PHS 398 (Rev. 9/04), Continuation Page s2
Principal Investigator/Program Director (Last, First, Middle): Mulshine, James Lawrence
F.2.6. Pilot and Collaborative Translational and Clinical Studies
F.2.6. A. Introduction. Academic medical centers (AMC) have traditionally been the incubator for the development of clinician-scientists and translational researchers. Current trends in clinical revenue streams, GME funding and research regulation have imperiled this aspect of the AMCs mission. Thus, it is fitting that a major emphasis in the Clinical Translational Science Award (CTSA) is to rejuvenate and prioritize this critically important activity.
Rush University Medical Center (Rush) has historically recognized the key role that such seed funding could provide and for many years has provided such support through small competitive grants ($10,000 to $20,000 per year) from the University Committee on Research and the Rush/John H. Stroger Jr. Hospital of Cook County Collaborative Research Program.
More robust funding mechanisms at Rush are essential to meet the demands of the current research climate. In addition, there is a broad realization that the existing process for administering institutional grants needs reengineering to create a new culture of accountability, transparency and achievement.
In the process of submitting our initial first round CTSA application the leadership of Rush understood the importance of these goals and set aside $500 thousand per year to fund a new category of translational two-year pilot projects for exceptionally promising translational research teams and programs. These funds were distributed in a competitive process in October, 2006 in advance of the review of our initial CTSA application, attesting to the strong institutional commitment to the promotion of translational research. In October 2008, a second round of two-year pilot projects were awarded and in October, 2009 a third round of one-year pilot projects will be awarded. The processes and procedures developed and subsequently refined to distribute these pilot funds serve as the basis for the program proposed in this CTSA application. In addition, the structure established for the distribution of these pilot funds and rigorous peer review was extended not only to the extant mechanisms of seed funding at Rush mentioned above, but also to targeted philanthropic initiatives including the Brian Piccolo/Gavers Cancer Research Fund, the Segal Foundation Research Award (women’s cancer), Bears Care (breast cancer research), and the Hasterlik Memorial Fund (capital equipment) (Table F.2.6.1.). As a result, during the 2007 - 2009 fiscal years, over $3.1 million in institutional funds were invested in pilot research projects. In addition, to foster collaborative interactions with our RTSC partner the Illinois Institute of Technology (IIT), a Request for Applications was issued in October 2009 for a one-year pilot project involving inter-institutional and interdisciplinary translational research. Each institution has set aside $25 thousand to award up to two projects. To be eligible for this award, at least one of the applicant PI’s must hold a primary faculty appointment at Rush or IIT (Assistant Professor or above) and at least one co-investigator should hold a primary faculty position at the partner institution.
In this CTSA application, a leveraging of the core RUSH pilot funding in translational research projects is requested.
F.2.6. B. Specific Aims
- Specific Aim #1: To support high quality, interdisciplinary translational studies that will serve as a nidus for sustainable research programs of the RTSC.
- Specific Aim #2: To identify promising early-career, health care researchers and provide support for their transition to scientific independence in translational research.
- Specific Aim #3: To build translational disease-focused research teams which collectively cover the spectrum of disciplines from basic research to community interventions.
F.2.6. C. Criteria for Funding Pilot Projects. The Scientific Leadership Council (SLC) of the RTSC is responsible for allocating research funds for promising pilot projects. Members of the SLC are accomplished in their field as validated by NIH grant awards or comparable designations. As such, they have served on NIH Study Sections and Review Panels and are familiar with the NIH criteria for evaluating research grant applications. Therefore, the SLC will adopt similar rigor for the review of pilot project proposals. A six-member subcommittee appointed by the SLC (the Pilot Project Evaluation Team, consisting of two basic researchers, two clinical researchers and two community-based researchers each of which will have two-year terms) containing experienced researchers/peer reviewers will be responsible for the selection of pilot projects to fund. One member of this team will serve as a liaison to the Community Advisory Board to insure that there is input from this body in the selection of the pilot projects. Ad hoc members of the team will be appointed if additional expertise is needed to evaluate a particular proposal.
TABLE F.2.6.1. Rush University Medical Center Intramural Research Grant Mechanisms
AWARD / ELIGIBILITY / AMOUNT/YEAR / NUMBER / DURATION / AMOUNT AWARDED FY07 - FY09Annual
RTSC Pilot Award / Member of RTSC / $25,000 – 100,000 / 4 – 7* / 1 – 2 Years / $1,422,622
University Committee on Research Award / Young Investigators / $20,000 / 5 – 6 / 1 Year / $319,724
Rush/Stroger Hospital / Collaborative Resarch Team from Both Institutions; Young Investigator / $10,000 / 2 – 3 / 1 Year / $123,840
Episodic/Philantropic
Hasterlik Memorial Fund / Capital Equipment / Up to $250,000 / 6 / Not Applicable / $1,207,689
Brian Piccolo/Gavers Cancer Fund / Breast Cancer / Up to $50,000 / 3 / 3 Years / $376,982
Segal Foundation / Women’s Cancer / Up to $50,000 / 1 - 3 / 3 Years / $398,909
Bears Care / Breast/Ovarian Cancer / $65,000 - $235,000 / 4 – 6 / 1 Year / $130,020
Haffner Fund / Nursing Faculty / $10,000 / Up to 5 / 1 year / $2,500
* This number will increase if this application is successfully funded
Specifically, the Pilot Project Evaluation Team (PPET) evaluates the significance and strategic value of the proposed project vis-à-vis the research portfolio of the RTSC; (i.e. the team will insure that the project is synergistic with ongoing research and addresses an important clinical problem that will advance scientific knowledge or clinical practice in terms of new technologies, treatments, preventions or interventions). The PPET will evaluate the approach to insure that the design, methods and analyses are appropriate to the aims of the project. The review also weighs innovation in hypotheses, methods, concepts, clinical paradigms and technology in the assessment of the proposed study.
The quality of the investigators is a key element in the evaluation of pilot proposals. The review team assesses whether the applicants are appropriately trained to carry out the research. The previous track record of the applicant is scrutinized to determine the likelihood of achieving extramural funding, particularly NIH R01 awards, for the project. The involvement of a full spectrum interdisciplinary team of investigators including basic, clinical and community-based investigators is encouraged to insure the clinical relevance and scientific rigor of the study. Finally, the scientific environment is assessed. Particular attention is paid to departmental support, including protected time for research activities, the availability of mentors and collaborators and the availability of necessary laboratory and/or clinical research infrastructure. Through the Request for Applications process, investigators will be encouraged to creatively leverage the infrastructure available in the Translational Technology and Resources Core (TTRC, section F.2.5) as well as the seed funding mechanisms described for the TTRC, the translational methodology development grants program and the “mini” sabbatical program (Section F.2.4,E.2. and F.2.4.E.3, Table F.2.6.4).
Priority will be given to projects that involve interwoven training or mentoring opportunities. The RTSC recognizes the critical importance of mentorship in the pathway to the development of independent investigators and, for the past two years, has distributed funds to allow key senior faculty the protected time necessary to mentor the most promising young investigators. The expectation with this process is to prepare talented investigators qualified for appointment to the K12 program (if the present application is successfully funded) and success in competition for RTSC-funded pilot projects.
To be eligible for funding, applicants are members of the RTSC, enrolled in or having completed the Masters in Clinical Research Program. Other young faculty with a proven track record in translational research are also eligible. In the review, preference is given to promising young investigators; to mature investigators who are pursuing new lines of translational research in areas that they have not been previously funded; and to promising interdisciplinary translational research with collaborative teams of basic scientists, clinicians and/or a community investigators. To insure a balanced portfolio of translational research, preference will also be given to projects that involve pediatric populations and projects that have a strong component of community-based research. In addition, the SLC actively recruits proposals from women and underrepresented minorities. There will be two types of pilots funded: 1) hypothesis-generating projects for up to $25 thousand for one year; and 2) hypothesis-driven projects for up to $50 thousand for each of two years. Proposals for pilot projects are reviewed and awarded yearly (for the larger, hypothesis-driven projects) or on a continuous basis (hypothesis-generating projects). Continued funding within the award period is contingent upon the demonstration of steady progress in the scientific aims of the project, as well as achievement of training goals. Each will be reviewed every six months by the PPET. Pilot projects under consideration by other granting agencies are eligible for funding within this mechanism. If an external grant is awarded, however, the project will no longer be eligible and any unused funds will be available for other pilot project proposals.
For all proposals, the PPET will insure provisions for the protection of human subjects, inclusion of women and underrepresented minorities, inclusion of children (if appropriate), protection of vertebrate animals and, for clinical trials, when appropriate, the establishment of a data and safety monitoring board. The PPET has an additional review procedure involving an evaluation of the rigor and appropriateness of the statistical methodology. This review is performed by the Design, Biostatistics Core, and Clinical Research Ethics (Section F.2.2). In addition, the PPET will insure that young investigators involved in these projects are enrolled in relevant coursework. The measures of success of these projects are assessed every six months and include the scientific output (national presentations, peer-reviewed publications); advanced degrees awarded (PhDs, Masters in Clinical Research); and ultimately the ability of grantees to establish an independent scientific career, including success in obtaining competitive extramural funding. The SLC annually reviews the overall performance of the pilot project program to ensure a balanced research portfolio and to identify any strategic gaps.
Using the criteria described above and summarized in Table F.2.6.2, the PPET has chosen the pilot studies described below. Rush investigators were asked for proposals that would, if successful:
1. allow clinical and translational trainees or researchers to generate preliminary data for submission of a federal research grant application;
2. seek to improve a process related to conducting translational research;
3. develop new technologies that have a bearing on translational research;
4. lead to the establishment of translational research teams or centers.
There is continued evaluation of the progress of these pilot programs, with particular emphasis on the scientific output and translational significance and plans. As noted above, these programs are evaluated at least every 6 months by the pilot project review team using the criteria enumerated in Table F.2.6.3. To date, all awarded projects have achieved satisfactory progress. Several projects achieved outstanding progress towards the objectives of the mechanism.
The Pilot and Collaborative Translational and Clinical Studies program is now in its third cycle and will be expanded upon successful receipt of this CTSA award. With the addition of institution funds, this allows the RTSC to increase the number of funded translational research projects and interdisciplinary translational research teams.
F.2.6.C.1 Evaluation
Tracking and evaluation of the effectiveness of the pilot project program is discussed in section F.3.2.B in the context of evaluating the progress of the RTSC as a whole. The Chair of the PPET with present quarterly reports to the SLC to update this body on the conformance of the activities to the proposed timeline and the outcomes of the funded projects. Particular attention will be paid to the balance of the distributed resources throughout the RTSC to ensure that pilot funds are being utilized to grow key strategic translational research programs. The effectiveness of the program will be based on quantitative metrics as much as possible including the sustainability of the research project, as measured by extramural grant awards, and the impact of the research on the field, as measured by the quantity and quality (impact factor) of the resulting publications. The outcome of each pilot project will be tracked by the PPET for at least two years since there is often a lag between the conduct of the research and its ultimate publication and/or its conversion into an extramural research grant. Outcomes of the pilot project award program during its early phases will inform the PPET and the SLC regarding the necessity for modifications in the duration and amount of awards, the review process and/or the strategic emphasis in the later phases of the program.
F.2.6.C.2 Cost Sharing
Currently Rush University Medical Center has been investing $500,000 per year in the Pilot Project Program and will continue to do so. In addition, the Illinois Institute of Technology has provided matching funds for projects in which one of the investigators has a primary faculty appointment at IIT. The tracking and evaluation process is intended to provide evidence of the return on investment of these funds to justify enhanced cost sharing with all the institutional partners of the RTSC, particularly IIT and Stroger Hospital.
Table F.2.6.2. Pilot Project Prioritization Review Criteria
Significance
What is the strategic scientific impact and level of overall quality of the project from a translational perspective?
What is the public health impact?
What will be the effect of the study on the concepts, methods, technologies, treatments, services, or preventive interventions that drive this field?
Investigator(s)
Are key personnel appropriately trained and well suited to carry out this work?
Is the proposed work appropriate to the experience level(s) of the PI and other researchers?