Innovative Cancer Drugs: Medicolegal Assessments on Efficacy and Necessity

Abstract

Objective: In recent years, targeted therapies and immune checkpoint inhibitors have provided significant gains in survival and quality of life compared to conventional chemotherapies. However, due to biomarker-based selectivity, high cost, and reimbursement restrictions, the concepts of “necessity” and “indication appropriateness” have become controversial from a medicolegal perspective. This study aimed to evaluate the clinical and forensic aspects of innovative cancer drug use by reviewing court case files.
Methods: A total of 455 case files submitted between January 1, 2022 and May 31, 2025, were retrospectively analyzed. Demographic data, cancer type and stage, histological subtype, performance status, previous treatments, innovative drug active agents, licensing and indication status, therapeutic intent, and council and board decisions were recorded. Data were evaluated using descriptive statistics.
Results: Of the cases, 53.6% were female and 46.4% male; the most common age group was ≥65 years. The most frequent malignancies were lung (35.2%) and breast (20.0%) cancers, and 69.5% of the patients were in stage IV disease. The most frequently used drugs were pembrolizumab (29.7%), nivolumab (16.0%), and atezolizumab (12.5%). Of all uses, 99.1% were licensed and 85.1% were indication-appropriate. Treatment intent was adjuvant in 48.6%, neoadjuvant in 10.1%, and post-progression in 41.1% of the cases. Multidisciplinary council decisions were documented in 47.3% of files, and the Forensic Board concluded that treatment use was “appropriate” in 97.6% of cases.
Conclusions: The findings indicate that innovative cancer drugs in Türkiye are mostly used within licensed and guideline-concordant indications. However, the lack of biomarker data and multidisciplinary council documentation constitutes an important limitation in medicolegal evaluations. Addressing these deficiencies would make therapeutic decisions more defensible both clinically and legally.

Keywords

• Innovative cancer drugs
• Smart drugs
• Cancer
• Indication appropriateness
• Medicolegal evaluation

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