Off-label use is when an approved pharmaceutical drug is used for an unapproved indication, in an unapproved patient group or dosage, or via an unapproved administration route.
The untapped potential of marketed drugs
Cancer therapies should be evaluated for their full potential, including their efficacy against less common or other cancer types. Adding new indications to existing treatments requires additional research and trials. This process costs money but is much faster than de novo drug development because costly investigations such as those concerning safety and toxicity do not need to be repeated for an approved drug.
Off-label use in cancer treatment
Off-label drug use is common in cancer treatment, because:
many cancer drugs are effective against more than one cancer type;
more than one approved therapy may be involved, even though the combination of therapies is not approved;
research uncovers new uses for drugs that are already approved for other uses;
- patients have exhausted all other approved options, as is often the case in rare cancers.
Difficulties of off-label use
Off-label use can complicate health insurance coverage and reimbursement, and puts the prescribing practitioner at a high level of clinical responsibility, as it is the practitioner that must approve off-label use. Because these drugs are not officially indicated for a specific disease, the patient often pays full price. Even more, there is a possibility that the producing company might take the drug off the market when it is no longer needed for its official indication.
Off-label use is not without any danger.
It could be harmful if:
there is limited evidence that the drug is effective against a specific type of cancer;
potential benefits are outweighed by the risks of giving the drug.
ACF’s view on off-label use
The Anticancer Fund strives to provide more evidence for off-label drug use by performing drug repurposing trials. After clinical trials provide evidence of an existing drug's effectiveness in an unapproved indication, patient group, dosage or administration route, it should become officially registered to minimise the difficulties and dangers of off-label use.