5. Off-label use across specific patient groups

5.2. Oncology (cancer)

The off-label situation is more extensive in oncology than in other therapeutic fields. Across almost all cancer types, many products are already commonly used off-label. Because oncology remains a therapeutic area of high unmet need, prescribers and patients are often willing to accept the use of medicines in circumstances in which their efficacy and safety profiles have not yet been clearly established. A systematic review of the literature showed that off-label use of oncology medicines was in the range of 18% to 41% for hospitalised patients and of 7% to 50% for patients treated ambulatory. In total, 13% to 71% of adult patients with cancer received at least one off-label chemotherapy during their treatment. This was more likely to happen in patients who had exhausted all standard lines of treatment. The main off-label uses were in an unauthorised indication for a specific tumour and in an unauthorised line of treatment (Saiyed, Ong and Chew, 2017 [1]).

The principal reason is the large number of different types of cancer. In fact, each cancer medicine may be useful in several different types of cancer. In practice, many widely used anti-cancer medicines are not authorised for all indications in  which they can be effectively employed.

In a 2021 policy brief titled ‘ Repurposing of medicines in oncology – the underrated champion of sustainable innovation’ [2] the World Health Organisation (WHO) describes an initiative for Non-commercial repurposing[3] of off-patent medicines for cancer treatment which should have the potential of addressing currently unmet needs in a cost-effective way, especially in areas that are not attractive for the industry, such as rare cancers.

[1] Saiyed, M., P. Ong and L. Chew (2017), “Off-label drug use in oncology: a systematic review of literature”, Journal of Clinical Pharmacy and Therapeutics, Vol. 42/3, pp. 251-258, http://dx.doi.org/10.1111/jcpt.12507.

[2] Repurposing of medicines – the underrated champion of sustainable innovation. Copenhagen: WHO Regional Office for Europe; 2021. Licence: CC BY-NC-SA 3.0 IGO.

[3] Repurposing is a strategy to identify new uses for approved or investigational medicines outside the scope of their original medical indication.