Phase 2 Proposal

Phase II, new thoughts.  The strengths of platform studies, exemplified by the STAMPEDE trial, are (1) the presence of a vision at the outset of a long-term research agenda that has the capacity to adapt during its conduct, and (2) a breadth of research questions that could be addressed by a cohesive research group working together to achieve a broad goal. Among the 8 “Big Ideas” applications, 3 target research topics (diabetes, PUMA, radiation health) that could be candidates for this approach and the other applications propose infrastructure that could facilitate it. This effort would benefit from all of the Big Ideas submitted!  

Steve Cummings’ proposal mentions Kirsten's interest in “planning a Center for Public Health (PH) that would work with local communities to provide accurate information on effects of environmental exposures and, potentially, initiate new studies.” Along these lines, I found inspiration at the U of Wisconsin-Madison website describing their “Institute for Research on Poverty,” https://www.irp.wisc.edu/. At the home page, we see their focus is on Wisconsin and on rural poverty; we could provide complementary focus on urban poverty. Under the “people” link, we see that they team with representatives of foundations and the private sector as well as with academics from other institutions -- including UC Davis, UCLA, UCSD, and Stanford; surely they need insights from UCSF biostatisticians and epidemiologists to round out their approaches. Under the “training” link, their 2018 ‘Teaching Poverty 101 Workshop’ provides a lot of valuable background information – indeed, the whole website is valuable.

It would like to see DEB not only conduct research that explains underpinnings of our selected problem – say, Kirsten’s idea – but also studies best approaches to preventing and reducing the problem and works with community partners to implement effective strategies in a sustainable way.

Objectives for Year 1:

1. Reach out to the UWM IRP group to see if we can join them and benefit from their infrastructure and organizational approaches.
2. Establish a working group of interested DEB members to refine target topics, and weigh their feasibility and interest to members, and select a final topic. Identify relevant readings, podcasts, and experts; meet regularly to review and discuss these.
3. Outline a strategic plan for the coming years, identifying many side options (to allow adaptation). Identify personnel needs and emerging leaders within DEB. Identify resources that could provide support.    

Budget:

My initial thought is that at least 3-4 faculty members would have 5%-10% of their time covered to launch this effort.   

Your thoughts on this agenda are most welcome.

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Phase-I submission. While teaching Biostat 226 this year, I was reminded of STAMPEDE, a multi-arm multi-stage randomized controlled trial cancer protocol that was selected as 1 of 2 RCTs of the year by the Society for Clinical Trials. Per doi:10.1093/annonc/mdx410: "...opening to recruitment with five research questions in 2005 and adding in a further five questions over the past 6 years, it has reported survival data on 6 of these 10 RCT questions over the past 2 years [1–3]. Some of these results have been of practice-changing magnitude [4, 5]." It appears that STAMPEDE also caught the eye of HDFCCC leadership, who sent this solicitation on May 10: "As part of a strategic planning process, the Cancer Center is conducting a broad scientific strategic review termed Cancer Research in 2030. Essentially, we are asking each of you to predict what cancer research will look like in 2030, and accordingly, what we will need to do to get there. [...This is] an effort to present scientific gap analyses and white papers to help Center leadership plan resource allocation and program design. I would like to meet with the leadership and members of your Site Committee at an appropriate time..."

Although STAMPEDE is tailored to research questions relevant to cancer that call for randomized assignments, I believe that whether randomization is employed or not, (i) a strategic visionary research plan that encompasses a range of linked individually led studies could move every clinical field forward very effectively, and (ii) DEB faculty could be valuable partners -- for example, with epidemiologists contributing expertise in behavioral measures and interventions and biostatisticians contributing expertise in study design, conduct, and analysis methods (as in STAMPEDE). Matching budgets from partner departments could cover time of faculty developing such an umbrella protocol. 

As we collectively gain experience in developing strategic plans, we also will identify specific areas of our own fields that need further development. Clinical research is moving toward use of Common Data Elements and PROMIS measures (doi: 10.1037/hea0000752); similarly, there is a need for standardization of many behavioral measures and methods to analyze them and need for implementation studies to optimally operationalize the platform protocols (Campbell MK et al, Health Technol Assess. 2007 Nov;11(48):iii, ix-105).

I foresee a potentially wide application of this visionary approach, with DEB faculty partnering with clinical colleagues to accomplish the goals.