The US Food and Drug Administration first released its “Framework for FDA’s Real-World Evidence Program” in December 2018; since then, professionals have worked to figure out how to make the most of using of RWE and real-world data (RWD) in their work. Employing RWE in drug development and approval now is being promoted by FDA commissioner Stephen Hahn.
Still, many experts remain skeptical of the RWE project, especially regarding contextualization of data. A study published in the October 2019 issue of the Journal of the American Medical Association reported RWD sources could only feasibly replicate 15% of clinical trials.
Outsourcing-Pharma (OSP) spoke with Hinco Gierman (HG), vice president of product development and scientific insights with Integra Connect, about the use of RWD and RWE throughout the pharmaceutical industry. The discussion talked about intelligent analysis of RWD, and how RWE can effectively supplement clinical trials.
OSP: Could you please explain differences between RWD and RWE—why a company would use one vs the other, and then some of the benefits and detractions of using each?
HG: RWD is best when you want to see the “real data as is;” RWE is using reliable RWD as clinical evidence. Integra Connect collects and de-identifies RWD from over 1 million cancer patients across the U.S. Using our proprietary algorithms, we clean, harmonize and analyze the data to then derive RWE from the RWD, assessing the effect on efficacy, toxicity and cost of care. The FDA emphasizes both the reliability and relevance of the RWD when evaluating RWE.
OSP: Could you please explain what you mean by “financial toxicity,” and how RWE counters the negative effects?
HG: Financial toxicity is a term used when the “out of pocket” costs for a patient becomes so “toxic” it threatens to reduce access to that care. Integra has used RWD/RWE to show drug manufacturers how reducing the out of pocket costs for patients could lead to more patients having access to their drugs, ultimately helping both the patients and the drug manufacturer.
OSP: In what ways can RWE bolster (if not replace) clinical trial efforts?
HG: There are several problems with clinical trials that RWE can help mitigate. Clinical trials study patients based on a strict inclusion/exclusion criteria, which results, for example, in many patients with common co-morbidities not being studied. However, in the “real world” outside of clinical trials, patients are often treated with approved therapies, which were excluded from the clinical trial itself. Therefore, RWE can show how other types of patients react to treatments. Finally, doctors participating in trials are often specialists, but most medical oncologists have to treat a wide range of cancer patients. RWE can thus help us understand how patients are treated in the “real world,” which can be different from the specialistic treatment patients receive in trials.
OSP: How specifically does RWE impact oncology drugs, novel therapies, and innovation?
HG: Prior to the 21st Century Cures Act, RWE was being used to assess toxicity (adverse event reporting). However, more recently RWE has been used as a “synthetic control arm,” for example for FDA approval of avelumab and blinatumomad. Both of these used historical data for the control arms (making them “synthetic”), and as these drugs are for rare cancer types, the recruitment of these patients would have been challenging. Thus, RWE is accelerating trial recruitment, drug approval, and making it easier to launch novel therapies for rare indications.
OSP: Could you please outline some of the ways R&D teams are using RWD, and the obstacles associated with that use?
HG: Integra Connect has been creating algorithms to create matched cohorts, while working with Medical Affairs and R&D teams at pharma companies. This allows us to compare similar groups of “matched” patients on similar but different drugs to compare efficacy, toxicity and cost of care. Thus we can identify those groups of patients, who most benefit from a certain drug within the group of similar drugs. We are publishing and sharing these results with the scientific community, for example as ASCO abstracts.
OSP: How can companies like yours help with the use and application of RWE?
HG: What make Integra Connect and our PrecisionQ business unit unique is that we combine data from different sources for our practices: EMR, claims and practice management systems. Being one of the largest providers of OCM analytics for U.S. oncologists, we have deep experience analyzing outcomes and cost, as well as using machine learning to create proprietary predictive analytics. This helps generate a very detailed picture of our patients, which benefits all parties involved. We are currently using our insights working with many of the top 25 pharma companies to help them better understand how cancer patients in the real world can benefit from improved treatments.