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RC: This can be defined as the use of an approved drug for something other than what it was intended for or what it was officially approved for. This is to be distinguished from the investigational use of unapproved drugs or compassionate import of foreign drugs. It is a complex area because it is not only a matter of therapeutic efficacy but involves regulatory and legal liability issues for the prescriber. Essentially, the US FDA (or other FDAs) intend drugs to be used according to the the details of the product's labelling (as in the package insert) but in reality, many oncology treatments (including chemotherapy) are conducted "off-label".

"Off-label" does not necessarily imply unproven or investigational, in fact there is usually good scientific investigation backing such an alternative use (at least in our practice). The real problem lies with the reality of business: gathering relevant data is very costly for manufacturers and they would need a powerful financial incentive to pursue a new indication. If the target population is small (e.g. some exotic cancers), drug is widely available as a generic (e.g. cimetidine), then the incentive does not exist.

Reality is that many drugs are used off-label or though this "side-usage" of approved drugs does put an increased level of liability on the presribing physician, especially if something goes wrong. A recent example is the unexpected cardiac side-effect of Fen-Phen diet pill combo now leaves many physicians at the mercy of case and money hungry attorneys. Fear of liability is perhaps why fairly common drugs (eg Celebrex, antibiotics, statins) with potential anticancer activity is not presribed more often or widely or aggressively by oncologists.

Although off-label prescription is not malpractice or negligence, it is still imperative that prescribers be able to justify their choice of off-label treatments such that it will could not be used as evidence for breech in standard of care. Towards this end, one must make sure that patients understands why they are prescribed a certain off-label treatment and all possible side-effects should be discussed and extra careful monitoring should be conducted. In some cases where potential side-effect is great, the patient should sign an informed consent before starting treatment.

Alternative cancer treatment frequently involve the off-label use of approved drugs and examples abound: common antibiotics (Ciprofloxacin for bladder cancer, biaxin for lung cancer, minocycline or tetracycline as antiangiogenic), anti-inflammatory cox-2 inhibitors such as celecoxib (Celebrex) as adjuncts in cancer prevention or treatment, cients ... to the use of the exotic orphan drug phenylbutyrate, the inhaled delivery of Leukine (GM-CSF) and so forth.

Patients or practitioners seeking to learn more about the off-label prescribing of drugs can read the dated by still insightful 1998 report by Emalee Murphy, Esq. There is also a nice official review on "Off-Label Use of Prescription Drugs" by the office of the Secretary of the US Dept of Health and Human Services published in the American Family Physician from 2003 (See Ref). In the field of cancer, J. Boos from Germany wrote an editorial "Off-label use - label off use?" in the Annals of Oncology in 2003 that is worth reading for a review of the issue. Below is a list of useful "off-label" drugs in cancer treatment:



Celecoxib (Celebrex)

Cimetidine (Tagamet)





Mumps vaccine


PPAR gamma agonists




Tetracyclines (Minocycline, Doxycycline)