Next-generation approaches to statistical significance

WHEN TRIAL DATA IS LACKING
The panel considered the potential for supplementary data sets – such as pharmacodynamic read-outs, histological evidence and historical controls – in enabling regulators to reach robust decisions.

Nick Sireau, of AKU Society, talked about his early experiences of trying to get new drugs approved for Alkaptonuria (AKU); his two sons both suffer from the ultra-rare disease. His organization relies heavily on grant funding and donations, with access to funds often difficult. It doesn’t help that the authorities’ requirements for clinical evidence have been quite restrictive up to now.