Sam - I hope you don't mind, but I have another question...

Sam - I hope you don't mind, but I have another question regarding the PPMD. It has been puzzling me why the FDA with a century of experience got into the problem with Sanofi and the adverse reactions to their drug, and since the PPMD seem to have a fair amount of influence at the FDA wouldn't it be likely that they were pushing pretty hard for the conditions that became the problem for Sanofi (and if so may well also be involved in helping to find a solution to it) ?

As the PPMD claim to have sprung hundreds of millions of dollars in funding to help get projects they support established I had been thinking along the lines that they may be able to help get the ANP funding sourced, rather than offering a small amount themselves to part fund a trial. I'm still quite convinced that Pat Furlong and her whole organisation is unrelentingly committed to getting early access through orphan drug designation or whatever else can be obtained as Uboy is suggesting, because their members (the parents, carers etc.) live in a world where taking risks to save time in their experience is always the best option for their boys. And if they can swing hundreds of millions maybe she could propose a similar deal to what the others did - make their help conditional on ANP getting an orphan drug trial set-up quickly. Also wonder if the American directors are aware that the TGA has the total responsibility of determining the procedures and conditions of how new drugs will be sold in Australia (I think the lady does).