Скачать или смотреть Real-world data from expanded access programs in health technology assessments

Expanded Access and Real-World Data are the focus of our latest video. Our new video is part of a special series where Tobias Polak, who is the director of Real-World Data at myTomorrows and a PhD candidate focusing on Expanded Access, explains 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. In each video of this series, Tobias will guide us through this group’s academic contributions — covering the introduction, methods, results, and discussion of the papers that they have written together.

In our previous video, Tobias discussed the group’s 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. Below you can find links to the video and the journal publication.

In today’s video, Tobias discusses their paper entitled, “Real-World Data from Expanded Access Programmes in Health Technology Assessments: A Review of NICE Technology Appraisals.” This paper was published in the Open Series of the BMJ (the British Medical Journal) in January 2022. You can read the full publication via the link below.  

Their last paper investigated the role that real-world data from expanded access plays in regulatory approvals. However, in some European countries, a regulatory approval does not necessarily mean that patients will be able to access the drug. Countries with a National Health System, such as the Netherlands, France, or the United Kingdom, have a system to make sure that drugs are cost effective.

For example, in the United Kingdom, the National Institute of Care and Excellence (also known as NICE) assess a drug’s cost-effectiveness in a “health technology assessment.”

In this new paper, Tobias and his colleagues investigated the use of data from expanded access programs in reimbursement dossiers, by reviewing all technology appraisals presented to NICE between 2010 and 2020. They sought to answer if, when, and how data from expanded access programs are leveraged in cost-effectiveness analyses. 

The video explores the following:

What is an expanded access program? For patients with life threatening or seriously debilitating diseases, who have no other treatment options and who cannot participate in trials, expanded access programs provide a pathway to access investigational medicine.

What is the goal of expanded access? The main goal of expanded access is to provide treatment. However, in addition to providing treatment, these programs may sometimes be used to collect data. These data are referred to as ‘real-world data’: data on safety and efficacy that are gathered outside of clinical trials.

This latest research project investigated the use of data from expanded access programs in reimbursement dossiers, by reviewing all technology appraisals presented to NICE between 2010 and 2020.

Tobias and his colleagues sought to answer if, when, and how data from expanded access programs are leveraged in cost-effectiveness analyses. 

Tobias and his colleagues in this collaboration were able to download 8,925 documents of all 380 technology appraisals with documentation available.

They discovered that an ‘expanded access’ term was present in 54% of the appraisals. In 21 %, that is 80 out of 380 appraisals, the expanded access program generated evidence for the appraisal.

Although they saw a significant increase in the absolute use of expanded access data, the relative use of these data did not show a significant trend.
To better understand how expanded access data may contribute to safety, efficacy, and or resource use, Tobias presents several examples.  
To gain a more in-depth understanding of what Tobias discusses in this video, you can read the full research paper here: https://bmjopen.bmj.com/content/12/1/...

Also, you can check out the first video in this series here:    • Expanded Access as a source of real-world ...  
and the collaboration’s first publication 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...