1. Preliminary Work Supporting an R01 Research Grant Donna Z. Bliss, PhD, RN, FAAN Professor Director, Pilot and Feasibility Study Core Center for Health Trajectory Research

2. Objectives At the end of this session, the participant will be able to: Describe the purposes of the preliminary studies section of and NIH grant Compare changes in NIH grant instructions regarding preliminary studies as of Jan 2010 Identify strategic content to be highlighted in report of preliminary studies in an R01 grant

3. Preliminary Studies Prior work of PI and Co-Is that supports the proposed study Especially important for –new investigators or those entering a new area and who are submitting an R01 –Highly innovative, ambitious proposals Findings of previous full, complete studies Pilot/partial work: feasibility, instrument testing etc.

4. Preliminary Studies Purpose Demonstrate expertise to conduct proposed work Demonstrate experience to successfully complete proposed work Direct hypothesis Groundwork* is completed PI knows how to do/is capable of research –Shows rigor of PI’s work, thoughtful preparation, commitment to topic Implies team can function effectively Inouye & Fiellin, 2005, Ann Int Med Bliss et al., 2000, Nutr Clin Pract

5. In the Old Days Separate Section (C) for Preliminary Studies No specific page limit 1-2 pages generally Content Summary and data Funding source Investigators involved Citation for article or abstract

6. Explicit vs. Implicit Balance of space and anticipated questions/concerns of reviewers New PI recommended to be more explicit

7. Groundwork Strategic Sample Info People eligible and targeted by study are available to meet power analysis requirements Recruitment and enrollment strategies are productive to achieve sample within proposed study period; not limited by eligibility criteria Collaborations for recruitment, services (e.g., lab measures) have been established and are functional Effect size, sample size, power are determined Random assignment is acceptable Estimates of attrition are reasonable

8. Groundwork Instruments are valid and reliable for population Resources and environment are optimal and supportive You can support your hypothesis You have your own data/experiential knowledge re: research question

9. Groundwork Pilot Studies Intervention is Feasible Reproductive Standardized Control group is appropriate Procedures are “worked out” Know most confounders and they are minimized Intervention fidelity possible Culturally acceptable Realistic time frame, budget Inouye & Fiellin, 2005 Ann Int Med

10. Preliminary Studies Include analysis data –Figures, charts, tables are helpful Analyze meaning of findings and relevance to proposed study –Address what was added, modified, dropped and why for current proposal

11. Groundwork Team Cover necessary areas with sufficient expertise “Get along” and have complementary expertise Theoretical, Analytical capability Published together Ongoing record of accomplishments Type of journal, recency, productivity

12. Preliminary Studies Opportunities Discuss lessons learned How overcome anticipated limitations/pitfalls Can articulate alternatives if problems occur (and they will) Experience PI gained Reference point to compare innovation of new proposal Prior funding shows interest of others in your work and your capability

13. Common Negative Critique of Reviewers Preliminary studies inadequately described Lack of attention to specifics of study No data included Lacking Unrelated to proposed study Inouye & Fiellin, 2005, Ann Int Med Bliss et al., 2000, Nutr Clin Pract

14. Sample Reviews Team This is a very large team of experienced investigators and outstanding consultants, which is warranted by the complexity of the study. The team has numerous grants and publications in the areas The PI is a highly productive investigator with extensive relevant clinical and research experience in the area. She has an international reputation in the area. PI is very well qualified to direct the proposed study. The PI is not an expert in this area but has however enlisted the help of numerous authorities in this area. The investigators have a wealth of experience conducting large- scale and multi-site studies in ethnically/racially and socioeconomically diverse communities and are widely recognized as leading researchers in the area. The investigator is highly experienced in conducting this type of research. She has assembled one of the most highly productive and respected teams in the country.

15. Sample Reviews Team Although a new investigator, she has a great deal of experience doing research with similar samples. PI is an accomplished investigator with appropriate background to conduct the work and the outstanding team is composed of senior investigators with experience directly applicable to the proposed project. Roles are well delineated and justified. The investigative team has broad expertise in the areas. Co-Investigator and statistician have extensive experience and requisite expertise in analyses proposed in this study. The roles of other Co-Investigators are well described and highly relevant to the specific aims. The track records of these investigators and the extensive targeted pilot data is extremely impressive. She has an outstanding bio-sketch for a young investigator and is clearly capable of conducting these studies. She has excellent support from a variety of outstanding co-investigators

16. Sample Reviews Environment The environment is an excellent site for the proposed study, affording many resources in support of the work. Research resources available at the applicant institution are very well described and are outstanding in the context of the scope and needs of the proposed study. There is substantial research infrastructure to support this work. The community network is developed to the extent that the aims of the project can be achieved. The investigator has forged the partnerships necessary with community agencies and referral networks to assure participant recruitment and retention. The environment and the team assembled by the PI are excellent. The environment to conduct these studies at the University of Minnesota and affiliated entities is outstanding. University of Minnesota is at the forefront of research on this topic.

17. Sample Reviews Prior Studies The investigator reports a number of prior studies using these sites demonstrating expected co-operation from them. The conceptual framework, design, methods, and analyses are well integrated and appropriate to the aims of the project. The PI now has adequately referenced prior investigations demonstrating the effect of the intervention. Formative research from the pilot project provides valuable information on the needs and wishes of participants. Results from the pilot project support the acceptability and feasibility of the intervention. Aims build logically from clear gaps in the literature, an explicit theoretical model, extensive preliminary work, and the expertise of the PI and collaborating investigators. They have pilot data demonstrating they can conduct this type of trial, and all the needed expertise and infrastructure in place to conduct the proposed studies. They have been especially sensitive to issues related to subject/patient burden, and have an outstanding strategy to reduce the overall burden.

18. New NIH Instructions Research Plan (Section D) replaced by Research Strategy (R01 = 12 pages, R21/R03 = 6 pages) Significance Innovation Approach Review Criteria: overall impact, significance, investigators, approach, environment

19. New NIH Instructions “Discuss preliminary studies as part of the Approach or integrate into any of 3 main sections” For latter, mark info clearly Preliminary data can be an essential part of a research grant application and help to establish the likelihood of success of the proposed project” “Reviewers use preliminary studies to assess your competence”

20. Guidance/Advice from NIH Consider streamlining, graphics, references Publications are important Limit of 3 publications not publicly available or accepted but not yet published in Appendix

21. Guidance/Advice from NIH Give detail For unique or new methods Convince reviewers you have enough resources and expertise If new PI, show you “can handle” an experimental method Show you (PI and/or CoIs) used methods before if no article publications

22. Preliminary Studies Additional New NIH Guidance Highlight successes of related grants Interpret findings critically The more complex the project, the more data you will need Need adequate data to support feasibility of innovation (too much innovation is not always better) Will particularly risky aspects be managed Tell how early work prepared you for proposed work

23. Preliminary Studies Additional New NIH Guidance Investigators Ongoing track record of advancing their field Appropriate expertise and training Environment Are institutional support, equipment and other resources adequate

24. Recommendations Preliminary Work Plan ahead; view preliminary work in context of current studies and include parts that support future, larger grant Address as many PARTS of proposal as possible –In approach section, ask yourself how you know the basis for what you propose –The more you can support approach using data from your own work, the better Build and work with your team Acquire and explain how you will utilize expertise from prior work Publish prior work

25. Recommendations In proposal Be strategic in content to include Anticipate reviewers’ questions and address Emphasize strengths and successes Tailor your emphasis to your situation; use detail as needed Citing your own publications from recent preliminary work is strong Be succinct and clear