Oireachtas Joint and Select Committees

Wednesday, 15 October 2025

Joint Oireachtas Committee on Health

Treatment of Rare Diseases: Discussion

2:00 am

Professor Michael Barry:

The pricing reimbursement assessment at the time looked at this. The drug was quite expensive, with Zolgensma costing €2 million. Essentially, it was based on the clinical evidence. One may agree or disagree with that but it was based on the fact that the clinical evidence suggested that, the younger a person was, the better they would respond to the drug. The 18-year timeframe was introduced at that stage. As I mentioned earlier, that can be reviewed if necessary but that is where the decision came from. It was based on the clinical trial data at that particular point in time. I have not looked at it recently in the sense of what that timeline should be but that is how it came about. It was based on the clinical trial data at the time that the reimbursement recommendation was made.

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