Agrawal, S;
Vamadevan, P;
Mazibuko, N;
Bannister, R;
Swery, R;
Wilson, S;
Edwards, S;
(2019)
A New Method for Ethical and Efficient Evidence Generation for Off-Label Medication Use in Oncology (A Case Study in Glioblastoma).
Frontiers in Pharmacology
, 10
, Article 681. 10.3389/fphar.2019.00681.
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Abstract
In oncology, preclinical and early clinical data increasingly support the use of a number of candidate “non-cancer” drugs in an off-label setting against multiple tumor types. In particular, metabolically targeted drugs show promise as adjuvant chemo and radiosensitizers, improving or restoring sensitivity to standard therapies. The time has come for large scale clinical studies of off-label drugs in this context. However, it is well recognized that high-cost randomized controlled trials may not be an economically viable option for studying patent-expired off-label drugs. In some cases, randomized trials could also be considered as ethically controversial. This perspective article presents a novel approach to generating additional clinical data of sufficient quality to support changes in clinical practice and relabeling of such drugs for use in oncology. Here, we suggest that a pluralistic evidence base and triangulation of evidence can support clinical trial data for off-label drug use in oncology. An example of an off-label drug protocol brought to the clinic for glioblastoma patients is presented, along with preliminary retrospective data from the METRICS study (NCT02201381). METRICS is a novel participantfunded, open-label, non-randomized, single-arm real-world study designed to gather highquality evidence on the safety, tolerability, and effectiveness of four off-label metabolically targeted medicines as an adjunctive cancer treatment for glioblastoma patients
| Type: | Article |
|---|---|
| Title: | A New Method for Ethical and Efficient Evidence Generation for Off-Label Medication Use in Oncology (A Case Study in Glioblastoma) |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.3389/fphar.2019.00681 |
| Publisher version: | https://doi.org/10.3389/fphar.2019.00681 |
| Language: | English |
| Additional information: | This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. https://creativecommons.org/licenses/by/4.0/ |
| Keywords: | off-label, glioblastoma, metformin, atorvastatin, mebendazole, doxycycline, metabolic targeting, pluralistic evidence |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > UCL BEAMS UCL > Provost and Vice Provost Offices > UCL BEAMS > Faculty of Maths and Physical Sciences UCL > Provost and Vice Provost Offices > UCL BEAMS > Faculty of Maths and Physical Sciences > Dept of Science and Technology Studies |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10077090 |
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