Building a trial around one patient, or letting one trial swap drugs in and out as evidence accumulates.
Bespoke antisense drugs have been made for single patients with rare neurological disease under an FDA pathway, and the same logic is being applied to private neoantigen and splice-targeting therapies in cancer. Adaptive platform trials such as I-SPY 2 in breast cancer graduate or drop arms on interim Bayesian analysis, which is the scalable version of the same idea. The obstacles are manufacturing, cost, and how to learn anything generalisable from a single case.
Either an intervention is designed for one person's mutation, or a master protocol keeps a shared control arm while arms enter and leave, so each new question costs less.
Query for this technology: (TITLE:"N-of-1 and rapid platform trials" OR ABSTRACT:"N-of-1 and rapid platform trials") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about N-of-1 and rapid platform trials, not a curated reading list.
Shares Response-adaptive randomisation, Seamless, adaptive and Bayesian trial designs, Master protocol (platform, basket and umbrella trials), Radical oncology: what could change the war by 2035 and the tags frontier, promising.
Shares Radical oncology: what could change the war by 2035, Personalised neoantigen (mRNA) vaccines and the tags frontier, promising.
Shares Radical oncology: what could change the war by 2035, Personalised neoantigen (mRNA) vaccines and the tags frontier, promising.
Shares Radical oncology: what could change the war by 2035, Functional (ex vivo) drug testing and the tags frontier, promising.
Shares Radical oncology: what could change the war by 2035 and the tags frontier, promising.
Shares Radical oncology: what could change the war by 2035 and the tags frontier, promising.
Shares Radical oncology: what could change the war by 2035 and the tags frontier, promising.
Shares Radical oncology: what could change the war by 2035 and the tags frontier, promising.