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In September 2016, the USA's Food and Drug Administration (FDA) approved a new drug for the treatment of Duchenne muscular dystrophy under its accelerated approval pathway. FDA approval was based on surrogate endpoints and, to date, no clinical benefit has been established.1 Internationally, there is considerable push to innovate therapeutics and one major feature of this is the acceleration of the process for new drug registrations, particularly in the field of cancer chemotherapy and for the management of rare diseases. In the USA, this is encapsulated in the 21st Century Cures Act currently going through the USA Senate; while in Europe, the European Medicines Agency …