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Everything You Wanted to Know About Expanded Access but Were Afraid to Ask, Part 2

By Alison Bateman-House, Hayley M. Belli, and Sage Gustafson

This series is adapted from a webinar hosted by PRIM&R on August 5, 2021: IRB Review of Expanded Access Protocols that Collect Real World Data: Considerations and Guidance. Read Part 1 here.

Part 2: Possible Value of “Real World Data” Collected from Expanded Access

Real world data (RWD) are data relating to patient health status and/or the delivery of health care such as medical bills/claims, electronic health records, and product/disease registries. RWD are derived from sources outside of randomized controlled trials (RCTs). Real world evidence (RWE) may be derived from the analysis of quality RWD.

The 21st Century Cures Act (passed in 2016) recommends the use of RWD to support FDA decision-making about product approvals. While RCTs are considered the “gold standard” for generating evidence about a product’s safety and efficacy, RWD can supplement RCT results. Furthermore, the collection of RWD from outside of RCTs may help to improve the understanding of how investigational products work in a population of patients that is more diverse than the sample of that population that participates in a clinical trial.

As U.S. and European regulators are increasingly willing to consider these data as part of the regulatory submission, the biopharmaceutical industry increasingly seeks to collect RWD from EA. At the same time, many patients are excited about the use of RWD from EA. Some patients altruistically hope to advance science by having RWD collected from their use of products via EA. Still others believe that data collection from EA offers an opportunity to learn about outcomes that were not studied in RCTs; for example, gauging quality of life measures in individuals taking an investigational drug. Finally, some patients believe that collecting RWD from EA opens the door to limiting or forgoing RCTs; however, we caution that when it comes to evaluating investigational products’ safety and efficacy, RWD cannot serve  as a substitute for a carefully designed RCT.

RWD collected from EA may include lab values, patient-reported outcomes, and functional assessments, among other things. Collecting RWD from EA can be of value by:

  • Providing safety information about an intervention
  • Generating hypotheses or informing the design of a later RCT
  • Identifying biomarkers
  • Determining characteristics for stratification when randomizing in a later RCT
  • Providing valuable data in the rare disease space where there are few patients. (Rare disease patients may obtain access to unapproved investigational products outside of clinical trials more often than inside the typically very small trials for these )
  • Providing valuable data in situations where RCTs are not feasible
  • Enhancing generalizability of RCTs as supplemental data

Key takeaway: EA was created to permit certain patients the option of trying medical products outside of clinical trials for which they are unable to participate, if the sponsor is willing to provide the requested item. It is not intended as research; nevertheless, in some cases there may be value in collecting “real world data” from Expanded Access. There is increasing interest in such data collection on the part of both the biopharmaceutical industry and patients.

The authors would like to thank Jan Jaeger for her contributions to this work.

The Petrie-Flom Center Staff

The Petrie-Flom Center staff often posts updates, announcements, and guests posts on behalf of others.

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