Access to orphan medicines
Deputy O'Sullivan highlighted Ireland's poor access to orphan medicines and cited the long delay in making ravulizumab available. Minister Donnelly said reforms to reimbursement, extra medicines funding and a new rare disease strategy were being pursued to improve access.
Following on from Deputy Troy's question, last month, data from the latest EFPIA WAIT indicator survey showed that just 14% of orphan medicines licensed by the European Medicines Agency since 2019 are available to patients in Ireland. This places us 26th out of the 36 European countries analysed. Treatments such as ravulizumab, which treats several forms of rare debilitating disease, have remained unavailable to patients in Ireland. In the case of the latter, it has been since its initial application in 2019, despite its availability in the majority of other countries in Europe. Reforms are often disregarded in Ireland as an option reserved to France and Germany. However, rare disease pathways are in place in several countries such as Lithuania, Latvia and Estonia, which operate ultra-orphan pathways for pricing and reimbursement, or Czechia, which operates a specialised assessment process for orphan drugs similar to what is proposed in my Bill. These processes take into account unique characteristics associated with medicines for smaller patient populations and recognise the reality apparent in Ireland. What further steps will be taken as a State to plan for future programmes in this regard?
Comment on this
I acknowledge the Deputy's ongoing advocacy and work in this area of rare diseases. The Deputy is aware of and inputting to the new rare disease strategy. We are improving the current reimbursement process through publication and implementation of the Mazars report and the recommendations therein, adding €30 million in new development funding for medicines this year. In parallel, we are developing the rare disease strategy. Through the combination of the approval and assessment process and the new strategy, we will seek better results in Ireland. To caveat, there are reports suggesting Ireland is far down the league but there are other reports that put us further up. Nonetheless, we must always strive to have the quickest possible access to the greatest number of medicines.