The notoriously complex healthcare system produces enormous quantities of data every moment. A typical care delivery network might generate petabytes of clinical, financial, laboratory, and pharmaceutical information on hundreds of thousands of patients each year – yet only a tiny fraction of this data is currently used to inform treatment decisions, support the development of new therapies, and foster better patient experiences and outcomes.
Government agencies, life science companies, care providers, payers, and patient advocates are all eager to change that ratio and make better use of the real-world data (RWD), and the real-world evidence (RWE) generated from it, that are pouring out of the care environment every second.
In fact, regulators are wasting no time in crafting an environment where rich, varied, and trustworthy RWD and RWE are central to the quest to create safer, more effective, more accessible therapies for a variety of disease groups, including cancer, said Sandy Leonard, Chief Commercial Officer at COTA during a recent webinar.
“We know that the regulatory environment is continuing to challenge us to move faster and find efficiencies while ensuring exceptional quality and value in the pursuit of better patient outcomes,” she said.
“To achieve those goals, we need multimodal RWD and RWE that can help us understand how actual patients engage with their care. By bringing together diverse data from multiple sources, such as EHR data, lab data, claims, pharmacy data, and even wearable device data, we can build out a multidimensional view of patients and better understand how they interact with and respond to specific treatments.”
Around the world, regulatory bodies are embracing the emerging role of RWD and RWE in pharmaceutical development and post-market surveillance. In the United States, the FDA and CMS are taking particularly active roles in setting new guidelines and creating roadmaps to help industry fulfill the goals of landmark laws, said Nirosha Lederer, Head of US Federal Government Programs at Aetion.
“RWD and RWE have been around for a while now. What’s new here is that there are significant policy developments at both FDA and CMS that are accelerating the demand for RWD and RWE and transforming them from a nice-to-have into a strategic imperative,” she explained.
For example, the 21st Century Cures Act of 2016 mandated that the FDA investigate how RWE can contribute to medical product approvals, which has catalyzed substantial investments in RWD and RWE by both FDA and industry, including RCT Duplicate, which looks at how and when RWE can answer regulatory questions.
The FDA is also actively involved in hosting public meetings, developing internal processes for using RWE, and issuing detailed guidance for industry on topics such as claims, EHR and registry data and how to use RWD for external controls.
“As a result, we continue to see an increase in the use of real-world data and real-world evidence in regulatory submissions in 2022,” said Lederer. “Based on our review of publicly available data, about 80% of approvals included real world evidence for either safety and or effectiveness. That’s huge, considering where we were only a few short years ago.”
CMS is also exploring how RWE can inform pricing, payment, and coverage decisions in light of the Inflation Reduction Act (IRA).
The FDA is also actively involved in hosting public meetings, developing internal processes for using RWE, and issuing detailed guidance for industry on topics such as claims, EHR and registry data and how to use RWD for external controls.
“Part of the law involves negotiating the prices of the most expensive products as ranked by CMS spend,” said Lederer. “There are some cut-and-dry equations involved in price setting, but there’s also room for more interpretive factors such as how a product compares against alternatives.”
“The good news is that we have transformative technologies like natural language processing, tokenization, and artificial intelligence to help us create fit-for-purpose multimodal data sets that assist with answering these questions. But we need to make sure we are building these data sets in an appropriate manner so we can trust the results we generate from them.”
Trust is the operative word when it comes to bringing together multiple data sources to create new assets for use in the life sciences environment, agreed Vera Mucaj, Chief Scientific Officer at Datavant.
“We don’t have a data volume problem, but we do have a fragmentation problem – and that can create trust problems if we’re not careful,” she said. “As we start looking at bringing multimodal data together for regulatory and decision-making purposes, we need to connect up these disparate silos in a compliant and privacy-preserving way.”
“We have to be able to tell that Patient ABC in one data set is the same as Patient XYZ in another data set without anybody needing to know that the patient’s name is Jane Doe and they live at 123 Main Street,” Mucaj continued. “This is important for regulatory reasons as we think about the need to access and share de-identified data in a HIPAA compliant manner while preserving the provenance and governance of the data. But more importantly, we need to protect the spirit of the law around patient privacy and respect the fact that these are very sensitive data elements.”
By employing technologies that can identify, aggregate, and synthesize data in a trusted and neutral manner, the health system can start to unlock previously unused data to augment the traditional clinical trial and post-market surveillance ecosystem.
“Multimodal RWD and RWE that we can understand and appropriately manipulate is critical for researchers and analysts, because we’re in serious need of richer, more comprehensive data sets that allow for really innovative longitudinal studies, prospective observational studies, and natural history studies,” Mucaj said.
Once healthcare and life science stakeholders have access to fit-for-purpose multimodal RWD, the questions they can answer are “almost limitless,” says COTA CEO Miruna Sasu.
“You can start asking about disease epidemiology and unmet medical needs, or the patient pathway from diagnosis to treatment,” she said. “How feasible is a given clinical protocol? What’s the safety and effectiveness of that protocol in the real-world? Are the clinical trial populations and the real-world populations different, and how does that affect adherence and outcomes?”
“If we have trustworthy RWD that is appropriately linked and curated, we can start to accelerate development timelines and see some of the efficiencies that we need to navigate the changing regulatory environment.”
Regulators are going to continue pushing the envelope on what’s possible with RWD and RWE in an ongoing effort to control unnecessary spending while improving patient outcomes, Sasu predicted. Life sciences leaders need to be prepared to start integrating RWD and RWE into their research and development programs – or risk getting left behind the curve.
“The world of RWD is moving so fast, and it’s great to see regulators and industry collaborating with each other to keep pushing innovation forward,” she said. “It’s a real honor to be part of that process and contribute to our ability to truly understand the patient’s journey so we can create more effective treatments in oncology and elsewhere.”
“Multimodal RWD and RWE are the linchpins of these efforts, and it is incredibly important that we continue to work together to develop knowledge and actionable strategies around using this data to improve outcomes.”
To learn more about how an evolving regulatory environment is shaping the use of multimodal RWD and RWE in the life sciences, watch the full webinar on demand by clicking here.
