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by myTomorrows myTomorrows | 15 Apr 2020

Expanded Access as a source of Real‐World Data: An overview of FDA and EMA approvals

The trend of leveraging data from Expanded Access to inform and improve drug development has captured the attention of the pharmaceutical industry.


At myTomorrows, we work at the forefront of research on Expanded Access and Real-World Data (RWD). That is why we set out to discover exactly how big of a role Real-World Data collected during Expanded Access Programs (EAPs) plays in regulatory approvals.


We collaborated in a scientific consortium with the Erasmus University Rotterdam's Department of Health Technology Assessment and the Erasmus Medical Center’s Department of Biostatistics. This research consortium focuses on Real-World Data from Expanded Access Programs, specifically on methods of incorporating such data in clinical development by biostatisticians, payors and regulators.


The results of the research, entitled ‘Expanded Access as a source of real-world data: An overview of FDA and EMA approvals´ has just been published in the British Journal of Clinical Pharmacology. The first author is Tobias B. Polak, RWD lead at myTomorrows, and the article is co-authored by Dr. Joost van Rosmalen and Prof. Carin A. Uyl – de Groot.  


In their research, they systematically assessed the role that Real-World Data collected during Expanded Access Programs plays during European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) approval procedures. The focus was on evidence supporting efficacy, as other scholars have already focused on safety signaling during EAPs. They identified 39 unique drug approvals where the RWD from EAPs contributed to regulatory decision making. In 13 cases, the evidence was considered to be ‘pivotal’. In 4 cases, EAPs formed the sole data source to constitute the efficacy profile. Additionally, they attempted to characterize the nature of such approvals. The majority of EA-backed approvals fall within the hemato-oncology and rare-disease space. Alongside that, they described data quality and data management issues.


The authors concluded: “Awareness of the potential value of RWD from EA should facilitate that these data are used appropriately. This helps pharmaceutical industry and regulators determine whether EA – and associated RWD – is useful. For patients, this would hopefully result in speedier access to more diverse treatments.“


This research comes amidst increasing interest in Real-World Data. It makes a strong case for the use of RWD in support of regulatory decision-making. This is to the benefit of drug developers, regulatory agencies, patients and other stakeholders.  The authors’ analysis is as follows:


What is already known about this subject


  • Patients are demanding faster access to investigational treatments
  • Industry and regulatory authorities are increasingly incorporating data collected outside of clinical trials in decision making
  • There is a massive opportunity to learn more about how, why, and whether Real-World Data from Expanded Access Programs may lead to regulatory approval



What this study adds


  • Techniques from artificial intelligence can be applied to process approval documentation for health policy analyses
  • Real-world Data from Expanded Access Programs are increasingly accepted as evidence by regulatory authorities when collecting data in controlled settings is infeasible
  • 39 FDA/EMA approvals rely on Real-World Data from Expanded Access Programs to constitute the efficacy profile of a treatment. In 4 cases, these data were the sole source of evidence.



myTomorrows is advancing change in the drug development process in order to allow patients with an unmet medical need, faster and more equitable access to drugs in development. Real-World Data is an integral part of this push for change. We are very much interested in engaging with all stakeholders. Should you have interest in learning more about our approach, please feel free to contact us.