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Frequently Asked Questions

FAQs for RFA-RM-11-022 Integrated Microphysiological Systems for Drug Efficacy and Toxicity Testing in Human Health and Disease (UH2/UH3) and RFA-RM-12-001 Stem/Progenitor Cell-Derived Human Micro-organs and -tissues (U18).

  1. What is meant by "Regulatory Science"?
  2. Who should we contact regarding questions related to organ-specific Microsystems?
  3. What kind of research and technologies fall under this initiative?
  4. Can an application contain more than one organ system and can applications be linked together?
  5. What is the connection between the U18 and the UH2/UH3? Can they be integrated?
  6. What is the distinction between UH2 and U18?
  7. What are acceptable transition plans from the UH2 to the UH3 phase of RFA- RM-11-022?
  8. What role does the NIH have in this cooperative agreement? What are the roles of DARPA and the FDA in this initiative?
  9. What are the points to consider when submitting an application in response to this FOA?
  10. Do I have to submit a letter of intent? What should be in it? How should I send it?
  11. How much preliminary data is required?
  12. When is the receipt date for sending in the application?
  13. Can I submit more than one application in response to either or both of these FOAs?
  14. How will applications be reviewed?
  15. Will I have an opportunity to submit supplementary information before my application is reviewed?
  16. Will my application(s) be reviewed by experts in my field?
  17. What sort of milestones will be expected for these proposals?
  18. How many meetings will project PIs/co-PIs be required to attend each year?
  19. Will site visits be conducted?
  20. I am an NIH intramural investigator. Am I eligible to apply?
  21. I am an FDA intramural investigator. Am I eligible to apply?
  22. Can an FDA scientist be part of my research team, as a collaborator or consultant? If so, what are allowable costs?
  23. I am not a U.S. citizen. Am I eligible to apply?
  24. My institution is not in the U.S. Am I eligible to apply?
  25. Can work be performed outside the U.S., and may I request funds for a foreign subcontract?
  26. Is it open to commercial, for-profit companies? Is there a benefit to having industry partners?
  27. Are there specific IP requirements in the FOAs?
  28. Will work within this grant restrict or constrain further commercial efforts by my firm? What are the "terms" with regard to the promotion and use of any technology that my firm would bring to this project or develop?
  29. Can additional PIs be added during the course of the Project Period?
  30. Can changes be made to the Research Plan during the Project period as new information is obtained?
  31. What are the plans for implementation and deployment of the technology and results from this program?
  32. Who will develop the milestones?
  33. Do I have to submit a detailed budget for the competing and noncompeting awards or can I use the modular format?
  34. Will the NGA for the Phase 1 (UH2) include the future commitments for the Phase 2 (UH3)?
  35. Should the indirect costs for any sub-awards be included in the cap amount ($675,000 for UH2 and 3 and $250,000 for U18)?
  36. Will a project be considered responsive if it uses animal cells at the beginning to develop a system but ultimately ends up using human-derived cells?
  37. With respect to DARPA, the platforms need to be solidified within the first 18 months of that program. What contractual mechanism is there to introduce additional requirements to that platform during that period? Is there any interaction with the Defense Threat Reduction Agency (DTRA) program?
  38. If an applicant responds to both DARPA and NIH with a substantially overlapping proposal, would there be an integrated funding decision between DARPA and NIH?
  39. What is preferred under NIH’s FOAs – deeper complexity of one system/organ vs. broad multi-systems/organs?
  40. Is a 2D cell model acceptable if one can recapitulate the function of the tissue?
  41. Which is more desirable - technology development or biological recapitulation of function?
  42. What does NIH expect in the Intellectual Property Management Plan?
  43. What is the structure of a U18 application?
  44. Would NIH review find it helpful if a potential applicant who plans to apply to both DARPA and NIH UH2/UH3 provides a detailed description on the differences between UH2/UH3 and DARPA applications?
  45. Is disease modeling necessary?
  46. Does the U18 require a multi-team approach?
  47. Does the U18 require using patient cells?
  48. How does one create a UH2/UH3 consortium with other applicants? Will there be a teaming website that NIH will set up or is there a mechanism to help integrate a multisystem platform before the application deadline of January 26, 2012?
  49. How should I structure the research strategy section to better inform review?

1. What is meant by "Regulatory Science"?
Regulatory science for the purposes of these FOAs (RFA- RM-11-022 and RFA-RM-12-001) is a specialized and interdisciplinary area of biomedical research that serves to generate new knowledge and tools for assessing experimental therapies, preventives, and diagnostics. Advancement in regulatory science will depend on the incorporation of cutting-edge science and evidence-based knowledge into the regulatory decision-making process. The research supported through this initiative should add to the scientific knowledge base by providing new methods, models or technologies that will inform the scientific and regulatory community about better approaches to evaluating safety, quality and efficacy in medical product development.  The emphasis of this initiative centers on innovation.

2. Who should we contact regarding questions related to organ-specific Microsystems?
The Common Fund programs are administered via a team of NIH program experts.  The Regulatory Science Microsystems Project Team members have microsystem-specific expertise and these members and their affiliations are listed below.

  • Guillermo Arreaza-Rubin, M.D. (NIH/NIDDK); Program Director, Clinical Immunology of Diabetes
  • David Balshaw, Ph.D. (NIH/NIEHS); Program Director, Center for Risk and Integrated Sciences, Division of Extramural Research and Training
  • Christine Colvis, Ph.D. (NIH/NIDA); Director of Program Integration
  • Holli Hamilton, M.D., M.P.H. (NIH/NIDCR); Senior Medical Officer, Division of Extramural Research
  • Rosemarie Hunziker, Ph.D. (NIH/NIBIB); Program Director, Division of Discovery Science and Technology
  • Nadya Lumelsky, Ph.D. (NIH/NIDCR): Director, Tissue Engineering and Dental and Craniofacial Regenerative Medicine Research Program, Integrative Biology and Infectious Disease Branch
  • Matthew McMahon, Ph.D. (NIH/NEI; Senior Advisor for Translational Research, Office of the Director
  • Richard Okita, Ph.D. (NIH/NIGMS); Program Director, Division of Pharmacology, Physiology, and Biological Chemistry
  • Thomas Palker, Ph.D. (NIH/NIAID); Program Officer, Division of Allergy, Immunology, and Transplantation
  • Douglas Sheeley, Sc.D. (NIH/NCRR); Deputy Director, Division of Biomedical Technology
  • Robert Star, M.D.(NIH/NIDDK); Director, Division of Kidney, Urologic, and Hematologic Diseases
  • Margaret Sutherland, Ph.D. (NIH/NINDS); Program Director,  Neurodegeneration
  • Danilo Tagle, Ph.D. (NIH/NINDS); Program Director, Neurogenetics
  • Hung Tseng (NIH/NIAMS); Health Scientist Administrator, Division of Skin and Rheumatic Diseases
  • Lois Tully, Ph.D. (NIH/NINR); Program Director, Division of Extramural Activities
  • Fei Wang, Ph.D. (NIH/NIAMS); Health Scientist Administrator, Division of Musculoskeletal Diseases
  • Anne Zajicek, M.D., Pharm.D,(NIH/NICHD); Acting Chief , Obstetric & Pediatric Pharmacology Branch

3. What kind of research and technologies fall under this initiative?
Please see Section I under Research Objectives of the FOAs. For the purposes of RFA- RM-11-022 and RFA-RM-12-001, the following definitions apply:

  • Microphysiological systems – circulatory, endocrine, gastrointestinal, immune, integumentary, musculoskeletal, nervous (including the eye), reproductive, respiratory and kidney and urinary tract 
  • 3D cell models –  culture of living cells within microfabricated devices having  structures that mimic tissue- and organ-specific microarchitecture that support tissue differentiation and recapitulate the tissue–tissue interfaces, spatiotemporal chemical gradients, and mechanical microenvironments of living organs
  • Stem cells – human embryonic, induced pluripotent or adult stem cells, which can self-renew by mitosis and which have the potential to differentiate along numerous cell fate pathways
  • Progenitor cells –  non-transformed multipotent or unipotent cells that are committed to a cell lineage
  • Primary cells – cells isolated directly from human tissue
  • Multicellular model – a system made up of more than one cell type, for example a multicellular  nervous system model consisting of neurons, astroctyes and interneurons
  • Disease models - models of genetic, idiopathic diseases associated with the microsystems studied.  These disease models could include, but are not limited to: cardiomyopathies; endocrinopathies; type 1 and type 2 diabetes; various inflammatory diseases such as inflammatory bowel disease; osteo- or rheumatoid arthritis, periodontal disease, and pancreatitis; autoimmune diseases; infectious diseases; liver disease; fibrotic diseases; muscular dystrophies; neuromuscular and musculoskeletal disorders; neurodevelopmental disorders; seizures; pulmonary hypertension and cystic fibrosis.  Disease models may also include those that model infectious diseases or models of environmental toxin exposure.

4. Can an application contain more than one organ system and can applications be linked together?
Yes.  A parallel announcement from DARPA requires integration of 10 systems, whereas the RFA- RM-11-022 FOA allows investigators to more fully develop the biology around a subset of microsystems.

5. What is the connection between the U18 and the UH2/UH3? Can they be integrated?

  • The U18 projects will run in parallel with UH2/UH3 projects. The U18 FOA is intended to accelerate advancements in current human stem- and progenitor-derived cell-type selectivity approaches through improvements in differentiation efficiencies, cell-type diversity, genetic complexity and utilization of 3D culturing approaches to enhance cellular microenvironments.
  • The cells and microsystems developed through the U18 mechanism can potentially become a part of an integrated platform if through the semi-annual interactions, the U18 differentiated cells or microsystems are deemed superior to what is currently available.

 

6. What is the distinction between UH2 and U18??
The U18 is geared towards stem cells and progenitor cells in terms of maturation protocols, used to seed the future device. The UH2/UH3 will support studies to develop modular Microsystems from a number of human organ systems that are functionally relevant and also reflect the multicellular, genomic and pathological complexity of the tissues of origin.

7. What are acceptable transition plans from the UH2 to the UH3 phase of RFA- RM-11-022?

  • It is conceivable that several UH2 projects could be linked or integrated during the UH3 phase.
  • NIH award recipients are also expected to participate in semi-annual meetings, where one of the goals will be to encourage investigators to form alliances when linking organ systems that each group is working on makes scientific sense for the UH3 phase.
  • During these semi-annual meetings, it will also be possible to interact with DARPA award recipients for possible collaborations for the UH3 phase. The choice of an integrated platform for the UH3 phase is not limited to what DARPA may have to offer. NIH awardees can also propose an alternative integrated platform.
  • Please bear in mind that the UH2/UH3 funding mechanism is a cooperative agreement such that specific details of the approaches and plans are subject to modification and approval by the trans-NIH Regulatory Science Microsystem Project Team during the post-award period. It is foreseeable that the specific objectives for each NIH award are likely to morph over time to reflect new collaborative activities established amongst funded investigators and through the partnership with DARPA.

8. What role does the NIH have in this cooperative agreement? What are the roles of DARPA and the FDA in this initiative?
The role of NIH staff in this cooperative agreement is spelled out in Section VI of the FOA and will further be defined in the terms of conditions of award. In brief, the NIH Regulatory Science Microsystem Project Team will have substantial scientific and programmatic involvement during the conduct of this activity through technical assistance, advice and coordination.  However, the role of NIH Regulatory Science Microsystem Project Team will be to facilitate and not to direct the activities.

DARPA and FDA, through their partnerships with the NIH, will provide guidance and recommendations to the NIH Regulatory Science Microsystems Project Team and its awardees.

9. What are the points to consider when submitting an application in response to this FOA?
The research supported through this initiative will recognize innovation that supports the development of new methods, models or technologies that will inform the scientific and regulatory community about better approaches to evaluating safety and efficacy in medical product development.  In preparing applications responsive to the NIH Regulatory Science initiative, the following points should be considered:

  • Will the new approach/methodology have a competitive advantage over existing/alternate approaches and have the potential to inform future medical product development?
  • Does the microsystem have the potential to be readily adaptable in drug efficacy and toxicity testing?
  • Will the expected results lead to advances in technologies used in the regulatory review of medical products (including drugs, biologics, and devices), and thus improvements regulatory practice and public health?
  • If the application focuses on a particular disease model, is the disease or health state adequately represented in the microsystem proposed?
  • Is the proposed use of particular cell types sufficient to capture the complexity and normal physiology of the target organ or tissue?
  • Does the application identify major technical risks, and are the proposed efforts to mitigate or address the risks clearly defined and feasible? Are the methods and procedures for characterizing and validating microsystems rational and clearly explained?
  • In the case of RFA- RM-11-022, does the proposed transition plan to the UH3 phase allow for flexibility and interoperability for integration of microsystems into alternative platforms? Is the transition plan complete and in a logical sequence to the elements of the phased UH2/UH3 as defined in Section 1?
  •  In the case of RFA- RM-11-022, do the evaluation plans, milestones and timelines proposed clearly identify successful completion of the UH2 and the appropriateness of advancement to the UH3 phase?

10. Do I have to submit a letter of intent? What should be in it? How should I send it?
No. Although a LOI is not required, the information that it contains allows NIH staff to anticipate the requisite expertise of reviewers, estimate the potential review workload and better inform the review process.
Prospective applicants are asked to submit a letter of intent that includes the following information:
Descriptive title of proposed research
Name, address, and telephone number of the Principal Investigator
Names of other key personnel
Participating institutions
Number and title of this funding opportunity

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 NIH CSR to estimate the potential review workload and plan the review. Letters of intent can be e-mailed, faxed, or sent as hard copy (e-mail is preferred). There is no need to send hard copy of a letter that has already been sent via e-mail or fax. All letters of intent should be sent to Dr. Margaret Sutherland at:
Margaret Sutherland, PhD for the Regulatory Science Microsystems Working Group
Program Director, Neurodegeneration
National Institute of Neurological Disorders and Stroke
6001 Executive Boulevard.
Neuroscience Center, Room 2203
Bethesda, MD 20892
Telephone: (301) 496-5680
Fax: (301) 480-1080
sutherlandm@ninds.nih.gov

11. How much preliminary data is required?
Sufficient preliminary data should be provided to determine the feasibility of the proposed approach. For RFA- RM-11-022, the UH3 transition plan should include gap analysis and is expected to be more conceptual to allow for the flexibility and interoperability that may be required for integration of microsystems into a common platform.  It is conceivable that the UH3 phase will be higher risk and therefore preliminary data for all aspects of the UH3 phase may not be available.

12. When is the receipt date for sending in the application?
The application receipt date is January 26, 2012. This is the receipt date and not a postmarked date. The application must be received by the Division of Receive & Referral of CSR on or before that date. There is no extension to that deadline. If you have served recently as a reviewer or advisory council member, normally you get an extension to your own submission deadline; however, extensions do not apply for this FOA.

13. Can I submit more than one application in response to either or both of these FOAs?
Yes, as long as they are non-overlapping and scientifically distinct.  Applicants may be PIs, co-PIs or subcontracts on other projects, so long as there is no scientific overlap between projects. In addition, a PI should not exceed 100% effort on all funding he/she has. Applications that are non-responsive to the specific requirements of the FOAs will not be considered.  Applications that are substantially identical to proposals funded in response to the DARPA BAA-11-73 will not be considered.

14. How will applications be reviewed?
Applications that are complete and responsive to the FOA will be evaluated for scientific and technical merit by a Special Emphasis Panel (SEP) convened by the Center for Scientific Review (CSR). Reviews most likely will take place during the month of April in 2012. Questions regarding the review process can be directed to Dr. Tom Peterson at petersonjt@csr.nih.gov .

15. Will I have an opportunity to submit supplementary information before my application is reviewed?
No.

16. Will my application(s) be reviewed by experts in my field?
Every effort will be made to ensure appropriate reviewer expertise; however, it is likely that not all of the reviewers assigned to your application will be considered experts in your specific field. We anticipate that investigators will submit applications on a wide range of topics. The SEP reviewers will be drawn from academia and industry, and have general expertise that is relevant to your application. It is extremely important to keep this in mind when you are writing your application. When describing what you want to do, avoid jargon, and use language that scientists in other fields can understand. You will also have to convince scientists in other fields that what you are proposing to do is exciting and exceptionally innovative or important to the field, and that the proposed research will have a profound impact. Please see Section V of RFA regarding review criteria.

17. What sort of milestones will be expected for these proposals?
The use of milestones should provide clear indicators of a project's continued success or emergent difficulties. Milestones are different from specific aims. The milestones must provide objective and quantitative outcomes by which to justify advancing the project. The application must include a strong rationale for the choice of models, parameters, and quantitative go/no-go decisions to be made by NIH staff, based upon accepted practices in the specific field.  The UH2 milestones should address the readiness of the microsystem for integration.  The transition plan for the UH2 to UH3 phase should emphasize where applicable the flexibility and interoperability of the microsystems to adapt to alternative platforms.

18. How many meetings will project PIs/co-PIs be required to attend each year?
Project PIs/co-PIs should budget for semi-annual meetings during the course of the award.

19. Will site visits be conducted?
It is possible that site visits will be conducted on a periodic basis as part of monitoring the progress of awarded grants.

20. I am an NIH intramural investigator. Am I eligible to apply?
Yes. Please see special application instructions under Section IV.6. “Applications Involving the NIH Intramural Research Program” of RFA- RM-11-022.

21. I am an FDA intramural investigator. Am I eligible to apply?
You would not qualify as a PI, but could be a collaborator. Because FDA researchers already carry out research relevant to regulatory science, it is unlikely that the project would be one outside of the work normally funded by the FDA.

22. Can an FDA scientist be part of my research team, as a collaborator or consultant? If so, what are allowable costs?
Yes, assuming the proper approvals have been received by the scientist from the FDA leadership. However since FDA is a PHS agency, no funds can be provided unless the project triggers "exceptional circumstances" as noted in the NIH GPS (see question 19).

23. I am not a US citizen. Am I eligible to apply?
Yes, if your place of business/institution is located within the US.

24. My institution is not in the US. Am I eligible to apply?
Investigators at non-US (foreign) institutions are not eligible to apply.

25. Can work be performed outside the US?
Yes, as a subcontract to the US-based institution.  Justification for inclusion of work to be performed outside the US must be provided in the grant application and if selected for funding, foreign clearance will be required prior to release of a Notice of Grant Award. Investigators at non-U.S. institutions are not allowed as paid collaborators.

26. Is it open to commercial, for-profit companies? Is there a benefit to having industry partners?
Yes, for-profit organizations are eligible to apply or serve as collaborators. The needs and scope of the proposed project should dictate and strongly justify the field of collaborators needed to accomplish the task. In general, the NIH highly encourages partnership with industry and a multidisciplinary team approach in accomplishing the goals of this FOA. 

27. Are there specific IP requirements in the FOAs?
Yes, please see Section IV.6 for details.  If access to third party IP is necessary to do the proposed work, this needs to be address in the IP management plan.

28. Will work within this grant restrict or constrain further commercial efforts by my firm? What are the "terms" with regard to the promotion and use of any technology that my firm would bring to this project or develop?
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. Please see Section IV.6 “Intellectual property management plan” of the FOA.

29. Can additional PIs be added during the course of the Project Period?
Yes. Addition of new key personnel is subject to approval by NIH and should not result in any increase in the approved budget.

30. Can changes be made to the Research Plan during the Project period as new information is obtained?
Since this is a cooperative agreement, the NIH Regulatory Science Microsystem Project Team will work with the investigators if minor changes to the research approach have to be made during the course of the project. However the research plan has to remain true to what was peer-reviewed.

31. What are the plans for implementation and deployment of the technology and results from this program?
Awardees will be encouraged to publish and publicly release and disseminate results, data and other products of the study, concordant with the study protocol and governance and the approved plan for making data and materials available to the scientific community and NIH. Awardee will work with the NIH to develop and implement an appropriate rapid data release policy. The path for knowledge transfer and commercialization of integrated organ system should be described in the application.

32. Who will develop the milestones?
This is a negotiated process between the Program Director, the Principal Investigator and Business Official. Once the milestones have been developed and approved, the business official should submit a signed copy of the milestones to the NIH Grants Management Office.

33. Do I have to submit a detailed budget for the competing and noncompeting awards or can I use the modular format?
The U18 applications may use the modular format requesting up to 2 years of funding. The UH2/UH3 must submit a detailed budget using the SF 424 requesting up to 5 years of funding.

34. Will the NGA for the Phase 1 (UH2) include the future commitments for the Phase 2 (UH3)?
No. An administrative review will be performed during the last half of Year 02 for the UH2 phase and if it is determined that the milestones have been successfully met, the Notice of Grant Award (NoGA) will be revised to reflect the UH3 Phase to include future funding for years 03-05.

The UH2--Only 2 years of funding will be indicated on the NoGA.
The UH3 will provide the remaining approved funding for years 03-05.

35. Should the indirect costs for any sub-awards be included in the cap amount ($675,000 for UH2 and 3 and $250,000 for U18)?
No. The cap in each program excludes the indirect costs of subawards.

36. Will a project be considered responsive if it uses animal cells at the beginning to develop a system but ultimately ends up using human-derived cells?
No, as pointed out in the FOA, the project must start with human cells.

37. With respect to DARPA, the platforms need to be solidified within the first 18 months of that program. What contractual mechanism is there to introduce additional requirements to that platform during that period? Is there any interaction with the Defense Threat Reduction Agency (DTRA) program?
The funding from NIH would be through a cooperative mechanism which involves programmatic involvement by NIH staff on project development, monitoring progress, and providing technical guidance when needed. The potential applicants are encouraged to contact the program staff of the NIH Microsystems team to shape their proposals and applications.

NIH has no formal relationship with the DTRA program.

38. If an applicant responds to both DARPA and NIH with a substantially overlapping proposal, would there be an integrated funding decision between DARPA and NIH?
DARPA and NIH cannot fund an identical application. If an identical application is funded by DARPA, it will be automatically be excluded from funding consideration by NIH. However, since these programs are intended to be synergistic but independent, applications may be partially overlapping if similar approaches are proposed. In this case, following NIH review and funding recommendations, NIH will work with the investigator and DARPA to identify non-overlapping research interests. The NIH funding decision will be made in May 2012, and DARPA’s decision will be made in February or March, 2012.

39. What is preferred under NIH’s FOAs – deeper complexity of one system/organ vs. broad multi-systems/organs?
The NIH encourages applications dealing with both a deeper and fuller representation of a particular organ in a microsystem and/or representation in an integrated platform of multiple organ systems.

40. Is a 2D cell model acceptable if one can recapitulate the function of the tissue?
The proposed system has to be something more than a simple model, far different from what a field currently has, and made up of more than one cell type.

41. Which is more desirable - technology development or biological recapitulation of function?
NIH does not intend to be prescriptive in what the applicants can propose but it would be beneficial for proposers to think about the overall goal of these FOAs; i.e., to create an environment to enable efficacy and toxicology studies using an in vitro system. The level of complexity, the type of readouts, and the functionality are all driving towards the overarching goal to address the needs of the biomedical research community and the FDA in finding the best predictors for safety and efficacy before going to clinic.

42. What does NIH expect in the Intellectual Property Management Plan?
Details about intellectual property (IP) involved with the proposed cell source, devices, or anything that may hinder commercialization of the finished product. More details are presented in Section 6 of the FOAs.

43. What is the structure of a U18 application?
The U18 uses the typical R01,12-page application structure.

44. Would NIH review find it helpful if a potential applicant who plans to apply to both DARPA and NIH UH2/UH3 provides a detailed description on the differences between UH2/UH3 and DARPA applications?
No. NIH and DARPA will not compare applications, so there is no need for such a detailed comparison between both proposed studies. NIH reviewers will not have access to DARPA applications.

45. Is disease modeling necessary?
A disease model is not a requirement under these RFAs but may be included depending on the maturity of the microsystem being proposed.

46. Does the U18 require a multi-team approach?
Applicant(s) need to demonstrate in the application that needed expertise is in place and should describe how it will be implemented.

47. Does the U18 require using patient cells?
No. However, if a project proposes to use patient cells, it is recommended, although not required, that the patients’ consent be obtained to allow for the commercialization of those lines.

48. How does one create a UH2/UH3 consortium with other applicants? Will there be a teaming website that NIH will set up or is there a mechanism to help integrate a multisystem platform before the application deadline of January 26, 2012?
The NIH will consider the suggestion for a teaming website prior to the application receipt date. Currently the plan is for the funded investigators to participate in semi-annual meetings where one of the goals will be to encourage investigators to form alliances to enable the linking of organ systems, if the alliances make scientific sense. During these semi-meetings, it is also possible to interact with DARPA award recipients for possible collaborations. An applicant(s) can also propose several UH2s and link them together during the UH3 phase.

49. How should I structure the research strategy section to better inform review?
Answer 1:  The Research Strategy section of UH2/3 proposals contains four sections unique to this announcement:

  • Description of module development (UH2) and integration (UH3)
  • Microsystem contribution to the evaluation of safety/efficacy/ADME for small molecules and biologics
  • Milestones with organization plans and timelines regarding milestone monitoring/reporting/evaluation
  • IP management plan

Clearly identifying each of these sections with a header will help in the review of your application.
Answer 2: UH2/3 proposals can contain up to 10 different organ systems. A table at the beginning of the Research Strategy session which lists the organ systems that are being proposed for modeling would assist in application review.

 

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FAQs for RFA-RM-10-006 Advancing Regulatory Science through Novel Research and Science-Based Technologies (U01)

  1. What is meant by "Regulatory Science"?
  2. What kind of research and technologies fall under this initiative?
  3. Can an application contain more than one of the five research categories and link to more than one research project?
  4. Is statistical methods development for adaptive designs or small clinical trials included within the scope of this FOA?
  5. Is this limited to pre-licensure safety, or is FDA and NIH interested in systems that might assist post-licensure safety evaluation on various events that occur with medication use?
  6. What are the points to consider when submitting an application in response to this FOA RFA-RM-10-006?
  7. Do I have to submit a letter of intent? What should be in it? How should I send it?
  8. What are the key differences in submitting an application in response to this FOA?
  9. When is the receipt date for sending in the application?
  10. Can I submit more than one application in response to this FOA?
  11. How will applications be reviewed?
  12. Will I have an opportunity to submit supplementary information before my application is reviewed?
  13. Will my application(s) be reviewed by experts in my field?
  14. What sort of milestones will be expected for these proposals?
  15. How many meetings will project PIs/co-PIs be required to attend each year?
  16. Will site visits be conducted?
  17. I am an NIH intramural investigator. Am I eligible to apply?
  18. I am an FDA intramural investigator. Am I eligible to apply?
  19. Can an FDA scientist be part of my research team, as a collaborator or consultant? If so, what are allowable costs?
  20. I am not a US citizen. Am I eligible to apply?
  21. My institution is not in the US. Am I eligible to apply?
  22. Can work be performed outside the US?
  23. Is it open to commercial, for-profit companies? Is there a benefit to having industry partners?
  24. Will work within this grant restrict or constrain further commercial efforts by my firm? What are the "terms" with regard to the promotion and use of any technology that my firm would bring to this project or develop?
  25. Can additional PIs be added during the course of the Project Period?
  26. Can changes be made to the Research Plan during the Project as new information is obtained?
  27. How will the FDA and NIH representatives be assigned to particular Projects?
  28. Is there a possibility for applying for continued support after 3 years to facilitate continued sharing of new reagents and models generated under this FOA?
  29. What are the plans for implementation and deployment of the results from this program?
     

1. What is meant by "Regulatory Science"?
Regulatory science is for the purposes of this FOA: RFA-RM-10-006 a specialized and interdisciplinary area of biomedical research that serves to generate new knowledge and tools for assessing experimental therapies, preventives, and diagnostics. This regulatory science initiative will foster the development, evaluation and availability of new or improved tools, methods, standards, and applied science that support a better understanding and improved evaluation of product safety, quality, effectiveness, and manufacturing throughout the product life cycle. Advances in this area depend on the incorporation of cutting-edge science and evidence-based knowledge into regulatory decision making. The research supported through this initiative should add to the scientific knowledge base by providing new methods, models or technologies that will inform the scientific and regulatory community about better approaches to evaluating safety and efficacy in medical product development.

2. What kind of research and technologies fall under this initiative?
Please see Section I under Research Objectives of the FOA RFA-RM-10-006. Basically the Research Scope includes the following in no order of priority: (1) New tools and methodologies for assessing medical product safety and efficacy (including drugs, biologics, and devices and point of care diagnostics); (2) Novel information technologies and statistical models that can improve product evaluation and inform regulatory decisions; (3) Strategic design of research in “omics” and systems biology to better inform regulatory decision-making and support product development; (4) Research on rare diseases/small sample size populations; and (5) Novel approaches addressing optimal study designs for clinical trials. This initiative will contribute to the overall goals of improving regulatory science by supporting research in at least one area of medical product development ranging from in vitro and in vivo product characterization and evaluation through clinical studies and to a manufactured, approved product.

3. Can an application contain more than one of the five research categories and link to more than one research project?
Yes.

4. Is statistical methods development for adaptive designs or small clinical trials included within the scope of this RFA?
Yes. See Section I of the FOA: RFA-RM-10-006 under Research Objectives for Research Scope. The projected outcome should be evidence-based and the results should have the potential to solve the identified problem and create significant value in informing the product evaluation pathway and regulatory decision-making process.

5. Is this limited to pre-licensure safety, or is FDA and NIH interested in systems that might assist post-licensure safety evaluation on various events that occur with medication use?
The research scope is not limited to pre-licensure activities but post-market safety assessment is also an important area of research.

6. What are the points to consider when submitting an application in response to FOA RFA-RM-10-006?
The research supported through this initiative should add to the scientific knowledge base by providing new methods, models or technologies that will inform the scientific and regulatory community about better approaches to evaluating safety and efficacy in medical product development.

  • Does the research address an important area of regulatory science and will it inform future medical product development and regulatory decision making?
  • Will the expected results lead to advances in technologies used in the regulatory review of medical products (including drugs, biologics, and devices), and thus improvements regulatory practice and public health?
  • Will the new approach or methodology have a competitive advantage over existing approaches?
  • Does the proposed research address an unmet area in regulatory science?
  • Does the research outcome have the potential to solve the identified problem and create significant value in informing the product evaluation pathway and regulatory decision-making process?

The research supported through this initiative should add to the scientific knowledge base by providing new methods, models or technologies that will inform the scientific and regulatory community about better approaches to evaluating safety and efficacy in medical product development.

  • Does the research address an important area of regulatory science and will it inform future medical product development and regulatory decision making?
  • Will the expected results lead to advances in technologies used in the regulatory review of medical products (including drugs, biologics, and devices), and thus improvements regulatory practice and public health?
  • Will the new approach or methodology have a competitive advantage over existing approaches?
  • Does the proposed research address an unmet area in regulatory science?
  • Does the research outcome have the potential to solve the identified problem and create significant value in informing the product evaluation pathway and regulatory decision-making process?

7. Do I have to submit a letter of intent? What should be in it? How should I send it?
Prospective applicants are asked to submit a letter of intent that includes the following information:

  • Descriptive title of proposed research
  • Name, address, and telephone number of the Principal Investigator
  • Names of other key personnel
  • Participating institutions
  • Number and title of this funding opportunity

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 NIH CSR to estimate the potential review workload and plan the review. Letters of intent can be e-mailed, faxed, or sent as hard copy (e-mail is preferred). There is no need to send hard copy of a letter that has already been sent via e-mail or fax. All letters of intent should be sent to Dr. Danilo Tagle at tagled@ninds.nih.gov.

8. What are the key differences in submitting an application in response to this FOA?
Applications will be submitted using the new version of the paper PHS 398 application form. The new application form and instructions can be downloaded from http://grants.nih.gov/grants/forms.htm. One key change concerns the bio-sketch which is required of all participants on the project. The bio-sketch contains a personal statement section, where you can elaborate on why you are particularly qualified to do what you are supposed to do. Another key change is in the 12 page limit to the application. Appendix material should be submitted on CD, and can include no more than 3 manuscripts in-press. The applications need not be hypothesis driven, however it is important to have strong development plan which include well defined measures of progress, how to validate success and how it compares to other resources that are available and underdeveloped.

9. When is the receipt date for sending in the application?
The application receipt date is April 27, 2010. This is the receipt date and not a postmarked date. The application must be received by the Division of Receive & Referral of CSR on or before that date. There is no extension to that deadline. If you have served recently as a reviewer or advisory council member, normally you get an extension to your own submission deadline; however, extensions do not apply for this RFA.

10. Can I submit more than one application in response to this FOA?
Yes, as long as they are non-overlapping and scientifically distinct.

11. How will applications be reviewed?
Applications that are complete and responsive to the FOA will be evaluated for scientific and technical merit by a Special Emphasis Panel (SEP) convened by the Center for Scientific Review (CSR). Reviews most likely will take place during the 2nd week of July.

12. Will I have an opportunity to submit supplementary information before my application is reviewed?
Supplementary information can be submitted to Dr. Joseph Rudolph (Joseph.Rudolph@nih.gov) no less than 30 days prior to review. There is a two-page limit on the supplementary information provided.

13. Will my application(s) be reviewed by experts in my field?
Every effort will be made to ensure appropriate reviewer expertise; however, it is likely that not all of the reviewers assigned to your application will be considered experts in your specific field. We anticipate that investigators will submit applications on a wide range of topics. The SEP reviewers will be drawn from academia and industry, and have general expertise that is relevant to your application. It is extremely important to keep this in mind when you are writing your application. When describing what you want to do, avoid jargon, and use language that scientists in other fields can understand. You will also have to convince scientists in other fields that what you are proposing to do is exciting and exceptionally innovative or important to the field, and that the proposed research will have a profound impact. Please see section V of RFA regarding review criteria.

14. What sort of milestones will be expected for these proposals?
The use of milestones should provide clear indicators of a project's continued success or emergent difficulties. Milestones are different from specific aims. The milestones must provide objective and quantitative outcomes by which to justify advancing the project. The application must include a strong rationale for the choice of models, parameters, and quantitative go/no-go decisions to be made by NIH staff, based upon accepted practices in the specific field.

15. How many meetings will project PIs/co-PIs be required to attend each year?
Project PIs/co-PIs should budget for two face-to-face meetings during the course of the 3 year award.

16. Will site visits be conducted
It is possible that site visits will be conducted on a periodic basis as part of monitoring the progress of awarded grants.

17. I am an NIH intramural investigator. Am I eligible to apply?
Yes. Please see special application instructions under Section IV.2. “Applications involving Federal Agencies” of the FOA.

18. I am an FDA intramural investigator. Am I eligible to apply?
You would not qualify as a PI, but could be a collaborator. Because FDA researchers already carry out research relevant to regulatory science, it is unlikely that the project would be one outside of the work normally funded by the FDA. Please see: http://grants.nih.gov/grants/policy/nihgps_2010/nihgps_ch17.htm.

In general within PHS, there has to be exceptional circumstances in order to allow support funding for an intramural project. It must clearly define and demonstrate that the proposed project is something outside of what is normally conducted under the intramural funding of an FDA investigator.

19. Can an FDA scientist be part of my research team, as a collaborator or consultant? If so, what are allowable costs?
Yes, assuming the proper approvals have been received by the scientist from the FDA leadership. However since FDA is a PHS agency, no funds can be provided unless the project triggers "exceptional circumstances" as noted in the NIH GPS.

20. I am not a US citizen. Am I eligible to apply?
Yes, if your place of business / institution is located within the US.

21. My institution is not in the US. Am I eligible to apply?
Investigators at non-US (foreign) institutions are not eligible to apply.

22. Can work be performed outside the US?
Yes, as a subcontract to the US-based institution.

23. Is it open to commercial, for-profit companies? Is there a benefit to having industry partners?
Yes, for-profit organizations are eligible to apply or serve as collaborators. The needs and scope of the proposed project should dictate and strongly justify the field of collaborators needed to accomplish the task. In general, the NIH highly encourages partnership with industry.

24. Will work within this grant restrict or constrain further commercial efforts by my firm? What are the "terms" with regard to the promotion and use of any technology that my firm would bring to this project or develop?
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.

25. Can additional PIs be added during the course of the Project Period?
Yes. Addition of  new key personnel is subject to approval by NIH and should not result in any increase in the approved budget.

26. Can changes be made to the Research Plan during the Project as new information is obtained?
Since this is a cooperative agreement, the NIH and FDA will work with the investigators if minor changes to the research approach have to be made during the course of the project. However the research plan has to remain true to what was peer-reviewed.

27. How will the FDA and NIH representatives be assigned to particular Projects?
Dr. Kathy Kopnisky is the designated NIH Project Coordinator, Dr. Danilo Tagle is the Program Officer and Dr. Vicki Seyfert-Margolis from the FDA is the key FDA staff member involved in the Regulatory Science program. Please see section VI 2A of the FOA regarding Roles and Responsibilities.

28. Is there a possibility for applying for continued support after 3 years to facilitate continued sharing of new reagents and models generated under this FOA?
This Regulatory Science program is currently funded for a 3 year period only.

29. What are the plans for implementation and deployment of the results from this program?
Awardee is encouraged to publish and publicly release and disseminate results, data and other products of the study, concordant with the study protocol and governance and the approved plan for making data and materials available to the scientific community and NIH. Awardee will work with the appropriate NIH and FDA individuals (if the NIH Program Officer conveys a NIH/FDA need) to develop and implement an appropriate rapid data release policy. The specific path for knowledge transfer to the FDA will be developed during the course of this program.

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