Ms Vicky McGrath:

I would love just to refer to one particular drug, EMA approval and what it said. During the study period, patients maintained similar progress to healthy subjects. This is in relation to a life-limiting condition. There was evidence of continued benefit on follow-up up to eight years. As it is a rare disease, the studies are necessarily small and the amount of data available on side effects is limited. Given the seriousness of the condition and the lack of existing treatments, the EMA decided that the drug's benefits are greater than its risks and that it can be authorised. By contrast, the NCPE's summary states that after reviewing the data, it concluded that it is not clear that the medicine works as well or better than other ways to manage the condition. We are talking about a life-limiting condition. "Other ways" means palliative care. The NCPE stated that the price of the medicine is too high compared with other ways to manage its condition and that it believes the medicine is very poor value for money. That says it all. As a patient, as somebody living with a rare disease, as a family member, you are looking at this and asking what is going on. It is very difficult for families to see that.