George Kumar, Senior Director at AstraZeneca, shared a post on LinkedIn:
“Off-Label, On Record: What 1,600 Patients Taught Us About Precision Oncology Beyond the Label
When a cancer drug is approved for one tumor type but a patient with a different cancer has the same genetic mutation – should they get it?
This happens constantly in oncology. It’s called off-label prescribing, and for most patients it happens in a data vacuum.
The DRUP trial from the Netherlands just published the largest prospective dataset on this question: 1,610 patients, 37 drugs, 8 years.
The headline numbers:
- 35% clinical benefit rate (response or stable disease ≥16 weeks)
- 16% objective response rate
- Median overall survival: 8.2 months
- Grade 3+ toxicity in 28% of patients
- 7% exceptional responders – meaningful, durable benefit
The honest takeaway: modest benefit overall, but real signal in defined molecular subgroups. And critically – this evidence actually influenced reimbursement decisions by Dutch regulators.
The study’s recommendation is direct: off-label precision medicine should only happen inside frameworks that capture outcomes, refine biomarkers, and build toward label expansion. Doing it any other way risks repeating futile treatments – on patients who have no other options.”
Title: Prospective evaluation of genomics-guided off-label treatment
Authors: K. Verkerk, A. C. Spiekman, S. F. Haj Mohammad, F. A. J. Verbeek, H. Timmer, M. A. van Maren, L. J. Zeverijn, B. S. Geurts, V. van der Noort, P. Roepman, A. M. L. Jansen, W. W. J. de Leng, H. Gelderblom, H. M. W. Verheul, E. E. Voest
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