Using Real-World Data and Biomarkers to Optimize Immunotherapy Use in Oncology Practice

Biomarker-driven patient selection using markers such as tsMHC-II or spatial immune profiling could help pharmacists better assess the risk–benefit of immune checkpoint inhibitors and counsel patients amid the substantial toxicity burden of these therapies.

Justin Balko said integrating these markers into approved assays or laboratory-developed tests that clinicians can order and rely on would make them useful tools in clinical oncology. Pharmacists can play a role by informing care teams about test availability and the value of results for treatment decisions, particularly for patients who proceed with immunotherapy.

Pharmacists should also be familiar with treatment algorithms and the signs and symptoms of severe immune-related adverse events so they can help detect problems early and initiate recommended interventions, such as corticosteroids, per guidelines.

Determining whether these biomarkers predict immunotherapy-specific benefit rather than general chemotherapy response is complicated by the lack of chemotherapy-only comparator arms in real-world cohorts, where patients typically receive both modalities. Historical datasets of patients treated with chemotherapy alone could provide context but are vulnerable to retrospective biases and cohort differences.

The recommended approach is to combine real-world data and historical controls to identify promising molecular markers, then validate those markers in randomized phase III trials that include chemotherapy-alone arms, such as NSABP B-59 and other controlled studies that compare chemotherapy with chemotherapy plus immunotherapy.

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