I think the biggest opportunity is designing trials around the questions that actually matter to patients. We now have multiple highly active immunotherapies, so it’s no longer simply about whether a therapy works. It’s increasingly about who should receive which therapy, when, and in what sequence. I’d like to see advocacy groups and pharma work together to incorporate quality of life, time at home, treatment burden, and access along with our traditional safety and efficacy endpoints...
I think the biggest opportunity is designing trials around the questions that actually matter to patients. We now have multiple highly active immunotherapies, so it’s no longer simply about whether a therapy works. It’s increasingly about who should receive which therapy, when, and in what sequence. I’d like to see advocacy groups and pharma work together to incorporate quality of life, time at home, treatment burden, and access along with our traditional safety and efficacy endpoints. We also need trials that better represent the patients that we treat in our everyday practice, including those who might be older, who might have some degree of renal impairment, extramedullary disease, and other high-risk features. And importantly, I would also like to see biomarker and translational studies built prospectively into these trials so ultimately we can become much more precise about selecting the right therapy for the right patient at the right time. To me, the biggest gap is between demonstrating that a therapy can work and understanding how to best use it in the real-world setting or in patients that we see in our everyday practice. Trials give us response rate and survival outcomes such as progression-free and overall survival. But clinicians and patients are asking very practical questions. Can this patient tolerate this therapy? How much time will they need to be in the hospital or in the clinic? Can treatment eventually be stopped should somebody achieve a very deep response? And the patients we see every day have comorbidities. They might have renal impairments, cytopenias, they might be frail or have rapidly progressive disease. So future trials need to tell us not only how effective a therapy is, but how feasible is this therapy? What burden does it place on our patients? And how does it perform in patients we actually treat in our everyday practice?
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