Sir, – While we still await a decision on granting Kaftrio, the ground-breaking drug to 35 children with cystic fibrosis, we note and welcome the ambition for reforms in the overall drug reimbursement system in Ireland. However, these do not go far enough, in our experience.
Minister for Health Stephen Donnelly recently stated there is need for greater transparency and communication, especially with patient groups around the drug therapy reimbursement process in Ireland. This ambition is warmly welcomed by Cystic Fibrosis Ireland. In his statement to The Irish Times on March 3rd, the Minister stated: “We need to avoid situations like we have seen with Kaftrio,” he said, referring to the standoff between drugmaker Vertex and the HSE over the cost of providing the cystic fibrosis medicine already available to 140 other children in Ireland to a 35-strong group of children aged between six and 11″.
Following the publication of the Mazar’s Report on Drug Reimbursement (completed in 2020 but only published in February 2023), Mr Donnelly also recently stated: “I want to see improvements in the overall transparency of the process, and I have asked the HSE to bring forward proposals in that regard as a matter of urgency. Greater engagement with stakeholders, including patient groups, will ensure that patients are aware of the process and have sight of various steps and timelines.”
This statement is again warmly welcomed by CFI. However, these ambitions are only a partial solution and we are likely to see further protracted disputes on drug reimbursement in Ireland for children and adults with rare diseases. Drug companies have their role to play in providing affordable drugs. But there also needs to be greater recognition of the huge costs and risks of bringing a drug from test-tube to reality.
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There remains no commitment by the Government to bring in orphan/rare drug designation. Under the existing legislation, there is no differentiation between orphan medicinal products and other products in Ireland. In recent years it has become clear that orphan/rare disease drugs continually struggle to secure reimbursement, often despite their wide ability elsewhere in Europe. The international EFPIA WAIT report is published every year provides data on Ireland’s performance on accessing orphan drugs compared with other European countries. For example, the rate of availability of Orphan drugs 2017-2022, measured by the number of medicines available to patients in European countries (as of January 1st, 2022) provides an important insight as to how we compare. Germany fares best with 95 per cent of available rare disease drugs; England is at 61 per cent; the EU average is 37 per cent, and Ireland is at 30 per cent.
Orphan Drug Designation came into the US in 1983. The European Medicines Agency enacted Orphan Drugs Designation in 2001. We are still waiting for Orphan Drug Designation in Ireland in 2023. We would call on the Government to enact Padraig O’Sullivan TD’s Private Member’s Bill on this issue with a matter of urgency. We need a level playing field for all who need life-changing drug therapies in Ireland, including those with rare diseases. – Yours, etc,
PHILIP WATT,
CEO Cystic Fibrosis Ireland,
Dublin 6.