We represent healthcare systems leaders and researchers who have partnered with the NIH Collaboratory Demonstration Projects. Through our experience, we have learned that the successful conduct of ePCTs requires shared vision and priorities, continuous communication and commitment, and, frequently, compromise. Our experience has led us to develop this section with recommendations for investigators who wish to conduct ePCTs.
The job of a healthcare system is to efficiently and affordably deliver high-quality healthcare to patient populations. Pragmatic clinical trials offer an opportunity to combine the work of caring for patients with the generation of real-world evidence that could improve the quality of care for patients in a reliable way. This may help create a cycle that leads to continual learning, i.e., the learning health system (Committee on the Learning Health Care System in America 2013). However, the conduct of research is an embellishment of the mission of most healthcare systems, not a primary obligation, and participation is voluntary.
“The purpose of the healthcare system is not to do research, but to provide good healthcare. Researchers often have a tail-wagging-the-dog problem. We assume that if we think something is a good idea, the healthcare system will too … We need to remember that we’re the tail and the healthcare system is the dog.” —Greg Simon, MD, MPH (PI of SPOT)
Healthcare systems have constrained bandwidth, and participating in learning activities involves significant costs and challenges: there are direct costs but also intangible costs, which can be substantial, such as personnel time, IT time, distraction of clinical staff, and the potential for supply chain issues (Sands 2018). Additionally, when partnering with researchers, it takes time to define relationships and develop trust; the process can be complex, lengthy, and costly (i.e., legal costs). Participating in an ePCT may also involve NOT participating in competing interventions or other quality improvement activities or practice changes. If operational and research timelines fall out of sync, communication and compromise is often necessary. Finally, for multi-site studies, communication between and within organizations can be challenging, as these delivery systems each have site-specific, complex communication structures, and often, differing priorities.
“We have to think about how the research that we will do will either interfere with or add value to the health system. Ideally, we would like to leave something behind to the people who have helped us, and to do that, we really need to understand the values, priorities, and expectations of the healthcare systems, and to realize that the people we are talking to about pragmatic clinical trials often have bigger fish to fry.” —Greg Simon, MD, MPH from the January 2020 Collaboratory Grand Rounds: What is a Pragmatic Trial and How do I Start?
Because of these challenges, the successful design and conduct of ePCTs is predicated on a strong partnership between clinical researchers and delivery systems stakeholders. In Real-World Advice for Generating Real-World Evidence, we establish 5 principles of partnership (Sands et al. 2019).
|5 Principles of Partnership
|Establish and maintain a durable partnership||This partnership should involve an empowered champion to provide enterprise leadership throughout the course of the study. Participation must provide benefit for each collaborating organization. Credit (such as authorship and presentations) and intellectual property should be equitably shared and determined ahead of time.|
|Select research topics of mutual interest||A research question that will interest a healthcare system must be clinically, operationally, or strategically important and provide value to the healthcare system, such as providing an adoptable solution to a problem or clarifying alternatives between different treatments or therapies.|
|Give precedence to operational imperatives||The activity should be largely transparent or similar to normal workflow and inserted into operational cadence; the schedule cannot be driven by research timing. Investigators may need to accommodate changing institutional priorities.|
|Recognize that data security is essential||Security systems should avoid transfer or duplication of data.|
|Build data systems for learning healthcare||Use standards that promote interoperability and aggregation of data, such as the CMS requirements for meaningful use.|
For healthcare system partners, the idea of improvement through systematic and scientific thinking is part of the value of participating. Healthcare systems leaders who volunteer to conduct embedded research may also participate for a variety of other reasons, including:
- Evidence generation is for the greater good and may improve the care of patients
- Early access to new knowledge of best practices
- Research is in keeping with the mission of the healthcare system
- The conduct of research is a market differentiator and/or a reputation builder
- As a strategy for physician recruitment
- As part of performance improvement initiatives/formal evaluation
- Core to being a learning healthcare system
- Research an important activity within the domain of corporate responsibility and advocacy
- Research is an extension of a commitment to “evidence based medicine.” (Sands et al. 2019)
When the National Academy of Medicine advanced the idea of a Learning Health System over a decade ago, they stated that a “strong synchrony of efforts” would be required between healthcare system stakeholders and clinical researchers (Committee on the Learning Health Care System in America 2013). If the partnership is symmetric, and “strongly synchronized,” then the embedded pragmatic research should be a “win-win”; it should feel equitable and be broadly perceived as such.
Watch a video where Dr. Greg Simon is tasked with convincing healthcare system leadership to invest in implementing the intervention from the Suicide Prevention Outreach Trial (SPOT). In this hypothetical situation, the trial is complete and the the intervention reduced suicide attempts in at-risk populations.
Committee on the Learning Health Care System in America, Institute of Medicine. 2013. Best Care at Lower Cost: The Path to Continuously Learning Health Care in America. Smith M, Saunders R, Stuckhardt L, McGinnis JM, editors. Washington (DC): National Academies Press (US). http://www.ncbi.nlm.nih.gov/books/NBK207225/. Accessed January 28, 2015.
Sands K, Platt R, Perlin JB. 2019 Sep 4. Real World Advice for Generating Real World Evidence. NEJM Catalyst. https://catalyst.nejm.org/doi/full/10.1056/CAT.19.0621.
Sands, Kenneth. 2018. Partnering with Stakeholders: Keys to Success. https://dcricollab.dcri.duke.edu/sites/NIHKR/KR/Panel-1-Combined.pdf Accessed February 26, 2020.