a few small examples with some success eg, Carotid stenting. Regulatory burdens should be reduced.

Solutions to enrollment challenges need to address physician and participant related barriers. NHLBI and AHA could lead a public private partnership and with patient advocacy groups (eg, Research America) and the media to enhance the understanding and acceptance of participation in clinical research.

To advance the efficiency of clinical trials NHLBI should support investigation of methodologies such as event driven trials, adaptive design, Bayesian approaches, multiple randomized groups, group/cluster randomized trials, maximizing information from subgroup analyses.

Name of idea submitter and other team members who worked on this idea AHA Staff & Volunteers

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6 net votes
8 up votes
2 down votes
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Goal 3: Advance Translational Research

Integrated Clinical Guideline on Comorbidities in Primary Care

The development of systematic evidence reviews (SER) that provide the evidence that partner organizations can use to develop an integrated clinical practice guideline for use by primary care providers for the treatment of patients with single and multiple conditions for the primary and secondary prevention of heart, lung, blood, and sleep (heart, lung, blood, sleep) diseases.

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

Details on the impact of addressing this CQ or CC

• Despite the success of single condition/disease guidelines, patients often have multiple conditions/risk factors that interact in various ways and can accelerate the development of atherosclerosis and chronic lung diseases. Effective management therefore requires a more integrated approach to assessment and treatment that spans all of relevant risk factors and conditions.
• The development of an integrated guideline has been recommended by participants in several NHLBI forums, including the NHLBI Strategic Planning process, the NHLBI Conference to Create a Cardiovascular Knowledge Network, and the Cardiovascular Disease Thought Leaders Meeting.

Feasibility and challenges of addressing this CQ or CC

Feasibility: • NHLBI currently participates with other Institutes in funding research on comorbidities, Behavioral Interventions to Address Multiple Chronic Health Conditions in Primary Care (R01, PA-12-024).
• An increasing number of scholarly articles are addressing the magnitude and cost of the problem and calling for guidelines that address this reality.

Challenges: .

• Few studies have focused on the management of patients with multiple chronic conditions.

• Clinical research often excludes persons with multiple chronic conditions.

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

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1 net vote
12 up votes
11 down votes
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Goal 3: Advance Translational Research

Clinical Trial Methodology

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:

 

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 Mitchell Grayson on behalf of the American Academy of Allergy, Asthma, and Immunology

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2 net votes
15 up votes
13 down votes
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Goal 3: Advance Translational Research

In-Vitro Assays to Predict Clinical Response

How can NHLBI support studies that produce in-vitro assays to predict clinical response and ways to translate those results into patient therapies through novel clinical trials, including those for small patient populations and rare diseases?

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 Cystic Fibrosis Foundation

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3 net votes
6 up votes
3 down votes
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Goal 3: Advance Translational Research

To Improve Clinical Practice Recommendations for Asthma

What are the strategies to improve the use of evidence-based clinical practice recommendations and thereby increase the quality of care and improve outcomes for people with asthma?

• Lack of provider awareness, knowledge, agreement, and/or self-efficacy in using the guidelines
• Inconsistent use of guidelines-based asthma care in clinical practice.
• Scarce/limited resources and limited access to target audiences.
•... 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

• Asthma is a chronic lung disease that affects 26 million people in the U.S., including more than 7 million children, at an estimated cost to the nation of $56 billion. Asthma accounts for 14.2 million missed work days, 10.5 million missed school days, 10.6 million physician office visits, and 1.8 million emergency department visits, and 439,000 hospitalizations each year.
• Despite widespread availability of evidence-based clinical practice guidelines for the diagnosis and management of asthma, only about half of individuals with asthma in the U.S. receive guidelines-based care.
• Identifying strategies to improve use of evidence-based clinical practice recommendations would 1) increase the number of people with asthma who receive evidence-based clinical care, 2) increase the number of health care providers who use (implement) evidence-based clinical practice recommendations, 3) increase the quality of care of people with asthma, and 4) improve outcomes and quality of life for people with asthma.

Feasibility and challenges of addressing this CQ or CC

• Investigators could evaluate guidelines-based implementation strategies in implementation settings such as community and regional health systems, private medical practices, federally qualified health centers and other safety-net clinics, and hospitals throughout the U.S.
• Documented successful and sustainable implementation strategies could be shared more broadly and applied to future efforts to improve asthma care and control as well as reduce asthma disparities.

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

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1 net vote
6 up votes
5 down votes
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