1542MO - Mapping the scale of off-label use in oncology: An important step in harmonising access

Background

Off-label use of anticancer drugs is often considered for treating rare tumours or advanced stages of disease when standard treatments are exhausted or ineffective. International reports suggest that 13%–71% of cancer patients receive at least one line of off-label chemotherapy. However, understanding the extent of off-label use in oncology remains limited. By mapping off-label indications, we can enhance our understanding of clinical needs, improve pathways for evidence assessment and facilitate access through reimbursement.

Methods

The off-label claim codes, approved by Dutch Healthcare Insurers for reimbursement without prior authorisation were analysed. Non-oncology ID- codes were excluded. The codes were categorised based on solid tumour or haemato-oncological indication, paediatric or adult indication, monotherapy/combination therapy, type and number of unique drugs, and data the positive reimbursement decision was based on (e.g. literature).

Results

Approximately 30% of the reimbursed oncological indications in the Netherlands are off-label. These indications consist largely of chemotherapy, (66% of total indications) especially in the treatment of solid tumours (48%). However, off-label use is also prevalent in other types of treatment, e.g. tyrosine kinase inhibitors (TKI), endocrine therapy and monoclonal antibodies (respectively 9%, 7% and 5%). Off-label use is common in treatment of children, with 26% of all indications being paediatric. Table: 1542MO

1. Different types of drug (e.g. TKI + chemotherapy)2. Data for reimbursement decision unknown

Conclusions

Our study underscores the scale and necessity of evaluating and reimbursing off-label use of oncology drugs, to provide equal access to effective treatment. This is particularly crucial for indications that may not be commercially viable for companies to pursue regulatory approvals due to small patient numbers or patent expiration. Reimbursement of off-label use enhances equitable access while also providing affordable and additional treatment options for patients.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Horizon Europe Cancer Mission, EC grant agreement no. 101104269 (PRIME-ROSE).

Disclosure

H. Gelderblom: Financial Interests, Institutional, Local PI: Deciphera, Cytovation; Financial Interests, Institutional, Coordinating PI: Boehringer Ingelheim, AmMax Bio, Debiopharm, Abbisko. All other authors have declared no conflicts of interest.