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The question of whether chimeric antigen receptor (CAR) T cell therapies should be used in earlier lines (after 1–2 prior lines of therapy) in patients with relapsed and/or refractory multiple myeloma remains unanswered. Herein, I argue that the use of surrogate end points that lack a robust correlation with overall survival, as well as suboptimal control arms and use of post-progression therapies, limit the ability to make definitive conclusions on the basis of the available data.
Certain subsets of patients with multiple myeloma or its precursor conditions are overtreated with current approaches to therapy. Herein, we highlight several key areas where we believe de-escalation of treatment is needed. Dedicated trials to assess these de-escalation approaches and urgent changes to current clinical practices are needed.
In 2023, the US FDA approved several new cancer drugs and biologic agents, including seven small-molecule inhibitors, four bispecific T cell engagers, two anti-PD-1 antibodies and one cell therapy product. Regulatory focus areas included analyses of biomarker-positive subgroups that drive efficacy, equipoise in randomized controlled trials and a new authority to require confirmatory trials be underway before accelerated approval.