Assessment of tumour-agnostic therapies in basket trials

There are notable differences between the two countries, for instance with regards to the French agency asking for prospectively planned real-world comparator data if randomised trials are not feasible, and the English HTA agency perhaps being more pragmatic for patient access to treatment and providing conditional approval and coverage with re-evaluation of additional evidence at a later date. Taking the example of the driver mutation in NTRK, one of the largest clinicogenomic oncology databases currently has less than 30 patients positive for this mutation out of over 58 000 patients.3 This number is smaller than the number of patients in the pivotal larotrectinib trial; therefore, indirect comparisons using this dataset would provide little reduction in uncertainty about clinical effectiveness. VS reports grants from Eli Lilly/Loxo Oncology, Blueprint Medicines Corporation, Turning Point Therapeutics, Boston Pharmaceuticals, and Helsinn Pharmaceuticals outside the submitted work; grants and advisory board or consultant positions with Eli Lilly/Loxo Oncology outside the submitted work; research grants from Roche/Genentech, Bayer, GlaxoSmithKline, Nanocarrier, Vegenics, Celgene, Northwest Biotherapeutics, Berghealth, Incyte, Fujifilm, D3, Pfizer, Multivir, Amgen, Abbvie, Alfa-sigma, Agensys, Boston Biomedical, Idera Pharma, Inhibrx, Exelixis, Blueprint Medicines, Altum, Dragonfly Therapeutics, Takeda, National Comprehensive Cancer Network, NCI-CTEP, University of Texas MD Anderson Cancer Center, Turning Point Therapeutics, Boston Pharmaceuticals, Novartis, Pharmamar, and Medimmune outside the submitted work; an advisory board or a consultant position with Helsinn, Incyte, QED Pharma, Daiichi-Sankyo, Signant Health, Novartis, Relay therapeutics, Roche, and Medimmune outside the submitted work; travel funds from Pharmamar, Incyte, ASCO, and ESMO outside the submitted work; and educational fund support from Medscape outside the submitted work.