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Joint Oireachtas Committee on Health: Treatment of Rare Diseases: Discussion (15 Oct 2025)

Maria Byrne: If the National Centre for Pharmacoeconomics is happy with the information, does that mean the drug will become readily available and what are the next steps?

Joint Oireachtas Committee on Health: Treatment of Rare Diseases: Discussion (15 Oct 2025)

Maria Byrne: Is Professor Barry hopeful that the NCP will hear within the next month from the company?

Joint Oireachtas Committee on Health: Treatment of Rare Diseases: Discussion (15 Oct 2025)

Maria Byrne: I want to discuss rare diseases and orphan drugs. Why is there a long waiting time of 710 days compared with other countries that seem to have a faster process? Is the process fit for purpose? Does it need a review or changes to be made to fast-track the process?

Joint Oireachtas Committee on Health: Treatment of Rare Diseases: Discussion (15 Oct 2025)

Maria Byrne: Professor Barry referred to the whole transparency side and decision-making. What steps should be taken or are there more steps that should be taken to improve that whole area?

Joint Oireachtas Committee on Health: Treatment of Rare Diseases: Discussion (15 Oct 2025)

Maria Byrne: Great. I have one other question to do with the oncology and the non-oncology orphan medicines that are not available in Ireland. What proportion have seen an application made to the reimbursement assessment process by the marketing authorised holders?

Joint Oireachtas Committee on Health: Treatment of Rare Diseases: Discussion (15 Oct 2025)

Maria Byrne: On the orphan drugs that fail the HTA due to restrictive cost-effectiveness, is the Department considering a different valuation framework or just a threshold for these medicines?

Joint Oireachtas Committee on Health: Treatment of Rare Diseases: Discussion (15 Oct 2025)

Maria Byrne: How are patient organisations and clinicians meaningfully involved in the HSE reimbursement process for orphan drugs?

Joint Oireachtas Committee on Health: Treatment of Rare Diseases: Discussion (15 Oct 2025)

Maria Byrne: I thank the witnesses.

Joint Oireachtas Committee on Health: Treatment of Rare Diseases: Discussion (15 Oct 2025)

Maria Byrne: I, too, want to raise the same issue. I have had dealings with a family whose grandchild lives in Wexford. The child is six years of age and is badly in need of givinostat. It is a fact that in April 2025 the European Medicines Agency granted conditional approval and on 6 June the European Commission granted conditional approval.

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