The Benefits of Using Real-World Evidence in Clinical Research

Real-World Evidence (RWE) is redefining how healthcare and drug development insights are generated. This blog explores the advantages utilizing RWE can offer, the factors driving its growing significance, and the benefits that utilizing it brings to regulators, patients, and pharma companies. 

What Is RWE? 

RWE is derived from analyzing Real-World Data (RWD)—information gathered from sources outside traditional clinical trials. These include electronic health records (EHRs), insurance claims, patient registries, and wearable devices. Unlike the tightly controlled environment of randomized controlled trials (RCTs), RWE reflects healthcare delivery and patient outcomes in everyday settings. Its goal is to supplement insights obtained from RCTs, addressing gaps and enhancing understanding of treatment effects across diverse populations and conditions. 

Why Is RWE Different from Traditional Clinical Data? 

RCTs are the gold standard for establishing causality due to their controlled design, but they often involve homogenous patient groups and limited sample sizes. In contrast, RWE captures the complexities of real-world clinical practice, offering:

1. Broader population coverage: Including underrepresented demographics such as older adults, patients with comorbidities, and those in under-resourced settings.
2. Longitudinal insights: Observing how treatments perform over extended periods in routine practice.
3. Diverse settings: Gathering data from various care settings can help enhance the generalizability of findings.

Why Is RWE Gaining Traction? 

The rising interest in RWE stems from its growing relevance in regulatory, clinical, and commercial domains. [1] Agencies like the FDA and EMA are increasingly open to using RWE to support regulatory decisions, from drug approvals to post-market safety assessments. For example, the FDA’s 2023 guidance emphasizes the need for high-quality RWD and outlines how RWE can complement traditional trials by assessing safety and efficacy in larger, more diverse populations. [2] 

Additionally, advanced technologies and data science techniques like machine learning and natural language processing (NLP) enhance the ability to analyze RWD, providing deeper insights into patient outcomes and treatment pathways.  

Below, Alice Wang, Associate Director of Data Science at Phastar, describes some of the key benefits that using RWE can bring during our recent webinar. 

Watch the full webinar on demand here 

What Specific Benefits Can Be Gained from Utilizing RWE? 

• Accelerating Approvals and Reducing Costs: The integration of real-world evidence (RWE) in early benefit assessments can potentially help expedite drug approvals by providing critical data that supplements traditional clinical trial methods. [3] Additionally, RWE contributes to cost efficiencies by reducing the need for certain components of traditional randomized controlled trials (RCTs), potentially lowering clinical trial expenses. [4] Together, these advantages highlight RWE’s potential to streamline both the approval process and the overall cost of drug development. 

• More Inclusive Research: Real-world evidence (RWE) plays a crucial role in identifying healthcare disparities related to factors such as race, ethnicity, gender, age, and socio-economic conditions. Recognizing these disparities is the first step in addressing them, and RWD can help ensure clinical trials are more inclusive by targeting historically underrepresented groups. By including diverse populations, clinical trials become more representative, improving the generalizability of results, and reducing the risk of perpetuating healthcare inequalities, fostering a more equitable approach to drug development. [5] 

• Tracking Long-term Safety and Efficacy: The long-term follow-up provided by RWE is essential for pharmacovigilance. RWE plays a crucial role in Post-Authorization Safety Studies (PASS) to assess drug safety in real-world settings after approval, along with post-marketing requirements (PMRs/PMCs) that gather additional safety data, monitor long-term effects, detect rare events, and evaluate risk mitigation strategies. [6] 

Conclusion 

Real-World Evidence is transforming the way we understand and evaluate healthcare interventions, offering deeper insights into diverse populations, real-world treatment pathways, and long-term outcomes. This, in turn, fosters more efficient delivery of effective, patient-centered therapies, contributing significant value to public health. 

Interested in how RWE can help achieve your organization’s goals? Our experts are ready to discuss innovative, tailored strategies that align with your needs. 

References

[1] Li, Y., Tian, Y., Pei, S., Xie, B., Xu, X., & Wang, B. (2023). Worldwide Trends in Registering Real-World Studies at ClinicalTrials.gov: A Cross-Sectional Analysis. International Journal of General Medicine, 1123-1136. https://doi.org/10.2147/IJGM.S402478  

[2] U.S. Food and Drug Administration. Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products. Published August 2023. Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-use-real-world-data-and-real-world-evidence-support-regulatory-decision-making-drug

[3] Schad, F., & Thronicke, A. (2022). Real-World Evidence—Current Developments and Perspectives. International Journal of Environmental Research and Public Health, 19(16), 10159. https://doi.org/10.3390/ijerph191610159

[4] National Academies of Sciences, Engineering, and Medicine, Health and Medicine Division, Board on Health Sciences Policy, & Forum on Drug Discovery, Development, and Translation. (2019). Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series (C. Shore, A.W. Gee, & B. Kahn, Eds.). Washington, DC: National Academies Press. https://doi.org/10.17226/25352

[5] Dagenais, S., Russo, L., Madsen, A., Webster, J., & Becnel, L. (2021). Use of real‐world evidence to drive drug development strategy and inform clinical trial design. Clinical Pharmacology & Therapeutics, 111(1), 77–89. https://doi.org/10.1002/cpt.2480

[6] Hammad, T.A., Afsar, S., Le-Louet, H., & Kugener, V.F. (2023). Navigating a transforming landscape: The evolving role of pharmacovigilance physicians in drug development and implications for future challenges and training requirements. Frontiers in Drug Safety and Regulation, 3, 1257732. https://doi.org/10.3389/fdsfr.2023.1257732