and Human Services (HHS)
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Participating Organization(s) |
National Institutes of Health (NIH) |
National Institute of Arthritis and Musculoskeletal and
Skin Diseases (NIAMS) |
|
Funding Opportunity Title |
Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers (U54) |
Activity Code |
U54 Specialized Center- Cooperative Agreements |
Announcement Type |
Reissue of RFA-AR-13-012 |
Related Notices |
None |
Funding Opportunity Announcement (FOA) Number |
RFA-AR-13-021 |
Companion Funding Opportunity |
None |
Catalog of Federal Domestic Assistance (CFDA) Number(s) |
93.865, 93.846, 93.853 |
Funding Opportunity Purpose |
The purpose of this Funding Opportunity Announcement (FOA) is to publicize the re-competition of Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers (MDCRCs). These Centers promote collaborative basic, translational and clinical research and provide important resources that can be used by the national muscular dystrophy research communities. The centers also provide an outstanding environment for the training and career development of new scientists electing to pursue careers conducting research in high priority areas of muscular dystrophy. Center investigators are expected to participate in important community outreach and education efforts to increase awareness and convey the importance and implications of their research activities to the general public. |
Posted Date |
July 11, 2013 |
Letter of Intent Due Date(s) |
August 13, 2013 |
Application Due Date(s) |
September 13, 2013 |
AIDS Application Due Date(s) |
Not Applicable |
Scientific Merit Review |
October/November, 2013 |
Advisory Council Review |
January, 2014 |
Earliest Start Date |
March, 2014 |
Expiration Date |
September 14, 2013 |
Due Dates for E.O. 12372 |
Not Applicable |
Required Application Instructions
It is critical that applicants follow the instructions in the PHS 398 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. While some links are provided, 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.
Looking ahead: NIH is committed to transitioning all grant programs to electronic submission using the SF424 Research and Related (R&R) format and is currently investigating solutions that will accommodate NIH’s multi-project programs. NIH will announce plans to transition the remaining programs in the NIH Guide to Grants and Contracts and on NIH’s Applying Electronically website.
Part 1. Overview Information
Part 2. Full Text of 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
The purpose of this Funding Opportunity Announcement (FOA) is to solicit applications for Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers (MDCRCs). These Centers promote collaborative basic, translational and clinical research and provide important resources that can be used by the national muscular dystrophy research communities. The Centers also provide an outstanding environment for the training and career development of new scientists electing to pursue careers conducting research in high priority areas of muscular dystrophy. Finally, Center investigators are expected to participate in important community outreach and education efforts to increase awareness and convey the importance and implications of their research activities to the general public.
The Muscular Dystrophy Community Assistance, Research, and Education Amendments of 2001 (the MD-CARE Act, Public Law 107-84) specified a number of provisions for expanding and intensifying research on muscular dystrophy. One provision of the MD-CARE Act was that the NIH establish centers of excellence for research on muscular dystrophy. The MDCRCs program was subsequently developed in honor of Senator Paul D. Wellstone, a champion of muscular dystrophy research. In the years following the MD-CARE Act, the NIH sponsored Requests for Applications in 2003, 2004, 2008 and 2009 and has supported six active MDCRCs.
Reauthorization of the MD-CARE Act in 2008 codified the MDCRCs as the Paul D. Wellstone MDCRCs. MDCRCs funded through this program have contributed toward the goal of improving the detection, diagnosis, and treatment of the muscular dystrophies.
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Institute of Neurological Disorders and Stroke (NINDS) are committed to continuing and enhancing the tradition of scientific excellence that has been fostered in the MDCRC program.
Muscular dystrophy refers to a group of hereditary, progressive degenerative disorders causing weakness of the skeletal or voluntary muscles. There are many different forms of muscular dystrophy, which differ in their age of onset, penetrance, severity, and pattern of muscles affected. Most types of muscular dystrophy are not simply muscle disorders, but rather multi-system disorders with manifestations in a variety of body systems, including the heart, gastrointestinal system, endocrine glands, skin, eyes, brain, and other organ systems. The major forms of muscular dystrophy include congenital, distal, Duchenne/Becker, Emery-Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and oculopharyngeal. Although some forms first become apparent in early childhood, others may not appear until middle age or later, but all have a significant clinical, economic, and psychosocial burden of disease.
While several therapeutic development strategies have shown promise in cell-based or animal models, and some early stage clinical trials in humans have begun, currently available therapies are very limited in their ability to change the clinical course of the diseases. Moreover, there is currently no consensus as to which of several potential therapeutic strategies, or combination of strategies, may ultimately prove successful in reducing patient morbidity and mortality. Symptomatic treatment, though not able to stop disease progression, may improve the quality of life for some individuals. Recent advances in genetics, pathogenic mechanisms, therapeutic technology, and patient diagnostics do provide compelling opportunities for advancing translational and clinical science in the muscular dystrophies. In addition to the need for new disease or symptom modifying therapies, there are gaps in understanding how the muscular dystrophies affect the lives of patients and their families. Filling these gaps in understanding may lead to novel strategies to lessen the burden of these diseases. For examples, better understanding of patient secondary conditions and neuropsychological and neurobehavioral profiles that affect quality of life and caregiver burden could lead to effective interventions.
The Action Plan for the Muscular Dystrophies (http://www.ninds.nih.gov/find_people/groups/mdcc/MDCC_Action_Plan.doc ), approved and released in January 2006 by the interagency Muscular Dystrophy Coordinating Committee (http://www.ninds.nih.gov/find_people/groups/mdcc/index.htm ), is a consensus document with input from patients, advocacy groups, basic scientists, clinicians, and Federal agencies that highlights many of these scientific opportunities and the need for broad cooperation in seeking effective treatments for the muscular dystrophies.
NIAMS, NICHD and NINDS seek to further develop the MDCRC program to further foster the translation of scientific findings and technological developments into the clinical research setting, and also to bring observations and findings from the clinical setting into the laboratory for more in-depth analysis and hypothesis testing. Under the FOA, each Center may contain a mixture of basic, translational, or clinical research, as long as efforts are directed toward the steps required for the development of therapies, which include drugs, biologics, exercise, behavioral and other interventions. The steps required for development of therapies may include, but are not limited to:
MDCRC applicants are encouraged to consider projects that address the high priority areas described in the Action Plan for the Muscular Dystrophies (http://www.ninds.nih.gov/find_people/groups/mdcc/MDCC_Action_Plan.doc ).
Applicants seeking support for projects that are stand-alone, single-component basic, translational, clinical studies or trials in muscular dystrophy should contact the Scientific/Research Contact listed in Section VII. Agency Contacts below for guidance on other, more appropriate funding opportunities.
Collectively and in cooperation with the NIH, the MDCRCs form part of a coordinated national program. Applicants are expected to emphasize new ideas, novel approaches, and state-of-the-art technologies to address the needs for effective treatments and other strategies to improve the lives of muscular dystrophy patients. Multidisciplinary collaborative efforts, in particular those involving basic scientists and clinicians with appropriate expertise, are required in each MDCRC. MDCRC applicants must also propose resource core facilities and training activities that will enhance muscular dystrophy research on a national or international level.
The organizational structure of the proposed MDCRC should facilitate the flow of new scientific findings and technologies into translational and clinical research. Each center may contain any combination of basic, translational, or clinical studies research with an emphasis placed upon moving the research field forward toward novel or improved therapies for any of the muscular dystrophies. Each center must include clinical research as defined in the PHS398 Supplemental Instructions Part II (http://grants1.nih.gov/grants/funding/phs398/phs398.html ). This includes patient-oriented research, for which an investigator directly interacts with human subjects, epidemiologic, behavioral studies, outcomes and health services research. Studies are not considered clinical research if they involve only human specimens without information identifying the subjects. Although guidance is provided in this FOA for interventional clinical trials in MDCRCs, clinical trials using investigational drugs or biologics are NOT a required component of an MDCRC.
The minimal structural requirements of a Wellstone MDCRC under this FOA are:
Projects
Each of the proposed research projects should address problems that require a substantial collaborative research effort to solve, and are best suited for a Center environment rather than a stand-alone grant. Collectively, the projects should involve synergistic teams of researchers with complementary expertise such as basic and clinical, skeletal muscle and other organ systems, primary data collection and bioinformatics, etc. Collaborations should be arranged to bring the best expertise to bear on a problem, whether the proposed collaborations are all on-site or utilize consortium agreements with off-site investigators at existing MDCRCs or off-site investigators not affiliated with an MDCRC. Although a clinical project is required, this need not be a clinical intervention trial. See section IV.2 for additional guidance on projects that propose a clinical trial.
Leadership
The Center Director and Co-Director should develop and maintain a center environment that fosters traditional and novel approaches to multi-disciplinary research collaborations and training. The Center Director and Co-Director cannot serve as Program Director/Principal Investigator of a project in another active MDCRC award.
Administrative Core
The Administrative Core should provide for the integration and management of activities within the MDCRC. Each Center should expect to form an external Center Advisory Committee (CAC) with scientific, clinical and patient advocate representation, composed of at least five members. The composition of the CAC will require NIH approval. The CAC should meet in-person or electronically approximately once a year, beginning in the first or second year of the Center award. Funded MDCRCs are expected to utilize the Administrative Core to establish and maintain a website to communicate the Center missions and the availability of training opportunities and Scientific Research Resource Core services. Applicants are encouraged to form the strongest teams to address the research questions, regardless of geography.
Shared Resource Core
The shared Scientific Research Resource Core should be designed and managed to support the research of the MDCRC, as well as serving as a resource for the national and perhaps international muscular dystrophy research community. Applicants may wish to consult the Action Plan for the Muscular Dystrophies (http://www.ninds.nih.gov/find_people/groups/mdcc/MDCC_Action_Plan.doc ) for consensus statements on infrastructure needs of the muscular dystrophy research community.
Investigator Development and Patient Outreach Core
As nationally recognized centers of excellence in muscular dystrophy, the MDCRCs are expected to play a leadership role in training new researchers for the muscular dystrophy field, contributing to the development of future research leaders and educating the patient and lay communities regarding research activities. Each center should include plans for a Investigator Development and Patient Outreach Core to provide support for pilot and feasibility projects conducted by mentored investigators, enhance the institution's environment for training of students, fellows and early-stage investigators in muscular dystrophy research, and to engage the patient and lay community in educational and research activities. Utilization and adaptation of existing training programs are encouraged. By conducting pilot and feasibility projects, the mentored researchers will become better prepared for careers as future independent investigators and leaders in the muscular dystrophy research community. This core may propose activities that enhance the training environment through a seminar program, retreats for presentation of trainee research, journal clubs or other activities that contribute to the preparation of junior investigators for careers in muscular dystrophy research. Students, fellows and early-stage investigators should be exposed to translational and clinical research whenever possible. Exposure to research at other Wellstone Centers is also encouraged. In order to promote awareness of muscular dystrophy research and the Wellstone Centers program in the patient and lay communities, this core should develop educational activities or materials such as seminars, web-based information, or lab tours involving patients and their families interacting with junior and senior investigators. Participation of patient advocacy groups in the planning and conduct of education and outreach activities is encouraged.
Recipients of MDCRC awards will become part of a national program in muscular dystrophy and will be expected to participate in MDCRC activities, including meetings of the Steering Committee (composed of MDCRC Directors and Co-Directors, and NIH staff) and an annual MDCRC meeting that rotates among the MDCRC sites.
Funding Instrument |
Cooperative Agreement: A support mechanism used when there will be substantial Federal scientific or programmatic involvement. Substantial involvement means that, after award, NIH staff will assist, guide, coordinate, or participate in project activities. |
Application Types Allowed |
New |
Funds Available and Anticipated Number of Awards |
The total commitment by the participating Institutes to this FOA is approximately $1.5M in FY 2014 to make no more than one award. |
Award Budget |
Applicants may request up to $1M in direct costs/year (exclusive of facilities and administrative costs of subcontracts with collaborating organizations). |
Award Project Period |
The maximum project period is 4 years. |
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.
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
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.
Applicant organizations must complete the following registrations as described in the PHS 398 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 Directors/Principal Investigators (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 least6 weeks prior to the application due date.
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.
Multiple PDs/PIs are not allowed in this FOA.
This FOA does not require cost sharing as defined in the NIH Grants Policy Statement.
Applicant organizations may submit more than one application, provided that each application is scientifically distinct.
NIH will not accept any application that is essentially the same as one already reviewed within the past thirty-seven months (as described in the NIH Grants Policy Statement), except for submission:
Applicants are required to prepare applications according to the current PHS 398 application forms in accordance with the PHS 398 Application Guide.
It is critical that applicants follow the instructions in the PHS 398 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.
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 to:
Glen H. Nuckolls, Ph.D.
Director, Muscle Disorders and Therapies Program
Musculoskeletal Diseases Branch
National Institute of Arthritis and Musculoskeletal
and Skin Diseases (NIAMS)
One Democracy Plaza
6701 Democracy Boulevard, Suite 800
Bethesda, MD 20892-4872
Telephone: 301-594-4974
Email: nuckollg@mail.nih.gov
Applications must be prepared using the PHS 398 research grant application forms and instructions for preparing a research grant application. Submit a signed, typewritten original of the application, including the checklist, and three signed photocopies in one package to:
Center for Scientific Review
National Institutes of Health
6701 Rockledge Drive, Room 1040, MSC 7710
Bethesda, MD 20892-7710 (U.S. Postal Service Express or regular mail)
Bethesda, MD 20817 (for express/courier service; non-USPS service)
At the time of submission, two additional paper copies of the application as well as an electronic version (preferable in pdf format) and all copies of the Appendix files must be sent to:
Kan Ma, Ph.D.
Scientific Review Officer
National Institute of Arthritis, Musculoskeletal and
Skin Diseases
National Institutes of Health, DHHS
One Democracy Plaza, Suite 800
6701 Democracy Blvd., MSC 4872
Bethesda, MD 20892
Telephone: 301-451-4838
Fax: 301-402-2406
Email: mak2@mail.nih.gov
All page limitations described in the PHS 398 Application Guide and the Table of Page Limits must be followed, in addition to the following page limitations to the Research Strategy section of each component of the application.
The following section supplements the instructions found in the PHS398 Application Guide, and should be used for preparing a multi-component application.
The application should consist of the following components:
All instructions in the PHS398 Application Guide must be followed, with the following additional instructions, as noted.
Face Page (Overall)
All instructions in the PHS 398 Application Guide must be followed.
Description, Project/Performance Sites, Senior/Key Personnel, Other Significant Contributors, Human Embryonic Stem Cells (Overall)
All instructions in the PHS 398 Application Guide must be followed, with the following additional instructions:
Senior/Key Personnel: Each applicant institution will name an MDCRC Center Director (Program Director/Principal Investigator) who will be the key figure in the administration, management, and coordination of the Center grant. The Director will be responsible for the organization and operation of the center. The Director should be a recognized scientific leader experienced in the field of muscular dystrophy research and must be able to coordinate, integrate, and provide guidance in the establishment of research programs. A Co-Director should also be named who will be involved in the administrative and scientific efforts of the Center.
Table of Contents (Overall)
All instructions in the PHS 398 Application Guide must be followed.
Detailed Budget for Initial Budget Period (Overall)
All instructions in the PHS 398 Application Guide must be followed, with the following additional instructions:
Applicants may request up to $1M in direct costs/year (exclusive of facilities and administrative costs of subcontracts with collaborating organizations).
Budget for Entire Proposed Period of Support (Overall)
All instructions in the PHS 398 Application Guide must be followed.
Biographical Sketch (Overall)
All instructions in the PHS 398 Application Guide must be followed.
Resources (Overall)
All instructions in the PHS 398 Application Guide must be followed.
Research Plan (Overall)
All instructions in the PHS 398 Application Guide must be followed, with the following additional instructions:
Research Strategy: The Overall Research Plan should describe the major theme of the Center, its goals and objectives, background information and the overall importance of the research to the development of therapies for the muscular dystrophies. Describe the rationale for the total proposed program. Explain the strategy for achieving the goals defined for the overall program and how each research project and core relates to that strategy. A successful Center grant application will include a well-integrated research plan that clearly shows how the proposed projects and cores will foster preclinical and or clinical development of novel therapeutics for muscular dystrophy. The program should be viewed as interrelated research projects, each of which is not only individually scientifically meritorious but is also complementary to the other projects and related to the overall theme developed for the Center. Provide justification in the application that: (a) the proposed projects are such that they require an intensive collaborative effort to succeed and (b) that key personnel will collaborate effectively.
Describe the roles of the Director and Co-Director and their qualifications as leaders of the Center.
Describe the organizational structure of the MDCRC including the components of the Center and any existing resources of the institution(s) that will be leveraged by the Center. Also describe any connections between the proposed Center and other organizations such as patient advocacy groups or industry partners. Explain how different components of the organization, including key personnel, will interact, why they are essential to accomplishing the overall goal of the research, and how combined resources create capabilities that are more than the sum of the parts.
For renewal applications, document achievement of the goals of the prior funding period.
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 PHS 398 Application Guide.
All instructions in the PHS398 Application Guide must be followed, with the following additional instructions, as noted.
Face Page (Administrative Core)
All instructions in the PHS 398 Application Guide must be followed.
Description, Project/Performance Sites, Senior/Key Personnel, Other Significant Contributors, Human Embryonic Stem Cells (Administrative Core)
All instructions in the PHS 398 Application Guide must be followed.
Table of Contents (Administrative Core)
All instructions in the PHS 398 Application Guide must be followed.
Detailed Budget for Initial Budget Period (Administrative Core)
All instructions in the PHS 398 Application Guide must be followed, with the following additional instructions:
The Administrative Core of the MDCRC application should include up to $5,000 per year direct costs for travel of the Director, Co-Director, and other Center investigators to the annual meeting of the Wellstone Network, and for visits of Center investigators or trainees to other MDCRCs or other collaborative sites to exchange scientific ideas, to plan multi-Center research projects, or to receive training in specialized techniques.
The Center Director and Co-Director should each have a minimum commitment of 20% (2.4 calendar months) effort to the MDCRC, which should include appropriate effort to the Administrative Core as well as other cores and/or projects.
Budget for Entire Proposed Period of Support (Administrative Core)
All instructions in the PHS 398 Application Guide must be followed.
Biographical Sketch (Administrative Core)
All instructions in the PHS 398 Application Guide must be followed.
Resources (Administrative Core)
All instructions in the PHS 398 Application Guide must be followed.
Research Plan (Administrative Core)
All instructions in the PHS 398 Application Guide must be followed, with the following additional instructions:
Research Strategy: The Administrative Core will be responsible for the management and administration of the overall Center. This section of the application should describe the strategies and processes that will be used to manage the Center and achieve the goals. This Core, led by the Center Director, will provide oversight for the projects and cores, promote coordination and collaboration within the Center and with investigators and organizations outside the Center. A narrative description should be provided that includes the planning and coordination of research activities; the integration of cross-disciplinary research; the oversight of fiscal and resource management; and the maintenance of ongoing communication. Indicate who will be responsible for each of these activities. Applicants should specify appropriate administrative/business management staff and oversight mechanisms by the Center Director, Center Co-Director, and a local Executive Committee.
When multiple performance sites are planned, the Administrative Core should include leadership and communication plans adequate to manage the multiple sites.
The Administrative Core should establish an Executive Committee, composed of members of the Center and a Center Advisory Committee, composed of people outside the Center. Describe how the Center Advisory Committee will contribute to oversight of the research projects, core facilities and training environment of the Center. The Center Advisory Committee should meet approximately once a year and brief reports of the proceedings of the meeting and recommendations of the committee should be included in the progress reports of the Center. Describe plans as to how you will work with the Center Advisory Committee, but only name the members of the Advisory Committee if applying for the renewal of a Center.
In order to assure active collaboration with other Centers, the MDCRC Director, Co-Director, and other staff should attend annual meetings of the MDCRC Network, contribute to the coordination of effort, and/or help to refine and standardize operating procedures among the Centers.
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 PHS 398 Application Guide
The Administrative Core should provide oversight of the resource sharing plans of the Center. A competitive application would be expected to provide a plan regarding the timely sharing of specimens, cells, animal models and redacted data generated with support from this award with other qualified research scientists, both within and outside the MDCRC network, and ensuring that such data are HIPPA compliant. Applicants should consider describing plans for tracking requests for resources and monitoring the timely accomplishment of the activities described in the resource sharing plans.
All instructions in the PHS398 Application Guide must be followed, with the following additional instructions, as noted.
Face Page (Investigator Development and Patient Outreach Core)
All instructions in the PHS 398 Application Guide must be followed.
Description, Project/Performance Sites, Senior/Key Personnel, Other Significant Contributors, Human Embryonic Stem Cells (Investigator Development and Patient Outreach Core)
All instructions in the PHS 398 Application Guide must be followed.
Table of Contents (Investigator Development and Patient Outreach Core)
All instructions in the PHS 398 Application Guide must be followed.
Detailed Budget for Initial Budget Period (Investigator Development and Patient Outreach Core)
All instructions in the PHS 398 Application Guide must be followed, with the following additional instructions:
The Investigator Development and Patient Outreach Core budget should include 10 percent of the total Center budget (up to $100,000/year). These funds should support three different activities of the core including 1) support for pilot and feasibility projects conducted by mentored researchers, 2) enhancements to the training environment of the center, and 3) outreach and research education activities for patients and their families. The majority of these funds should support pilot and feasibility projects.
Budget for Entire Proposed Period of Support (Investigator Development and Patient Outreach Core)
All instructions in the PHS 398 Application Guide must be followed.
Biographical Sketch (Investigator Development and Patient Outreach Core)
All instructions in the PHS 398 Application Guide must be followed.
Resources (Investigator Development and Patient Outreach Core)
All instructions in the PHS 398 Application Guide must be followed.
Research Plan (Investigator Development and Patient Outreach Core)
All instructions in the PHS 398 Application Guide must be followed, with the following additional instructions:
Research Strategy: The Investigator Development and Patient Outreach Core should enhance the environment within the Center for recruiting and training the next generation of muscular dystrophy researchers. Included in this core budget should be support for several pilot and feasibility projects per year that are conducted by mentored researchers and relate to the theme of the Center. All pilot and feasibility projects must be focused on advancing understanding or treatment of one or more of the muscular dystrophies. Describe the processes that will be used by the Center to publicize these research opportunities, solicit applications, involve the Center Advisory Committee in evaluation of the applications, select among the candidates and monitor their progress. Award of support for pilot and feasibility projects will require NIH approval and documentation of the merits of the proposed research, qualifications of the investigator and the recommendation of the CAC. Individuals from underrepresented racial and ethnic groups, individuals with disabilities, and individuals from disadvantaged backgrounds are always encouraged to apply to such programs.
Describe the internal institutional plans and procedures to ensure that all pilot and feasibility projects supported from this award will comply fully with all applicable Federal regulations, policies, and guidelines for research involving human subjects, including the evaluation of risks and protections in project applications, appropriate ethical oversight and funded projects, and plans for data and safety monitoring for clinical trials, if applicable.
Describe activities through which this core will enhance the training environment of the Center. Examples of such activities may include a seminar program, retreats for presentation of trainee research, journal clubs or other activities that contribute to the preparation of junior investigators for careers in muscular dystrophy research. Since the other Wellstone Centers will also be engaged in activity to enhance training, applicants should consider ways to coordinate and develop synergy with other Centers, which can be implemented after the Center grant is awarded.
Describe plans for activities that educate and/or engage patients and their advocates in the research conducted by the Center and the field of muscular dystrophy research in general.
For renewal applications, document the past performance of the Training and Education Core, including the progress of predoctoral and postdoctoral researchers supported, enhancements to the training environment of the center and outreach activities to the patient communities.
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 PHS 398 Application Guide.
All instructions in the PHS398 Application Guide must be followed, with the following additional instructions, as noted.
Face Page (Shared Scientific Resource Core)
All instructions in the PHS 398 Application Guide must be followed.
Description, Project/Performance Sites, Senior/Key Personnel, Other Significant Contributors, Human Embryonic Stem Cells (Shared Scientific Resource Core)
All instructions in the PHS 398 Application Guide must be followed.
Table of Contents (Shared Scientific Resource Core)
All instructions in the PHS 398 Application Guide must be followed.
Detailed Budget for Initial Budget Period (Shared Scientific Resource Core)
All instructions in the PHS 398 Application Guide must be followed.
Budget for Entire Proposed Period of Support (Shared Scientific Resource Core)
All instructions in the PHS 398 Application Guide must be followed.
Biographical Sketch (Shared Scientific Resource Core)
All instructions in the PHS 398 Application Guide must be followed.
Resources (Shared Scientific Resource Core)
All instructions in the PHS 398 Application Guide must be followed.
Research Plan (Shared Scientific Resource Core)
All instructions in the PHS 398 Application Guide must be followed, with the following additional instructions:
Research Strategy: Describe the function of the core as a resource to the program. This section must clearly present the facilities, techniques, and professional skills that the core will provide. A core is principally designed as a service or resource component; it would be highly unusual to include research in a core (a possible exception would be methodology development). Please contact the Institute staff if you require guidance on this issue.
Describe the role of the core as a resource to the program as a whole. Discuss ways in which these centralized services will produce an economy of effort and/or savings in overall costs compared to their inclusion as part of each project in the program. To aid in the review of your application it is recommended that you prepare in tabular form information concerning the research projects that each facility core unit would serve and the proportion of the cost of the facility core unit associated with each research project involved.
Cores are seldom created specifically for the MDCRC and more often already exist in some form prior to the application. When proposing support for an already existing core, describe how the MDCRC award would enhance the resources or services already available through new innovation and technology development, expanded availability, increased throughput, etc.
Each Center should contain a scientific core that provides services and/or resources that are shared with other investigators nationally and perhaps internationally. These resources could be reagents, specimens, services or technical expertise that will help to accelerate progress of multiple projects toward the development of therapies or other strategies for improving the lives of patients with muscular dystrophy. Describe how this shared core will meet the needs of the national/international muscular dystrophy research community. If cores providing similar resources are already available, explain the need for this additional core.
Additional cores may be proposed if they are needed to advance the local research effort and if they fit within the budget limits described elsewhere in this FOA. For each scientific core, the applicant should identify projects that will depend on the services and/or resources proposed. Projects outside the Center that would use the core should be described in general terms and investigators outside the Center should not be contacted, as this would lead to conflicts of interest during peer review.
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 PHS 398 Application Guide.
All instructions in the PHS398 Application Guide must be followed, with the following additional instructions, as noted.
Face Page (Project)
All instructions in the PHS 398 Application Guide must be followed.
Description, Project/Performance Sites, Senior/Key Personnel, Other Significant Contributors, Human Embryonic Stem Cells (Project)
All instructions in the PHS 398 Application Guide must be followed.
Table of Contents (Project)
All instructions in the PHS 398 Application Guide must be followed.
Detailed Budget for Initial Budget Period (Project)
All instructions in the PHS 398 Application Guide must be followed.
Budget for Entire Proposed Period of Support (Project)
All instructions in the PHS 398 Application Guide must be followed.
Biographical Sketch (Project)
All instructions in the PHS 398 Application Guide must be followed.
Resources (Project)
All instructions in the PHS 398 Application Guide must be followed.
Research Plan (Project)
All instructions in the PHS 398 Application Guide must be followed, with the following additional instructions:
Research Strategy: Each project should clearly state its overall objective and explain its relevance to the central theme of the Center. In addition, an explanation should be included describing how the project relates to and both complements and enhances the other research projects and cores of the program. Why the project is best suited to be carried out in the Center environment should be highlighted. Specify the overall biomedical significance of the work proposed. Specify the niches filled by each project in advancing treatments for muscular dystrophy.
Each MDCRC should contain at least one clinical study, but this study is not required to be an interventional clinical trial. Options for clinical studies include but are not restricted to evaluation of natural history, development of biomarkers, or development/validation of endpoint measures. Knowledge from such clinical studies is essential to direct subsequent clinical trials and can be invaluable for the muscular dystrophy field.
For each project that includes an interventional clinical trial, the description of the significance should support the potential value and feasibility of successfully completing the study within the term of the grant, including preclinical rationale. The preclinical rationale should provide evidence that the rigor of preclinical efficacy studies and the level of effect of the agent are both sufficient to warrant clinical testing of the agent (see http://grants.nih.gov/grants/guide/notice-files/NOT-NS-11-023.html ). The approach section should describe evidence that regulatory requirements will be met in a timely manner, evidence of drug/biologic availability for use in a trial, and agreement of all participating clinical/corporate partners. Clinical trials involving the testing of new investigational therapeutics, new indications for FDA-approved drugs, or other medical interventions under a research protocol should be performed under an IND, unless otherwise agreed upon by the FDA. If not exempt, the applicant must provide the NIH with the name and organization of the IND/IDE holder, the date the IND/IDE was filed with the FDA, the FDA IND/IDE number, and any comments from the FDA regarding this protocol. Studies will not be funded unless necessary regulatory approval has first been obtained; regulatory approval at the time of application is preferred. In studies where a pharmaceutical/biotechnology company is providing the study agent, a written agreement by a company official affirming this arrangement must be provided with the application as a letter of support. It is strongly encouraged that clinical studies utilize established Common Data Elements (CDEs; see http://www.commondataelements.ninds.nih.gov/#page=Default ) to ensure comparability with other clinical trials in muscular dystrophy.
Because of the budget limitation of the overall Center and requirements for other components, any interventional clinical trials proposed as part of the MDCRCs are likely to be phase 0/exploratory IND, phase 1, or early stage proof of concept trials. Any clinical trial proposed within MDCRCs should be designed to validate the therapeutic target or candidate therapeutic (phase 0 trial) or to provide specific data that will be necessary to design a subsequent definitive efficacy trial (phase 1 or early stage proof of concept trial). The proposed study must address questions that, when answered, will optimize the design of the eventual definitive clinical trial rather than simply address the clinical question with lower power. Underpowered efficacy trials that are unlikely to advance the development of a candidate therapeutic should be avoided. Examples of relevant clinical research include, but are not limited to, the following:
Protection of Human Subjects: Subjects who participate in MDCRC clinical research projects should be fully informed, using appropriate consent procedures. The consent form for funded projects should specifically address the following: (1) disclosure that biological materials and clinical data will be distributed to other researchers; (2) assurance that such data will be de-identified and stored and maintained without personal identifiers; (3) disclosure that analyses of these data will be conducted by other scientists currently not included within the current research team, potentially including those with commercial interests; (4) that the data collected by the researchers may be used to study their specific disorder as well as other disorders.
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 PHS 398 Application Guide.
Do not use the Appendix to circumvent page limits. Follow all instructions for the Appendix (please note all format requirements) as described in the PHS 398 Application Guide.
Part I. Overview Information contains information about Key Dates.
Information on the process of receipt and determining if
your application is considered on-time is described in detail in the PHS 398
Application Guide.
Applicants may track the status of the application in the eRA Commons, NIH’s electronic system for grants
administration.
This initiative is not subject to intergovernmental review.
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.
Applications must be received on or before the due dates in Part I. Overview Information. If an
application is received after that date, it will not be reviewed.
Upon receipt, applications will be evaluated for
completeness by the Center for Scientific Review and responsiveness by components of participating organizations,
NIH. Applications that are incomplete and/or nonresponsive will not be
reviewed.
Applicants are required to follow the instructions for post-submission materials, as described in NOT-OD-10-115.
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.
For this particular announcement, note the following:
Centers should be designed to include the following components: two or more scientific project(s), an Administrative Core, a Shared Scientific Research Resource Core with national impact, and a Research Investigator Development and Patient Outreach Core. Applications may include additional core facilities within the overall budget cap. After the review of the individual components, an overall impact/priority score will be assigned to the center application. The overall score will reflect a) the scientific merits of the research project(s), b) the overall effectiveness and adequacy of core resources and facilities, c) the qualifications of the Center Director and Co-Director, d) the quality of the plans for management and oversight of the Center, e) the institutional commitment, and f) the synergy among the components and overall impact of the Center. The overall score for the center application may be higher or lower than the average of the individual components based on the assessment of whether the whole is greater than the sum of its parts.
Reviewers will provide an overall impact score to reflect their assessment of the likelihood for the Center 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 Center proposed).
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 Center that by its nature is not innovative may be essential to advance a field.
Significance
Does the Center address an important problem or a critical barrier to progress in the field? If the aims of the Center 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?
Investigator(s)
Are the PD(s)/PI(s), collaborators, and other researchers well suited to the Center? 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/PI, do the investigators have complementary and integrated expertise; are their leadership approach, governance and organizational structure appropriate for the project?
Are the qualifications, experience, and commitment of the MDCRC Director and Co-Director adequate to lead the Center program and are they devoting sufficient time/effort to achieve Center goals? Does the Center Director have sufficient authority and credibility within the institution and broader community as a base for serving a national leadership role in muscular dystrophy? Does the Center Co-Director have appropriate complementary and integrated experience/expertise to help lead a multi-disciplinary center effort? Are there adequate plans for interaction among Center personnel and any off-site investigators?
Innovation
Does the application challenge and seek to shift current research or clinical practice paradigms by utilizing novel theoretical concepts, approaches or methodologies, instrumentation, or interventions? Are the concepts, approaches or methodologies, instrumentation, or interventions novel to one field of research or novel in a broad sense? Is a refinement, improvement, or new application of theoretical concepts, approaches or methodologies, instrumentation, or interventions proposed?
Approach
Are the overall strategy, methodology, and analyses
well-reasoned and appropriate to accomplish the specific aims of the Center?
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?
If the Center involves clinical research, are the plans for 1) protection of
human subjects from research risks, and 2) inclusion of minorities and members
of both sexes/genders, as well as the inclusion of children, justified in terms
of the scientific goals and research strategy proposed?
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 tangible institutional commitment to the establishment and growth of the MDCRC? Will the institution provide incentives and rewards to promote the mission of team-based research? Is there substantial institutional commitment to tenured faculty positions, dedicated space and other resources, and sufficient time release to allow the investigators to pursue the goals of the MDCRC? Is the physical distribution of Center investigators and core resources conducive to the synergy necessary for a successful MDCRC? Will existing NIH-supported core facilities be shared with the MDCRC? Do the institutional administration and environment provide opportunities for Center growth? If applicable, are there sufficient commitment and support on the part of institutions associated with the MDCRC through consortium agreements?
As applicable for the Center proposed, reviewers will evaluate the following additional items while determining scientific and technical merit, and in providing an overall impact score, but will not give separate scores for these items.
Center Justification
Is there strong justification that this will be a successful Center, with each of the projects and cores not only individually scientifically meritorious but also complementary to the other components, and related to the overall theme developed for the Center? Is there justification in the application that: (a) the proposed projects are such that they require an intensive collaborative effort to succeed and (b) that key personnel will collaborate effectively? Are there appropriate plans for the Center to collaborate and otherwise contribute to the national MDCRC program, through participating in the annual meeting, workshops, training, collaborative efforts, or other MDCRC-wide activities?
Will the Center be effective in significantly accelerating progress toward effective treatments or other improvements in the lives of muscular dystrophy patients through coordinated and synergistic research and infrastructure activities? Are the aggregate quality and level of innovation of the Center’s research base sufficient and are the proposed research projects highly relevant to the overall goal of advancing therapeutic development in muscular dystrophy?
For research that involves human subjects but does not involve one of the six categories of research that are exempt under 45 CFR Part 46, the committee will evaluate the justification for involvement of human subjects and the proposed protections from research risk relating to their participation according to the following five review criteria: 1) risk to subjects, 2) adequacy of protection against risks, 3) potential benefits to the subjects and others, 4) importance of the knowledge to be gained, and 5) data and safety monitoring for clinical trials.
For research that involves human subjects and meets the criteria for one or more of the six categories of research that are exempt under 45 CFR Part 46, the committee will evaluate: 1) the justification for the exemption, 2) human subjects involvement and characteristics, and 3) sources of materials. For additional information on review of the Human Subjects section, please refer to the Human Subjects Protection and Inclusion Guidelines.
Inclusion of Women, Minorities, and Children
When the proposed Center involves clinical research, the committee will evaluate the proposed plans for inclusion of minorities and members of both genders, as well as the inclusion of children. For additional information on review of the Inclusion section, please refer to the Human Subjects Protection and Inclusion Guidelines.
Vertebrate Animals
The committee will evaluate the involvement of live vertebrate animals as part of the scientific assessment according to the following five points: 1) proposed use of the animals, and species, strains, ages, sex, and numbers to be used; 2) justifications for the use of animals and for the appropriateness of the species and numbers proposed; 3) adequacy of veterinary care; 4) procedures for limiting discomfort, distress, pain and injury to that which is unavoidable in the conduct of scientifically sound research including the use of analgesic, anesthetic, and tranquilizing drugs and/or comfortable restraining devices; and 5) methods of euthanasia and reason for selection if not consistent with the AVMA Guidelines on Euthanasia. For additional information on review of the Vertebrate Animals section, please refer to the Worksheet for Review of the Vertebrate Animal Section.
Biohazards
Reviewers will assess whether materials or procedures proposed are potentially hazardous to research personnel and/or the environment, and if needed, determine whether adequate protection is proposed.
Reviewers will provide an overall impact 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). An application does not need to be strong in all categories to be judged likely to have major scientific impact. For example, a Center that by its nature is not especially innovative may be essential to advance a field.
Reviewers will consider each of the review criteria below in the determination of scientific merit, and give a separate score for each.
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?
Is the proposed project sufficiently novel and meritorious and the research plan feasible, in addressing one or more stages in the development of therapies or other strategies to improve the lives of muscular dystrophy patients? For disease mechanism/therapeutic target identification and validation projects, is a plan provided as to how these efforts help to support the therapeutic development pipeline?
For clinical studies or trials: Is there compelling evidence in support of the therapeutic target and candidate therapeutics? Is there a clear need for the data or resource for future clinical trials or is there clear justification for moving the selected candidate therapeutic(s) forward with proof of concept or safety trials? If the aims of the application are achieved, how will scientific knowledge or clinical practice be advanced? What will be the effect of these studies on the concepts, methods, technologies, treatments, services, or preventative interventions that drive this field? Has the applicant addressed both the significance of the eventual definitive clinical trial AND the significance of this study in providing knowledge needed to proceed to the definitive clinical trial? Is there a sufficient body of high quality preclinical or clinical research that supports the rationale for the proposed study? What is the state of equipoise in the medical and patient communities with respect to the proposed intervention? What is the potential for the proposed intervention to have a powerful influence on patient care and quality of life? If the aims of the study are achieved, how will these results contribute to the design and implementation of the definitive clinical trial?
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/PI, do the investigators have complementary and integrated expertise; are their leadership approach, governance and organizational structure appropriate for the project?
Do the collaborative efforts require substantial, and not token, contributions from the partners for successful completion? For studies aimed at developing therapeutics, are there plans for involving industry partners that would enhance the research, accelerate progress or increase the likelihood of developing a product that improves the lives of dystrophy patients?
Innovation
Does the application challenge and seek to shift current research or clinical practice paradigms by utilizing novel theoretical concepts, approaches or methodologies, instrumentation, or interventions? Are the concepts, approaches or methodologies, instrumentation, or interventions novel to one field of research or novel in a broad sense? Is a refinement, improvement, or new application of theoretical concepts, approaches or methodologies, instrumentation, or interventions proposed?
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?
If the Project involves clinical research, are the plans for 1) protection of
human subjects from research risks, and 2) inclusion of minorities and members
of both sexes/genders, as well as the inclusion of children, justified in terms
of the scientific goals and research strategy proposed?
For preclinical translational research projects, is there a clear step-by-step plan, including adequate milestones, to track and evaluate the therapeutic development effort?
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?
Do the problems to be addressed require both a center atmosphere and an intensive collaborative effort for successful completion?
The Administrative Core provides for leadership and management of all Center activities. Reviewers will consider each of the review criteria below in determining the scientific merit of this core. Each assigned reviewer will provide one score for the Administrative Core. The scientific merit of the Administrative Core should be considered by reviewers in determining overall score for the Center.
Purpose
Is the proposed Administrative Core well matched to the needs of the overall center?
Management
Is the management proposed appropriate for scientific administration as well as fiscal administration, procurement, property and personnel management, planning, budgeting, etc.? Is the Center scientific and administrative structure sufficient, including its internal and external procedures for monitoring and evaluating the proposed research projects and core facilities/resources? Are there appropriate plans for establishing the Center Advisory Committee and will this Committee contribute to the oversight of Center research projects, the Shared Resource Core, the Investigator Development and Patient Outreach Core and other components?
Leadership
Do the Director and Co-Director have the leadership and research qualifications to lead a Wellstone Center? Do they have the collective expertise to identify and focus research projects on clinically relevant issues? Are there plans for establishing an Executive Committee and interacting with this committee in a way that will promote the success of the Center?
Communication
Is there an appropriate plan for establishing and maintaining effective communications and cooperation among Center investigators and with investigators outside the Center? Are there appropriate plans for establishing and maintaining a website for the Center that provides useful information to the research community about shared resources and helps to inform the patient community about the research and other activities of the Center?
Environment
Is the environment for the Administrative Core adequate and appropriate to support the overall Center as proposed? Is there evidence of institutional support for the management of the Center?
The Investigator Development and Patient Outreach Core provides support for pilot and feasibility projects conducted by mentored researchers, enhances the training environment of the Center and engages in outreach activities for the muscular dystrophy patient communities. Reviewers will consider each of the review criteria below in determining the scientific merit of this core. Each assigned reviewer will provide one score for the Core. The scientific merit of the Investigator Development and Patient Outreach Core should be considered by reviewers in determining Overall Impact score for the Center.
Purpose
Will the Core achieve the goals of promoting the development of future leaders in muscular dystrophy research by supporting significant, innovative and well designed pilot and feasibility projects, enhancing the training environment for the Center and engaging in outreach activities to increase understanding of the research by patients and their families?
Pilot and Feasibility Project Program
Are there adequate plans to publicize opportunities for support of pilot and feasibility projects, solicit applications, involve the Center Advisory Committee in evaluation of the proposals, select among the applications and monitor progress? Will the majority of funds for this core be applied to the pilot and feasibility projects and will the proposed budget for the core be appropriate to support several such projects per year? Will the Center have a sufficient pool of well-qualified, mentored researchers to conduct these pilot and feasibility projects?
Training Environment Enhancement
Are there well-developed plans for activies that will significantly enhance the training environment of the Center and to leverage existing programs and activities?
Patient Outreach
Are there well-developed plans for significant activities that will engage patients in research and educate patients and their families and/or advocates about the research being conducted by the Center inestigators?
Leadership
Does the application demonstrate that the Director of this Core is dedicated to the training of students and fellows, with a track record of successful mentoring? How strong is the aggregate training record of the MDCRC investigators?
Innovation
Will the Core develop and implement innovative strategies for enhancing the training students and fellows in various aspects of basic, translational and clinical research in the muscular dystrophies? Are innovative outreach activities proposed that will engage patients and their families?
Environment
Are the institutional environment and resources conducive to the development of future leaders in muscular dystrophy research and engaging patients in research and education activities?
Reviewers will consider each of the review criteria below in determining the scientific merit of this core. Each assigned reviewer will provide one score for the Scientific Research Resource Core. The scientific merit of the Scientific Research Resource Core should be considered by reviewers in determining Overall Impact score for the Center.
Purpose
Are the core activities capable of effectively and efficiently supporting research productivity and collaborations and are they essential to the mission of the Center? Is there adequate scientific and technical merit to justify the core? If other, similar cores are already available to the research community, is there still a significant need for this core?
Quality and Quality Control
Is there a strong commitment to provide services to the national muscular dystrophy community and are plans for oversight and prioritization of user requests for core services adequate and fair?
Utilization Potential
Is there strong evidence via the core description that at least one Scientific Research Resource Core will serve as an important national/international resource for the muscular dystrophy research community? Is there sufficient likelihood that this core facility will be used by researchers within and outside the Center, and that the resources provided will significantly accelerate progress on the projects that make use of this core?
Leadership
Does the core provide adequate leadership and technical expertise to ensure that it meets its stated goals?
Cost Effectiveness
If the core facility is already established and supported by funding other than an MDCRC, how will the MDCRC award enhance the resources available? If designed as a fee-for-service facility, are the projected fees appropriate for recovery of only the variable costs (supplies, service contracts, etc.) and not the costs of personnel and equipment?
Environment
Are the staffing, allocated space, equipment, and other resources that are available to the core sufficient to meet the anticipated demand on its services?
As applicable for the Center proposed, reviewers will evaluate the following additional items while determining scientific and technical merit, and in providing an overall impact score, but will not give separate scores for these items.
Protections for Human Subjects
For research that involves human subjects but does
not involve one of the six categories of research that are exempt under 45 CFR
Part 46, the committee will evaluate the justification for involvement of human
subjects and the proposed protections from research risk relating to their
participation according to the following five review criteria: 1) risk to
subjects, 2) adequacy of protection against risks, 3) potential benefits to the
subjects and others, 4) importance of the knowledge to be gained, and 5) data
and safety monitoring for clinical trials.
For research that involves human subjects and meets the criteria for one or
more of the six categories of research that are exempt under 45 CFR Part 46,
the committee will evaluate: 1) the justification for the exemption, 2) human
subjects involvement and characteristics, and 3) sources of materials. For
additional information on review of the Human Subjects section, please refer to
the Human
Subjects Protection and Inclusion Guidelines.
Inclusion of Women, Minorities, and Children
When the proposed Center involves clinical research, the committee will evaluate the proposed plans for inclusion of minorities and members of both genders, as well as the inclusion of children. For additional information on review of the Inclusion section, please refer to the Human Subjects Protection and Inclusion Guidelines.
Vertebrate Animals
The committee will evaluate the involvement of live vertebrate animals as part of the scientific assessment according to the following five points: 1) proposed use of the animals, and species, strains, ages, sex, and numbers to be used; 2) justifications for the use of animals and for the appropriateness of the species and numbers proposed; 3) adequacy of veterinary care; 4) procedures for limiting discomfort, distress, pain and injury to that which is unavoidable in the conduct of scientifically sound research including the use of analgesic, anesthetic, and tranquilizing drugs and/or comfortable restraining devices; and 5) methods of euthanasia and reason for selection if not consistent with the AVMA Guidelines on Euthanasia. For additional information on review of the Vertebrate Animals section, please refer to the Worksheet for Review of the Vertebrate Animal Section.
Biohazards
Reviewers will assess whether materials or procedures proposed are potentially hazardous to research personnel and/or the environment, and if needed, determine whether adequate protection is proposed.
Resubmissions
For Resubmissions, the committee will evaluate the application as now presented, taking into consideration the responses to comments from the previous scientific review group and changes made to the Center.
Renewals
For Renewals, the committee will consider the progress made in the last funding period. The productivity of the Center and its impact on the muscular dystrophy research field over the period of Center funding will be taken into consideration when determining the Overall Impact score.
Revisions
Not Applicable
As applicable for the Center 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 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
Reviewers will comment on whether the following Resource Sharing Plans, or the rationale for not sharing the following types of resources, are reasonable: 1) Data Sharing Plan; 2) Sharing Model Organisms; and 3) Genome Wide Association Studies (GWAS).
Budget and Period of Support
Reviewers will consider whether the budget and the requested period of support are fully justified and reasonable in relation to the proposed research.
Applications will be evaluated for scientific and technical merit by (an) appropriate Scientific Review Group(s) convened by the NIAMS, in accordance with NIH peer review policy and procedures, using the stated review criteria. Assignment to a Scientific Review Group will be shown in the eRA Commons.
As part of the scientific peer review, all applications:
Appeals of initial peer review will not be accepted for applications submitted response to this FOA.
Applications will be assigned on the basis of established PHS referral guidelines to the appropriate NIH Institute or Center and will compete for available funds with all other recommended applications submitted in response to this FOA. Following initial peer review, recommended applications will receive a second level of review by the NIAMS Advisory Council. The following will be considered in making funding decisions:
After the peer review of the application is completed, the PD/PI 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.
If the application is under consideration for funding, NIH
will request "just-in-time" information from the applicant as
described in the NIH
Grants Policy Statement.
A formal notification in the form of a Notice of Award (NoA) will be provided
to the applicant organization for successful applications. The NoA signed by
the grants management officer is the authorizing document and will be sent via
email to the grantee’s business official.
Awardees must comply with any funding restrictions described in Section IV.5. Funding Restrictions. Selection
of an application for award is not an authorization to begin performance. Any
costs incurred before receipt of the NoA are at the recipient's risk. These
costs may be reimbursed only to the extent considered allowable pre-award costs.
Any application awarded in response to this FOA will be subject to the DUNS, SAM
Registration, and Transparency Act requirements as noted on the Award
Conditions and Information for NIH Grants website.
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.
The following special terms of award are in addition to, and not in lieu of, otherwise applicable OMB administrative guidelines, HHS grant administration regulations at 45 CFR Parts 74 and 92 (Part 92 is applicable when State and local Governments are eligible to apply), and other HHS, PHS, and NIH grant administration policies.
The administrative and funding instrument used for this program will be the cooperative agreement an "assistance" mechanism (rather than an "acquisition" mechanism), in which substantial NIH programmatic involvement with the awardees is anticipated during the performance of the activities. Under the cooperative agreement, the NIH purpose is to support and stimulate the recipients' activities by involvement in and otherwise working jointly with the award recipients in a partnership role; it is not to assume direction, prime responsibility, or a dominant role in the activities. Consistent with this concept, the dominant role and prime responsibility resides with the awardees for the project as a whole, although specific tasks and activities may be shared among the awardees and the NIH as defined below.
The PD(s)/PI(s) will have the primary responsibility for:
The PD(s)/PI(s) will have the primary responsibility for: defining objectives and approaches, and to plan, conduct, analyze, and publish results, interpretations, and conclusions of their studies. The Principal Investigator will serve as Center Director and together with a Center Co-Director, will be responsible for the integration and management of activities within the MDCRC.
The Center Director shall be responsible for organizing a local Executive Committee for day-to-day management of the MDCRC, and an external Center Advisory Committee, with scientific, clinical and patient advocate representation (final membership to be approved by the NIH). The role of these Committees will include the solicitation, review, and selection of proposals for predoctoral and postdoctoral research positions and collaborative projects, and the selection and prioritization of projects that will use resources and services that are provided for through the MDCRC.
The Center Director and Co-Director of each MDCRC also serve as members of the MDCRC Steering Committee (see below) and are required to participate in its activities, to include regular conference calls and an annual MDCRC face-to-face meeting.
Awardees agree to participate in the overall coordination of NIH research efforts in muscular dystrophy. This participation may include collaboration and consultation with other NIH awardees, the sharing of information, data, and research materials, and participation in NIH efforts to standardize and harmonize pre-clinical and clinical data collection.
Awardees with a clinical trial component in their MDCRC agree to review of associated data, abstracts, and other publications by the DSMB and the NIH prior to their release.
Awardees will retain custody of and have primary rights to the data and software developed under these awards, subject to Government rights of access consistent with current HHS, PHS, and NIH policies.
NIH staff have substantial programmatic involvement that is above and beyond the normal stewardship role in awards, as described below:
An NIH Project Scientist will have substantial programmatic involvement that is above and beyond the normal stewardship role in awards, as described below.
Each MDCRC will have the support of Project Scientists, representing each participating Institutes, and a Program Official from NIH staff who are assigned administrative roles for the muscular dystrophies being studied and have expertise in the implementation of the MDCRC Program.
The NIH Project Scientists will have substantial scientific-programmatic involvement during conduct of this activity, through technical assistance, advice, and coordination above and beyond normal program stewardship for grants. The NIH Project Scientists also will assist in the interaction between the awardee and investigators of other institutions, as well as between the awardee and other Federal agencies and/or potential commercial sponsors. The NIH Project Scientists will be members of the MDCRC Steering Committee. The NIH Project Scientists retain the option to recommend additional research endeavors within the constraints of the approved research and negotiated budget.
An important part of the NIH MDCRC Program is the coordination of research efforts across different funding mechanisms and research structures, and coordination among efforts aimed at different muscular dystrophies. The NIH Project Scientists will have the primary responsibility for this overall coordination.
The NIH may appoint an MDCRC External Advisory Committee (EAC), consisting of scientific and public members. The EAC may function in an advisory role to the NIH as necessary on issues that arise related to the MDCRC program. EAC members may participate in the annual meeting of the Steering Committee and be consulted as necessary.
The NIH Project Scientists representing NINDS, NIAMS, NICHD, and NHLBI will, collectively, have a single NIH vote on the MDCRC Steering Committee (see below). NIH Project Scientists will abstain from voting on any issue where they are unable to reach a consensus.
The NIH will establish one or more Data Safety Monitoring Boards to provide oversight and to advise the NIH on any clinical trials that are supported by the MDCRC awards.
Additionally, an NIH Program Official will be primarily responsible for program oversight. The Program Official assigned to each MDCRC will exercise the normal stewardship responsibilities of an NIH Program Official, including assessment of the progress of the projects toward the accomplishment of specified objectives. NIH Program Officials also retain the option of recommending termination of studies if technical performance falls below acceptable standards, or when specific lines of research cannot be effectively pursued in a timely manner. This Program Official will be responsible for the normal scientific and programmatic stewardship of the award and will be named in the award notice.
Areas of Joint Responsibility include:
Overall coordination of the MDCRC Program will be done by a Steering Committee. The MDCRC Steering Committee will make strategic decisions with regard to goals and research implementation, including the establishment and development of collaborations.
The Steering Committee will consist of the Center Directors and Co-Directors of each MDCRC, NIH Project Scientists and a public member. The Steering Committee will be chaired and co-chaired by MDCRC Center Directors, who are elected by vote of the Steering Committee for staggered two-year terms. The Chair and Co-Chair will be responsible for conduct of regular conference calls. The Steering Committee will hold a face-to-face meeting at least annually. Each MDCRC and the public member will have one vote on the Steering Committee and the NIH Project Scientists, collectively, will have a single NIH vote.
Each full member will have one vote. Awardee members of the Steering Committee will be required to accept and implement policies approved by the Steering Committee.
Dispute Resolution:
Any disagreements that may arise in scientific or programmatic matters (within the scope of the award) between award recipients and the NIH may be brought to Dispute Resolution. A Dispute Resolution Panel composed of three members will be convened. It will have three members: a designee of the Steering Committee chosen without NIH staff voting, one NIH designee, and a third designee with expertise in the relevant area who is chosen by the other two; in the case of individual disagreement, the first member may be chosen by the individual awardee. This special dispute resolution procedure does not alter the awardee's right to appeal an adverse action that is otherwise appealable in accordance with PHS regulation 42 CFR Part 50, Subpart D and DHHS regulation 45 CFR Part 16.
When multiple years are involved, awardees will be required to submit the Non-Competing Continuation Grant Progress Report (PHS 2590 or RPPR) 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.
We encourage inquiries concerning this funding opportunity and welcome the opportunity to answer questions from potential applicants.
GrantsInfo (Questions regarding application instructions and
process, finding NIH grant resources)
Telephone: 301-710-0267
TTY 301-451-5936
Email: GrantsInfo@nih.gov
eRA Commons Help Desk (Questions regarding eRA Commons
registration, tracking application status, post submission issues)
Telephone: 301-402-7469 or 866-504-9552 (Toll Free)
TTY: 301-451-5939
Email: commons@od.nih.gov
Glen H. Nuckolls, Ph.D.
National Institute of Arthritis and Musculoskeletal and Skin
Diseases (NIAMS)
Telephone: 301-594-4974
Email: nuckollg@mail.nih.gov
Tiina Urv, Ph.D.
Eunice Kennedy Shriver National Institute for Child Health
and Human Development (NICHD)
Telephone: 301-402-7015
Email: tu36j@nih.gov
John D. Porter, Ph.D.
National Institute of Neurological Disorders and Stroke
(NINDS)
Telephone: 301-496-5739
Email: porterjo@ninds.nih.gov
Kan Ma, Ph.D.
National Institute of Arthritis, Musculoskeletal and Skin
Diseases (NIAMS)
Phone: 301-451-4838
Email: mak2@mail.nih.gov
Sheila Simmons
National Institute of Arthritis and Musculoskeletal and Skin
Diseases (NIAMS)
Telephone: 301-594-9812
Email: simmonsS@mail.nih.gov
Bryan S. Clark, M.B.A.
Eunice Kennedy Shriver National Institute of Child Health
and Human Development (NICHD)
Telephone: 301-435-6975
Email: clarkb1@mail.nih.gov
Tijuanna DeCoster, MPA
National Institutes of Neurological Disorders and Stroke
(NINDS)
Telephone: 301-496-9231
Email: decostert@ninds.nih.gov
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.
Awards are made under the authorization of Sections 301 and 405 of the Public Health Service Act as amended (42 USC 241 and 284) and under Federal Regulations 42 CFR Part 52 and 45 CFR Parts 74 and 92.
Weekly TOC for this Announcement
NIH Funding Opportunities and Notices
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