PT  - JOURNAL ARTICLE
AU  - Luca Mazzarella
AU  - Stefania Morganti
AU  - Antonio Marra
AU  - Dario Trapani
AU  - Giulia Tini
AU  - Piergiuseppe Pelicci
AU  - Giuseppe Curigliano
TI  - Master protocols in immuno-oncology: do novel drugs deserve novel designs?
AID  - 10.1136/jitc-2019-000475
DP  - 2020 Mar 01
TA  - Journal for ImmunoTherapy of Cancer
PG  - e000475
VI  - 8
IP  - 1
4099  - http://jitc.bmj.com/content/8/1/e000475.short
4100  - http://jitc.bmj.com/content/8/1/e000475.full
SO  - J Immunother Cancer2020 Mar 01; 8
AB  - The rapid rise to fame of immuno-oncology (IO) drugs has generated unprecedented interest in the industry, patients and doctors, and has had a major impact in the treatment of most cancers. An interesting aspect in the clinical development of many IO agents is the increasing reliance on nonconventional trial design, including the so-called ‘master protocols’ that incorporate various adaptive features and often heavily rely on biomarkers to select patient populations most likely to benefit. These novel designs promise to maximize the clinical benefit that can be reaped from clinical research, but are not without costs. Their acceptance as solid evidence basis for use outside of the research context requires profound cultural changes by multiple stakeholders, including regulatory bodies, decision-makers, statisticians, researchers, doctors and, most importantly, patients. Here we review characteristics of recent and ongoing trials testing IO drugs with unconventional design, and we highlight trends and critical aspects.