Скачать или смотреть Expanded Access as a source of real-world data: An overview of FDA and EMA approvals

Our latest video features Tobias Polak, the director of Real-World Data at myTomorrows and a PhD candidate focusing on Expanded Access. This video is part of a special series where Tobias will explain his research on Expanded Access and Real-World Data.

The research presented here is part of a public-private collaboration between Erasmus Medical Center, myTomorrows, and Erasmus University. Tobias discusses their first paper, entitled “Expanded Access as a Source of Real-World Data: An Overview of FDA and EMA Approvals.” This paper was published in the British Journal of Clinical Pharmacology in March 2020, and below you can find a link to it.

In our video, Tobias explores the following:

What is an Expanded Access Program? (Expanded Access Programs provide patients with life-threatening or seriously debilitating diseases, who are not eligible for registered treatment options or trials, a pathway to access investigational medicines.)
What other names are used to describe Expanded Access? (compassionate use, managed access, early access, etc.)
Next to providing treatment, Expanded Access Programs may sometimes be used to collect data. These data are called Real-World Data (RWD). RWD are data on safety and efficacy that are gathered outside of clinical trials.

There have been some examples of how RWD have been used by regulators in their decision-making, however, a systematic overview was lacking.
In this paper, Tobias and his colleagues set out to identify and characterize all FDA and EMA approvals that relied (in part) on data from Expanded Access Programs to inform the profile of clinical efficacy.
Tobias and his colleagues analyzed hundreds of documents from the FDA and EMA to identify approvals that relied (in part) on data from Expanded Access Programs.
They identified 49 FDA and/or EMA approvals that used data from Expanded Access Programs up to 2020.


Through their research, Tobias and his colleagues have added some facts to a discussion that has been mainly anecdotal in the past. You can check out the full paper, which was published in the British Journal of Clinical Pharmacology, here: https://www.ncbi.nlm.nih.gov/pmc/arti...

At myTomorrows, we are dedicated to helping patients and their physicians discover and access all possible treatment options. We are working to make drug development serve everyone better. We hope you found our video useful.

Learn more about myTomorrows at: https://mytomorrows.com/en



Curious to learn more about Real-World Data? Hear more from Tobias on our podcast, Shared Experiences in Oncology & Rare Diseases:

Spotify: https://open.spotify.com/show/5LbbOmV...

Apple Podcasts: https://podcasts.apple.com/nl/podcast...