A novel trial methodology to test interventions with very large effect sizes: the case of dostarlimab in mismatch repair-deficient, locally advanced rectal cancer

Dostarlimab (Jemperli, GlaxoSmithKline) is an anti-programmed death receptor-1 monoclonal antibody (anti-PD-1) recently tested in a non-randomized, phase II trial (NCT04165772) which included patients with mismatch repair-deficient, locally advanced rectal cancer. Among the first 12 patients treated with dostarlimab, 100% achieved a clinical complete response with no patients experiencing progression or recurrence to date. Most impressive, none required chemotherapy, radiotherapy or surgery the prevailing standard of care. In this paper, we discuss the impressive results of this trial and how they relate to cancer policy, as well as propose a novel trial methodology to assess dostarlimab.