In October, the IMPACCT: Real World Evidence Summit took a deep dive into the use of real-world evidence for regulatory submissions and improved clinical trial design. In preparation, conference organizer Hanson Wade interviewed speaker Kelly Zou. We share it with you here.
With draft FDA guidelines calling for more RWE to be implemented in clinical trials, what area of the trial process do you think RWE will have the biggest impact on?
Real-world evidence provides the opportunities for patient-centric innovations, patient-journey mapping, and outcome assessment. According to the US Food and Drug Administration, “Decentralized clinical trials hold promise to reduce patient and sponsor burden and increase accrual and retention of a more diverse trial population …” Specifically, the FDA’s Project Equity “aims to ensure that FDA-approved medical products work for all,” while Project Silver aims “to increase representation of older adults (65 years and older) …” Furthermore, “evaluating the COVID-19 experience with remote assessments is important to develop best practices when deploying [decentralized clinical trials] modifications in future prospective [decentralized clinical trials] designs.” Thus, the biggest impact of real-world evidence may result from disease burden assessment, trial design optimization, external control arm construction, and decentralized clinical trials implementation to complement clinical trials, just to name a few.
What is the biggest challenge in implementing real-world evidence for regulatory approvals?
According to the FDA’s real-world evidence framework, real-world data “are the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. Real-world data can come from a number of sources.” Given such a wide variety of data types, such as “electronic health records, claims and billing activities, product and disease registries, patient-generated data including in-home use settings, and data gathered from other sources that can inform on health status such as mobile devices,” regulatory-grade data that are fit for purpose are critical.
In addition, the European Commission has proposed the European Health Data Space following its General Data Protection Regulation, as well as the artificial intelligence act. The European Health Data Space “provides a specific ecosystem comprised of rules, common standards and practices, infrastructures, and a governance framework.” However, there are challenges in assessing data quality, given that data can be siloed to be interoperable. Thus, it is important to be able to develop ways to evaluate data standards before embarking on regulatory pathways to harness real-world data and real-world evidence.
What do you see as the future landscape for real-world evidence in a patient-centric digital era?
Several co-editors, coauthors, and I recently published a new book, Real-World Evidence in a Patient-Centric Digital Era. We focused on statistical and analytic methodologies in real-world evidence to generate insights on health care, with a special focus on the pharmaceutical industry. We examined timely topics of high relevance to industry such as bioethical considerations, regulatory standards, and compliance requirements. We highlighted emerging and current trends and provided guidelines for best practices. We illustrated methods through examples and use-case studies to demonstrate impact. Finally, we provided guidance on software choices and digital applications for successful analytics. Here, it is worth emphasizing the patient perspectives, along with multiple stakeholders, when harnessing real-world, big data, digital innovation, and artificial intelligence.
Editor’s Note: Zou is an employee of Viatris. The views expressed here are her own.
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