Showing 7 ideas for tag "regulatory"

Goal 2: Reduce Human Disease

The role of Extracorporeal Photopheresis (ECP) in the prevention and treatment of rejection of heart and lung transplants

According to the ISHLT, more than 4,000 patients undergo a heart transplant each year, and almost 4,000 receive single or double lung transplants. Their prognosis depends heavily on the avoidance of rejection, which claims the majority of their lives. For heart transplant recipients, the median survival is 11 years, while for lung transplant recipients, it is approximately 5 years. The current most common anti-rejection... 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

Patients who are fortunate to receive a matched heart or one or two lungs transplants are at high risk of dying from rejection early and even years after the operation. Thus, they are given cocktails of highly toxic anti-rejection drugs for the rest of their lives. Unfortunately, despite compliance with their drug regimens, many patients still suffer repeated episodes of rejection that may be fatal. In addition, they develop serious side-effects such as diabetes, infections, malignancies, renal failure, etc. ECP has been shown efficacy in preventing and treating cardiac transplant rejection, but the data are limited. ECP appears to benefit such patients by causing an increase in the number of circulating T regulatory (“T regs”) cells. T regs are known to mediate immune tolerance, the ultimate goal of a long-term successful transplant. The role of ECP in lung transplantation is mostly unknown. Very preliminary data have been gathered from retrospective studies. We suspect that patients with early bronchiolitis obliterans syndrome (“BOS”) will benefit from ECP prior to developing irreversible pulmonary damage. In both types of transplants, however, it is unknown when should ECP be started, how often it should be employed (treatment schedule), and for how long. Finally, the most compelling argument to use ECP in heart and lung transplantation is its excellent side-effect profile. Furthermore, ECP may allow a decrease in the number of drugs needed to prevent rejection.

Feasibility and challenges of addressing this CQ or CC

Many patients with heart and lung transplants develop severe and often fatal rejection despite the current drug options to prevent rejection. ECP could be added to their treatment regimens and decrease side-effects, improving long-term survival.

ECP is generally well tolerated and complications are extremely infrequent.

There is a great potential for multi-disciplinary collaboration between Apheresis Medicine, Cardiology, and Pulmonary specialists.

It is conceivable that manufacturers of ECP instruments will be interested in contributing to the design and support of these studies.

Such studies could shed light in the mechanism of action of ECP in heart and lung transplantation.

There is a need to develop standardized treatment regimens based on well designed clinical trials to further optimize the use of ECP. Development and standardization of measurable outcomes is critical for the success of clinical studies in apheresis in general, and ECP in particular.

Challenges:

  1. Limited number of institutions providing ECP treatment.
  2. Cost of ECP procedures.
  3. Small number of animal models available for apheresis research. Thus, limited studies of ECP mechanism(s) of action. However, understanding pathological mechanisms and their relationship to response to apheresis is critical for optimization and advancement of patient care in heart and lung transplantation.
  4. Lack of infra-structure for apheresis research.

Name of idea submitter and other team members who worked on this idea Marisa Marques on behalf of ASFA

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80 net votes
102 up votes
22 down votes
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Goal 3: Advance Translational Research

Facilitating the translation of discovery science into proof of concepts in preclinical models

What steps can the research community take to facilitate the translation of discovery science into proof of concepts in preclinical models and in humans for diagnosis, prevention, and treatment?

• Current regulatory environment
• Lack of communication between discovery and clinical research worlds
• Lack of training
• Getting industry, academia, and NHLBI to partner; and the business model to make it happen.
• Limited... 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

• Increased number of novel therapeutics, diagnostics and devices in early phase clinical trials
• Increased impact in rare diseases and unmet needs
• Increased number of licensed IPs from academic centers

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

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17 net votes
25 up votes
8 down votes
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Goal 1: Promote Human Health

Gene regulatory networks in HLB disease mechanisms

What is the level of population variation of gene regulatory networks in heart, lung, and blood and sleep disease mechanisms? Can we use different animal model strains to identify key factors in networks and modifier genes?

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

Details on the impact of addressing this CQ or CC

Improve our understanding of the pathophysiology of disease and perhaps identify novel targets for intervention.

Feasibility and challenges of addressing this CQ or CC

Advances in high throughput technology have made addressing these questions possible.

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

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10 net votes
22 up votes
12 down votes
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Goal 3: Advance Translational Research

NHLBI Cardiovascular Engineering Strategy

Most impressive and impactful advances in CV diagnostics and therapies came in the last 50 years from CV engineering, including implantable devices and imaging technology. CV engineers are developing next breakthrough technology including tissue engineering and flexible electronics. However, organizational structure of NIH does not have an entity responsible for strategic development of CV engineering. NIBIB does not... 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

Cardiovascular Science produced numerous fundamental ideas, which frame our approaches to diagnostics and therapy of heart disease. However, translating these ideas to clinic very often requires engineering approaches. Examples of such breakthrough therapies are implantable pacemakers and defibrillators, stents, MRI, CT and many other imaging modalities. NIBIB supports many fields of biomedical engineering, except cardiovascular! NHLBI lacks a branch responsible for strategic development of cardiovascular engineering as a critical pathway to translation of basic science ideas. There is no study section or review group focusing on cardiovascular engineering. As a result, most of CV Engineering grants are reviewed by CV biologists, who lack engineering background and have quite different priorities and vision of the field. Next breakthrough developments will happen in tissue engineering, flexible/stretchable/biodegradable electronics, novel imaging modalities, computational physiology, and other classical biomedical engineering sub-fields. Unfortunately, they are less likely to happen in cardiovascular field, because NHLBI lacks corresponding organizational structure. A working group should be formed to frame NHLBI's vision for the future of cardiovascular engineering as an indispensable component for translation from CV biology to CV therapy.

Feasibility and challenges of addressing this CQ or CC

Biomedical engineering has trained several generations of professionals in academia and industry, which pursue basic and translational research and development with great degree of success. CV Engineering is a standard component in numerous BME Departments. Large number of senior and junior CV engineers have been recognized for their significant contribution to CV health. There is enormous CV engineering expertise and experience, which should be leveraged by NHLBI, in order to broadly define institutional strategy not only for CV biology but also for CV engineering, which are equally important in development of future breakthrough therapies for CV disease. Currently, support for CV engineering is scattered across numerous mostly biology focused groups, lacking strategic vision and coherent policy. A number of talented CV engineers are forced to leave the field to pursue other areas of biomedical engineering, which enjoy better-organized professional group support.

Name of idea submitter and other team members who worked on this idea Igor Efimov

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8 net votes
17 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

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

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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2 net votes
3 up votes
1 down votes
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