When Real-World Evidence met Patient-Centricity
If real-world evidence and patient-centricity teamed up could they cure all of pharma’s ills?
Real-world evidence (RWE) and patient-centricity have a lot in common. Both were concepts that filtered slowly into pharma’s collective consciousness. Each was derided by many, embraced by others, yet they have has since grown to become powerful forces driving companies right across the industry.
Where they meet is an exciting place, says Sandy Leonard, VP, Medical Evidence and Observational Research, AstraZeneca. “From a clinical trial perspective, there has been a lot of interest in two key areas – how RWE can help improve clinical programs and how patient-centricity can ensure our trials are attractive and fit-for-purpose for patients. Yet, at times, they are very much one and the same.”
They share the same ultimate goal, she says – getting treatment options to patients as efficiently and safely as possible. “Patient input and feedback on our clinical trial proposals and concepts is essential, but there are many ways to gather the patient voice – through patient surveys or by convening patient advisory boards for clinical trial design and development.”
Leonard has been looking at some of AZ’s collaborations. “We've looked at both our patient-centric collaborations and some of our RWE collaborations to see if there are projects we can do to together, utilizing electronic medical records (EMR) and registry data and as well as patient-driven data to deliver real insights. There is a huge amount that can be done there.”
Such insights can help to deliver truly patient-centric clinical programs, she says. “The more we use real-world data such as EMRs, the less potential burden on patientspatients and physicians in delivering our clinical as well as medical evidence research programs. Even with some prospective observational studies, there is still the potential that a patient would be asked to do a couple extra PROs or to see a doctor, but if we can use retrospective data to answer those questions, we lessen the burden on patients.”
Patient insight gained through RWE has broad applications, says Leonard. “Real-world data sources allow us to ask the questions that matter at different points across the product lifecycle. At the beginning, in early development and with business development opportunities, RWE can have a significant impact as we ask important questions like, What is the disease epidemiology? What is the unmet medical need? Answering these questions with RWE allows us to determine, in a world of limited resources, where to best invest our research dollars [and] where we will have the greatest opportunity to deliver an innovative therapy to truly impact patient outcomes.”
Greater understanding of patient experiences and attitudes can deliver huge productivity improvements in clinical development and provide exceptional value by adding depth and context to RWE. “As you go through the lifecycle, using RWE to understand the patient’s journey can help you develop a better clinical program, to set up better trials, to know where it makes sense to introduce a therapy or treatment option. We can consider fundamental questions like, Where do patients receive care, the larger academic trial centers or perhaps local community centers? Does it make sense to go where the patients are?” she asks.
Moving along the lifecycle, you reach the pigeon-hole that most people put RWE in. “When people think about RWE, they think about studies around launch or post-launch to ask, How is the medicine is used in the real world, what impact is it having, what does it look like when patients are not fully compliant or where there are comorbidities?”
Here, RWE can replace numbers with real people. “In the shift from volume to value, the role of RWE is tell the story of the value. A health economic model can give you the numbers, but the value of a medicine is far greater than that, and that needs the patient perspective. We need to know what’s valuable to a patient and it is hard to get that from an RCT alone,” says Leonard.
“A RWE study reflects different types of patients; you’re not getting the ‘healthy’ patient with cancer, you’re getting the patient with several different comorbidities and all the human foibles that we all possess that leads patients to not always be completely compliant with their medications. It expands value from just an economic discussion to really knowing the value to the patient, which, in turn, impacts the value to the physician who, ultimately, want to find the right option for their patient, bringing about the best patient outcome.”
Delivering an evidence plan that encompasses all these applications, however, is not easy. “It is a challenge. In large organizations, and AZ is not unique here, it’s about making sure that the capability and expertise is utilized right across the different clinical teams. The good news is that as RWE becomes more recognized externally, my feeling from colleagues internally is that the appreciation and demands for RWE in the clinical program will increase as well.”
As important internal alignment might be, Leonard also has her eyes firmly fixed beyond the walls of AstraZeneca. “One of the key areas we’ve identified to accelerate our RWE capability and our evidence generation as a whole has been finding partners. We’ve been collaborating for many years with many partners but one of the most significant is in diabetes, where we’ve been working with the American College of Cardiology since 2014 on the Diabetes Collaborative Registry (DCR).”
At the time, it was clear that no large data set existed, in spite of the size and importance of the disease area. “We needed a large data set to ask and answer the many different questions we had around standard of care or how diabetics with multiple comorbidities go through their patient journey. Starting with the data the ACC already had in their clinical registry and pulling together multiple partners – including all the other key professional societies and organizations that wrap around a patient with diabetes – we identified the different data elements that would allow researchers to answer many of these questions as well provide quality measure back to the DCR participants. We’re now able to progress studies more quickly because the data exists.”
Collaboration is essential for the future, but not just the future of pharma. “Health systems need the data to understand their quality of care; if you don’t have the data, it’s very difficult to show you’re having an impact on patient care. Without collaboration, each entity goes out and builds its own registry or data source and you get a disparate landscape with different pockets of data. With a large disease state like diabetes, having a large data asset that really reflects the population allows the broad research community, not just AZ or the ACC, to answer relevant questions to drive forward better patient outcomes.”
The next step is to combine medical data with patient perspectives, says Leonard. “We’ve been working with PatientsLikeMe, the web-based community where patients connect with others and share their health data to track their personal progress as well as allow it to be used in research. Together we’ve asked, Can we look at the patient from multiple perspectives? The medical aspect comes from DCR while the patient perspectives come from the community. This is important because a patient’s perspective on their diabetes care, for example, may be is different from how it is recorded in their EMR. In diabetes, we talk about patients working towards an HBA1c goal but sometimes their understanding of the goal, whether they’ve reached it and the potential consequences of poor control depends on how it has been communicated to them, and is very different, and more impactful on health outcomes, than what may be captured in clinical records. This kind of collaboration that brings together RWE and the patient voice is essential to answer one of the most important questions of all, How can we support and enable patients to be more informed about their own care?"
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