Off-label despite high-level evidence: a clinical practice review of commonly used off-patent cancer medicines
- Several 'old' cancer medicines remain off-label despite strong scientific evidence supporting their use in specific settings.
- Off-label prescriptions often carry administrative and legal burdens for physicians, affecting workflows and potentially patient access to safe and effective therapies.
- Results from large phase III trials should lead to regulatory applications for new therapeutic indications but financial and market disincentives hinder this process for generic medicines.
- Most studies on well-known, off-label anticancer products do not trigger major clinical or regulatory issues, suggesting the need for early regulatory advice to address gaps and ensure efficacy.
- European initiatives are in place to support patient access to off-label medicines with proven value, such as the STAMP framework and EMA’s repurposing framework.
- The study emphasizes the need to streamline the legal and regulatory framework to update indications of off-patent medicines, thereby optimizing treatment options for patients.
Cancer is among the leading causes of death and an ongoing challenge for health care systems worldwide. During the last decade, a plethora of new medicines have been approved by regulatory bodies for the treatment of neoplastic diseases and in most countries, medicines are reimbursed according to their labeling indication. However, the off-label use of medicines is quite common, especially in oncology. According to the results of an American Society of Clinical Oncology (ASCO) survey, ∼50% of patients with cancer have received chemotherapy in an off-label indication during their disease course.
At the same time, the off-label use of antineoplastic high-cost medicines is increasingly implemented based on identified plausible molecular targets, despite support of clinical benefit by low-level evidence only, or none....
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