At the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, the usual avalanche of clinical trial data dazzled attendees — but amidst the blinding white were data that looked remarkably similar to trial-level data. These data were from expanded access programs, and data on the safety of an investigational therapeutic were not the only data coming out of reports. Efficacy data from these protocols were reported during the meeting, too.

“While expanded access programs are no replacement for true research studies, we have seen a surge of interest from oncology companies seeking to bolster their path to market with real-world efficacy evidence from expanded access,” said Jess Rabourn, founder and CEO of WideTrial, a company that helps pharmaceutical companies manage and collect data from expanded access programs.

Expanded access, also known as compassionate use or managed access, is a pathway through which patients with life-threatening conditions who are ineligible for a clinical trial can receive an investigational agent. The pathway began as a way to treat patients, and while that still remains the primary goal of the studies, it is also a means for collecting real-world data.1

During an interview with Cancer Therapy Advisor, Hayley Belli, PhD, assistant professor of biostatistics in the department of population health at NYU School of Medicine, said there has been a “push” recently to access real-world data, which are data collected outside of a clinical trial. Real-world data can be gleaned from numerous sources, such as electronic health records, claims activities, wearable devices, and expanded access programs.2


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“[People] have been concerned for years about these clinical trials that have super restrictive inclusion/exclusion criteria,” said Dr Belli. “Expanded access programs now are this opportunity to collect real-world data.”

The push for real-world data began with that passage of the 21st Century Cures Act in 2016, which tasked the US Food and Drug Administration (FDA) with creating a framework for evaluating real-world evidence for the approval of an investigational agent.3

Later, the FDA displayed their openness to considering expanded access data in regulatory submissions when they hosted a public meeting in 2018 about the topic. The report from the meeting explicitly stated that expanded access data can used to supplement clinical trial data in regulatory review, inform off-label use, and drive label expansions.4

“The FDA still pushes first and foremost for clinical trials,” said Dr Belli, “because just as a rule of thumb in statistics, if we don’t have randomization or a controlled comparison arm, you really can’t draw true efficacy.”

Despite lacking a control arm, expanded access data reported at ASH 2020 from 11 studies provided a glimpse into how patients in the real world respond to therapies, most often confirming clinical trial results, but on occasion, adding nuance.

For example, while the final results of the expanded access study RIALTO (ClinicalTrials.gov Identifier: NCT02187354), do not affect practice, the data are still useful.5 RIALTO allowed 110 children with relapsed or refractory B-precursor acute lymphoblastic leukemia (ALL) access to blinatumomab, which has been used as a bridging therapy for years.

“It adds to the body of evidence about the benefit of blinatumomab, partly because a lot of our data [are] still in the adult setting,” said Shilpa Shahani, MD, assistant clinical professor, department of pediatrics, City of Hope, Duarte, California, during an interview with this publication.