Department of Health and Human Services

Part 1. Overview Information
Participating Organization(s)

National Institutes of Health (NIH)

Components of Participating Organizations

National Center for Advancing Translational Sciences (NCATS)

Funding Opportunity Title

A Pre-application for the NIH-Industry Pilot Program:  Discovering New Therapeutic Uses for Existing Molecules (X02)

Activity Code

X02 Pre-application

Announcement Type

New

Related Notices

  • May 12, 2014 - This PAR has been reissued as PAR-14-213.
  • July 20, 2012 - See Notice NOT-TR-12-008. Clarification for the NIH-Industry Pilot Program providing clarification regarding application requirements

Funding Opportunity Announcement (FOA) Number

PAR-12-203

Companion Funding Opportunity

RFA-TR-12-004, Limited Competition for the NIH-Industry Pilot Program: Discovering New Therapeutic Uses for Existing Molecules (UH2/UH3); RFA-TR-12-005, Limited Competition for the NIH-Industry Pilot Program: Discovering New Therapeutic Uses for Existing Molecules (UH3)

Number of Applications

See Section III. 3. Additional Information on Eligibility.

Catalog of Federal Domestic Assistance (CFDA) Number(s)

93.350  

Funding Opportunity Purpose

The National Center for Advancing Translational Sciences (NCATS) seeks to develop a therapeutics discovery pilot program to explore new therapeutic uses for proprietary drug candidates (Agents) across a broad range of human diseases. This innovative program will match Agents and associated data from pharmaceutical company partners with the best ideas for new therapeutic uses from the biomedical research community. 

This FOA encourages X02 pre-applications for the NIH-Industry Pilot Program: Discovering New Therapeutic Uses for Existing Molecules.  The X02 pre-application is the first step in the application process for RFA-TR-12-004 and RFA-TR-12-005; applicants must read both of the companion FOAs.  The X02 pre-applications will be evaluated by a panel of outside experts for scientific and technical merit.  

Investigators whose X02 pre-applications are judged to be the most meritorious will be notified of the opportunity to submit a UH2/UH3 or UH3 application under the Limited Competition for RFA-TR-12-004 or RFA-TR-12-005.  No awards will be made under this FOA.  

Key Dates
Posted Date

June 12, 2012

Open Date (Earliest Submission Date)

July 14, 2012

Letter of Intent Due Date

July 14, 2012

Application Due Date(s)

August 14, 2012 , by 5:00 PM local time of applicant organization.

AIDS Application Due Date(s)

Not Applicable

Scientific Merit Review

September 2012

Advisory Council Review

Not Applicable

Earliest Start Date(s)

Not Applicable

Expiration Date

August 15, 2012

Due Dates for E.O. 12372

Not Applicable

Required Application Instructions

It is critical that applicants follow the instructions in the SF 424 (R&R) Application Guide, except where instructed to do otherwise (in this FOA or in a Notice from the NIH Guide for Grants and Contracts). Conformance to all requirements (both in the Application Guide and the FOA) is required and strictly enforced. Applicants must read and follow all application instructions in the Application Guide as well as any program-specific instructions noted in Section IV. When the program-specific instructions deviate from those in the Application Guide, follow the program-specific instructions. Applications that do not comply with these instructions may be delayed or not accepted for review.

Table of Contents

Part 1. Overview Information
Part 2. Full Text of the Announcement
Section I. Funding Opportunity Description
Section II. Award Information
Section III. Eligibility Information
Section IV. Application and Submission Information
Section V. Application Review Information
Section VI. Award Administration Information
Section VII. Agency Contacts
Section VIII. Other Information

Part 2. Full Text of Announcement

Section I. Funding Opportunity Description

Background

On December 23, 2011, Congress created a new center at the National Institutes of Health (NIH), the National Center for Advancing Translational Sciences (NCATS, Public Law 112-74 (amending the Public Health Service Act, 42 U.S.C. § 287)).  One aspect of the NCATS mission is to focus on developing innovative methods and tools to reduce or eliminate barriers to drug and diagnostic development.  By developing new methods that can be adopted across the entire medical product development sector, NCATS will enhance others’ ability to bring safe and effective products to patients.  One important way that NCATS will do this is to develop innovative public-private partnerships with pharmaceutical companies and the biomedical research community.  To this end, NCATS has developed the NIH-Industry Pilot Program: Discovering New Therapeutic Uses for Existing Molecules to match discontinued proprietary drug candidates from pharmaceutical company partners with the best ideas from the biomedical research community for new therapeutic uses. 

In April 2011, NIH convened an NIH-Industry Roundtable that included a group of senior leaders and experts from the pharmaceutical industry, government, academia, and the non-profit sector to explore opportunities to foster new NIH-industry partnerships that facilitate drug rescue and repurposing.  Some of the challenges that were identified include:  resource implications (the time and resources for a pharmaceutical company to maintain, update, and organize their compound libraries for drug rescue and repurposing); patent considerations (off-patent compounds or compounds whose patents are close to expiring, may not be attractive to industry because the financial return and market incentives for the product may be limited); and transactional hurdles related to developing, negotiating and implementing appropriate legal agreements among the parties, including addressing such concerns as intellectual property rights and liability.  The Roundtable participants agreed that more can and should be done to increase engagement and partnerships in drug rescue and repurposing and to enhance the success of these efforts.  In response to one of the recommendations from the meeting, NCATS has developed the NIH-Industry Pilot Program:  Discovering New Therapeutic Uses for Existing Molecules. 

The goal of this NCATS program is to enable therapeutics discovery research through public-private collaborations between NIH, pharmaceutical company partners, and the biomedical research community in order to benefit public health.  NCATS is especially interested in identifying projects that address areas of unmet medical need.  NCATS plans to initiate the research program through this FOA as a pilot with a limited set of high-quality drug candidates (Agents) and pharmaceutical company partners.  If successful, the program may be expanded to include additional pharmaceutical and biotechnology company partners, Agents, and new therapeutics discovery projects. 

Objectives of the Program

The Program intends to crowd-source promising Agents with the best minds in the biomedical research community to investigate their potential for use in new therapeutic areas.  The goal of the program is to discover new therapeutic uses for Agents that are ready to move quickly into proof of concept trials in the selected patient population.  Proof of concept trials can include, as examples:  use of the Agents as stand-alone interventions, or as adjunctive interventions (if there is no evidence of drug-drug interactions with the proposed standard of care treatment).  Strategies to inform the selection of patients for proposed new uses of the Agents are of interest.

For this FOA, Phase Ib and Phase IIa trials are defined as follows.  Phase Ib trials are defined as studies usually conducted in the target patient population to establish feasibility (e.g., target engagement, pharmacodynamics/pharmacokinetics (PD/PK), optimal dosing of the Agent) for a Phase IIa proof of concept trial.  Phase IIa proof of concept trials are defined as studies designed to explore new hypotheses and to assess whether the Agent demonstrates an early signal of efficacy in the targeted patient population, typically 150 subjects or less.  In addition to clinical benefit, Phase IIa trials also include assessments of safety, tolerability, and PD/PK response of the Agent.

Through the Limited Competition for the NIH-Industry Pilot Program: Discovering New Therapeutic Uses for Existing Molecules FOAs, RFA-TR-12-004 (UH2/UH3) or RFA-TR-12-005 (UH3), investigators will have an opportunity to investigate Agents with known pharmacologic mechanisms of action for clinical efficacy in new therapeutic areas.  Investigators whose X02 pre-applications are judged to be the most meritorious will be notified of the opportunity to submit a UH2/UH3 or UH3 application under the Limited Competition FOAs.

RFA-TR-12-004 (UH2/UH3).  The UH2/UH3 is a single application in two stages.  The UH2 (Stage 1) will support milestone-driven feasibility studies to provide evidence for the biological relevance of the Agent's pharmacologic mechanism/target in pre-clinical disease models and/or in Phase Ib clinical trials for a period which varies in time from three months up to one year.  UH2 studies should establish a link between the Agent's mechanism/target and the biology of the proposed disease.  UH2 projects that have met the scientific milestones and feasibility requirements, as evaluated by NIH administrative review, will be eligible for rapid transition to the second UH3 stage.  The UH3 (Stage 2) will support milestone-driven Phase IIa proof of concept trials to assess early signals of efficacy of the Agent as a therapeutic intervention in the proposed disease population and assess its potential for further clinical development.  The project period for the UH3 stage is up to two years.

RFA-TR-12-005 (UH3).  This FOA will support UH3 Phase IIa proof of concept trials in which no initial feasibility studies are needed to establish optimal dosing or tolerability of the Agent in the selected patient population.  As in the companion FOA, the UH3 will support milestone-driven Phase IIa proof of concept trials to assess early signals of efficacy of the Agent as a therapeutic intervention in the proposed disease population and assess its potential for further clinical development.  The project period for the standalone UH3 stage is up to two years.  If invited to apply to the Limited Competition FOAs, applicants for the UH3 standalone FOA will need to justify the rationale and availability of sufficient preliminary/feasibility data to move directly into a Phase IIa proof of concept trial.

Application Process

The submission of an X02 pre-application is a necessary first step in applying for an award under RFA-TR-12-004 or RFA-TR-12-005; applicants must also read both companion FOAs prior to submitting an X02 pre-application.  Pre-applications submitted in response to this FOA will be evaluated by outside experts.  X02 applicants will receive feedback on the scientific merit, technical feasibility, administration and management plans, and overall potential of the science proposed. Investigators whose X02 pre-applications are identified as being highly meritorious and relevant to program priorities will be notified of the opportunity to submit a UH2/UH3 or UH3 application under RFA-TR-12-004 or RFA-TR-12-005, respectively.  No awards will be made for X02 pre-applications under this FOA.

Proposed studies are anticipated to vary based on the selected Agent's stage of clinical development and the strength of the evidence supporting the biological rationale for the proposed therapeutic use. For instance, it may be possible for an Agent to be taken rapidly into clinical trials, or pre-clinical studies may be needed to provide evidence for the new therapeutic use.  The applicant's team of investigators should have the appropriate pre-clinical models in place, the ability to recruit patients for the specified disease, and experience and expertise in conducting clinical trials with the proposed disease population to demonstrate feasibility of expeditiously carrying out the proposed research.

Partnership Information

NCATS has executed a Memorandum of Understanding (MOU) with each of the pharmaceutical company partners to provide a framework under which the specified proprietary Agents will be provided by these partners to the program awardees.  Template agreements have been developed for this Program:  Confidential Disclosure Agreements (CDAs) and Collaborative Research Agreements (CRAs) between the pharmaceutical company partner and the applicant.  These template agreements have been developed to streamline interactions among the parties, expediting the indications discovery efforts.  X02 applicants should consider their willingness to agree to the conditions in the appropriate CRA for the selected Agent prior to submitting a pre-application.  The investigator is strongly encouraged to consult early with their institutional technology transfer or sponsored research office in order to confirm that the terms and conditions of the CDA and CRA for the selected Agent are acceptable to their institution.  A successful UH2/UH3 or UH3 application will be contingent on the applicant’s ability to provide the NIH with documentation of access to the selected Agent and associated data needed for conducting the proposed pre-clinical studies and for filing an investigator-sponsored IND in order to conduct the proposed clinical trials (e.g., an executed CRA or letter from the pharmaceutical partner).   

Agents Available for the Program

The list of Agents and non-confidential information can be found at http://www.ncats.nih.gov/research/reengineering/rescue-repurpose/therapeutic-uses/directory.html.  The non-confidential information available for the Agents should be sufficient for applicants to prepare X02 pre-applications.

Timeline for the Program

August 14, 2012

Submission date for X02 pre-applications

September 2012 

Evaluation by outside experts for X02 pre-applications

Late September 2012 

Notification of opportunity to submit UH2/UH3 or UH3 applications

December 17, 2012

Submission date for UH2/UH3 and UH3 applications

March 2013 

Scientific merit review for UH2/UH3 and UH3 applications

May 2013

NCATS Advisory Council review of UH2/UH3 and UH3 applications

July 2013 

Earliest possible start date for awarded grants


Technical Assistance Webinar for Applicants

A technical assistance teleconference will be held on June 25, 2012 from 1:00 to 2:30 PM EST.  All prospective applicants are invited to participate.  In order to support the number of participants on the conference call, participants are encouraged to register by June 22 via e-mail at Therapeutics.Discovery@nih.gov.  Please submit questions to be addressed at this webinar on or before June 22.  Answers to the questions will be available on the NCATS Webinar page after the call. 

Please also refer to the FAQ page as an additional source of information about the program.

Section II. Award Information
Funding Instrument

The X02 pre-application submitted in response to this announcement will not result in an award using any of the traditional NIH mechanisms. A highly meritorious pre-application will result in notification of the opportunity to submit a UH2/UH3 or UH3 cooperative agreement application (i.e., RFA-TR-12-004 or RFA-TR-12-005).  For tracking purposes, each pre-application will be assigned a number that will use the X02 mechanism.

Application Types Allowed

New

The OER Glossary and the SF 424 (R&R) Application Guide provide details on these application types.

Funds Available and Anticipated Number of Awards

No awards will be made under this announcement.  Through the associated FOAs, RFA-TR-12-004 and RFA-TR-12-005, NCATS expects to commit up to $20 million in FY2013 to fund six to eight awards.

Award Budget

Not Applicable

Award Project Period

Not Applicable  

NIH grants policies as described in the NIH Grants Policy Statement will apply to the applications submitted and awards made in response to this FOA.

Section III. Eligibility Information

1. Eligible Applicants

Eligible Organizations

Higher Education Institutions

The following types of Higher Education Institutions are always encouraged to apply for NIH support as Public or Private Institutions of Higher Education:

Nonprofits Other Than Institutions of Higher Education

For-Profit Organizations

Governments

Other

Foreign Institutions

Non-domestic (non-U.S.) Entities (Foreign Institutions) are not eligible to apply.
Non-domestic (non-U.S.) components of U.S. Organizations are not eligible to apply.

Foreign components, as defined in the NIH Grants Policy Statement, are allowed.

Required Registrations

Applicant organizations must complete the following registrations as described in the SF 424 (R&R) Application Guide to be eligible to apply for or receive an award. Applicants must have a valid Dun and Bradstreet Universal Numbering System (DUNS) number in order to begin each of the following registrations.

All Program Director(s)/Principal Investigator(s) (PD(s)/PI(s)) must also work with their institutional officials to register with the eRA Commons or ensure their existing eRA Commons account is affiliated with the eRA Commons account of the applicant organization.

All registrations must be completed by the application due date. Applicant organizations are strongly encouraged to start the registration process at least 4-6 weeks prior to the application due date.

Eligible Individuals (Program Director(s)/Principal Investigator(s))

Any individual(s) with the skills, knowledge, and resources necessary to carry out the proposed research as the Program Director(s)/Principal Investigator(s) (PD(s)/PI(s)) is invited to work with his/her organization to develop an application for support. Individuals from underrepresented racial and ethnic groups as well as individuals with disabilities are always encouraged to apply for NIH support.

For institutions/organizations proposing multiple PD(s)/PI(s), visit the Multiple Program Director(s)/Principal Investigator(s) Policy and submission details in the Senior/Key Person Profile (Expanded) Component of the SF 424 (R&R) Application Guide.

For these X02 pre-applications, the PD/PI listed on the X02 pre-application must be the same PD/PI listed on a subsequent UH2/UH3 or UH3 application.   For X02 pre-applications proposing multiple PD(s)/PI(s), the contact PD/PI listed on the X02 pre-application must be the same PD/PI listed on a subsequent UH2/UH3 or UH3 application.  The contact PD/PI is strongly encouraged to continue the multiple PD(s)/PI(s) leadership identified in the X02 pre-application if notified of the opportunity to submit a UH2/UH3 or UH3 application.

NIH Intramural Research Program (IRP) investigators are eligible to apply for the X02 FOA.  IRP investigators can apply as PD(s)/PI(s), as multiple PD/PIs in conjunction with extramural investigators (http://grants.nih.gov/grants/multi_pi/index.htm), or as collaborators with extramural investigators from the biomedical research community.  For more information, see Section 6, Other Submission Requirements and Information.    

2. Cost Sharing

This FOA does not require cost sharing as defined in the NIH Grants Policy Statement.

3. Additional Information on Eligibility

Number of Applications

Applicant organizations may submit more than one application, provided that each application is scientifically distinct.

NIH will not accept any application in response to this FOA that is essentially the same as one currently pending initial peer review unless the applicant withdraws the pending application. NIH will not accept any application that is essentially the same as one already reviewed.

Section IV. Application and Submission Information

1. Requesting an Application Package

Applicants must download the SF424 (R&R) application package associated with this funding opportunity using the “Apply for Grant Electronically” button in this FOA or following the directions provided at Grants.gov.

2. Content and Form of Application Submission

It is critical that applicants follow the instructions in the SF424 (R&R) Application Guide, except where instructed in this funding opportunity announcement to do otherwise. Conformance to the requirements in the Application Guide is required and strictly enforced. Applications that are out of compliance with these instructions may be delayed or not accepted for review.

For information on Application Submission and Receipt, visit Frequently Asked Questions – Application Guide, Electronic Submission of Grant Applications.

Letter of Intent

Although a letter of intent is not required, is not binding, and does not enter into the review of a subsequent application, the information that it contains allows IC staff to estimate the potential review workload and plan the review.

By the date listed in Part 1. Overview Information, prospective applicants are asked to submit a letter of intent that includes the following information:

The letter of intent should be sent by email to:  Therapeutics.Discovery@nih.gov   

Required and Optional Components

The forms package associated with this FOA includes all applicable components, mandatory and optional.  Please note that some components marked optional in the application package are required for submission of applications for this FOA. Follow all instructions in the SF424 (R&R) Application Guide to ensure you complete all appropriate “optional” components.

Page Limitations

All page limitations described in the SF424 Application Guide and  must be followed, with the following exceptions or additional requirements:

PHS 398 Research Plan Component

All instructions in the SF424 (R&R) Application Guide must be followed, with the following additional instructions:

Specific Instructions for Preparing an X02 Pre-application

1. Applicants must read both this FOA and RFA-TR-12-004 and RFA-TR-12-005 prior to submitting an X02 pre-application.

2. In order to ensure an effective review, the application should be clearly laid out and well organized.

3. See Section 6, Other Submission Requirements and Information for additional information.

4. Pre-applications are limited to 6 pages for the Research Strategy.  Do not include a Specific Aims page.

Research Strategy

Within the 6-page Research Strategy, provide the following information. 

1. Background & Significance

a. Identify the proposed Agent from among those made available for this Program that you are interested in using, its known pharmacologic mechanism of action or target, and the new therapeutic use which will be investigated.

b. Clearly describe the patient population to be studied.

c. Provide the scientific rationale for selection of the Agent and its proposed new therapeutic use.

d. Describe the potential impact of the novel therapeutic use of the Agent for the disease or disorder.

e. Address the global burden of disease, which patients may benefit, how they may benefit, how use of the Agent might be superior to current therapy options, potential for impact on public health.  

2. Preliminary Studies

a. The Preliminary Studies section may contain data and information that validate feasibility for conducting the proposed studies. The amount of preliminary data available may depend on the level of previous investigation for the proposed Agent.

b. Examples may include but are not limited to:

3. Approach

a. Provide a plan to assess the validity of the biological hypothesis for use of the Agent in the new disease area. 

b. Provide a timeline as well as the steps required to determine whether a proposed new use of the Agent can be tested in human trials.

c. Define go/no go decision points and milestones for advancing the Agent to Phase Ib and/or Phase IIa proof of concept trials to validate the new therapeutic use.

d. Describe the design of clinical trials to assess efficacy of the Agent.

e. Describe the plan for expedited Institutional Review Board (IRB) approval of the Phase Ib and IIa proof of concept trials in the UH2 and/or UH3 stages.

f. Describe the plan for timely accrual of subjects that links to the expedited timeline for patient recruitment in the UH3 stage.

4. Administration and Management

a. Provide an operational plan for managing the pre-clinical studies and clinical trials necessary to fully develop a new use for the Agent.

b. Explain the proposed contribution of each of the administrative components to achieving the objectives of the program. 

c. Describe relevant experience and knowledge of public-private partnerships.

d. Describe the qualifications and experience of the key personnel (investigative team) involved in the UH2 and/or UH3 stages of the proposed project.

e. Describe the team's experience in the methods and approaches for design and implementation of human clinical trials and readiness to test the hypothesis.

f. Describe the team's requisite competencies and experience with clinical trials recruitment and execution.

Resource Sharing Plan

Individuals are required to comply with the instructions for the Resource Sharing Plans (Data Sharing Plan, Sharing Model Organisms, and Genome Wide Association Studies (GWAS)) as provided in the SF424 (R&R) Application Guide, with the following modification:

Appendix

Do not use the Appendix to circumvent page limits. Follow all instructions for the Appendix as described in the SF424 (R&R) Application Guide. Appendices are not allowed for this FOA and will not be accepted. Do not use the other attachments section.

Foreign Institutions

Applicants may have subcontracts with foreign institutions if they provide novel approaches or patient populations. If included, foreign (non-US) institutions must follow policies described in the NIH Grants Policy Statement, and procedures for foreign institutions described throughout the SF424 (R&R) Application Guide.

3. Submission Dates and Times

Part I. Overview Information contains information about Key Dates. Applicants are encouraged to submit in advance of the deadline to ensure they have time to make any application corrections that might be necessary for successful submission.

Organizations must submit applications via Grants.gov, the online portal to find and apply for grants across all Federal agencies. Applicants must then complete the submission process by tracking the status of the application in the eRA Commons, NIH’s electronic system for grants administration.

Applicants are responsible for viewing their application in the eRA Commons to ensure accurate and successful submission.

Information on the submission process and a definition of on-time submission are provided in the SF424 (R&R) Application Guide.

4. Intergovernmental Review (E.O. 12372)

This initiative is not subject to intergovernmental review.

5. Funding Restrictions

All NIH awards are subject to the terms and conditions, cost principles, and other considerations described in the NIH Grants Policy Statement.

Pre-award costs are allowable only as described in the NIH Grants Policy Statement.   

6. Other Submission Requirements and Information

Applications must be submitted electronically following the instructions described in the SF 424 (R&R) Application Guide.  Paper applications will not be accepted.

Applicants must complete all required registrations before the application due date. Section III. Eligibility Information contains information about registration.

For assistance with your electronic application or for more information on the electronic submission process, visit Applying Electronically.

Important reminders:
All PD(s)/PI(s) must include their eRA Commons ID in the Credential field of the Senior/Key Person Profile Component of the SF 424(R&R) Application Package. Failure to register in the Commons and to include a valid PD/PI Commons ID in the credential field will prevent the successful submission of an electronic application to NIH.

The applicant organization must ensure that the DUNS number it provides on the application is the same number used in the organization’s profile in the eRA Commons and for the Central Contractor Registration (CCR). Additional information may be found in the SF424 (R&R) Application Guide.

See more tips for avoiding common errors.

Upon receipt, applications will be evaluated for completeness by NCATS, NIH. Applications that are incomplete will not be reviewed.

At the time of submission, send an electronic copy as one pdf document on a CD to: 

Bonnie B. Dunn, Ph.D.
Scientific Review Officer
Office of Scientific Review
National Center for Advancing Translational Sciences
National Institutes of Health
6701 Democracy Blvd., Room 1066
Bethesda, MD 20892-4874
Courier service zip code 20817
301-435-0824 (direct line)
301-435-0811 (Office of Review)
Email:  dunnbo@mail.nih.gov

Additional Instructions for X02 Applicants

1. Biographical sketch. Use the sample format on the Biographical Sketch Format Page to prepare this section. Include biographical sketches of all senior/key personnel and other significant contributors. Describe the role(s) of each key participant in the project.  The Biographical Sketch may not exceed four pages per person. This four-page limit includes the table at the top of the first page.

2. Letters of Support. Include a letter from an appropriate institutional official, generally a dean or provost, documenting institutional commitment to the project, including provision of resources, space, and available faculty.  Include in the letter confirmation of the Institution’s willingness to engage in the necessary negotiations with the pharmaceutical company regarding the terms and conditions of the template CDA and CRA for the selected Agent.  A successful UH2/UH3 or UH3 application will be contingent on the applicant’s ability to provide the NIH with documentation of access to the selected Agent and associated data needed for conducting the proposed pre-clinical studies and for filing an investigator-sponsored IND in order to conduct the proposed clinical trials (e.g., an executed CRA or letter from the pharmaceutical partner).

3. If multiple institutions are involved in an application, a letter should come from each institution.

4. Human Subjects Sections. Do not include any attachments that deal with human subjects even if human subjects will be involved.

5. Vertebrate Animals. Do not use this attachment even if vertebrate animals will be involved.

6. Budget: Do not include any budget information.

7. Consortium/Contractual Arrangements. Do not use this attachment.

8.  Multiple PD(s)/PI(s) Leadership Plan: Applications designating multiple PD(s)/PI(s) must include a Multiple PD/PI Leadership Plan describing the rationale for choosing the multiple PD/PI approach, and the governance and organizational structure of the leadership team.

Additional Instructions for NIH Intramural Research Program (IRP) Applicants

1. IRP investigators can apply as PD(s)/PI(s), as multiple PD(s)/PI(s) in conjunction with extramural investigators (http://grants.nih.gov/grants/multi_pi/index.htm), or as collaborators with extramural academic or biotechnology company investigators, pending the availability of intramural funds to support the project.

2. An official letter from the Scientific Director, which indicates approval of the IRP scientist's role as PD/PI or as collaborator in the project, must be included as a letter of support in the submission of the X02 pre-application. 

3. If selected, NIH IRP scientists, in conjunction with their respective technology transfer representative, will need to contact the pharmaceutical company partner providing the selected Agent made available through this FOA to:  execute a CDA to exchange confidential information, and negotiate a PHS Cooperative Research and Development Agreement (CRADA), Clinical Trials CRADA, or other similar type of agreement, to incorporate, as appropriate, the terms of the CRA.

Post Submission Materials

Applicants are required to follow the instructions for post-submission materials, as described in NOT-OD-10-115, with the following modifications: No post-submission materials will be accepted.

Section V. Application Review Information

1. Criteria

Only the review criteria described below will be considered in the review process. As part of the NIH mission, all applications submitted to the NIH in support of biomedical and behavioral research are evaluated for scientific and technical merit through the NIH peer review system.

Overall Impact

Reviewers will provide an overall impact/priority score to reflect their assessment of the likelihood for the project to exert a sustained, powerful influence on the research field(s) involved, in consideration of the following review criteria and additional review criteria (as applicable for the project proposed).

Scored Review Criteria

Reviewers will consider each of the review criteria below in the determination of scientific merit, and give a separate score for each. An application does not need to be strong in all categories to be judged likely to have major scientific impact. For example, a project that by its nature is not innovative may be essential to advance a field.

Significance

Does the project address an important problem or a critical barrier to progress in the field? If the aims of the project are achieved, how will scientific knowledge, technical capability, and/or clinical practice be improved? How will successful completion of the aims change the concepts, methods, technologies, treatments, services, or preventative interventions that drive this field?  Does the proposed novel use of the Agent have the potential to affect pharmacologic intervention for a disease or disorder with unmet medical need? Is there a strong biological rationale for the proposed clinical trials?  

Investigator(s)    

Are the PD(s)/PI(s), collaborators, and other researchers well suited to the project? If Early Stage Investigators or New Investigators, or in the early stages of independent careers, do they have appropriate experience and training? If established, have they demonstrated an ongoing record of accomplishments that have advanced their field(s)? If the project is collaborative or multi-PD(s)/PI(s), do the investigators have complementary and integrated expertise; are their leadership approach, governance and organizational structure appropriate for the project? What are the qualifications, experience, and commitment of the personnel involved in the proposed project? Do the PD(s)/PI(s) have the scientific and organizational vision and experience to serve effectively as the Director(s) of a therapeutic discovery effort? Is there evidence of sufficient management capabilities that include fiscal administration, personnel management, planning, and budgeting? Does the investigative team have the requisite competencies and experience with clinical trials planning, recruitment and execution? Do the PD(s)/PI(s) demonstrate relevant experience and knowledge of public-private partnerships? 

Innovation

Not applicable for this pre-application because of limited information available at this stage about the proposed Agent.

Approach

Are the overall strategy, methodology, and analyses well-reasoned and appropriate to accomplish the specific aims of the project? Are potential problems, alternative strategies, and benchmarks for success presented? If the project is in the early stages of development, will the strategy establish feasibility and will particularly risky aspects be managed? Have the PD(s)/PI(s) provided an operational plan for managing the necessary collaborations between and among preclinical and clinical plan investigators, and the pharmaceutical company partner? Have the PD(s)/PI(s) discussed a plan for submitting an IND application for the proposed clinical trials, IRB approval, and patient recruitment? Is there a plan for moving expeditiously to Phase IIa proof of concept trials once a new therapeutic use has been assessed for biological validity and feasibility?

Environment

Will the scientific environment in which the work will be done contribute to the probability of success? Are the institutional support, equipment and other physical resources available to the investigators adequate for the project proposed? Will the project benefit from unique features of the scientific environment, subject populations, or collaborative arrangements? Is there evidence of institutional commitment to the project?  Is there institutional confirmation that the terms and conditions of the CDA and CRA for the selected Agent are acceptable to the technology transfer or sponsored research office (for example, through the letter documenting the Institution's willingness to enter into the necessary agreements with the pharmaceutical company partner)? Does the scientific environment indicate the potential for a multi-disciplinary approach involving teams of investigators?  

Additional Review Criteria

As applicable for the project proposed, reviewers will evaluate the following additional items while determining scientific and technical merit, and in providing an overall impact/priority score, but will not give separate scores for these items.   

Protections for Human Subjects

Not Applicable.

Inclusion of Women, Minorities, and Children 

Not Applicable.

Vertebrate Animals

Not Applicable.

Biohazards

Not Applicable.

Resubmissions

Not Applicable.

Renewals

Not Applicable.

Revisions

Not Applicable.

Additional Review Considerations

As applicable for the project proposed, reviewers will consider each of the following items, but will not give scores for these items, and should not consider them in providing an overall impact/priority score.

Applications from Foreign Organizations

Not Applicable.

Select Agent Research

Reviewers will assess the information provided in this section of the application, including 1) the Select Agent(s) to be used in the proposed research, 2) the registration status of all entities where Select Agent(s) will be used, 3) the procedures that will be used to monitor possession use and transfer of Select Agent(s), and 4) plans for appropriate biosafety, biocontainment, and security of the Select Agent(s).

Resource Sharing Plans

Not Applicable.

Budget and Period of Support

Not Applicable.

2. Review and Selection Process

Applications will be evaluated for scientific and technical merit by (an) appropriate Scientific Review Group(s), convened by NCATS, in accordance with NIH peer review policy and procedures, using the stated review criteria. Review assignments will be shown in the eRA Commons.

As part of the scientific peer review, all applications:

Applications will be assigned to NCATS with potential secondary assignment, based on the proposed disease population, to the appropriate NIH Institute or Center.

Investigators whose X02 pre-applications are identified as being highly meritorious and relevant to program priorities will be notified of the opportunity to submit a UH2/UH3 application under RFA-TR-12-004 or a UH3 application under RFA-TR-12-005.

3. Anticipated Announcement and Award Dates

After the peer review of the application is completed, the PD(s)/PI(s) will be able to access his or her Summary Statement (written critique) via the eRA Commons

Information regarding the disposition of applications is available in the NIH Grants Policy Statement.

Section VI. Award Administration Information

1. Award Notices

Not Applicable.

2. Administrative and National Policy Requirements

All NIH grant and cooperative agreement awards include the NIH Grants Policy Statement as part of the NoA. For these terms of award, see the NIH Grants Policy Statement Part II: Terms and Conditions of NIH Grant Awards, Subpart A: General  and Part II: Terms and Conditions of NIH Grant Awards, Subpart B: Terms and Conditions for Specific Types of Grants, Grantees, and Activities. More information is provided at Award Conditions and Information for NIH Grants.

Cooperative Agreement Terms and Conditions of Award

Not Applicable.

3. Reporting

When multiple years are involved, awardees will be required to submit the Non-Competing Continuation Grant Progress Report (PHS 2590) annually and financial statements as required in the NIH Grants Policy Statement.

A final progress report, invention statement, and the expenditure data portion of the Federal Financial Report are required for closeout of an award, as described in the NIH Grants Policy Statement.

The Federal Funding Accountability and Transparency Act of 2006 (Transparency Act), includes a requirement for awardees of Federal grants to report information about first-tier subawards and executive compensation under Federal assistance awards issued in FY2011 or later.  All awardees of applicable NIH grants and cooperative agreements are required to report to the Federal Subaward Reporting System (FSRS) available at www.fsrs.gov on all subawards over $25,000.  See the NIH Grants Policy Statement for additional information on this reporting requirement. 

Section VII. Agency Contacts

We encourage inquiries concerning this funding opportunity and welcome the opportunity to answer questions from potential applicants.

Application Submission Contacts

Grants.gov Customer Support (Questions regarding Grants.gov registration and submission, downloading or navigating forms)
Contact Center Phone: 800-518-4726
Email: support@grants.gov

GrantsInfo (Questions regarding application instructions and process, finding NIH grant resources)
Telephone 301-435-0714
TTY 301-451-5936
Email: GrantsInfo@nih.gov

eRA Commons Help Desk (Questions regarding eRA Commons registration, tracking application status, post submission issues)
Phone: 301-402-7469 or 866-504-9552 (Toll Free)
TTY: 301-451-5939
Email: commons@od.nih.gov

Scientific/Research Contact(s)

We encourage inquiries and welcome the opportunity to answer questions from potential applicants.

For technical questions regarding the FOA (mechanisms, partnerships, application content or scientific management of the cooperative agreements), please contact:

Christine Colvis, Ph.D.
Therapeutics Discovery Program
National Center for Advancing Translational Sciences, NIH
6701 Democracy Blvd., Democracy I, Rm. 924
Bethesda MD 20892-4874 (Regular mail)
Bethesda MD 20817 (FedEx)
Phone: See Contact List
E-mail: ccolvis@mail.nih.gov
Mailbox: Therapeutics.Discovery@nih.gov

FAQs: http://www.ncats.nih.gov/research/reengineering/rescue-repurpose/therapeutic-uses/therapeutics-faq.html

Heng Xie, M.D., M.P.H., Ph.D.
Supervisory Medical Officer
Division of Clinical Innovation
National Center for Advancing Translational Sciences, NIH
6701 Democracy Blvd., Democracy I, Rm. 908
Bethesda MD 20892-4874 (Regular mail)
Bethesda MD 20817 (FedEx)
Phone: 301-443-8063
Fax: 301-480-3661
E-mail: XieHe@mail.nih.gov

For questions related to proposed new therapeutic uses of the Agents in specific disease areas, Institute or Center contacts can be found at http://www.ncats.nih.gov/research/reengineering/rescue-repurpose/therapeutic-uses/contacts.html.

Peer Review Contact(s)

Examine your eRA Commons account for review assignment and contact information (information appears two weeks after the submission due date).

Bonnie B. Dunn, Ph.D.
Scientific Review Officer
Office of Scientific Review
National Center for Advancing Translational Sciences
National Institutes of Health
6701 Democracy Blvd., Room 1066
Bethesda, MD 20892-4874
Courier service zip code 20817
Phone: 301-435-0824 (direct line)
Phone: 301-435-0811 (Office of Review)
Email: dunnbo@mail.nih.gov

Financial/Grants Management Contact(s)

Susan Lowenthal
Office of Grants Management
National Center for Advancing Translational Sciences, NIH, DHHS
6701 Democracy Blvd, Room 1051
Bethesda, MD 20892 (20817 for express delivery)
Phone: 301-435-0848
Fax: 301-480-3777
lowenths@mail.nih.gov

Section VIII. Other Information

Recently issued trans-NIH policy notices may affect your application submission. A full list of policy notices published by NIH is provided in the NIH Guide for Grants and Contracts. All awards are subject to the terms and conditions, cost principles, and other considerations described in the NIH Grants Policy Statement.

Authority and Regulations

Awards are made under the authorization of Sections 301, 479 and 480 of the Public Health Service Act as amended (42 USC 241, 287 and 287a) and under Federal Regulations 42 CFR Part 52 and 45 CFR Parts 74 and 92.


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