Well the authorities have now approved the drug but unfortunately we do seem to be dragging our feet in making it available to the few patients and families who could benefit. The disease is ultra-rare, at the last count in 2010 affecting about 170 people in UK, of whom 140 are thought to be in England. Some 10-15 new cases present each year in UK. There are about 50 patients like the lady I was chatting to, people who are locked into lifelong dialysis who will never receive a transplant until eculizumab can be used to protect their new kidney .
The aHUS patient support group and the wider kidney community had been hoping the drug would be available from January this year but the Department of Health ducked the issue deciding instead to defer national funding and refer the case to NICE for further consideration of the affordability before reporting to the new commissioning board that is now called NHS England. Well I hope that NHS England does not let the kidney community “get left behind “ to quote the new planning guidance . The evidence for benefit is clear, yes the costs are high but the benefits are truly life changing for people and families with aHUS . If we, the system, get our act together its quite a reasonable aspiration to expect a successful live donor transplant for the lady I met and a holiday in the sun (wearing sun block of course) shortly thereafter. One could also wonder “how come she hasn’t been able to get away for a break or a holiday while on dialysis“ but that’s another even longer running story that for now I will leave for another day .