The Future of RWE in Clinical Trial Design and Recruitment

How we’ve conducted clinical trials in the past will not be sustainable in the future. Less than four percent of Americans participate in clinical studies; 30 percent of patients drop out; and 80 percent of studies do not meet enrollment deadlines. We brought together three leaders who are working to change these statistics by bringing recruitment to the patient and leveraging real-world evidence (RWE) to innovate trial design and accelerate recruitment. MaryAnne Rizk, PhD, Chief Strategy Officer, Medable, Kushal Gohil, Chief Strategy Officer, Parexel, and  Tony Clapsis, SVP & General Manager, Clinical Trial Services, CVS Health, joined Clarify’s CEO, Jean Drouin, MD on stage at CNS Summit 2022 to discuss the critical role of RWE in bringing their vision to life: improving patient access, increasing diversity, and doing clinical trials more efficiently. Below are highlights and key takeaways from the panel.

Question: How has the democratization of real-world data (RWD) changed the clinical trial landscape?

Dr. Rizk provided an example of how we’re getting better at meeting patients where they are. She described a middle-aged man, named Malik, who is in a rural location in the Midwest. He can now walk into a local regional pharmacy, like a CVS, Walgreens, or Walmart, and get educated about an upcoming trial that might be applicable to him.

Prior to him walking in, the sponsor and pharmacy already have a holistic view of his patient journey and understand factors affecting his lifestyle, such as whether he buys cigarettes or lacks transportation. And by bringing recruitment to the patient, it gives them a better understanding of clinical research so they can make informed decisions, and be more likely to participate, engage, and share data.

“I think this is probably the most exciting time for all of us here to be in clinical research because we are seeing a convergence of data from clinical settings with data from the real-world. Life sciences and healthcare are becoming united. We’re able to combine information from claims data, EHR data, even lifestyle data,” said Dr. Rizk.

Additionally, Mr. Clapsis spoke about the role CVS Health is playing in capturing and using RWD for clinical research: “What’s really exciting now is that we have the ability to generate patient reported outcomes (PROs) and other observational data as part of the normal course of healthcare. We generate PROs on all our specialty pharmacy patients as part of what we do to really help them manage their conditions.” What these data mean is that sponsors might not have to run a new observational study—the data already exists, it’s been standardized, and it can easily be linked to other data sets. “We can actually design and stand up really large-scale RWE studies by going directly to the members,” said Mr. Clapsis. “And just in the last year, we’ve educated 25 million members and that number’s only going to go up as we bring recruitment and other opportunities directly to patients.”

Question: How are real-world and patient journey data used today for clinical trial design and what’s different from 10 years ago?

Mr. Clapsis described two main catalysts that have propelled the use of RWE over the past ten years. The first is the 21st Century Cures Act signed into law in 2016 and the second is the COVID-19 pandemic, which drove the use of RWD and RWE, at scale. Mr. Clapsis commented on the remarkable rise of sponsors leveraging RWD and RWE in regulatory submissions: “I think there’s a lot to be proud of. Ninety percent of all Biologics License Applications (BLAs) had elements of RWE and RWD if you look back over the last couple years, and that’s steadily been rising.” Additionally, he went on to say, “The work in synthetic control arms is pretty cool. About 10% of pharma companies at this point have active efforts but we’ve started to see some approvals. And, as you get into more specialty and rare conditions, the need from a cost, ethical, and frankly just a workflow perspective is only going to increase,” Mr. Clapsis added.

That said, Mr. Clapsis thinks the opportunity is still ahead of us and we’re just getting started. He described the application of RWD and RWE for protocol design, particularly understanding feasibility (i.e., testing inclusion and exclusion criteria), as well as for precision targeting of sites and patients. He sees a lot of the opportunity for RWD and RWE to optimize study design upfront and avoid having to rescue a failing study at the end. He said, “I think the additional data that’s come online in the last two, three years gives you the ability to do that at a level you’ve never been able to do before.”

Dr. Drouin, the moderator, added to the discussion: “Five years ago, it was not possible to bring together 300 million lives annually of patient journey data with claims, lab, prescription, and social determinants data linked at the patient level. Today, we’re stitching together real-world patient journey data and applying data sciences methods to deeply understand the patient journey.” Dr. Drouin illustrated an example of how this helps in clinical research. He said, “It’s possible that an academic medical center in a lower-income area is conducting a trial, but most of the individuals who live right there choose to go elsewhere for their care. With real-world data, you can see that pattern clearly and more precisely recruit the right patients into the trial.”

According to Dr. Rizk, there’s evidence that leveraging RWE and launching decentralized clinical trials is improving clinical research. She said, “Medable partnered with Tufts, showing evidence of 14X ROI when decentralized methods were applied to create the convenience of access to care and also being able to streamline some of the activities in the traditional data collection.”

Question: What’s the FDA’s approach to unlocking the full promise of real-world evidence?

Mr. Gohil said, “When we look at the FDA, since 2016, the industry overall has seen a good amount of adoption. I think the pandemic sped that up a bit. I think their acceptance of pharmacovigilance is quite solid, I think it’s quite mature. In other areas, based on our discussions through the Association of Clinical Research Organizations (ACRO) or through our direct engagement, I think they are still a bit hesitant  but more open since the pandemic.”

Mr. Gohil believes that to unlock the full promise of RWE, the life sciences industry needs to give the FDA more confidence in RWE and ensure it’s fool-proof and that patient safety isn’t impacted. “It’s not just the FDA,” he said. The industry needs to come together to solve for this, but I think the pandemic has shown us that it is feasible, so I’m hopeful.”

Question: Why is now the right time for CVS Health and other retail pharmacies to enter the clinical trials business?

Mr. Clapsis said it makes perfect sense for CVS Health to bring research to its members and normalize participation. CVS already has clinics, it is building primary care assets, and has the largest in-home health network. He explained the thought process behind the decision: “The broader vision of the role CVS is playing in members’ lives starts to beg the obvious question, ‘if you’re going to take care of their health needs, why has research always been outside of the equation?’ And so that’s a very natural way for us to really think about how we bring research to members and norm it as an opportunity.” Today, CVS is working to build a nationwide network of principal investigators, hundreds of sites, technology, and home research capabilities.

Question: What’s the future for clinical research organizations (CROs)? How will they need to adapt as retail pharmacies continue to play in the clinical trial landscape?

Mr. Gohil shared that he believes the future for CROs hinges on strengthening partnerships between all the players and leveraging the innovations that have burgeoned during the pandemic—decentralized clinical trials (DCTs), risk-based quality management (RBQM), RWE, etc. He said Parexel’s partnerships with CVS Health and Medable have become part of the organization’s operating model. He also commented on how CROs need to ensure end-to-end data flow. He said, “I think the responsibility on our side is to button up the end-to-end data and document flow because the client does not want to interact with 10 different vendors.”

Question: In 5 years, what will be most different about the way in which clinical trials are conducted?

Mr. Gohil said, “More trials will be a hybrid between traditional sites and communities. I think by design, diversity will improve, that will also improve scientific outcomes.”

Dr. Drouin, the moderator, shared an example: “Traditionally, we had to collect just enough data from patients to power the study, and the information had to be perfect and pristine, understandably. The world of big data is different. You can now have a congestive heart failure study and look at that cohort of hundreds of thousands of journeys and filter through the noise with data science methods. We can reduce the number of patients that we need to recruit and speed up the time with which we do it.”

Mr. Clapsis concluded that he thinks we’re already at a point where RWD in R & D should be ubiquitous. He believes that generating evidence, not just through tokenization and external control arms, but through patient-centric models will give us a fundamentally different way to provide the data quality that regulators and others are looking for.

You can watch the full panel on-demand here.