The Duke Margolis Center for Health Policy and U.S. Food and Drug Administration (FDA) recently hosted a public workshop to discuss how to effectively use real-world evidence (RWE) – the insights gleaned from information collected outside of a clinical trial, such as electronic medical records—in drug development and regulatory decision-making. PCORI and its innovative initiative, PCORnet, the National Patient-Centered Clinical Research Network, were front and center at the event, with several of our renowned experts sharing how PCORnet is ready to put RWE practices into action and propel healthcare forward.
The opportunity: a living textbook of medicinal products
With RWE, there is a massive opportunity to achieve a totality of information that a single study or trial will never allow, said Dr. Adrian Hernandez, director, Health Services and Outcomes Research, Duke Clinical Research Institute (DCRI) and panel participant at the workshop. He added that by partnering with the FDA and other stakeholders to develop the right framework for using real-world data (RWD) such as electronic medical records, we can more effectively use RWE to complement other kinds of research and create a living textbook about medicinal products and the experiences of patients who use them.
Dr. Hernandez was a panelist at the workshop’s session that spotlighted matching RWE to regulatory use cases. To illustrate how RWE can fill crucial knowledge gaps, he used a personal example: his own mother, a premature coronary disease patient who wants to help other patients like her but finds clinical trials inconvenient for her lifestyle. Dr. Hernandez explained how pragmatic clinical trials like PCORnet’s ADAPTABLE study are engaging participants electronically to better suit patients’ lifestyles and needs.
According to Dr. Hernandez, another key element to effectively leverage RWE lies in understanding the benefit to risk ratio. By creating a parallel system that addresses safety and benefits that matter to patients (e.g. will this improve my quality of life, not just length of life?), we can create a learning healthcare system where patients, clinicians, healthcare systems, and regulators use RWE to generate outcomes that matter most to patients.
Joe Selby, executive director, PCORI, also spoke at the workshop, discussing how to chart a collective path as we move forward with the use of RWD and RWE. He shared key elements he believes are essential to the future success of RWE.
First, he touched on the importance of data linkage and bringing stakeholders and partners together to understand data. He also discussed the need for a societal change. Patients must become aware of barriers to improving their care and have platforms where they can use their own voices to solve these issues.
Dr. Selby also stressed that we aren’t going to learn anything unless we actually dive in and start doing research. Take, for example, PCORnet demonstration studies. Aimed to answer critical research questions, demonstration studies give researchers a chance to compile a list of learnings and trail blaze a new path for collecting data. PCORI is currently funding several of these demonstration studies.
Dr. Selby also touched on standardization and the need to bring stakeholders together to discuss measures that haven’t yet been incorporated into any common data model. He closed with a mention of the need to develop an infrastructure to conduct pragmatic trials in the real world, something that PCORnet and its teams have in their sights for the coming years.
By closing gaps in data, we will close gaps in care
Dr. Kevin Haynes, clinical epidemiologist in translational research for HealthCore, a PCORnet Health Plan Research Network, was a panelist at the workshop highlighting the development of fit-for-purpose RWD. Throughout his discussion, he stuck to a common theme: “We need to close the gaps in data necessary to close the gaps in evidence and ultimately gaps in care.”
We need to close the gaps in
data necessary to close the gaps
in evidence and ultimately gaps in care.
Dr. Kevin Haynes – Clinical Epidemiologist, HealthCore
Dr. Haynes stressed the need to integrate data, and he proposed two forms of data that must be linked: longitudinal data, such as Medicare data, and the data that is held deep inside a clinical system over defined periods of time that is unavailable to today’s administrative claims. HealthCore is one of two PCORI-funded health plan networks partnering with PCORnet’s CDRNs and PPRNs to take the lead in developing a stepwise approach toward successful linkages of claims and EHR data.
You can find additional information about the regulatory use of real-world evidence in this white paper, which both Adrian Hernandez and Richard Platt, co-Principal Investigator of PCORnet’s Coordinating Center, helped compose as contributing authors.
Were you unable to catch the event? Watch the webinar recording and see what you missed!
PCORnet, the National Patient-Centered Clinical Research Network, is an innovative initiative of the Patient-Centered Outcomes Research Institute (PCORI). The goal of PCORnet is to improve the nation’s capacity to conduct clinical research by creating a large, highly representative network that directly involves patients in the development and execution of research. More information is available at www.pcornet.org.
The Patient-Centered Outcomes Research Institute (PCORI) is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions. PCORI is committed to continually seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.