As healthcare data collection and governance capabilities continue to expand, real-world data (RWD) have become invaluable to drug development, approval, and post-market monitoring. In fact, one study found that all 111 medicines authorized in Europe from 2018 to 2019 referenced utilizing RWD, and real-world evidence (RWE) derived from it, in at least one stage of development.1
RWD is gathered from routine clinical care and can include various sources such as registries, electronic health records, lab results and medical claims. The RWD toolkit can now include linkage to clinical study participants for even deeper insights. Longitudinal real-world patient data that spans patients’ pre- and post-study healthcare journeys can provide strategic evidence development opportunities. By leveraging patient tokenization and robust data practices, researchers can create a rich and comprehensive dataset that reaches beyond the temporal bounds of traditional clinical trial designs.
The linkage of clinical study patient data to longitudinal RWD offers numerous advantages for sponsors. Among other benefits, elements of this linked data can contextualize study data, help to close the efficacy-effectiveness gap, and identify patterns that can influence product strategy post-approval.
Contextualizing study data with RWD linkage
Linking a patient’s RWD to clinical study data yields several key insights. It allows for a comparison of the patient’s healthcare resource utilization (HCRU) before, during and after a clinical study, providing an essential foundation for assessing how a new therapy and patient healthcare journeys interact across different stages. For example, contrasting pre-study healthcare journeys with a patient’s HCRU during and after a study can demonstrate a treatment’s impact on doctor visit and hospitalization frequency and length.
Linking to pre-study medical histories may also reveal potential directions for further research. Among others, these include identification of biomarkers that could be used for earlier patient diagnosis, and better understanding of the diagnostic journey and disease progression leading to clinical study participation.
In addition to leveraging pre-trial medical histories, clinical study data can be enriched through linkage with concurrent RWD, providing opportunities to contextualize adverse events, study discontinuation, and study dropout. Being able to assess outside factors, in addition to data points from within the bounds of the study itself, enables an improved understanding of the causes of such events and enhances the interpretation of study outcomes.
Closing the efficacy-effectiveness gap
A common challenge for sponsors is the discrepancy between a drug’s efficacy in clinical trials and its effectiveness in real-world clinical settings.2 Understanding the underlying causes of this gap is crucial for optimizing uptake, adherence and persistence, and increasing confidence among patients and providers.
If linked, longitudinal patient data from study participants allow for benchmarking against the broader patient population, offering a better understanding of elements that may impact real-world outcomes. For instance, the structure of a clinical trial may support better adherence than what patients experience in typical treatment settings, or the drug’s efficacy may vary in patient groups that were excluded from trial participation. These factors become easier to discern when it is possible to analyze the efficacy in patients during and after clinical trials, and compare this data with that of the general patient population.
Identifying post-market patterns
Post-market studies may be a regulatory requirement or may be optional. Continuing to collect safety data after market approval is often a critical consideration for many new drugs. Data linkage can provide an attractive strategic opportunity for sponsors, particularly in the case of chronic diseases.
Linking clinical study data to RWD allows sponsors to gather information that might not be seen in clinical studies, such as real-world dosing and administration, and patterns in patients’ adherence or decisions to switch to other treatments. Study participants can be compared to real-world populations for deeper understanding. Such information can give sponsors insights into which usage scenarios are most beneficial to patients and identify potential barriers to continued use. Crucially, if a therapeutic is under scrutiny for safety signals that arise in the real world, sponsors may be able to identify important differences between the real-world population and clinical study population where the signal was not observed. This could result in a label change based on patient sub-populations, versus a regulatory recall.
Leveraging a comprehensive dataset
The collection of longitudinal data from clinical trial participants provides a wealth of insights for drug developers, even beyond what has been discussed here. Enriching clinical studies with rich longitudinal patient data provides unique opportunities to gain a more comprehensive and long-term view of patient health and healthcare.
To make the best use of longitudinal RWD, sponsors must employ a strong data interoperability strategy that includes patient consent and tokenization, well-thought-out data sourcing and standardization, and robust data governance. Learn more about the use of longitudinal RWD in clinical trials, and strong data interoperability strategies to support it, in UBC’s case study: Enriching clinical studies with longitudinal real-world data.
About UBC
United BioSource LLC (UBC) is the leading provider of evidence development solutions with expertise in uniting evidence and access. UBC helps biopharma mitigate risk, address product hurdles, and demonstrate safety, efficacy, and value under real-world conditions. UBC leads the market in providing integrated, comprehensive clinical, safety, and commercialization services and is uniquely positioned to seamlessly integrate best-in-class services throughout the lifecycle of a product.
About the Author
Judy Lytle serves as the Executive Director of Evidence Development Study Solutions for UBC. Dr. Lytle joined UBC in 2023, bringing more than 15 years of experience in life science and healthcare strategy development, implementation, and execution. With a background in medical affairs and real-world evidence, she brings together differentiated study design and evidence generation solutions for value demonstration. She also has oversight of epidemiology, patient and physician services, scientific/clinical strategy, and medical writing teams.
Dr. Lytle holds a PhD in Neuroscience from Georgetown University as well as a Master of Biotechnology Enterprise & Entrepreneurship (MBEE) from Johns Hopkins University. A fellow of the American Association for the Advancement of Science (AAAS), and certified Project Management Professional (PMP), her approach is systematic and grounded in science.
References
- Eskola, Sini Marika, et al. “Use of Real‐World Data and Evidence in Drug Development of Medicinal Products Centrally Authorized in Europe in 2018–2019.” Clinical Pharmacology & Therapeutics, vol. 111, no. 1, 2022, pp. 310–20, https://doi.org/10.1002/cpt.2462.
- Rudrapatna, V. A., & Butte, A. J. (2020). Opportunities and challenges in using real-world data for health care. The Journal of clinical investigation, 130(2), 565–574. https://doi.org/10.1172/JCI129197
