Advisory Council Concept Clearances

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 Advisory Council concept clearances by year:


Clinical and Translational Science Awards Program Operations Support

Jan. 16, 2020

Clare K. Schmitt, Ph.D., presented a contract concept for operations support for the CTSA Program–related grantee and NCATS staff activities. The aims of the CTSA Program are to accelerate translation of research discoveries into improved care for patients by addressing systemwide clinical and translational research problems collaboratively; disseminate the expertise, tools, training and clinical research innovations for effective treatments; and support a diverse translational science workforce.

The goal of this operations support contract concept is to provide effective and efficient management of this large, complex national CTSA Program, including the ongoing administrative tasks (e.g., supporting the consortium-wide agendas and large-scale Program deliverables). Specific operations support tasks will include transfer of the NCATS Specific, Measurable, Achievable, Realistic and Timetable (SMART) institutional review board (IRB) platform from the current grantee for long-term management and receipt and distribution of funds from other NIH Institutes and Centers and sister agencies for emergent clinical trial needs. In addition, the operations support contract will provide meeting and conference support and manage data safety monitoring-related activities in clinical trials.

The expected outcomes will be the consolidation of multiple activities under one contract and addition of needed tasks, allowing grantees to focus their intellect and efforts on addressing the CTSA Program goals and, therefore, NCATS goals. Regarding plans to promote and ensure sustainability, the activities and platforms will be hosted on the NCATS cloud platform, so a transition plan is required.

Project/Program Officer:
Clare K. Schmitt, Ph.D.
Deputy Director
Division of Clinical Innovation
National Center for Advancing Translational Sciences
Phone: 301-827-3787

Supporting Clinical Trials of a Prime Genome Editor in Multiple Genetic Diseases

Jan. 16, 2020

P.J. Brooks, Ph.D., presented the concept of supporting clinical trials of a prime editor (e.g., CRISPR) for the treatment of multiple genetic diseases. The current approach to gene-editing clinical trials is focusing on one disease at a time. This approach is based on commercial considerations, which is not optimal for addressing the thousands of rare monogenic diseases that exist. In 2019, researchers discovered a modified CRISPR Cas9 (CRISPR-associated protein 9) technology that has the potential to correct 90 percent of human disease–causing mutations without introducing double-strand DNA breaks or increasing the risk of cancer. The DCI and ORDR are interested in expanding these findings to NCATS programs and clinical trials.

The concept goals are to assess the feasibility of clinical trials of a prime editor in multiple rare monogenic diseases and identify and overcome any feasibility challenges for such trials. Dr. Brooks emphasized that the concept would leverage ongoing efforts, such as the NCATS CTSA Program and the NIH Common Fund Somatic Cell Genome Editing program. This initiative will use the general framework of the CCIA program and will support clinical trials that focus on defects in a somatic cell type to which a single prime editor can be delivered (e.g., liver hepatocytes). Depending on the technology delivery method, potential regulatory issues involving the FDA Center for Biologics Evaluation and Research may need to be addressed.

Several outcomes are expected, including identifying and overcoming challenges in conducting these types of clinical trials, increasing the number of rare disease patients in trials, and maximizing the clinical impact of somatic genome editing. If successful, this project will fundamentally change the way genome editing in rare diseases clinical trials are conducted. Results will be disseminated to both the gene editing and rare disease research communities via the CTSA Program and publications. Ultimately, the goal is to identify a regulatory pathway for clinical trials of a prime editor that includes rare disease patients.

Project/Program Officer:
Philip John (P.J.) Brooks, Ph.D.
Program Director
Office of Rare Diseases Research
National Center for Advancing Translational Sciences
Phone: 301-443-0513

SaME Therapeutics—Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Rare Diseases

Jan. 16, 2020

Dr. Brooks presented the concept of supporting rare diseases clinical trials of drugs targeting SaME therapeutics. Although thousands of rare diseases exist, the number of underlying causes (i.e., etiologies) is small. The SaME approach is the focus of ongoing drug development. In addition, drugs targeting SaME in different cancers are considered state-of-the-art in oncology, and the approach has established a regulatory pathway for these types of FDA drug approvals. Although discussion and interest in SaME for rare diseases have increased in the Collaborative Forum on Rare Diseases and the International Rare Disease Research Consortium, no clinical trials are ongoing.

The goals of the concept are to adapt the oncology SaME approach to rare diseases and identify and overcome any challenges. It is anticipated that recruiting for clinical trials based on SaME will dramatically increase the number of rare disease patients in trials, translating to more treatments delivered to more patients more rapidly. The concept will leverage the RDCRN, and the results will be disseminated to the community via this Network and publications. The expected outcome will be the ability to stratify patients according to underlying etiology rather than traditional clinical characteristics, thus transforming rare disease clinical trials.

Project/Program Officer:
Philip John (P.J.) Brooks, Ph.D.
Program Director
Office of Rare Diseases Research
National Center for Advancing Translational Sciences
Phone: 301-443-0513


Rare Diseases Are Not Rare! Challenge 2.0

Sept. 19, 2019

Alice Chen Grady, M.D., explained that the first NCATS rare diseases prize competition, Challenge 1.0, sought creative ways to raise awareness about rare diseases and the need for expanded research, new treatments and patient support. The competition opened in 2018, and nearly 50 submissions were received. Participation was widespread and generated innovative works of art that educated the public about rare diseases. For details on the three winning submissions and honorable mentions, visit the NCATS Rare Diseases Are Not Rare! Challenge Winners and Honorable Mentions page of this site.

The overarching goals of Challenge 2.0 are to change the public perception of “rare” and foster collaborations across the rare disease community. Metrics for success will be defined in advance and could possibly inform ORDR public-facing programs. Children can directly submit entries with the appropriate consent. In addition to increasing public awareness about rare diseases, the many people affected, and common challenges encountered, NCATS could use the Challenge 2.0 submissions to strongly convey the message about the ongoing need for rare diseases research and new treatments. This concept builds on the success of the NCATS 2018 prize competition and aligns with some ongoing rare diseases activities, such as the patient organization-sponsored competitions and the emphasis of the NCATS RDCRN to study multiple diseases at a time and include patient groups as partners in research.

Project/Program Officer:
Alice Chen, M.D.
Medical Officer
Office of Rare Diseases Research
National Center for Advancing Translational Sciences
Phone: 301-827-2015

Ethical, Legal and Societal Implications in Translational Research

May 16, 2019

Emerging discoveries and technologies raise potential ethical and legal issues that also may have societal implications. Evidence suggests that the familiarity and collaboration of translational researchers and bioethics and legal scholars can be beneficial. To advance discoveries to impact health responsibly, engagement and collaboration of researchers, scholars, communities and the public are needed. Major obstacles that need to be addressed regarding ongoing research and activities in this area include the establishment of the Neuroethics Working Group within NIH’s Brain Research through the Advancing Innovative Neurotechnologies (BRAIN) Initiative and the National Human Genome Research Institute’s Ethical, Legal and Social Implications Research Program.

The objectives of this initiative are to increase recognition of and research on these issues, encourage research on these challenging questions to collect data, foster collaboration between the two communities, and provide empirical data and frameworks for addressing these issues. The outcomes will be increases in projects, scholarship, research collaborations and stakeholder engagement in these areas.

Project/Program Officer:
Elaine Collier, M.D.
Senior Advisor to the Director
Office of the Director
National Center for Advancing Translational Sciences
Phone: 301-814-4286

Non-Viral Delivery Technologies for Somatic Genome Editing Therapeutics

May 16, 2019

Somatic genome editing has the potential to treat a large number of rare genetic disorders and has therapeutic implications for common diseases. Therapeutic benefit is maximized if the genome editing machinery has broad coverage in many somatic cell types. For many diseases, the ability to deliver genome editors to relevant cell types is the limiting factor. Non-viral delivery methods allow transient expression of genome editors, which is expected to reduce toxicity.

The objective of this initiative is to support the development of non-viral technologies to deliver genome editing machinery to a wide variety of cells and tissues, especially in cell types with no effective delivery technology currently available.

Project/Program Officer:
Philip John (P.J.) Brooks, Ph.D.
Program Director
Office of Rare Diseases Research
National Center for Advancing Translational Sciences
Phone: 301-443-0513

Alternatives to Commercially Available Cell Culture Insert Membranes and Manufacturing Techniques

May 16, 2019

Despite the increased focus on the use of more complex cellular and tissue models (e.g., 3-D tissue printing) to provide physiologically relevant platforms for enhancing the development of therapeutics for patients, challenges exist. Biodegradable cell culture-insert membranes that also provide structural support are needed, as existing commercially available alternatives are insufficient.

The objective of this initiative is to a support a small business, via an SBIR contract mechanism, that can provide a commercial solution to manufacture cell culture insert membranes in an automated and reproducible manner.

Project/Program Officer:
Samuel G. Michael
Chief Information Officer
Information Technology Resources Branch
Office of Administrative Management
National Center for Advancing Translational Sciences
Phone: 301-827-7796

Multidisciplinary Approaches to Shortening the Diagnostic Odyssey for Rare Diseases

May 16, 2019

Many patients with a rare disease experience years-long delays in receiving a correct diagnosis (i.e., the “diagnostic odyssey”), lending to considerable anxiety and despair for patients and families as well as potential delays in treatment. Current approaches to the rare disease diagnostic odyssey typically occur through idiosyncratic referrals to a small number of disease experts at tertiary care centers.

The objective of this concept is to improve diagnostic accuracy and accelerate diagnosis for these patients through a multidisciplinary process that can be performed at the primary or secondary care levels by front-line providers. Novel approaches need to be developed that should focus on an integrated combination of clinical, computer-assisted and genomics assessments to speed diagnosis and improve accuracy. Some current activity exists in each of these areas, but no comprehensive methods have been developed that are using multiple approaches simultaneously that have been adopted by front-line clinicians. The outcome will be the development of a broadly adaptable, facile process with the potential impact of advancing the International Rare Diseases Research Consortium’s 2027 goals of achieving an accurate diagnosis within one year of a rare disease patient presenting for medical evaluation.

Project/Program Officer:
Anne R. Pariser, M.D.
Office of Rare Diseases Research
National Center for Advancing Translational Sciences
Phone: 301-402-4338


Drug Development Collaboratory

Sept. 27, 2018 

The proposed Drug Development Collaboratory would include the intramural Therapeutics Development Branch – within the NCATS Division of Preclinical Innovation – to conduct Investigational New Drug (IND)-enabling studies, develop regulatory strategy and allow changes in drug delivery and formulation. The goals of the Collaboratory are to develop a strategic process for applicants to collaborate across multiple NCATS programs; optimize drug delivery method, formulation and regulatory strategy; develop milestones; assist with clinical trial study design; and provide continuity of support across existing programs.

NCATS Therapeutics Development Branch will provide IND-enabling data and expertise. Applicant institutions will provide access to disease-specific knowledge, expertise, access to models/assays and access to patient populations that are available at applicant institutions. NCATS Drug Development Partnership programs will support preclinical through early-stage clinical trials that are conducted at applicant institutions.

HEAL Pain Effectiveness Research Network (HEAL Pain-ERN)

Sept. 27, 2018 

The evidence for optimal pain management, such as long-term opioid use for management of chronic pain, is often insufficient. The NIH Help End Addiction Long-term (HEAL) Pain-ERN would conduct clinical trials and studies to establish interventions or programs to manage, reduce or prevent acute and chronic pain in ways that reduce risk for addiction and provide evidence to inform practice-based guidelines. The HEAL Pain-ERN will leverage the existing Clinical and Translational Science Award Trial Innovation Network (TIN) to implement studies of interest to multiple NIH Institutes and Centers (ICs).

The proposed infrastructure would include TIN Clinical and Data Coordinating Centers and a Pain Leadership Group that would report to entities within NIH, including the Pain IC Councils and the NIH Pain IC Directors. The National Institute of Neurological Disorders and Stroke will provide the repositories for clinical data and biosamples.

A trans-NIH group will write the FOAs. The trials and studies will use standard outcome measures whenever possible, and all data will be stored centrally to ensure data sharing. The initiative will help coordinate pain research across different disciplines, help resolve the problem of having limited outcome measures for pain and provide more functional pain measures.

CTSA Program: Competitive Supplement Applications to Develop, Demonstrate and Disseminate Translational Science Advances

Sept. 27, 2018 

This program will provide supplements to translational science projects that address the goals of the Clinical and Translational Science Awards (CTSA) Program. These are peer-reviewed competitive supplements that could extend beyond the scope of the parent grant.

The supplements will allow projects to respond to emerging scientific opportunities, stimulate high-priority translational science areas and provide the opportunity to extend new projects, activities and collaborations across the CTSA Program consortium. The supplements will allow the development of new approaches, the demonstration of their efficacy and the dissemination of the findings. Success will be measured in terms of the ability of the proposed activities to advance translational science and increase and broaden the overall impact of the CTSA Program.

CTSA Program Collaborative Projects Program

May 10, 2018 

The objective of the Clinical and Translational Science Awards (CTSA) Program Collaborative Projects Program is to support innovative and sustainable approaches to overcome roadblocks in translation by building upon strengths at individual CTSA Program hubs. The program will support dissemination of successful methods and processes developed at individual hubs, thereby strengthening and enhancing the impact of the CTSA Program.

The program is expected to have a sustainable and transformative impact on multiple domains of translational science. Examples of challenges to be addressed include the lack of underrepresented minorities participating in clinical trials, the lack of natural language processing capacity in translational research, and variations in training opportunities across the CTSA Program. For more information, please see the CTSA Program Collaborative Innovation Awards.

Non-Polydimethylsiloxane Biocompatible Alternatives for Organs-on-Chips 

May 10, 2018 

Polydimethylsiloxane (PDMS) is widely used in the construction of tissue chips. However, PDMS sometimes displays undesirable properties, such as drug absorption, which can lead to drug/compound loss and/or cross-contamination of surrounding areas or tissues. What is needed is an alternative material, in whole or in part, for fabricating tissue chips.

The objective of the Non-Polydimethylsiloxane Biocompatible Alternatives for Organs-on-Chips initiative is to find such alternative materials that would improve the in vivo predictive capabilities and reliability of various microfluidics platforms. Limited supplemental funding has previously been provided to tissue chip project researchers to develop PDMS alternatives; however, this initiative would expand the opportunity to a broader community.

Synthetic Technologies for Advancement of Research and Therapeutics

May 10, 2018

The cost of developing a new drug now exceeds $1 billion. One factor exacerbating the situation is that libraries of compounds have exhausted the limits of structural diversity; it is increasingly difficult to find new compounds with chemical activity. Use of natural products for therapies has been limited due to low yields in their host organism, limited supply of some host organisms, the complexity of natural compounds’ structures and the difficulty of modifying them. Synthetic biology can generate novel, biologically active compounds through advances in scientific disciplines (e.g., gene editing, gene synthesis, automation, metabolomics) to enhance the diversity of drug libraries.

The Synthetic Technologies for Advancement of Research and Therapeutics (START) program would formulate natural, biologically relevant pathways to engineer new and safer therapies; expand the current catalog of naturally occurring compounds and their analogues; identify, characterize and synthesize novel bioactive compounds; and enhance productivity and yield of biological systems that produce natural compounds. The START program has the potential to catalyze the field of synthetic biology for drug development.

Universal Medium/Blood Mimetic for Use in Integrated Organs-on-Chips

May 10, 2018

For the Tissue Chip for Drug Screening program to continue to be successful, organ chips need to be integrated to form multi-tissue platforms. This was a goal from the outset of the program. Integrating systems is challenging, however, due to the tissue chips’ 3-D structure, heterogeneity of cell types and fluid flow.

The objective of the Universal Medium/Blood Mimetic for Use in Integrated Organs-on-Chips initiative is for small businesses, using the NCATS Small Business Innovation Research (SBIR) contract mechanism, to develop a universal medium/blood mimetic that could be used in integrated systems and maintain cell viability and function for at least one month. Having a universal medium will enable researchers to link many new tissue types, increasing the utility of the chips and expanding access to the technology to a wider community for commercialization and dissemination. Also, such a medium could be used in other systems across laboratories and could have utility in other tissue-engineered systems, such as 3-D bioprinting.

Clinical Trial Readiness for Rare Diseases

Jan. 11, 2018

The objective of the Clinical Trial Readiness for Rare Diseases initiative is to support clinical studies that address obstacles to the design of trials needed for rare diseases. Such trials are critical in the development and evaluation of new treatments for rare diseases. This initiative aims to support studies that address these bottlenecks by focusing on specific gaps in natural history data and biological and clinical outcome measures.

Addressing bottlenecks in rare diseases clinical development will accelerate progress from discovery to patient benefit and thus is directly aligned with the mission of NCATS and NIH. Read the full Concept Clearance (PDF - 282KB).


Rare Diseases Clinical Research Network Program

Sept. 7, 2017

The Rare Diseases Clinical Research Network (RDCRN) program was established in late 2003, with expansion and renewal occurring in 2009 and 2014. Through this reissuance, this cooperative agreement research program will continue to facilitate identification of biomarkers for disease risk and disease severity/activity and measures of clinical outcome applicable to clinical trials. It also will encourage development of new approaches to diagnosis, prevention and treatment of rare diseases.

The RDCRN will consist of all funded Rare Disease Clinical Research Consortia (RDCRC) and a single Data Management and Coordinating Center. It will support the continuation of a collaborative and coordinated network of RDCRCs composed of investigators at multiple institutions/sites and patient advocacy groups committed to investigation of rare diseases working in partnership to enhance clinical research focusing on natural history studies, providing training to young investigators, and sharing of resources in a multidisciplinary approach. Read the full Concept Clearance (PDF - 63KB).


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 preclinical 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).

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).

Clinical and Translational Science Data Metrics Coordinating Center

Jan. 14, 2016

The Clinical and Translational Science Awards (CTSA) Program supports a national network of medical research institutions — called hubs ― that work together to improve the translational research process to get more treatments to more patients more quickly. Ensuring that this investment transforms the entire spectrum of clinical and translational science, including multi-site clinical trials, requires CTSA Program hubs to catalyze innovation in training, research tools and processes.

Achieving this goal requires significant coordination and collaboration across the CTSA Program hubs. The functions of the proposed center are integral to the success of the entire CTSA Program. Read the full Concept Clearance (PDF - 87KB).

Collaborative Innovation Pilot Projects for the CTSA Program

Jan. 14, 2016

The purpose of this initiative is to allow teams of investigators from different Clinical and Translational Science Awards (CTSA) Program hubs to carry out collaborative, innovative projects to evaluate novel approaches to important translational science questions.

This exploratory grant program will allow some novel approaches to be quickly evaluated for feasibility and will provide a potential source of funding for collaborative innovative approaches that are not well suited to the Collaborative Innovation Awards (PAR-15-172 and PAR-15-173). Read the full Concept Clearance (PDF - 66KB).


Drug Repurposing/Repositioning

Sept. 3, 2015

The purpose of this concept is to develop a set of funding opportunity announcements (FOAs) to support robust, preclinical studies (to establish rationale for a clinical trial), clinical trial planning and clinical trial implementation. The preclinical studies will serve as “use cases” to demonstrate the usefulness of the drug-indication pairing method. Appropriate FOAs will be issued to complement the ongoing NCATS New Therapeutic Uses initiatives.

While the goal of an individual project will be to explore the potential new use of existing investigational and Food and Drug Administration-approved drugs, NCATS seeks to identify strategies that may improve the efficiency of drug repurposing studies. Funding will be used to repurpose drugs where the hypothesis originates from the use of a publicly available method for identifying new indications for existing drugs such as independent crowdsourcing strategies for investigational drugs or computational algorithms. Read the full Concept Clearance (PDF - 34KB).

Ethical, Legal and Social Implications of Biomedical and Translational Research

Sept. 3, 2015

As technology and science advance, members of the research community confront evolving and recurring questions about the ethical, legal and social implications (ELSI) of their research and its translation to improve human health. ELSI arise across the entire spectrum of biomedical and translational research, including basic, preclinical, clinical, behavioral, implementation and dissemination science as well as population health. This collaborative initiative with multiple NIH Institutes and Centers (ICs) would seek research projects that address the ELSI of biomedical and translational research of high importance to the participating NIH ICs.

Applications to conduct empiric research as well as those that propose to develop new conceptual frameworks would be allowed. Interdisciplinary and collaborative projects using multiple approaches would be strongly encouraged. ELSI research is critical in the biomedical and translational science domain. The results of projects under this initiative are expected to contribute knowledge that will enhance the ethical conduct and social value of biomedical and translational research in support of the NIH mission. Read the full Concept Clearance (PDF - 18KB).

Translational Research Informatics and Operations Support

Sept. 3, 2015

The purpose of this contract requirement is to provide coordinated and comprehensive scientific and technical support for NCATS translational and clinical research operations and management in order to harmonize and centralize activities across NCATS. Activities are wide-ranging and will include translational and clinical research operations support, information management, safety oversight and administrative support.

This contract will provide clinical, operational and administrative support for the Clinical and Translational Science Awards Program to achieve its goals to catalyze clinical and translational science. The contractor will assist NCATS staff in managing the clinical and operational activities and provide information management of NCATS’ clinical activities. A centralized information system will allow NCATS to quantify and describe characteristics of individual clinical studies to analyze the overall clinical program. Additionally, the awardee will provide support for ensuring human subject safety and quality monitoring of NCATS clinical studies. Read the full Concept Clearance (PDF - 26KB).

Collaborative Innovation Supplements for CTSA Program

June 18, 2015

This supplement program will allow NCATS to provide administrative supplement funds to investigators from different Clinical and Translational Science Awards (CTSA) hubs to carry out collaborative, innovative demonstration projects and move towards dissemination. The focus is aligned with that of the Collaborative Innovation Awards but intended to be smaller in scale and shorter in duration. This supplement program will allow novel approaches to be quickly evaluated for feasibility and provide a potential source of funding for collaborative innovative approaches that are not well suited to the Collaborative Innovation Awards program (PAR-15-172 and PAR-15-173).

The purpose of the administrative supplement is to allow teams of investigators from different CTSA hubs to carry out collaborative, innovative projects to evaluate novel approaches to important translational science questions. Read the full Concept Clearance (PDF - 22KB).

Development of SmartPlate Technology

June 18, 2015

The term “smartplate” was adopted to imply flexibility to the technology and multiple applications of the smartphone. Once a platform was built to create a phone that could perform a variety of functions, as opposed to simply one, a huge amount of innovative ideas sprang forth. The key goal of this Small Business Innovation Research solicitation is to fundamentally transform the idea of a microtiter plate from a single-use laboratory ware for an experiment to nearly becoming an instrument that could provide more data about the tested samples by in-line monitoring and sensing technologies.

Instead of limiting these plates to a variety of plastics with a lifespan of one use, if different materials and manufacturing techniques were utilized, it could greatly affect the purpose(s) for which a plate could be used. Imagine the plate as a multilayer circuit, for example; it could be possible for a variety of monitor and control applications be built directly into the plate, such as temperature, relative humidity, and CO2 and O2 levels, instead of relying on external pieces of instrumentation to perform these measurements. A key goal tied to this technology is to greatly improve the utility of a microtiter plate being completely disposable and instead treat each plate as a fully integrated device that can be used many times. Read the full Concept Clearance (PDF - 39KB).

Development of Stem Cell- or iPS Cell-Based Assays for Compound Toxicity Evaluation

June 18, 2015

There is a growing interest in testing environmental chemicals by using human stem cell or cells derived from induced pluripotent stem (iPS) cells, because transformed and immortal cell lines lack xenobiotic metabolic capability and fail to represent normal physiology and pathophysiology. For the phase I contract, the goal is to develop toxicologically related assays in a homogenous format that can be used in human stem cell or iPS-derived cells with short-time compound treatment. For phase II contracts, the goal is to miniaturize the assays into 384-well and 1,536-well plate formats. Assays with various endpoints using iPS-derived cells in a 1,536-well plate format will greatly speed up the capacity of screening thousands of environmental chemicals. Also, using human stem cells and/or iPS-derived stem cells will make this screening approach even more relevant and the data more valuable in establishing predictive models of how these chemicals compounds affect human tissues and pathways, ultimately making this technology the basis for future screening for the Toxicology in the 21st Century program and other quantitative high-throughput screening initiatives.

The purpose of the Small Business Innovation Research (SBIR) contract solicitation is to use assays developed from the SBIR contract proposals to test toxicologically related targets in a large compound collection of environmental chemicals and pharmaceutical compounds. Read the full Concept Clearance (PDF - 104KB).

NCATS Exploratory Clinical Trials for Small Business

June 18, 2015

This proposed funding opportunity announcement (FOA) will use small businesses under the Small Business Innovation Research/Small Business Technology Transfer grant program to support early and exploratory clinical trials. Examples of appropriate clinical studies under this FOA include those whose primary aim is to evaluate and optimize the dose, formulation, safety, tolerability or pharmacokinetics of an intervention or diagnostic in healthy volunteers or the target population; conduct prospective clinical validation of a therapeutic intervention; and evaluate whether an intervention produces sufficient evidence of short-term activity (e.g., biomarker activity, dose-response trends, pharmacodynamic response) in a human proof-of-concept trial.

The purpose of the FOA is to support applications from small businesses for clinical trials (i.e., phase I and II clinical studies) of drugs, biologics, devices or diagnostics — as well as surgical, behavioral or rehabilitation therapies — that contribute to the justification for and provide the data required to design a future trial to confirm efficacy (i.e., a phase III clinical trial). Read the full Concept Clearance (PDF - 46KB).

R&D Contract Support for NCATS Translational Sciences

June 18, 2015

NCATS is interested in advancing collaborative research projects across the phases of the translational science spectrum. These research projects are designed to overcome key obstacles and inefficiencies in the translational process. While the mechanisms for overcoming obstacles differ, multiple NCATS programs require access to contract resources to achieve their missions. For example, the Therapeutics for Rare and Neglected Diseases (TRND) and Bridging Interventional Development Gaps (BrIDGs) programs heavily rely on contract research organizations (CROs) to provide manufacturing, pharmacology, toxicology, regulatory and clinical operations services to achieve milestones of their projects. All projects within TRND and BrIDGs programs require services compliant with Good Manufacturing Practice and Good Laboratory Practice, which cannot be performed within NCATS’ own laboratories. Other NCATS programs, such as Chemistry Technology and Matrix Combination Screening, regularly use CROs to profile molecules that are being investigated internally.

Under this proposed initiative, NCATS will request proposals from vendors to provide long-term contract services for NCATS programs in various preclinical and clinical therapeutic development areas.

The purpose of the request for proposals is to obtain long-term contract laboratory services in support of the mission of the NCATS Division of Preclinical Innovation. Read the full Concept Clearance (PDF - 87KB).

Small Business Translational Science Innovation Award Program

June 18, 2015

This proposed Small Business Innovation Research/Small Business Technology Transfer program announcement (PA) will focus on innovative translational science. Translating biomedical discoveries into clinical applications is essential to improving human health. It is also a complex process with high costs and substantial failure rates that can result in delays of years or decades before improved patient outcomes result from discoveries in biomedical research. The scientific and operational issues that underlie most translational inefficiency are not specific to a particular disease, discipline, institution or geographic locale. Rather, they are systematic issues, which require systematic and generalizable solutions. Further, every stage of the translational process currently is fraught with ineffectiveness and in need of bold, innovative new solutions. Through this PA, we aim to enlist the small business community in this effort. We anticipate that the efforts supported through this PA will proceed in parallel with and synergize with ongoing activities in the Clinical and Translational Science Awards (CTSA) consortium under PAR-15-172 and PAR-15-173.

The goal of the proposed PA is to incentivize small businesses to develop products to address bold and innovative new solutions to problems in translational sciences, as outlined in PAR-15-172 and PAR-15-173. Another goal is to stimulate partnerships between small businesses and the CTSA network. Read the full Concept Clearance (PDF - 47KB).


Innovative Collaborations for the CTSA Consortium

Sept. 19, 2014

Turning discoveries into clinical advances is a long and inefficient process. The proposed initiative aims to accelerate translational research in specific areas of research and at the same time to more broadly advance our understanding of translational science, the field focused on best scientific and operational methods. The focus of the innovative collaborations initiative is less on the development of technologies de novo, but more on the demonstration that innovative approaches can be applied to translational research problems, or can help address roadblocks. The projects should define the positive outcome, describe how it is measured, and include plans for next steps under different outcome scenarios. At project completion, the data generated should allow for a decision as to whether the approach should be abandoned in favor of alternatives, or whether it should be optimized and disseminated more widely in support of translational research. Therefore, data quality is critical, and robust experimental approaches are encouraged. When feasible, approaches might for example include the randomization or cluster randomization to different interventions that would allow for comparisons between contemporary arms or between pre- and post-intervention scenarios. Projects under this initiative are demonstration projects, and will be considered successful if they provide high-quality data on an innovative and generalizable approach so that the field moves forward. The projects are thus expected to have an impact almost regardless of their outcome by accelerating the uptake of an effective innovation, or by alternatively making it clear which approaches should be discontinued in favor of alternative investments.  As appropriate, projects will include pre-defined milestones to monitor progress.

The goal of this initiative is to stimulate collaborative research in the CTSA consortium. Building on the existing strengths at the CTSA hubs, and utilizing resources already funded by NCATS under the CTSA program, the collaborative projects under the proposed initiative will address important gaps in clinical and translational research, and will advance innovative solutions with the goal to get more treatments to more patients more quickly. Read the full Concept Clearance (PDF - 122KB).

Proposed CTSA Initiatives

May 16, 2014

The Clinical and Translational Science Awards (CTSA) program has evolved continually since its inception in 2006. In 2011, the CTSA program was positioned under the leadership of NCATS to build upon its existing strengths and to ensure its further development in alignment with the NCATS mission to enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions. An Institute of Medicine (IOM) study of the CTSA program, released in June 2013, recommended that NCATS take a more active role in the program’s governance and direction, formalize the evaluation processes of the program, advance innovation in education and training programs, and ensure community engagement in all phases of research, among other recommendations. In December 2013, NCATS tasked a Working Group of its Advisory Council with developing measurable goals for the CTSA program. The Working Group reported to the NCATS Advisory Council in May 2014.

NCATS is proposing to develop a number of new initiatives starting in Fiscal Year 2015 that are aimed at further strengthening the CTSA program, taking into account the IOM and Council Working Group reports. The proposed set of initiatives will emphasize measurable progress in overcoming barriers to translational efficiency across the translational science spectrum and will include, but not be limited to, preclinical research, clinical research and training.

Platform Delivery Technologies for Nucleic Acid Therapeutics

Jan. 16, 2014

Nucleic acids have shown proof of concept as therapeutics in preclinical models of numerous genetic diseases. The major hurdle is finding effective methods to deliver nucleic acids to specific cell types, particularly to the nervous system. Platform-type nucleic acid delivery technologies that are safe and effective could, in principle, enable therapies for multiple genetic diseases for which there are no treatments, but also increase therapeutic options for more common conditions, such as viral infection. The goal of the proposed funding opportunity will be to encourage SBIR /STTR applications for platform technologies to deliver nucleic acid therapeutics (siRNAs, miRNAs, antisense oligonucleotides, splice-switching oligonucleotides, plasmid/chromosomal DNAs, or messenger RNA) to specific cell types in humans. To be responsive to this solicitation, technologies must be, in principle, applicable to the treatment of multiple diseases.


Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) Opportunities

May 17, 2013

NCATS is interested in the development of innovative tools, technologies and intervention (drug, device, diagnostic) platforms that would support the creation of novel therapeutics and/or diagnostics, especially for rare and neglected diseases. The NCATS SBIR/STTR program proposes to initiate new funding opportunity announcements (FOAs) for small businesses on topics relevant to the Center’s mission.

NCATS SBIR/STTR set-aside funds will support opportunities for the following topics using SBIR Phase I and Phase II award mechanisms:

  • Development of neurocognitive pediatric tools for measuring and analyzing clinical study endpoints in rare neurocognitive disorders
  • Development of biomarkers for rare diseases as endpoints for clinical trial measurements

As part of the U.S. Public Health Service’s 2014-1 SBIR Contract Solicitation call for topics, NCATS will develop solicitations for the topics listed below. All research topics for this contract solicitation must have received concept review. Topics selected for Phase I project awards must have the potential to continue to Phase II.

  • Portable parathyroid hormone pump and calcium monitoring device
  • Development of improved genome-editing technologies
  • Development of droplet detection system for high-throughput screening
  • Platforms for the rapid development of cell-based assays for rare diseases

Ethical Challenges in Translational Research: Evidence-Based Research

Jan. 23, 2013

Researchers face many ethical challenges in the translation of basic findings into clinical testing and, ultimately, implementation in the clinic. Evidence-based research is needed to provide data to inform solutions. This concept proposes to help the Clinical and Translational Science Awards community and NIH-funded networks by integrating evidence-based ethics research into current research activities. The concept will be supported with bioethics funding from the NIH Office of the Director.

Increasing National Capacity for Clinical and Translational Research

Jan. 23, 2013

Industry, academia and regulatory bodies exhibit willingness to change currently inefficient practices in translational research. However, all in the biomedical research community need a national environment that supports innovation. This concept will provide opportunities to pilot expansion of innovative and effective practices, methodologies and technologies across multiple sites in real time to support a sustainable national capacity for translation research.


Strengthening Community-Engaged Research in the Clinical and Translational Science Awards Program

Sept. 14, 2012

The development of innovative approaches to engage communities in research efforts and to improve the dissemination and implementation to the community of insights from NIH research are important for developing and implementing new preventives, diagnostics and therapeutics. This concept will identify major challenges to effective community-engaged clinical research, especially those encountered by institutes and centers at the National Institutes of Health, and will solicit focused, highly innovative demonstration projects to test strategies to overcome these barriers.