Official websites use. Share sensitive information only on official, secure websites. Correspondence to: Prof. Off-label use of medicines is generally discouraged. However, several off-patent, low-cost cancer medicines remain off-label for indications in which they are commonly used in daily practice, supported by high-level evidence based on results of phase III clinical trials.
This discrepancy may generate prescription and reimbursement obstacles as well as impaired access to established therapies. A list of cancer medicines that remain off-label in specific indications despite the presence of high-level evidence was generated and subjected to European Society for Medical Oncology ESMO expert peer review to assess for accountability of reasonableness.
These medicines were then surveyed on approval procedures and workflow impact. The most illustrative examples of these medicines were reviewed by experts from the European Medicines Agency to ascertain the apparent robustness of the supporting phase III trial evidence from a regulatory perspective. A total of 47 ESMO experts reviewed 17 cancer medicines commonly used off-label in six disease groups. We highlight the common use of off-patent essential cancer medicines in indications that remain off-label despite solid supporting data as well as generate evidence on the adverse impact on patient access and clinic workflows.
In the current regulatory framework, incentives to promote the extension of indications of off-patent cancer medicines are needed for all stakeholders. Generic, off-patent cytotoxic cancer medicines are commonly used off-label due to failure to update their formal indications. The study highlights the administrative, regulatory and workflow challenges associated with the prescription of these medicines. Incentives to promote the extension of indications of these off-patent cancer medicines are needed for all stakeholders.
Cancer is among the leading causes of death and an ongoing challenge for health care systems worldwide. Most of these medicines have new clinical applications based on large-scale phase III clinical trials, with sufficient scientific data to support their safety and effectiveness. For example, oxaliplatin, formally approved for use in patients with colorectal cancer, is commonly administered in an off-label context for patients with localised and advanced gastric and pancreatic adenocarcinomas, based on survival benefits.
