Q: The Crohn’s & Colitis Foundation of America (CCFA) announced the launch of a large-scale integrated research platform called “IBD Plexus” in May. Can you explain briefly what this initiative will do?
IBD Plexus is a research and information exchange platform whose goal is to accelerate research and transform the care of patients with inflammatory bowel disease (IBD). IBD Plexus will unite clinicians, patients, academia, and industry to answer questions that are critically important to advance the field of IBD research.
IBD Plexus centralizes patient registries to capture clinical, patient-reported, and linked biosample data, housed in a biobank. It will utilize a centralized analytics lab to conduct omics and expression analysis, a large data management platform to house, organize, link, and disseminate data, and a researcher portal that provides access to aggregate de-identified data sets. The prep-to-research tools will enable hypothesis generation and cohort search and selection, ability to submit research proposals and high performance cloud computing to conduct data analysis, and analytics activities. As of mid-September 2015, contracts have been authorized for data management, biobanking, cloud computing services, and subject enrollment sites to launch this initiative.
Within three years, the platform will contain clinical information and patient-reported data from more than 40,000 IBD patients along with multi-omic and microbial profiles from 7,000 patients who will be followed over time. This has been in the planning stages for two years. IBD Plexus is conceived as an integrated program encompassing numerous vendors and patient centers. We’ve launched the implementation phase just this year with lead support from the Helmsley Charitable Trust and commitment from numerous industry partners. Our partners believe a well-coordinated information exchange will enable the uniting of basic and clinical research and help drive translational research.
Q: What was the need you perceived that this project is intended to address?
IBD researchers and patients need, like many TRAIN participating organizations, a centralized research exchange platform that provides the infrastructure to facilitate important researcher-specific IBD questions and allow for effective collaboration between stakeholders through shared research goals, including accelerating progress toward precision medicine.
We designed IBD Plexus by gathering leading academic and industry experts in the field of IBD and having them help us identify this need and then the infrastructure necessary to meet the need. The Plexus platform will grow and become more powerful as researchers continue to contribute their own findings, enabling early dissemination and utilization by other researchers. Novel data-sharing guidelines will accelerate knowledge sharing through providing a process for expedited data deposition prior to manuscript submission.
In addition, to help overcome intellectual property and ownership issues that have stymied other industry-academic consortiums and cross-institution collaborations, the Helmsley Charitable Trust recognized having CCFA at the helm of this program takes advantage of our unique ability to foster the development of cross-disciplinary research collaborations and forge lasting relationships with academia, industry, and patient communities with a common goal of accelerating discovery and transforming the care of patients.
Q: What have been the biggest challenges thus far in planning and launching this effort? What lessons have you learned?
In order to meet the "team science" objective, we need to get past hurdles that have stymied other consortia and share the data across institutions and industry. Possibly the most critical success factor for IBD Plexus will be ensuring end users trust that the data is of the highest value and is reusable. For the clinical and patient-reported data we will be installing an IBD SmartForm directly into sites' Electronic Medical Record (EMR) system. The form allows clinicians to continue their routine care regimen with minimal disruption, while it also allows sites to capture critical phenotypic data and outcomes and process measures that cannot be captured through ICD 9/10 codes.
To make it as easy as possible for scientists, biosamples will be stored and processed at a centralized biobank where sites will use standard operating procedures for collection. These longitudinally collected reagent grade samples are likely to be highly coveted for a condition like IBD in which disease progression is highly variable.
A challenge that we faced is optimizing use of these samples across our researcher community, specifically changing their mind frame that it's not the physical biosamples that have value but the data derived from the biosamples. We have begun to assess use of a centralized analytic lab to standardize molecular analysis performed on the biosamples. This will not only provide the opportunity to optimize sample use but also ensure that derived data is high quality, reusable, and standardized, and mitigate against batch errors through use of common software, protocols, and normalization techniques.
Q: Are there early successes you can point to?
IBD Plexus will include distinct patient cohorts, listed below. While each cohort is distinctive, we will standardize and link data across multiple cohorts through activities such as creation of a master patient index and creation of a data dictionary and common vocabulary to facilitate integration and data extraction. In addition, when applicable, we will encourage sites to promote enrollment of their patients into the other IBD Plexus cohorts.
- Adult Prospective Clinical Cohort: A new 15-center prospective study targeted to enroll 7,000 patients and collect patient-reported data, clinical data, and biosamples with the primary objectives of finding predictors of response to therapy and relapse.
- Pediatric Risk Stratification Study: An existing study with 1,200 newly diagnosed pediatric Crohn’s patients collecting clinical data and biosamples, with the primary objective to find biomarkers to predict disease severity at time of diagnosis.
- CCFA Partners Patient-Powered Research Network (PPRN): An Internet-based cohort of over 14,000 adult IBD patients and over 800 pediatric patients that collects patient-reported data through validated surveys and patient-generated data collected through mobile health apps and wearables.
- CCFA Quality of Care program: A 30-center initiative targeted to enroll 30,000 patients over the next three years and collect clinical and patient-reported data.
- Real World Evidence Registry: We will leverage CCFA’s Clinical Research Alliance network of 60-plus sites to create an additional prospective cohort focused on collecting safety endpoints to make post-marketing studies more efficient.
Q: What advice would you give other foundations that might be interested in undertaking a similar effort?
When undertaking an initiative such as IBD Plexus, having all stakeholders participate in the planning and design phase is crucial. Over a two-year planning period, from ideation to implementation, CCFA established multiple ways to harness the voice of academia, patients, and industry. We conducted an extensive needs assessment where we interviewed academia, industry, and patients to gain insights and feedback to the governance of the network and ensure solution functionality met their needs. We also established an Industry Affairs work group where we united representatives from seven companies to help inform the design and operations of IBD Plexus, guide roadmap development, advise on policies, and assist in developing the platform's value proposition. We leveraged our PPRN patient governance committee to improve the patient experience and find ways to better reach patients who are interested in research and self-monitoring. In addition, we selected nine academic vanguard sites from our IBD Plexus cohort to aid in development of site contracts, data and biosample sharing strategies, study design, and publication policies. Finally, we worked with all stakeholders to understand how sustainability, income opportunities, and other sources of support could be developed to provide ongoing operational support for Plexus after the build-out is complete.
In addition to understanding your stakeholders, is it important to identify similar initiatives ongoing in your space. This is important when developing long-term strategic and sustainability plans for the initiative and finding opportunities to cultivate additional partnering organizations. We are building out IBD Plexus to not only produce tangible short-term outcomes but also to have the agility to adapt to the expected advances in precision medicine as new technologies arise and new companies form.