Professor Michael Barry:

No, it was a few days ago. I just wanted to give the committee a flavour of what is going on. There were five meetings in 24 days and it is testimony to the significant work done by committee members, who do such work on their own time and in an unpaid capacity.

The committee has focused on two medicines to date, which are sapropterin or Kuvan for phenylketonuria, and nusinersen or Spinraza for the treatment of spinal muscular atrophy, SMA. As an example, the committee recommended to the HSE drugs group that access to sapropterin be considered under a managed access programme. In other words it would occur where clinical criteria were laid down for starting and stopping the medication if it was not working. It was taken by the HSE drugs group, which made a positive recommendation with regard to Kuvan, which is good news. The funding issue must be decided on by leadership. Currently we are preparing a rare diseases technology review committee recommendation for nusinersen for spinal muscular atrophy and we hope to submit it to the drugs group shortly. That is a flavour of how the new committee is working.