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A New Method for Ethical and Efficient Evidence Generation for Off-Label Medication Use in Oncology (A Case Study in
Samir Agrawal1,2, Padman Vamadevan3, Ndaba Mazibuko4
1Blizard Institute, Queen Mary University of London, London, United Kingdom.
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 participant-funded, open-label, non-randomized, single-arm real-world study designed to gather high-quality evidence on the safety, tolerability, and effectiveness of four off-label metabolically targeted medicines as an adjunctive cancer treatment for glioblastoma patients.
Insights
Investigating off-label drugs for cancer treatment requires new study designs. A novel approach using a pluralistic evidence base and real-world data from the METRICS study supports off-label drug use in oncology.
Area of Science:
- Oncology
- Clinical Pharmacology
- Evidence-Based Medicine
Background:
- Preclinical and early clinical data suggest "non-cancer" drugs, particularly metabolically targeted agents, show promise as adjuvant chemo- and radiosensitizers in oncology.
- Investigating these off-label drugs in large-scale clinical studies is crucial, but high-cost randomized controlled trials (RCTs) are often economically unviable or ethically controversial for patent-expired drugs.
Purpose of the Study:
- To propose a novel approach for generating high-quality clinical data to support off-label drug use in oncology, enabling potential changes in clinical practice and drug relabeling.
- To present an example of an off-label drug protocol for glioblastoma and preliminary data from the METRICS study.
Main Methods:
- Suggests a pluralistic evidence base and triangulation of evidence to support clinical trial data for off-label drug use.
- Presents preliminary retrospective data from the METRICS study (NCT02201381).
- METRICS is a participant-funded, open-label, non-randomized, single-arm real-world study.
Main Results:
- The METRICS study aims to gather high-quality evidence on the safety, tolerability, and effectiveness of four off-label metabolically targeted medicines.
- Preliminary retrospective data from the METRICS study are presented as an example.
Conclusions:
- A pluralistic evidence base, including real-world data, can support the evaluation of off-label drugs in oncology.
- Novel study designs like the METRICS study are essential for gathering robust evidence on the efficacy and safety of off-label drugs, potentially leading to new therapeutic options for cancer patients.
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