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:

2021

Contract Support for NCATS Intramural R&D Activities

June 11, 2021

R. Dwayne Lunsford, Ph.D., presented a renewal concept for contract support of the NCATS intramural research and development (R&D) activities. NCATS proposes to renew this concept to complement in-house scientific capabilities of the DPI scientists—who require a variety of support services—and also to maintain access to mission-critical research services. DPI advances translational science by decreasing the risk of investing (i.e., de-risking) in promising new targets and therapies for indications that otherwise lack private-sector funding.

Since the original concept was approved in 2015, the DPI has generated data for regulatory filings (pre- IND and IND stage), contributing to 19 INDs. The DPI also developed diverse therapeutic targets and drug candidates (e.g., small molecules, peptides, bio-therapeutics). Regarding implementation and impact, NCATS will issue new requests for proposals (RFPs) addressing the relevant technical service areas. It is expected that contracts will allow seamless continuity of operations supporting ongoing and future collaborations and will include a Determination of Exceptional Circumstances (DEC) clause to protect the IP rights of the collaborators.

Project/Program Officer:
R. Dwayne Lunsford, Ph.D.
Deputy Director
Therapeutic Development Branch
Division of Preclinical Innovation
National Center for Advancing Translational Sciences
Phone: 301-402-8462
Email: robert.lunsford@nih.gov

LitCoin Prize Competitions

June 11, 2021

Christine M. Colvis, Ph.D., informed the Council that the LitCoin concept was developed with the desire to help researchers share their data in a model that meets NIH requirements and the needs of investigators. NCATS will pilot test the project with the anticipation that the NIH National Library of Medicine and journal publishers will lead the efforts on a broader scale.

Tyler F. Beck, Ph.D., presented the concept for establishing LitCoin prize competitions. This concept addresses three main issues. First, data are not machine-readable. Second, generating computationally accessible data is costly and involves manual curation. Third, few incentives exist to promote data-sharing. In addition, early-career researchers have few mechanisms to share reproducible results outside of their respective disciplines. NCATS proposes to incentivize and enhance the sharing of machine-readable knowledge from biomedical publication free text.

Dr. Beck described the LitCoin conceptual framework, which consists of four key roles of engagement: author, NCATS, publisher, and other researchers. The author makes a discovery, writes a short publication, and uploads the text to the LitCoin server. NCATS facilitates a natural language processing (NLP) tool to build assertions and displays them back to the author, who verifies and then submits to the publisher. The publisher reviews the assertions and approves them for publication. The NLP deposits the information to a dedicated database. Other researchers can then cite the published findings and the data remain available and open to the public.

NCATS proposes two challenge competitions. Challenge 1, LitCoin NLP Challenge, will be a software contest to generate highly accurate, computationally accessible data from free text. The competition is planned for fall 2021 and winners will be required to grant a broad, permissive license to the NIH to use, alter, and redistribute the software. Challenge 2, LitCoin Concept Challenge, will encompass a competition to generate end-to-end plans to build the LitCoin submission platform, commencing in spring or summer 2022. NCATS anticipates combining ideas from multiple winners to inform and plan the next step, a LitCoin Development concept.

To engage stakeholders, NCATS, in collaboration with NLM and four publisher partners, hosted a virtual LitCoin Stakeholder Feedback Workshop June 17–18, 2021. The DDPP sought advice from the Council on key factors to enable success of this program and ideas on enhancing adoption and dissemination of LitCoin.

Project/Program Officer:
Tyler F. Beck, Ph.D.
Scientific Program Officer
Office of the Director
Drug Development Partnership Programs
National Center for Advancing Translational Sciences
Phone: 301-827-1943
Email: tyler.beck@nih.gov

Clinical and Translational Science Award (UM1)

June 11, 2021

Erica K. Rosemond, Ph.D., presented the CTSA UM1 award concept. NCATS proposes this concept to support an integrated research and training environment for clinical and translational science. The objectives and areas of emphasis align with the six main objectives of the Program. This concept addresses the RFI responses to decrease applicant administrative burden, increase CTSA Hub flexibility specialization opportunities, and introduce a distinct clinical and translational science research project.

NCATS proposes to simplify the application and budget, enhance review quality, and streamline award actions. With the UM1 mechanism, applications will be simpler, less repetitive, more organized, and have a single budget. The Hub career development and optional training components will be separate, allowing for independent scores and reviews. The administrative adjustments will speed processing the awards. In terms of flexibility, this concept will require fewer elements, emphasize unique qualities, and create a Hub-specific research project.

The activities of the CTSA will be retained, reorganized and grouped more appropriately, and balanced and focused on the Hub’s activities. These enhancements will allow the Hubs to showcase unique strengths and capabilities. The unique clinical and translational science research project will address existing roadblocks to translational research. This concept builds on the existing CTSA Program and nurtures innovations in clinical and translational science.

Project/Program Officer:
Erica K. Rosemond, Ph.D.
Acting Deputy (Division) Director and Acting (Branch) Chief
Division of Clinical Innovation
Clinical and Translational Science Awards Program Branch
National Center for Advancing Translational Sciences
Phone: 301-594-8927
Email: Erica.Rosemond@nih.gov

Specialized Innovation Programs (SIPs) (RC2)

June 11, 2021

Erica K. Rosemond, Ph.D., presented the new SIPs RC2 concept, which aligns with the RFI responses to increase flexibility and diversity across Hubs to leverage strengths and drive innovation, allow awardees to specialize, and balance local efforts with Consortium efforts. The aim of this concept is to provide support to SIPs through an RC2 funding mechanism, with the goal of catalyzing clinical and translational science locally through the support of unique activities, resources and/or expertise at CTSA Hubs. The proposed innovation ecosystem will consist of a CTSA UM1 Hub (local and national collaborations), RC2, Collaborative Innovation Awards (R21/U01), and Consortium-wide centers (U24), all focusing on improving health.

The goals of the SIPs are to support highly specialized capabilities or resources—with local impact and considerations for early dissemination—and to create a streamlined program-level tracking of outcomes and impact. SIPs will be peer reviewed separately and only Hub awardees will be eligible to apply. Examples of SIPs include telehealth, regulatory science, clinical informatics, genetics and genomics, pragmatic trials, dissemination and implementation, rural health and health disparities, community outreach and engagement, and other specialized programs.

Project/Program Officer:
Pablo Cure, M.D., M.P.H.
Program Director
Division of Clinical Innovation
Clinical and Translational Science Awards Program Branch
National Center for Advancing Translational Sciences
Phone: 301-827-2014
Email: pablo.cure@nih.gov

National Research Service Award (NRSA) Institutional Predoctoral and Postdoctoral Research Training Grants (T32)

June 11, 2021

Mercedes Rubio, Ph.D., presented the NRSA Predoctoral and Postdoctoral Research Training Grant (T32) concept, which aligns with NCATS Strategic Goal 3 to develop and foster innovation in translational training and a highly skilled, creative, and diverse translational science workforce.

This NRSA T32, which replaces the CTSA Program TL1, has the objectives to customize research training opportunities, provide high-quality research training, develop characteristics and attributes of successful translational scientists, and promote evidence-informed mentoring practices. In FY 2020, 181 postdoctoral trainees, 305 predoctoral trainees, and 34 short-term/summer training positions were supported. This concept is expected to enhance the career and training education opportunities available to the clinical and translational science workforce and nurture the clinical and translational science field.

Project/Program Officer:
Mercedes Rubio, Ph.D.
Program Director
Division of Clinical Innovation
National Center for Advancing Translational Sciences
Phone: 301-480-8957
Email: rubiome@mail.nih.gov

Institutional Career Development Award (K12)

June 11, 2021

Joan Davis Nagel, M.D., M.P.H., presented the K12 concept. The CTSA Institutional Career Development Program (KL2) offers postdoctoral scholars and junior faculty advanced training in clinical and translational science research and allows 75 percent of protected time for research. The proposed K12 will provide customized career development and education opportunities to align with local institutional strengths and resources; and will promote flexible, innovative learning models to engage scholars in team science, individual development plans, and advanced research training. The concept also will promote evidence-informed mentoring practices and includes a leadership and management component. This concept aligns with NCATS Strategic Goal 3 to develop and foster innovation in translational training and a highly skilled, creative, and diverse translational science workforce.

NCATS anticipates that the K12 will nurture the characteristics of a translational scientist, with enhanced tracking of scholar outcomes and measures of impact. The K12 is expected to create a clear and sustainable career pathway for junior faculty, enable scholars to acquire the knowledge and skills needed to cross translational science hurdles, and expand the field of translational science. This concept will support the next generation of diverse clinical and translational scientists who have the knowledge, skill sets, and abilities to advance discoveries across the translational science spectrum to improve health.

Project/Program Officer:
Joan Davis Nagel, M.D., M.P.H.
Medical Officer
Division of Clinical Innovation
National Center for Advancing Translational Sciences
Phone: 301-827-2512
Email: joan.nagel@nih.gov

Research Education Grant (R25)

June 11, 2021

Jamie Mihoko Doyle, Ph.D., presented a concept for the CTSA Research Education Grant (R25). The NIH R25 grant supports activities that complement or enhance workforce training; enhance diversity; help recruit individuals with specific specialty or disciplinary backgrounds; and foster a better understanding of biomedical, behavioral, and clinical research and its implications. Currently, 25 R25 funding opportunities are active across the NIH. Very few training programs have short-term agendas. The current CTSA TL1 programs can include predoctoral, postdoctoral, and short-term positions.

This NCATS R25 concept provides an opportunity to expand, enhance, and meet local needs with the objective to support short-term clinical and translational research experiences (10 to 15 weeks) not available through formal NIH training mechanisms. The overarching goal is to build a pathway for a translational science workforce of the future.

Project/Program Officer:
Jamie Mihoko Doyle, Ph.D.
Program Director
Division of Clinical Innovation
National Center for Advancing Translational Sciences
Phone: 301-402-0403
Email: doylejm@mail.nih.gov

Helping to End Addiction Long-termSM Initiative or NIH HEAL InitiativeSM Translational Science Training

Jan 15, 2021

Dr. Colvis presented the NIH HEAL InitiativeSM translational science training concept. The NIH HEAL Initiative, a trans-NIH effort to speed scientific solutions to stem the national opioid public health crisis with a focus on developing novel pharmacotherapies to treat pain, opioid use disorder and overdose. It is well known that therapeutic discovery, development, and deployment is a complex process. Traditional training often occurs within a small segment of this process. In addition, workplace diversity in biomedical research is less than optimal, particularly within therapeutic discovery and development.

The objective of this concept is to provide NIH HEAL Initiative funds to support early- and mid-career scientists who are expert in pain or opioid use disorders research to receive in-depth training in therapeutic development in an academic or government translational research center or in an industry setting. The area of emphasis directly addresses the lack of workplace diversity in drug development by specifically providing translational training to individuals from underrepresented groups. The goal is to build a workforce of investigators better equipped to translate scientific discoveries into clinical breakthroughs.

The NCATS anticipates that this opportunity will provide the fields of pain, dependency, and overdose with scientists better equipped to design experiments with an emphasis on translating discoveries to affect health. Importantly, incorporating a translational perspective in the early- and mid-career stages will promote the guiding NCATS principle of bringing “more treatments to more patients more quickly.”

Project/Program Officers:
Christine M. Colvis, Ph.D.
Director, Drug Development Partnership Programs
Office of the Director
National Center for Advancing Translational Sciences
Phone: 301-451-3903
Email: ccolvis@mail.nih.gov

Steven T. Pittenger, Ph.D.
Program Officer
Office of the Director
Drug Development Partnership Programs
National Center for Advancing Translational Sciences
Phone: 301-827-5810
Email: steven.pittenger@nih.gov

Technological Development and Validation of Remote Measures for Use in Clinical Trials in Individuals with Rare Diseases

Jan 15, 2021

Tiina K. Urv, Ph.D., and Lili Portilla, M.P.A., presented an SBIR contract concept on technological development and validation of remote measures for use in clinical trials in individuals with rare diseases.  Rare disease patients face significant barriers to participation in clinical trials due to travel limitations, health issues, as well as financial difficulties. Although virtual clinical trials (e.g., remote or decentralized trials) leverage technologies to overcome these barriers, validated measures specific for use in remote clinical trials and studies are missing. This concept proposes to use U.S. small businesses to develop and validate sensitive and specific outcome measures that are technologically viable for use in clinical trials in patients with rare diseases.

The objectives are two-fold: (1) facilitate virtual studies by developing and validating outcome measures that can be assessed remotely and that are suitable for use in clinical trials and (2) develop robust, user-friendly technologies to collect these assessments. This research will capture accurate and replicable data remotely in the patient’s family environment (e.g., adaptive life skills, strength). It also will collect continuous variables and provide a more accurate assessment of the patient’s real condition through new measurements made possible by technology.

Consistent with NCATS mission, this concept supports the three “Ds” to develop, demonstrate and disseminate innovative interventions that benefit public health, enabling a more diverse group of individuals to participate in rare disease clinical trials.  Because of the success of NCATS SBIR-funded projects in stimulating technological innovation and meeting research and development needs, commercialization of these technologies is expected to result in significant patient benefit.

Project/Program Officers:
Tiina K. Urv, Ph.D.
Program Director
Office of Rare Diseases Research
National Center for Advancing Translational Sciences
Phone: 301-827-2746
Email: urvtiin@mail.nih.gov

Lili M. Portilla, M.P.A.
Director, Office of Strategic Alliances
National Center for Advancing Translational Sciences
Phone: 301-827-7170
Email: lili.portilla@nih.gov

CTSA Consortium-Wide Centers: Resources for Rapid Demonstration and Dissemination (C3-R2D2)

Jan 15, 2021

Audie A. Atienza, Ph.D., presented a concept on establishing the CTSA Consortium-Wide Centers: Resources for Rapid Demonstration and Dissemination (C3-R2D2) initiative, which proposes to leverage capabilities and expertise across the CTSA Program to advance Clinical and Translational Science (CTS). Innovations within CTS require multidisciplinary teams with diverse and unique skill sets. Three centers currently share capabilities and expertise across the consortium: the CTSA Data to Demonstrate Health (CD2H), Centers supported through the Trial Innovation Network (TIN), and the Center for Leading Innovation and Collaboration (CLIC).

Over the past few years, CTSAs’ successes have expanded beyond coordinating communications, facilitating data harmonization and data sharing, and using innovative approaches in clinical trial recruitment. NCATS proposes the C3-R2D2 initiative to build on these successes and focus on research demonstration and dissemination at the consortium level. The administrative tasks for the CTSA Program are in the process of being transferred to a support contract mechanism approved by NCATS Council during the January 2020 meeting.

The objective of this concept will be to leverage subject matter expertise across the CTSA Program to develop and provide consortium-level resources and capabilities for specific categories of innovative approaches. Potential areas of emphasis may include health information technology (e.g., mobile health/telemedicine, electronic health records interoperability and data exchange), research-impact quantification, trial designs and recruitment approaches, and platforms/processes that facilitate satisfying regulatory requirements. This initiative also will be responsive to emerging high-priority topics.

In establishing the C3-R2D2 initiative, NCATS anticipates overcoming clinical and translational science roadblocks; accelerating dissemination methodologies across the CTSA consortium for uptake of knowledge, research tools, methods, and data; and identifying, implementing, and disseminating consortium-wide resources for faster adoption to positively affect patients and public health. The overarching goal is to rapidly accelerate CTSA consortium-wide collaborations toward innovative, game-changing, high-impact clinical and translational science advances.

Project/Program Officer:
Audie A. Atienza, Ph.D.
Program Director
Division of Clinical Innovation
National Center for Advancing Translational Sciences
Phone: 301-435-0198
Email: audie.atienza@nih.gov

Clinical Trial Readiness (CTR) for Rare Diseases

Jan 15, 2021

Alice Chen Grady, M.D., presented a renewal concept for the CTR for Rare Diseases, Disorders and Syndromes grants program, aiming to support studies that are addressing critical gaps in rare diseases clinical trials, including lack of (1) validated biomarkers and clinical outcome assessments necessary for selecting appropriate endpoints in rare diseases and (2) a trial readiness priority area needed by regulatory agencies.

NCATS proposes this renewal concept to support projects focused on efficiently and effectively advancing candidate rare diseases therapeutics (or diagnostics) into clinical trials and to increase their likelihood of success in upcoming clinical trials. The program focuses on rare diseases that currently (or soon will have) candidate therapeutics or diagnostics ready for testing in clinical trials and projects that leverage partnerships and existing NCATS resources (e.g., RDCRN).

The first CTR grants were awarded in summer of FY 2019. To date, NCATS and the NICHD have collaborated to fund two Small Research Grant (R03) awards and 16 Exploratory/Developmental Research Grant (R21) awards, through three funding cycles. Regarding accomplishments, nine publications citing this relatively young CTR program have been reported in the NIH Research Portfolio Online Reporting Tools, and several projects show promising data. For example, Children’s Hospital of Philadelphia investigators are working to validate an objective outcome measure of therapeutic response in a pediatric rare disease population.

NCATS and the NICHD recognize an ongoing need to provide FOAs for studies that address specific gaps in understanding the natural history of disease, suitable biomarkers, and appropriate clinical outcome measures.

Project/Program Officer:
Alice Chen Grady, M.D.
Program Officer
Office of Rare Diseases Research
National Center for Advancing Translational Sciences
Phone: 301-827-2015
Email: alice.chen2@nih.gov

2020

Small Research Grants for NCATS Clinical and Translational Science Award (CTSA) Program

Sept. 17, 2020

Erica K. Rosemond, Ph.D., presented a concept to establish small research grants within the CTSA Program. These grants will address the challenge of speeding up the clinical and translational science research process that typically takes multiple steps and time to deliver a solution to any single operational problem.  To speed up this research process, NCATS is proposing small clinical and translational science research projects, which can be leveraged to advance science more quickly and identify “fast-fail” decision points in the project.

The objectives and emphasis will be that these small, self-contained research projects can (1) address general roadblocks in science or operations that limits the efficiency and effectiveness of translation, and (2) develop, demonstrate or disseminate innovative solutions or new or improved treatments that will have an impact on improving the health of patients. In addition, the small research grants will provide an opportunity to support the transition of clinical and translational scientists to achieve independent investigator status.

In implementation, these grants will help research support projects get over a roadblock, catalyze a tangible scientific outcome and allow projects to fail fast resulting in a change in approach.  The overall impact will be assisting young (i.e., early-career) investigators in building their independent research portfolios and transitioning to careers as independent academic translational scientists.

Project/Program Officer:
Erica K. Rosemond, Ph.D.
Deputy Director, CTSA Program Hubs
Division of Clinical Innovation
National Center for Advancing Translational Sciences
Phone: 301-594-8927
Email: rosemonde@mail.nih.gov

Translational Ethics Collaboratories

Sept. 17, 2020

Elaine Collier, M.D., presented a concept of a translational ethics collaboratories program, a next phase in the NCATS ethics research program. Although novel discoveries and emerging technologies offer potential to improve health, the possibility for harm to health or to the norms of individuals, families and communities—as well as of society—remain. The goals of the concept are threefold: (1) support institution-independent collaborative transdisciplinary teams to conduct research on ethical, legal and societal issues related to the application of a novel discovery or emerging technology to improve health, (2) provide research ethics consultation outreach to the research community in a selected area of emphasis, and (3) collect data on this consultation model.

The collaboratory area of emphasis (e.g., synthetic biology, gene modification or data aggregation) must present an ethical issue involved in translation and requires a research consultation project. NCATS is the only NIH Institute or Center that targets systemic issues agnostic of disease, specialty or age across the entire research spectrum, from basic and clinical research to research focused on health care and public health. The translational ethics collaboratories will fill a gap in the knowledge and practice necessary to advance novel discoveries to improve human health in emerging areas. NCATS anticipates that the collaboration of translational scientists, ethicists, legal scholars and social scientists becomes comfortable and routine and facilitates interdisciplinary translational ethics research and consultation. The outcome will provide evidence on the usefulness of research ethics consultation and the effectiveness of models.

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
Email: colliere@mail.nih.gov

Genetic and Rare Diseases (GARD) Information Center

Sept. 17, 2020

Eric Wk Sid, M.D., M.H.A., presented a renewal concept for the GARD Information Center. GARD was launched in response to the Rare Diseases Act of 2002 and congressional mandate to provide consumer health information on rare diseases. Over the past 5 years, the demand for information— with contact center inquiry volume doubling and research evidence exponentially increasing — has outpaced GARD staff’s capabilities to manually maintain and expand content. The GARD Information Center currently is the most visited NCATS webpage, and the traffic (2 million users per month) has at times paralleled that of the NIH home page.

The goal of this concept renewal is to modernize the GARD program and enhance NCATS’ public health role in delivering reliable information about rare diseases to patients and caregivers. The objectives will be to sustainably audit research on the more than 7,000 current and newly identified rare diseases and enhance the ability of GARD to be of maximum use to patients and caregivers. The key areas of emphasis include developing both a research dashboard to quantify and visualize the research evidence in the rare disease portfolio and a translational science framework to educate consumers on rare disease evidence interpretation. A total of 34 public datasets comprising 3.8 million data points are now integrated into GARD’s database from existing NCATS programs and other rare diseases resources. In this next phase, efforts will focus on investigating translational science approaches to leverage these data.

NCATS anticipates that this GARD modernization will clear the contact center’s inquiry backlog by automating processes to stay abreast of new research findings and improve the ability of rare disease patients and caregivers to make decisions using these data, with the outcome of shortening the diagnostic journey for patients with rare diseases.

Project/Program Officer:
Eric Wk Sid, M.D., M.H.A.
Program Director
Office of Rare Diseases Research
National Center for Advancing Translational Sciences
Phone: 301-827-3073
Email: eric.sid@nih.gov

Scanning for Conditions with Electronic Nose Technology (SCENT)

Sept. 17, 2020

Danilo Tagle, Ph.D., M.S., presented the SCENT concept. The goal is to develop a noninvasive and portable diagnostic device that provides rapid and accurate diagnosis of a variety of medical conditions, facilitating treatment of patients. The most pervasive method of sensing in nature, detection of scents and odors, has scarcely been used in the diagnosis of disease. This concept aims to reverse engineer a bio-mimic of the sense of smell found in canines for disease diagnostics, involving several steps — from the detection of the smell/odor, to data acquisition and processing, to comparisons of known patterns, and the readout via a mobile device or portable system.

Leah Tolosa Croucher, Ph.D., explained that the objectives of the concept are to use volatile organic compounds (VOCs) that are released through the skin and/or breath as the key substrate, integrate the components of the various SCENT instrumentation and software, and establish VOC signatures unique for each disease. The SCENT devices will adhere to the Affordable, Sensitive, Specific, User-friendly, Rapid and robust, Equipment-free and Deliverable to end users (ASSURED) criteria outlined by the World Health Organization (WHO). Machine learning and artificial intelligence will be incorporated to rapidly analyze the VOC signatures unique for each disease and condition, and to ensure that the WHO ASSURED criteria are met.

The anticipated result would be a market-ready handheld/wearable, noninvasive diagnostic device that is expected to decentralize diagnostic testing, bringing it closer to patients and resulting in improved care and patient outcomes.

Project/Program Officers:
Danilo A. Tagle, Ph.D., M.S.
Associate Director for Special Initiatives
Office of the Director
Office of Special Initiatives
National Center for Advancing Translational Sciences
Phone: 301-594-8064
Email: Danilo.Tagle@nih.gov

Leah Tolosa Croucher, Ph.D.
Program Officer
Office of the Director
Office of Special Initiatives
National Center for Advancing Translational Sciences
Phone: 240-701-2580
Email: Leah.Croucher@nih.gov

Novel Explorations in Rare Diseases (NERD)

Sept. 17, 2020

Dr. Pariser presented the novel explorations in rare diseases concept and noted the rationale for such a program. Extracting rare disease patient information from existing health care system databases is exceedingly challenging and often unreliable. The available tools are not readily usable for rare diseases, especially multi-disease explorations/analyses. Data extraction is unique to individual data environments (e.g., electronic health record (EHR) system, region/location). NCATS is proposing this concept to quantify the impact, including cost and health care utilization, of rare diseases on patients and health care systems.

The objectives are threefold: (1) develop methodology to accurately and broadly estimate the prevalence of rare diseases and associated health care utilization, (2) develop patient journey maps across different diseases to objectively describe and quantify the diagnostic odyssey and (3) adapt machine learning software and tools to derive this information directly from primary source data. Key focus areas include identifying, with reproducibility, patients with rare diseases within different health care databases, quantifying the overall impact, and working with partners and/or vendors to develop the necessary methodology and tools.

Regarding implementation, a NERD 14-disease pilot is being conducted intramurally, and some efforts are in progress externally with academic and industry partners in the United States and Western Australia. NCATS envisions this concept as having the transformative potential to improve understanding of the magnitude of rare diseases and their impacts on the health care and research systems and on the well-being of patients and their survival.

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

Rare Diseases Informatics Platform (RDIP)

Sept. 17, 2020

Dr. Pariser presented a concept to establish a rare diseases informatics platform, which builds on the NCATS GARD and NERD proposals. This concept will address several needs. First, most rare disease information routinely used are estimates and are not easily traceable back to the primary data sources. Second, current data analysis efforts are discrete, siloed and do not comprehensively meet the perquisite for timely rare diseases information. Third, the ability to collect and integrate rare disease information from multiple sources (e.g., insurance, EHR or research) is lacking. To address these needs, NCATS proposes this concept to support the collection, integration and analysis of rare diseases data from diverse sources. This information will inform NCATS’ rare diseases research initiatives and priorities.

The objectives are to create and staff an RDIP or knowledge center; collect, integrate and analyze rare diseases data from diverse sources to provide objective information for multiple purposes; and incorporate ongoing ORDR data initiatives within one central location. NERD is being designed as a model for building the RDIP, which will harmonize approaches or data models to integrate with or leverage other ongoing NCATS informatics programs, such as GARD, DCI-data initiatives and the Biomedical Data Translator.

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

Bespoke Gene Therapy Consortium (BGTC) Coordinating Center

Sept. 17, 2020

P.J. Brooks, Ph.D., presented a concept on the BGTC Coordinating Center. Many monogenic rare diseases could benefit from gene therapy using adeno-associated virus (AAV) vectors. For diseases of no commercial interest, navigating the multiple steps (e.g., vector production and toxicity testing) necessary to get to a clinical trial, as well as the clinical trial itself, is a major challenge. To address this challenge, NCATS, the FDA Center for Biologics Evaluation and Research, and the Foundation for the NIH (FNIH) are establishing the BGTC, a major component of the FNIH Gene Therapy Accelerated Medicines Partnership (AMP).

NCATS proposes a BGTC Coordinating Center to support these multiple steps using contract mechanisms, all under the direction of the Coordinating Center. The objectives are to coordinate contract activities for the BGTC and evaluate novel collaborative approaches to support gene therapy clinical trials for rare diseases. In addition to accelerating rare diseases clinical trials, other areas of emphasis for this concept include collaboration with multiple stakeholders (e.g., FDA, private industry, nonprofits and rare diseases PAGs) and commitment to ensuring that the diseases chosen for study will include those affecting racial and ethnic minority populations.

The BGTC Coordination Center and its activities are expected to be synergistic with other NCATS programs and initiatives, such as the Platform Vector Gene Therapy (PaVe-GT) project. In the long-term, knowledge gained and best practices will be parlayed into the AAV-based gene therapy clinical trials for rare diseases handbook and broadly disseminated.

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
Email: pj.brooks@nih.gov

A Specialized Platform for Innovative Research Exploration—ASPIRE-ing Beyond HEAL

May 14, 2020

Danilo A. Tagle, Ph.D., M.S., presented a new concept to expand the ASPIRE Program. He reminded the Council that the ASPIRE initiative originally was proposed in September 2017 to address key translational challenges: (1) the uninterrogated vast chemical space (1063) of potential pharmacologically active molecules, (2) the undrugged biological space (5 × 105), and (3) an outdated reaction toolkit for accessing the relevant chemical space. To address these issues, NCATS convened the “Workshop on Automated Chemical Synthesis” on October 19–20, 2017, to identify the associated research opportunities, challenges and roadblocks. Subsequently, NCATS officially launched ASPIRE in 2018, as a pilot program funded by the Helping to End Addiction Long-termSM Initiative, or NIH HEAL InitiativeSM with an extramural component through prize competitions and challenges, and an intramural component to build an automated chemistry infrastructure.

For this new concept that goes beyond the initial HEAL investment, Dr. Tagle noted the goals will be to establish a collaborative structure involving multidisciplinary expert teams from NCATS intramural and extramural scientists that takes advantage of the validated open-source infrastructure as well as tools and technologies within NCATS Division of Preclinical Innovation toward general translational challenges in synthetic chemistry.

Samuel Michael described the intramural components of the ASPIRE modular platform, which includes automation, informatics, consumables, chemistry, analytical, and biology modules, as well as technology development. The initial platform will be a 16-module design with integrated automation and technology components.

Dr. Tagle highlighted that the outcome will be a disseminatable platform for automated chemical synthesis, biological testing, and machine learning–driven optimization. NCATS anticipates that this platform will broadly enable the identification and synthesis of novel biologically active chemical structures with drug-like characteristics. The potential exists for a much-needed expansion of the chemical space toward new and improved therapeutics, which would catalyze an innovative and collaborative ecosystem.

Project/Program Officers:
Danilo A. Tagle, Ph.D., M.S.
Associate Director for Special Initiatives
Office of the Director
Office of Special Initiatives
National Center for Advancing Translational Sciences
Phone: 301-594-8064
Email: Danilo.Tagle@nih.gov

Samuel G. Michael
Chief Information Officer
Information Technology Resources Branch
Office of Administrative Management
National Center for Advancing Translational Sciences
Phone: 301-827-7796
Email: michaelsg@mail.nih.gov

Artificial Intelligence in Health Care

May 14, 2020

Karlie R. Sharma, Ph.D., presented a concept on advancing artificial intelligence (AI) in health care. The concept was developed in collaboration with Christine M. Cutillo, M.M.C.i. and will leverage NCATS’ existing AI and informatics efforts. The importance of using AI in health care is well emphasized in the clinical community, but translating such innovative tools for clinical applications remains a challenge. In recent weeks, many laboratories and companies have attempted to use AI for COVID‑19 treatments. These, in addition to other recent examples, illustrate the urgent need for new paradigms for the development and application of these innovative AI-based tools before and as they are deployed into clinical care settings, because misdiagnoses from AI systems can result in fatal consequences.

Dr. Sharma highlighted several unique challenges that have impeded the progress of AI in health care; these challenges include barriers to data sharing, ineffective implementation into clinical care workflows, and transparency in ethical issues. To address these issues, NCATS sponsored a workshop focusing on machine intelligence in health care in July 2019. Three key topics emerged from the discussion that rose to the top as timely and aligning with the NCATS purview: testing algorithms in the real world prior to their implementation, prioritizing reproducibility and robustness of AI systems, and improving communication across groups developing these systems and the community that will use them. This concept aims to (1) develop a pipeline for testing algorithms prior to implementation in the clinic, and (2) in the long-term, improve trust in and uptake of AI systems in the health care setting. In a proof-of-concept approach, NCATS will develop and test the pipeline with use case algorithms and demonstration projects (Stage 1), then use the tested pipeline to assess newly developed algorithms focused on urgent clinical needs in a variety of disease areas (Stage 2).

The outcomes of the research will be (1) a fully formed and vetted pipeline equipped with standard operating procedures, creating a framework for comprehensive algorithm assessment in real-world environments; (2) distributable algorithms that can be implemented across health systems worldwide; and (3) dissemination of results and resources to the broader research community to facilitate the creation of other AI pipelines by the community modeled after the initiative-developed pipeline. NCATS anticipates that the incorporation of these algorithms into clinical practice will create new sources of reliable information for clinicians to better diagnose and treat patients.

Project/Program Officers:
Karlie R. Sharma, Ph.D.
Program Officer
Office of the Director
Drug Development Partnership Programs
National Center for Advancing Translational Sciences
Phone: 301-451-4965
Email: karlie.sharma@nih.gov

Christine M. Cutillo, M.M.C.i.
Data Science Lead
Office of the Director
National Center for Advancing Translational Sciences
Phone: 301-402-4006
Email: cutilloc@mail.nih.gov

Multisensory Virtual/Augmented Reality (VR/AR) Systems and Rehabilitation for Rare Disease Patients

May 14, 2020

Mr. Michael presented the SBIR contract concept on the topic multisensory VR/AR and rehabilitation for patients with rare diseases. The senses (e.g., audio, visual and hand haptics) are limited in current VR/AR systems but could benefit from taking advantage of other senses that include full-body haptics (e.g., TESLASUIT), smell and taste. Research has shown that VR/AR immersive experience can reduce pain and discomfort in rehabilitation and, potentially, in clinical settings. In addition, the AI and machine learning algorithms can generate individualized immersive experiences, which must be verified for efficacy, user needs, and preferences. The challenge is that individuals respond differently to these environments and systems must be tailored.

This contract concept will support the development of multisensory VR/AR systems that adapt intelligently to each individual for the assessment and rehabilitation of patients with rare diseases. The aim is to assess the efficacy of the systems in clinical and rehabilitation settings for these patients through biofeedback readings and user self-reports. Developing such systems could benefit the ORDR by enabling technology-based applications for the rare diseases community. Applications include pain management and rehabilitation, remote monitoring and data capture for clinical trials, and diagnostic assistance for rare diseases with motor components.

NCATS will solicit SBIR applications to develop a virtual environment proof-of-concept system that incorporates dynamic audio, visual, full-body haptic and olfactory stimuli. The system should incorporate runtime logfile capabilities for remote patient monitoring and performance analytics and a human-in-the-loop system model that addresses patient personalization and biofeedback. The outcome will be a system that provides users a VR/AR pipeline to influence the accessibility, affordability and accuracy of therapeutic treatment and clinical use; design guidelines and efficacy considerations for existing VR/AR platforms; a multisensory tool or suite of tools for remote patient monitoring; and an AI-driven user behavioral model for adapting rehabilitation stimuli. The intent is that these remote-based systems would assist with clinical monitoring and rehabilitation efforts for patients with rare diseases, especially in cases where travel is impossible or difficult. The systems are intended to benefit the entire research community.

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
Email: michaelsg@mail.nih.gov

Platform for Rapidly Deployable Autonomous Laboratory

May 14, 2020

Mr. Michael presented the SBIR contract concept on the topic of a platform for a rapidly deployable autonomous laboratory, which aligns with the NCATS ASPIRE initiative. The COVID-19 pandemic—and subsequent physical distancing and closing of research laboratories—has led to the postponement of critical experiments necessary for developing novel diagnostics and potential therapeutic interventions. The purpose of this SBIR contract concept is to develop next-generation distributed, AI-enabled, fully automated laboratories that are linked to a cloud-based virtual research organization (VRO) to acquire, harmonize, store, analyze and share data generated during experimentation.

The goals are to create a platform consisting of modular automated devices capable of performing laboratory tasks for both diagnostic and therapeutic discovery purposes. NCATS will solicit SBIR applications to develop the following: (1) a distributed, modular, autonomous lab instrumentation platform that focuses on such areas as HTS for drug discovery, next-generation sequencing, high content imaging, and polymerase chain reaction diagnostics and (2) a cloud-based VRO that connects each automated laboratory and AI methods that integrate the lab data into the VRO, allowing real-time data collection and analysis. NCATS will leverage its collaboration with Kebotix, Inc., on AI/machine learning models and HTS.

Carleen Klumpp-Thomas, M.S. described an example of use of the proposed platform. In collaboration with the National Institute of Biomedical Imaging and Bioengineering (NIBIB), one of its a small laboratory without automated equipment was upgraded. The automation increased the laboratory’s analysis capacity from 500 to 10,000 samples, and copious data were collected in real time and exported into a VRO.

Mr. Michael noted that the outcomes of the SBIR contract concept will be a system that provide users (1) a platform that enables the on-demand initiation of physical experiments across laboratories, (2) a cloud‑based VRO that houses data suited for analysis, and (3) a modular platform that can quickly add or scale up additional resources necessary for responding to public health emergencies. NCATS anticipates that using SBIR contract mechanisms will create opportunities for small businesses to facilitate an emergency commercialization for rapidly scalable instrumentation.

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

Carleen A. Klumpp-Thomas
Research Services Core Lead
Division of Preclinical Innovation
National Center for Advancing Translational Sciences
Phone: 301-827-1789
Email: klumppc@mail.nih.gov

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
Email: clare.schmitt@nih.gov

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
Email: pj.brooks@nih.gov

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
Email: pj.brooks@nih.gov

2019

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
Email: alice.chen2@nih.gov

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
Email: colliere@mail.nih.gov

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
Email: pj.brooks@nih.gov

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
Email: michaelsg@mail.nih.gov

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.
Director
Office of Rare Diseases Research
National Center for Advancing Translational Sciences
Phone: 301-402-4338
Email: anne.pariser@nih.gov

2018

Drug Development Collaboratory

Sept. 27, 2018

The proposed Drug Development Collaboratory would include the intramural Therapeutic 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 Therapeutic 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).

2017

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).

2016

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).

2015

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).

2014

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.

2013

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.

2012

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.