By Brittany Wade 

January 3, 2023 | In recent years, pharmaceutical and life science companies have leveraged real-world evidence (RWE) to maximize patient recruitment and formulate clinical trial criteria. After recognizing that real-world data (RWD)-based trials are faster and cheaper than their randomized controlled counterparts, many companies also discovered new opportunities to engage with patients and develop better-performing products for the market. 

As a self-described “data hippie,” Christopher Boone, Ph.D., vice president and global head of health economics and outcomes research at AbbVie, has had a front-row seat to watch real-world data and evidence significantly impact clinical research and patient outcomes. 

“We’ve progressed tremendously over the last several years,” Boone tells Deborah Borfitz, Clinical Research News senior writer and host of the Scope of Things podcast. “We’re at a place where folks are coming up with novel use cases for how real-world data can be utilized to generate real-world evidence… [The fact that] we’ve done so much more in R&D has been a pleasant surprise.” 

While pharmaceutical companies slowly allowed RWE to influence their developmental processes, the United States federal government made its own shifts. In 2016, the U.S. Food and Drug Administration (FDA) launched the 21st Century Cures Act, a law that permits using RWE and clinical outcomes assessments to “speed the development and review of novel medical products.” Under the Act, the FDA crafted specific criteria dictating the circumstances under which RWE could meet regulatory standards and best practices for tracking drug efficacy in a real-world context. 

“The 21st Century Cures Act was probably the single biggest catalyst for the expanded use of RWE, especially on the R&D side of the house,” says Boone. “[It] gave the industry the license to think innovatively about how we can better utilize RWE going forward.” 

Boone points to the COVID vaccine as a downstream benefit of the Cures Act and credits RWE as one of the primary reasons scientists developed the vaccine in record time. What historically took 10 to 15 years, scientists whittled down to months, thanks to the new clinical landscape spurred by the Cures Act. 

A Disruptive Innovation 

Traditional clinical trial evidence will likely always be the gold standard, but it is no longer the sole guiding force for drug discovery and development strategies. Instead, RWE informs the decisions of big-name companies, buyers, payers, physicians, and nearly every other stakeholder in the industry. “You have to think of [RWE] as… a disruptive innovation. It has the potential to challenge the status quo, the way that we’ve done drug discovery and development in the past, and even the way we commercialize many therapies,” says Boone.  

He asserts that the general push toward more inclusive, decentralized, and community-based clinical trials would not be feasible if RWE, the expansion of digital technology, and the resurgence of artificial intelligence and machine learning as advanced data analytical tools were not impacting the industry on a mass scale.  

The standard protocols for data curation are also changing. RWE enables multiple interdisciplinary studies from one data set due to the advent of health data platforms and patient-generated data repositories. He says, “Much of the data we care about is captured outside the walls of the provider environment… Now, with all the digital technology, you can really understand the [patient’s] environment and experience in real time.” 

Boone believes that one of the last few hurdles to widespread RWE adoption is employing a “learning-based culture,” where companies take on a curious approach to drug development with a willingness to be flexible and learn new things. He admits this approach may be challenging for most pharmaceutical companies: “We’ve done clinical trials the same way for the last five decades, and we’ve mastered that. Asking that to be disrupted is a tough pill to swallow.” 

Integrating RWE into clinical trials also brings new concerns for patients. As companies collect and gather more RWD from various sources, the general public becomes increasingly wary of how their data is used and why. As a result, most life science companies must change their thought processes around data, including how they think about data privacy and security on the patient’s behalf. 

Boone insists that stakeholder mindsets, company culture, and trial design must change to suit the growing use of RWE as the industry campaigns for more patient-centric trials. Nevertheless, experts like Boone are committed to transforming clinical research on every front, including how the products resulting from RWE are commercialized and deemed profitable for their respective parent companies. 

Recently, Boone was appointed to the Patient-Centered Outcomes Research Institute Board of Governors, representing "a broad range of perspectives and… scientific expertise" to equip patients with actionable knowledge to make better-informed health decisions. In early February of this year, he will be a speaker at Clinical Research News’ Summit for Clinical Ops Executives Summit focusing on the novel ways RWE allows the industry to reimagine clinical discovery and development efforts. He says, “I hope to initiate a dialogue around the science of building these functions and capabilities and how we can do it at scale.”