Embleton-Thirsk, Andrew;
(2021)
Identifying, profiling and learning from non-commercial clinical trials used in regulatory submissions.
Doctoral thesis (Ph.D), UCL (University College London).
Abstract
Regulatory approval of a medicine that expands treatment options available to patients is arguably, one of the most impactful results a researcher can have. Non-commercial trials, such as those sponsored by public-bodies or charities, are widely perceived as not being appropriate to be used as key evidence in licencing submissions when compared to company-led trials. Isolated case studies of non-commercial trials being used in submissions are known to experienced researchers, but are rarely published in the academic literature. Consequently, little formal knowledge exists of the experience gained from attempts to increase the number of approved medicines available for patients utilising these types of trials. I was part of the trial team for ICON6, which was a non-commercial trial unexpectedly used as the pivotal trial in the submission of Zemfirza to the European Medicines Agency (EMA). Retrospective verification of data was performed in order to 'ready' the trial for use. These activities made immaterial differences to the conclusions but nevertheless the application was withdrawn by the company who were sponsoring the application due to a negative interim decision from the EMA. I searched the traditional literature for other examples of non-commercial trial use to learn from, with limited results. Consequently, I used the EMA public assessment reports, as an alternative source of data to assess this. These reports were downloaded and systematically searched, producing 25 examples of pivotal non-commercial trials. I then served as a Visiting Expert to the EMA in order to utilise the confidential internal reports, to learn more about the identified applications. Reviewing these applications, I highlight that the approval rate of non-commercial trials compares well with those utilising only company-led trials. Despite this there are lessons to be learnt with respect to the adherence to, and demonstration of, Good Clinical Practice (GCP) in non-commercial trials. I sought and gained access to further, novel inspection data not available to researchers. Using this I expanded upon specific areas of GCP that will need focus from non-commercial trialists if a trial is considered for use in a submission in the future.
| Type: | Thesis (Doctoral) |
|---|---|
| Qualification: | Ph.D |
| Title: | Identifying, profiling and learning from non-commercial clinical trials used in regulatory submissions |
| Event: | UCL (University College London) |
| Language: | English |
| Additional information: | Copyright © The Author 2021. Original content in this thesis is licensed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International (CC BY-NC 4.0) Licence (https://creativecommons.org/licenses/by-nc/4.0/). Any third-party copyright material present remains the property of its respective owner(s) and is licensed under its existing terms. Access may initially be restricted at the author’s request. |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology > Comprehensive CTU at UCL |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10123485 |
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