Concepts describe the basic purpose, scope and objectives of proposed initiatives and represent an early planning stage for potential NCATS activities. Concepts are discussed with the NCATS Advisory Council and Cures Acceleration Network (CAN) Review Board and through other public venues. Council approval of a concept does not guarantee it will become an initiative. That decision is made based on scientific and programmatic priorities and the availability of funds.
View approved CAN Review Board concept clearances by year:
CTSA Program Data to Health Initiative
Sept. 15, 2016
The objective of NCATS’ CTSA Program Data to Health initiative is to demonstrate and disseminate through its Clinical and Translational Science Awards (CTSA) Program the advances in informatics that can help catalyze the translation of discoveries into health benefits. To maximize its potential impact on human health, NCATS, through the CTSA Program, should promote the collaborative collection, management and analysis of biomedical research data from diverse sources, including CTSA Program-affiliated researchers, health care organizations, mobile devices, patients and/or caregivers. Read the full Concept Clearance (PDF - 95KB).
NIH-Industry Program: Discovering New Therapeutic Uses for Existing Molecules
Sept. 15, 2016
NCATS launched the Discovering New Therapeutic Uses for Existing Molecules program in May 2012. The objective of a re-issued initiative is to support the exploration of new therapeutic uses for investigational drugs or biologics (assets) from across a broad range of human diseases. Assets have undergone significant pre-clinical and safety testing in humans and are ready for additional testing in patient populations. Bringing together the best assets from pharmaceutical companies with the best new ideas from academic researchers could produce new treatments much more quickly than starting from scratch. The overarching goal is to enable an efficient drug repurposing partnership model that is adopted broadly by the biomedical research community. Read the full Concept Clearance (PDF - 71KB).
Development of Drone Labware
June 13, 2016
The objective of this contract is to develop an autonomous drone capable of taking a laboratory consumable (such as a well plate) from one station to another. NCATS has had success in using the Small Business Innovation Research contract mechanism to address needs for improvement in the high-throughput screening (HTS) realm. NCATS believes that there would be a potential market opportunity for a small business to develop lab drone technology for HTS applications because:
- Drones have a much greater range of motion than stationary robotic arms and are cheaper to maintain.
- In an HTS setting, it is difficult to have robotic arms in shared workspaces, due to synchronization concerns and the possibility of collision. Lab drones can occupy the same airspace, allowing for the coordination of multiple drones in the same work area.
- The open-source community is constantly developing new tools to make drones more efficient and cheaper.
- While there is a great deal of drone technology in the marketplace, its use in the lab is limited and not available to the research community, specifically in the HTS field.
Read the full Concept Clearance (PDF - 60KB).
NIH-CASIS Coordinated Program in Tissue Chip Systems for Translational Research in Space
June 13, 2016
This proposed initiative seeks to leverage recent advances in tissue engineering and microfabrication to create microphysiological systems and organ-on-chip technology platforms that recapitulate human physiology, to better determine the molecular basis of human disease and/or the effectiveness of diagnostic markers and therapeutic intervention for disease treatment. The initiative will focus on the deployment and further development of tissue chip technology to facilitate space-related research at the International Space Station and integrate results from that research into an improved understanding of human physiology. This initiative will advance biomedical research approaches and clinical technologies for use on Earth and in space and for research in Earth- and space-based facilities that could improve human health.
It is now widely known that accelerated aging occurs in space, due to muscle wasting, osteoporosis, reduced cardiopulmonary function, immune response, and other factors, but that these conditions are reversible when astronauts return to Earth. It is anticipated that this initiative by NCATS and the Center for the Advancement of Science in Space will delve into the molecular basis, including epigenome changes, for these human conditions and provide information for novel targets for drug development. Read the full Concept Clearance (PDF - 20KB).
The NIH/NCATS Registry Program
June 13, 2016
The objective of this initiative is to provide a coordinated and comprehensive approach for NCATS to promote standardized patient registries that are feasible and sustainable and that result in high-quality data to advance clinical research and therapy development.
The awardee(s) will provide centralized operational, informatics, and data and project management support to ensure a sound and efficient approach to supporting high-quality and high-impact patient registries for NCATS. Patient registries, collections of standardized information, are an indispensable resource in rare diseases research, since they contribute to multiple phases of the research lifecycle, including participant recruitment for research studies (contact registries); development of datasets to better understand disease progression, biomarkers and clinical outcomes (natural history registries); and collection of safety and efficacy data after regulatory approval (post-marketing registry). Read the full Concept Clearance (PDF - 22KB).
3-D Bioprinting of Human Live Tissues for Drug Screening
Dec. 11, 2015
Bioprinting of architecturally defined and physiologically relevant human live tissues is emerging as a key enabling technology for drug discovery. 3-D bioprinting of human live tissues has the potential to accelerate the drug discovery process, enabling treatments to be developed faster and at a lower cost by bridging the predictability gap between in vitro and in vivo assays and positive clinical outcomes. The major reason for the low success rate in drug development is the lack of efficacy in clinical trials. This failure in the late stages of clinical development is in large part due to the use of simplistic in vitro cell assays and non-predictive in vivo animal models during the drug discovery and development process. 3-D bioprinting of human live tissues derived from human stem cells is expected to provide data that are more relevant to the whole body response than traditional studies with two-dimensional cell cultures.
The purpose of this initiative is to generate architecturally defined human tissues that closely resemble in vivo human tissues for drug screening by integrating groundbreaking tissue bioengineering, 3-D printing, cell development, stem cell and disease biology, and noninvasive detection technologies. This program will create the infrastructure necessary to enable 3-D bioprinting for the fabrication of tissues at NCATS and to establish collaborations with the research community to advance and disseminate its use for drug discovery. Read the full Concept Clearance (PDF - 25KB).
Increasing Access to Compounds and Tox Data
Dec. 11, 2015
The underlying mechanism of toxicity discovered in or after Phase I trials often is not investigated. NCATS would work with multiple pharmaceutical companies, the Food and Drug Administration and companies that develop predictive toxicology tools. This initiative would broker relationships between pharmaceutical companies and academic researchers who could conduct research to better understand and perhaps help to overcome toxicities detected in drugs that gave a safety signal in Phase I trials that were not predicted based on pre-clinical studies. The research would help answer the question of why pre-clinical tools sometimes fail to predict toxicity by providing researchers with access to the compounds, as well as associated pre-clinical and clinical data.
The goal of this initiative is to increase access to compounds that did not have a safety signal in pre-clinical studies but were later shown to have toxicity in humans. The program would investigate underlying mechanisms for the human toxicity and explore potential reasons why pre-clinical tools failed. The information would be incorporated into predictive modeling to benefit drug development. Read the full Concept Clearance (PDF - 33KB).
Proof of Principle (POP) Awards
Dec. 11, 2015
The proposed program is aimed at pre-clinical research projects that develop, demonstrate or deploy interventions to improve human health. Often, prospective grantees have applied for NIH support but did not receive funding because they lack a specific piece of translational data. The program would fund generation of the needed data to make the project more competitive for subsequent funding or otherwise move the project forward.
The program would strengthen applications for programs across NIH, and perhaps at a future stage, across the entire translational research enterprise. Applications to be considered for potential funding will be those that have a broad and significant impact, and each project will be completed in a relatively short time. Read the full Concept Clearance (PDF - 44KB).
Proteome Profiling in the Clinic
Dec. 11, 2015
The Human Genome Project cannot be used fully for precision medicine without profiling the proteome and its dynamically regulated post-translational modifications (e.g., phosphorylation, ubiquitination). Genomic tools do not allow the analysis of post-translational modifications at all. Indeed, the lack of well-established protein markers might explain some of the failures in clinical trials that are solely based on genetic data. New sensitive clinical tests, reliable panels of protein biomarkers and quantifiable assays are urgently needed in the clinic.
The initiative will establish new clinical tests and protein biomarkers based on quantitative proteomics, phosphoproteomics and validated antibodies; optimize technical and analytical tools and easy-to-use resources and databases for physicians and clinical staff; and perform combined analysis of genetic and proteomic data for decision making in personalized health care. Quantitative read-outs will promote better understanding and longitudinal monitoring of pathophysiology and drug effects. Read the full Concept Clearance (PDF - 31KB).
SaME Therapeutics: Targeting Shared Molecular Etiologies Underlying Multiple Diseases to Accelerate Translation
Dec. 11, 2015
While the number of disorders with a known molecular basis continues to increase rapidly, the number with an effective treatment continues to lag far behind. What is needed to overcome this translational roadblock is a fundamental change in the current symptom-based, one-disease-at-a-time approach to drug development and clinical trials. An explicit focus on identifying SMEs for translation represents such a fundamental change, which ultimately will bring more rationally designed treatments to more patients more quickly.
In contrast to the current approach to disease based on clinical presentation, the concept of SaME therapeutics is to focus on shared molecular etiologies underlying multiple diseases using systems biology as a framework for drug development and clinical trials. An important part of this initiative will be to develop a matrix of diseases and molecular etiologies to identify shared molecular etiologies (SMEs) underlying multiple diseases, and to stimulate novel clinical trials of SME-targeted drugs based on grouping patients by SME rather than clinical phenotype. Read the full Concept Clearance (PDF - 41KB).
Sensors and Devices to Detect Clinical Outcomes
Dec. 11, 2015
Many sensors and devices are available; however, the clinical utility of these is limited. This proposal will focus on solving technical, computational, engineering, social and cultural barriers to collecting, integrating and analyzing data from multiple devices and sensors and patients’ health care data in the context of addressing a pilot study of a compelling clinical question that could not be answered without such data integration.
A diverse collaborative team (technology leaders, patients, data scientists, etc.) is required to uniformly collect and analyze sensor and device data for assessing clinical outcomes that could not be answered without data integration. Read the full Concept Clearance (PDF - 31KB).
Tissues-on-Chips: Part II
Dec. 11, 2015
The goal of Part I of the Tissue Chip for Drug Screening program was to develop bioengineered micro-devices that represent functional units of the 10 major human organ systems: circulatory, respiratory, integumentary, reproductive, endocrine, gastrointestinal, nervous, urinary, musculoskeletal and immune. In the first part of this program, several unique and novel in vitro platforms have demonstrated human organotypic physiological functions and responses to drug exposure, ensuring that safe and effective therapeutics are identified sooner and ineffective or toxic ones are rejected early in the drug development process. These micro-fabricated devices also have proven to be useful for modeling human diseases, and they may prove to be sufficient alternatives to animal testing. Despite these successes, there is a clear need to advance the technology to fully exploit the use of the tissues-on-chips not only at the pre-clinical stage but also as a clinical tool.
The purpose of the proposed request for applications is to foster a multitude of new research applications including, but not limited to, studies in personalized medicine, environment exposures, reproduction and development, autoimmune disorders, infectious diseases, cancer, countermeasures for chemical warfare, immune responses and neuro-inflammation. Read the full Concept Clearance (PDF - 123KB).
Micro-Awards for Researchers Who Need to Get Past a Small Hurdle
Sept. 19, 2014
The concept is based on experience with the several NCATS programs, which revealed that some applicants lacked specific critical pieces of data to present competitive proposals. Gap analysis showed that a few programs at NIH exist to meet this need, but they do not focus on the translational space, nor are they aimed at projects that have already undergone the NIH review process.
This concept would provide proof-of-principle (PoP) micro‑awards to investigators who had undergone NIH review to fund the generation of predominantly pre-clinical data needed to make a project more competitive or otherwise move the project forward. Measures of success could include receipt of funding, or achievement of relevant milestones such as the creation of intellectual property or the preparation of an Investigational New Drug package. If PoP awards are successful, the approach could be expanded across the entire translational research spectrum or beyond NIH.
Devices and Sensors to Detect Clinical Outcomes
Sept. 19, 2014
An array of devices and sensors are available to collect physiological, environmental or patient‑reported information in real time, but their use is limited by a lack of information about how to collect, manage, analyze and interpret the data. There also is a need for best practices and standards for the integration of sensor and device data with medical record and other data to describe clinically relevant outcomes.
This concept would focus on integrating real-time data from multiple sources in order to characterize patients or disease status in a clinically meaningful way. The emphasis would be on devices that are already available and the data would be made publicly available at the end of the program.
Access to Compounds, Toxicology/PK Data, Patient Populations
Sept. 19, 2014
The underlying mechanism of toxicity discovered in phase 1 trials often goes without further investigation. This concept would focus on uncovering such toxicity mechanisms, helping answer the question of why pre-clinical tools sometimes fail to predict toxicity. Researchers would be provided with the compounds as well as associated pre-clinical and clinical data. Once the mechanisms are identified, it could be possible to build complementary models, assays, or tools to increase prediction success rates and improve safety.
The concept entails collaboration with pharmaceutical firms, the FDA, and companies that develop predictive toxicology tools. Measurable outcomes could include the number of compounds brought into the program and the number of toxicity mechanisms elucidated.