The case for tumour-agnostic reimbursement of dual immunotherapy

Barjesteh van Waalwijk van Doorn-Khosrovani and colleagues raise important questions about this recommendation for tumour-agnostic reimbursement of nivolumab and ipilimumab in Australia. We respectfully offer a counterpoint and endorse the broad coverage. In 2025, Pharmaceutical Benefits Advisory Committee (PBAC) proposed that prescribing nivolumab and ipilimumab be guided by clinical judgement informed by the best available evidence for undefined "immunotherapy-sensitive" advanced or metastatic cancers, and, in January 2026 made a similar recommendation for pembrolizumab. Patients with rare and ultra-rare cancers, including but not limited to angiosarcoma, alveolar soft part sarcoma, gestational trophoblastic disease, small cell carcinoma of the ovary (hypercalcemic type), and adrenocortical carcinoma, face profound unmet need. Within many of these diseases, a subset of patients derives substantial benefit from immunotherapy. For example, the SWOG DART (Dual Anti-CTLA-4 and Anti-PD-1 Blockade in Rare Tumors) trial, the largest prospective evaluation of nivolumab plus ipilimumab in rare/ultra-rare cancers, demonstrated clinically meaningful activity across multiple histologies, without biomarker selection. Notably, some patients with metastatic disease remain disease-free years after treatment, outcomes rarely achieved with chemotherapy or supportive care in refractory settings. Therefore, concerns that biomarker-agnostic approval signals an evidentiary retreat overlook both our incomplete understanding immune sensitivity and the global access limits to comprehensive genomic testing. Importantly, clinical judgement is not arbitrary; it reflects training, experience, ethical reasoning, and patient-centred decision making. The question is whether demanding perfect evidence should prevent access to potentially transformative treatment. Grounded in evidence and expertise, PBAC's decision prioritises meaningful access to effective therapy for patients with few alternatives.