Quite right Saus. There is always a story behind the numbers. I...

Quite right Saus. There is always a story behind the numbers. I saw a trial Mum on one of the Rett org videos. They had a 4.5hr drive to the medical centre, 6 hrs in clinic and 4.5hrs home. Her daughter had diarrheoa and they dropped out. Stastically they may have dropped out because of the bowel problems but I'm sure its not that simple. Had the lived around the corner from the clinic they may have decided to tolerate the diarroheoa. Even more importantly to our current discussions they have now been prescribed Daybue and are working through dosage titration to fine tune the levels.

So there is no way that you can draw a direct line from the dropout rate from a tough trial, which itself imposed great strain on many participant familes, to projections of uptake of a drug that can be taken at home in a fine tuned dose.

Rather than saying "well 40% dropped out of the trial so the true long term market is therefore 60% of sufferers" I'd be turning that on its head.

Of the 40% who dropped out what percentage
- found the effort of participation placed too much strain on the family?
- figured out they were on the placebo anyway so why tolerate the travel and side effects?
- weren't able to titrate the dose to reduce the side effect because of the trial design?
- found that diarrheoa in a non-verbal child which might be managable at home was just unbearable while travelling or staying in hotels?

My best guess. The vast majority of those 40% have already seen their primary care doctor about a prescription for Daybue.