Pa ystyriaeth y mae Ysgrifennydd y Cabinet wedi'i rhoi i ddefnyddio cofrestrfa ffibrosis systig y DU i alluogi mynediad dros dro at Orkambi, ac a fyddai hyn yn ffordd briodol i atal rhagor o oedi mewn triniaeth i bobl â ffibrosis systig yng Nghymru, o gofio ei fod bellach wedi'i drwyddedu ar gyfer tair blynedd?
I note the Cystic Fibrosis Trust propose to carry out a pilot project using the UK CF Registry to collect data to monitor the clinical impact of Orkambi® on patients, in addition to the clinical trails conducted previously. Registry data can help us understand the real world outcomes of innovative treatments and I therefore welcome the Trust’s proposal.
As the Trust acknowledges, any such project would need to be “underpinned by an acceptable interim commercial access arrangement”. Any commercial arrangement would need to reflect the significant uncertainty identified by NICE in its appraisal of Orkambi®. Furthermore an agreement would need to ensure the price NHS Wales pays for Orkambi® could be considered to be within the bounds of what would normally be considered cost-effective by NICE and other UK appraisal bodies, as is accepted by the UK pharmaceutical industry.