From the Director | What's New at NCATS? | Research Opportunities Volume 04 • Issue 03 • March 26, 2015

Director's Message

Christopher Austin

Often in today's world, the word "chemical" conjures up a toxic, man-made substance that is undoubtedly harmful to human health. The growing consumer demand for "natural" foods and cosmetics illustrates this trend. Similarly, the word "drug" can have a different meaning depending on whether it's considered dangerous (heroin) or helpful (aspirin). In the scientific arena, however, the distinctions between these terms are largely artificial. For example, the widely used stroke and heart attack prevention drug warfarin (trade name Coumadin) has another common use as a rat poison.

Read more in the latest Director's Message.

Christopher P. Austin, M.D.

What's New at NCATS?

Tox21 Collaboration Generates Innovative Platform for Chemical Sensitivity Testing

NCATS Hosts Event for NIH Researchers on Pfizer's Centers for Therapeutic Innovation

CTSA Initiative Yields Standard Agreement for Industry-Sponsored Clinical Trials

Matching CTSA Investigators with NIH Research Opportunities

NIH Postdoctoral Scholars Learn How to Translate Their Research at NCATS Workshop

Rare Disease Day Emphasizes Teamwork and Dedication

Improving Patient Recruitment: The Accrual to Clinical Trials Initiative

Creating a National Institutional Review Board Agreement

NCATS Hosts Inaugural Assay Guidance Manual Workshop

Creating Review Standards for Investigator-Initiated Research Studies

Establishing Good Clinical Practices for Clinical Study Personnel

NIH Plan for Increasing Access to Scientific Publications and Digital Scientific Data

Upcoming Events

NCATS in the News

Collaborate with NCATS Scientists

Toxicology in the 21st Century robot

Tox21 Collaboration Generates Innovative Platform for Chemical Sensitivity Testing

What if we could predict whether an individual will have an adverse reaction to everyday chemicals, such as those in laundry detergent or perfume? NCATS researchers and collaborators, as part of the Toxicology in the 21st Century (Tox21) program, have published a groundbreaking study in the Jan. 13, 2015, issue of Environmental Health Perspectives. Read the full feature. View the NCATS Director's message.

NCATS Hosts Event for NIH Researchers on Pfizer's Centers for Therapeutic Innovation

On April 13, 2015, NCATS will host a seminar for NIH intramural researchers interested in pursuing collaborative opportunities with Pfizer's Centers for Therapeutic Innovation (CTI) network. The seminar will take place from 2 to 5 p.m. in Lister Hill Auditorium, Building 38A on the NIH campus in Bethesda, Maryland. Interested scientists also can participate in one-on-one meetings by appointment on April 14, 2015. The NIH-CTI collaboration is designed to help bridge the gap between early scientific discovery and its translation into new medicines through public-private resource sharing. The program pairs leading researchers with Pfizer resources to pursue scientific and medical advances through joint therapeutic development. Learn more.

Two men sitting at a conference room table.

CTSA Initiative Yields Standard Agreement for Industry-Sponsored Clinical Trials

A standard agreement intended to reduce the contract negotiation time for industry-sponsored multisite studies, including Phase IIb and Phase III trials, is now available to download. The Accelerated Clinical Trial Agreement (ACTA) is the result of an initiative supported by NCATS' Clinical and Translational Science Awards (CTSA) that sought to reduce clinical trial contracting delays by developing efficient contract models for sponsor-initiated multisite trials.

At the February 2015 CTSA principal investigators' meeting, the Mayo Clinic's Sundeep Khosla, M.D., shared progress on ACTA. Composed of legal experts from 25 CTSA institutions, the team collaborated with industry to develop a standardized clinical trial agreement that could be used voluntarily by participating institutions and sponsors. ACTA addresses issues such as recruitment, record retention, patient safety, HIPAA/confidentiality, right to terminate, patient billing, publications, limit of liability, data use, intellectual property and indemnification. More than 60 institutions (57 affiliated with CTSAs) have accepted ACTA.

A scientist working in a lab.

Matching CTSA Investigators with NIH Research Opportunities

In January 2015, NCATS began a series of webinars and conference calls between Clinical and Translational Science Awards (CTSA) investigators and staff from NIH Institutes and Centers (ICs) on research topics and opportunities of high interest to both parties. The goal is to encourage collaboration between the NIH ICs and the CTSA network for high-priority multisite clinical trials.

The first webinar highlighted the upcoming TACT2 study to assess chelation therapy in patients who have diabetes and who have had a heart attack — a topic of importance to the National Heart, Lung, and Blood Institute and the National Center for Complementary and Integrative Health. Gervasio Lamas, M.D., chairman of medicine at Mount Sinai Medical Center, explained that this new study builds upon beneficial findings from an earlier trial assessing chelation therapy in heart attack survivors, which was published in JAMA in March 2013. Findings from that study showed that an intravenous chelation regimen reduced the risk of adverse cardiovascular events in heart attack survivors, particularly those with diabetes. Now Lamas plans to test this hypothesis in a follow-on study with diabetes patients, and he hopes that CTSA hubs will participate.

Through the connections made via these webinars and conference calls, NCATS hopes to uncover more opportunities for the ICS and the CTSA network to partner on important multisite clinical trials. The next webinar in May 2015 will focus on opportunities to collaborate with select ICs through Small Business Innovation Research/Small Business Technology Transfer grants.

People meeting in a boardroom

NIH Postdoctoral Scholars Learn How to Translate Their Research at NCATS Workshop

"How can I translate my basic research into a medical innovation that will benefit patients?" That was the question on the minds of 20 NIH postdoctoral research fellows who attended the NCATS Translational Science Training Program (TSTP) workshop on March 4, 2015. Sponsored by NCATS in coordination with the NIH Office of Intramural Training and Education, the event was designed to provide young researchers at NIH with career development information and show how observations in the laboratory can be turned into interventions that improve public health. The workshop focused on pre-clinical methods and technologies and featured scientific presentations, a tour of NCATS research facilities, and small group discussions with experienced drug development scientists.

NCATS Director Christopher P. Austin, M.D., kicked off the event by speaking about the immense challenges and opportunities in translational science and the unique environment NCATS has created to develop, demonstrate and disseminate solutions. Other presentations included topics such as target identification and validation with RNA interference technology, assay design and development for screening chemical compounds, and NCATS resources that help investigators de-risk drug candidates and conduct studies required to initiate clinical trials. In the small group discussions, NCATS intramural researchers provided expert feedback on the participants' ideas for developing their own translational science projects. During the tour of NCATS intramural facility, participants saw the cutting-edge equipment and tools used to discover and develop new therapies and innovative technologies.

Matt Might, Ph.D., president of NGLY1.org, discusses a community-building model for rare diseases research.

Rare Disease Day Emphasizes Teamwork and Dedication

On Feb. 27, 2015, more than 400 rare disease patients, advocates, researchers and interested members of the public attended the Rare Disease Day at NIH event on campus in Bethesda. Rep. Leonard Lance (New Jersey-7th District), co-chair of the Rare Disease Congressional Caucus, set the tone for an informative and moving program, saying, "Today is about you, the tireless advocates for rare diseases."

Sponsored by NCATS and the NIH Clinical Center, the event also included remarks by NIH Director Francis S. Collins, M.D., Ph.D.; NCATS Director Christopher P. Austin, M.D.; NIH Clinical Center Director John I. Gallin, M.D.; Food and Drug Administration Office of Orphan Products Development Director Gayatri Rao, M.D., J.D.; and other collaborators.

The day featured presentations on successful team and community building, the critical role of technology transfer and strategic alliances in advancing treatments, and NCATS' Rare Diseases Clinical Research Network advances. The underlying "teamwork" theme underscored how the rare diseases community is continuing to build momentum by working together.

Watch an archived videocast of the event, and check out photos from the event on NCATS' Flickr page.

In the photo from the event, Matt Might, Ph.D., president of NGLY1.org, discusses a community-building model for rare diseases research.

A health care professional treating a patient.

Improving Patient Recruitment: The Accrual to Clinical Trials Initiative

During the annual Clinical and Translational Science Awards (CTSA) principal investigators' meeting in February 2015, University of Pittsburgh's Steven Reis, M.D., provided an update on the Accrual to Clinical Trials (ACT) initiative. The NCATS-supported effort, which was launched in September 2014 to overcome clinical trial roadblocks, aims to create a network of sites that share electronic health record (EHR) data to further multisite study feasibility and recruitment efforts. The initiative builds on existing platforms (i2b2/SHRINE) and operating models to create a "federated" network with common standards, data terminology and shared resources. Over the last several months, CTSA ACT investigators have been working on:

Reis reported that the team had met the four-month key milestones: All 13 CTSA hubs from the first phase implemented the technical platform, all 13 sites received institutional review board approval and 8 more hubs were selected for the second phase. Plans are underway to include more hubs and coordinate with PCORNet to synergize the two national efforts. Read the related January 2015 feature.

A man presenting his computer screen to colleagues.

Creating a National Institutional Review Board Agreement

Before starting multisite clinical trials, investigators often must go through multiple institutional review board (IRB) processes at each study site. To overcome redundancies and improve efficiencies, NCATS' Clinical and Translational Science Awards (CTSA) program supports an initiative designed to create a national IRB reliance agreement. At a February 2015 meeting of CTSA principal investigators, participants learned about IRBrely, which aims to create a national IRB reliance agreement by building on the expertise of existing regional IRB models.

Alan Green, M.D., of Dartmouth College, reported on the project's progress to date and gave some examples of regional IRB models. So far, the project team has shared a draft agreement with the legal departments of the participating institutions, has identified key system requirements to support IRB reliance and sharing functions and is developing a centralized portal and homepage for a national IRB reliance agreement using open source software. Next steps include feasibility testing using a low-risk multicenter clinical trial and evaluation. Read a related story in the November 2014 e-newsletter.

People in a conference room.

NCATS Hosts Inaugural Assay Guidance Manual Workshop

NCATS' Assay Guidance Manual, an eBook developed in coordination with an international editorial board and colleagues at Eli Lilly and Company, is a 32-chapter how-to guide for researchers on developing and using assays in high-throughput screening projects. In February 2015, the manual's editors held an inaugural workshop, "Assay Guidance for High Throughput Screening and Lead Discovery," at the NCATS laboratories in Rockville, Maryland. The goal of the workshop, which was aimed at scientists who are new to the drug discovery community, was to provide a broad introduction and baseline information for developing assay methods.

Participants included intramural researchers from all NIH Institutes and Centers and the Food and Drug Administration. Activities included presentations about critical fundamental concepts relevant to researchers with diverse backgrounds and interests, demonstrations and tours, and small group discussions. Due to overwhelming interest, plans for the next workshop are underway.

Creating Review Standards for Investigator-Initiated Research Studies

The NCATS Scientific Review Standards initiative aims to develop a consensus Clinical and Translational Science Awards (CTSA) scientific review process that can ensure feasibility and scientific validity for research projects before reviews by institutional review boards. At the annual CTSA principal investigators' meeting in February 2015, Tufts University's Harry Selker, M.D., M.S.P.H., reported on this initiative and the progress made by the team leading the effort. Initially, the team is focusing on CTSA-funded, investigator-initiated clinical studies, such as pilot awards and projects by TL1 trainees and KL2 scholars. Selker said the goal is to recommend a scientific review committee (SRC) process that is acceptable to the CTSA consortium, conduct a pilot test to evaluate the approach and create a plan to disseminate the results once the final product is agreed upon. The team has developed plans for SRC operational processes and procedures, information technology infrastructure, and evaluation. Next steps include finalizing the consensus report and testing the SRC impact for speed, efficiency and quality in a pilot study before implementing it across the consortium.

Two health care professionals shaking hands.

Establishing Good Clinical Practices for Clinical Study Personnel

At the February 2015 Clinical and Translational Science Awards (CTSA) principal investigators' meeting, Thomas Shanley, M.D., of the University of Michigan, shared progress and next steps for the CTSA program's Good Clinical Practice (GCP) initiative. This initiative was established to ensure that clinical study personnel are qualified and acquire necessary competencies to implement safe and efficient studies. Shanley explained that in the first phase, the project team is focusing on standardizing GCP training for clinical study team personnel across the CTSA consortium.

To start, the team identified key competencies and evaluation metrics, which were then shared with all 62 CTSA hubs. The team drafted GCP recommendations and had them endorsed by the CTSA consortium and forwarded to NCATS leadership. Once these are finalized, staff at the CTSA hubs will demonstrate the effectiveness of the GCP training standards within their institutions and then disseminate the standards broadly. For the second phase of the initiative, the team plans to develop a competency-based training curriculum for clinical research professionals. View an earlier article on this subject.

NIH Plan for Increasing Access to Scientific Publications and Digital Scientific Data

For the past two years, NIH and other federal agencies have been developing plans to increase access to scientific publications and digital data for the benefit of the biomedical research community and the patients they ultimately serve. NCATS strongly supports data transparency and public release to speed the translation of new knowledge into improved human health.

The final NIH plan, released in February, requires no changes to NIH's current public access policy for scientific publications that result from NIH-funded research. The new plan does include several actions NIH will take to increase access to scientific data from funded studies:

Visit the Department of Health and Human Services (HHS) Open Government website for access to plans for NIH and other HHS components.

Upcoming Events

April

Pfizer's Centers for Therapeutic Innovation (CTI) Seminar and One-on-One Meetings for NIH Intramural Researchers

On April 13, 2015, NCATS will host a seminar as well as one-on-one meetings for NIH intramural researchers interested in Pfizer's Centers for Therapeutic Innovation (CTI) collaborative opportunities. The CTI model is the first NIH-wide biologics initiative with a pharmaceutical partner that NCATS will coordinate on behalf of all NIH intramural researchers.

Translational Science 2015

On April 16–18, 2015, the Association for Clinical and Translational Science and the American Federation for Medical Research are sponsoring the Translational Science 2015 Annual Meeting to bring together all of the disciplines involved in clinical and translational research, for the shared benefits of networking and education. Presenters include Petra Kaufmann, M.D., M.Sc., director of NCATS' Division of Clinical Innovation, and Robert M. Califf, M.D., newly named deputy commissioner for Medical Products and Tobacco, Food and Drug Administration, and former principal investigator at the Duke Translational Medicine Institute, which is supported by the Clinical and Translational Science Awards program.

International Society for Extracellular Vesicles (ISEV) Annual Meeting

On April 22, 2015, the NIH Extracellular RNA Communication Consortium will host an Education Day as a prelude to the ISEV annual meeting on April 23–26, 2015. The ISEV meeting will feature cutting-edge research presentations and keynote speakers, including NIH Director Francis S. Collins, M.D., Ph.D., and Xandra O. Breakefield, Ph.D., extracellular vesicle pioneer and Harvard University professor of neurology.

May

Second EATRIS Conference: Building Bridges in Translational Medicine

On May 27–28, 2015, the EATRIS (European Infrastructure for Translational Medicine) conference will bring together key international stakeholders in translational research, providing a unique opportunity to interact with a wide range of key stakeholders: patient organizations; research funders; and representatives from government, industry and regulatory agencies. NCATS Director Christopher P. Austin, M.D., will be one of the keynote speakers on May 28 during Session 4: Global Collaboration in Translational Medicine.

June

NCATS Advisory Council/CAN Review Board to Meet June 17–18

On June 17–18, 2015, NCATS will hold a joint meeting of the NCATS Advisory Council and the Cures Acceleration Network (CAN) Review Board on the NIH campus in Bethesda, Maryland. The meeting will feature reports from NCATS Director Christopher P. Austin, M.D., and other presenters about the Center's initiatives, policies, programs and future direction. For more information, visit NCATS Advisory Council and CAN Review Board.

NCATS in the News

Collaborate with NCATS Scientists

NCATS researchers are seeking collaborators in the following areas:

NCATS Chemical Genomics Center (NCGC)

NCATS offers biomedical researchers access to large-scale screening capacity and medicinal chemistry and informatics expertise to develop chemical probe molecules. These resources can help scientists study the functions of genes, cells and biochemical pathways. For inquiries or to obtain NCGC probe molecules, contact Ajit Jadhav.

NCATS researchers also seek collaborators for assay development and high-throughput screening, chemistry and chemistry technology, automation, and informatics. Learn more.

NIH RNA Interference (RNAi) Initiative

Through the NIH RNAi initiative, NCATS provides state-of-the-art, high-throughput RNAi genome-wide screens for humans and mice. This resource is available only to NIH researchers. For more information, contact Madhu Lal-Nag, Ph.D.

Toxicology in the 21st Century (Tox21) Program

The goal of the Tox21 program is to test 10,000 chemicals and evaluate their potential to cause health problems. Any investigator may propose the development of biological assays for high-throughput screening. To suggest an assay, submit a nomination form to Menghang Xia, Ph.D. Proposed assays must be compatible with the high-throughput screening guidelines described in the assay guidance criteria.

Research Opportunities and Announcements

Visit the NCATS Open Opportunities page for a complete list of funding and program announcements.

Reminder: NIH Grant Applications and the NIH Genomic Data Sharing Policy • NOT-OD-15-083

Notice Announcing Frequently Asked Questions (FAQs) for RFA-RM-15-001 "Metabolomics Core for the Undiagnosed Diseases Network (UDN) (U01)" • NOT-RM-15-013

New Form to Capture Additional Indirect Costs in Multi-project Grant Applications • NOT-OD-15-081

Notice Regarding Requirement of Grantees and Contractors to Submit Invention Disclosures, Related Reports and Documents Via iEdison • NOT-OD-15-080

Use of Updated Inclusion Enrollment Format Now Required for Successful Submission of RPPR • NOT-OD-15-078

Notice of National Center for Advancing Translational Sciences (NCATS) Participation in RFA-CA-15-006 "Big Data to Knowledge (BD2K) Advancing Biomedical Science Using Crowdsourcing and Interactive Digital Media (UH2)" • NOT-TR-15-010

Notice of Extension of Public Comment Period for Notice of Proposed Rulemaking for Clinical Trials Registration and Results Submission under FDAAA • NOT-OD-15-072

Notice of NIH Participation in the Cyber-Physical Systems Initiative (CPS) • NOT-EB-15-003

Notice of Intent to Publish a Funding Opportunity Announcement for Pre-applications for Collaborative Innovation Award, Clinical and Translational Science Award Program (X02) • NOT-TR-15-005

Notice of Intent to Publish a Funding Opportunity Announcement for Collaborative Innovation Award, Clinical and Translational Science Award Program (U01) • NOT-TR-15-006

NIH Interim General Grant Conditions Implementing New HHS Grants Regulations (Uniform Guidance) • NOT-OD-15-065

Direct Phase II SBIR Grants to Support Biomedical Technology Development • PAR-14-088

Registration Open for the 2015 NIH Regional Seminar on Program Funding and Grants Administration - Baltimore, MD • NOT-OD-15-063

Metabolomics Core for the Undiagnosed Diseases Network (UDN) (U01) • RFA-RM-15-001

NIH Big Data to Knowledge (BD2K) Enhancing Diversity in Biomedical Data Science (R25) • RFA-MD-15-005

Notice of Intent to Publish a Funding Opportunity Announcement Trial Innovation Centers - TICs for the Clinical and Translational Science Award Network (U01) • NOT-TR-15-001

Notice of Intent to Publish a Funding Opportunity Announcement for Recruitment Innovation Centers (RICs) for the Clinical and Translational Science Award Network (U01) • NOT-TR-15-003

Biomedical Data Science Training Coordination Center (U24) • RFA-ES-15-004

Notice To Extend PA-14-071 "PHS 2014-02 Omnibus Solicitation of the NIH, CDC, FDA and ACF for Small Business Innovation Research Grant Applications (Parent SBIR [R43/R44])" • NOT-OD-15-036

Notice To Extend PA-14-072 "PHS 2014-02 Omnibus Solicitation of the NIH for Small Business Technology Transfer Grant Applications (Parent STTR [R41/R42])" • NOT-OD-15-037

HHS Changes Standard Due Dates for SBIR/STTR Grant Applications • NOT-OD-15-038

Update: New Biographical Sketch Format Required for NIH and AHRQ Grant Applications Submitted for Due Dates on or After May 25, 2015 • NOT-OD-15-032

Reminder: NIH Requires the Research Performance Progress Report (RPPR) for All Type 5 Progress Reports • NOT-OD-15-014

Onsite Tools and Technologies for Heart, Lung, and Blood Clinical Research Point-of-Care STTR (R41/R42) • RFA-HL-14-017

Onsite Tools and Technologies for Heart, Lung, and Blood Clinical Research Point-of-Care SBIR (R43/R44) • RFA-HL-14-011

Platform Delivery Technologies for Nucleic Acid Therapeutics (R41/R42) • PA-14-308

Platform Delivery Technologies for Nucleic Acid Therapeutics (R43/R44) • PA-14-307

Bioreactors for Reparative Medicine (R41/R42) • RFA-HL-15-004

Bioreactors for Reparative Medicine (R43/R44) • RFA-HL-15-008

We Want to Hear from You

We welcome your feedback to ensure that we are meeting the needs of all of our stakeholders. Please e-mail us directly at info@ncats.nih.gov, follow us on Facebook and Twitter, view our YouTube channel, and join the NCATS e-mail list for other Center announcements.

For language access assistance, contact the NCATS Public Information Officer.