Extent and outcomes of off-label drug use in oncology practice: a systematic review of literature

Aim/Background

Use of a drug outside the regulatory recommendations is denoted as off-label use. Off-label uses in oncology setting are widespread but its real magnitude and outcomes is still unknown. Hence, this review aims to assess extent and clinical outcomes of off-label drug use in oncology setting

Methods

A systematic search was made using Pubmed from 1975 to 2015. Studies addressing the extent and clinical outcomes of off-label use in different oncology settings, different chemotherapeutic drugs used and different cancer types were identified.

Results

Of the 191 eligible papers retrieved, 41 studies were included in this systematic literature review. Off-label use in inpatients ranged from 18 to 41%. Among adult cancer patients, 15 to 85% received a minimum of one off-label chemotherapy. The main reason for off-label use was unapproved drug for specific tumour and modified applications. Metastatic cancers and palliative care patients received the most off-labelled drugs. Off-label use of drugs such as sunitinib and sorafenib for thyroid cancer, combination of nab paclitaxel and gemcitabine for advanced pancreatic cancer showed real world outcomes comparable with control trials.

Conclusions

Off-label use in cancer therapy is highly prevalent. Greater scrutiny of this practice to establish the benefit-risk ratio for patients at the time of prescribing is required. In addition, robust guidelines working at each level of oncology care is highly needed to facilitate rational off-label prescribing.

Clinical trial identification

Disclosure

All authors have declared no conflicts of interest.