Gino Kenny (Dublin Mid West, People Before Profit Alliance) | Oireachtas source

I thank everybody for their statements. It is a fascinating debate, which was prevalent in the previous Dáil with regard to access to orphan drugs, particularly scenarios where families were campaigning to get access to these transformative medications.

Other Deputies and I got to know organisations, particularly those families campaigning for the drug Kuvan to treat phenylketonuria, PKU, Orkambi for cystic fibrosis, CF, and Spinraza for spinal muscular atrophy, SMA. All these families campaigned hard, for years in some cases, in difficult circumstances. They obtained the medication eventually through much hard work and many hard years of lobbying with regard to the criteria, which are quite stringent.

It is my understanding that the National Centre for Pharmacoeconomics gave the price structure for some of these orphan drugs, which are sometimes made in Ireland. These medications can be at very high cost, however. The pharmaceutical companies probably do not see where they can make a lot of money on these cases, which they can in most instances. That needs to be looked at with regard to the cost of orphan drugs, particularly in respect of the National Centre for Pharmacoeconomics.

I am aware that many of the families stated that the criteria around the reimbursement was very protracted and went on for years in some cases. My question is underpinned by the 2013 legislation, which is the Health (Pricing and Supply of Medical Goods) Act 2013. Other members who were Deputies in the previous Dáil have said that this Act needs to be amended to be cognisant of orphan drugs that need to be accessible to those rare conditions. The narrative, which was very prevalent and true, is that these drugs were transformative. The great thing about modern medicine is that these medications can be life-changing yet, at the same time, people cannot gain access to them. I find it very difficult to comprehend that there is a medication out there that could be of great benefit but it cannot be obtained because of price. We do not live in a civilised society if a child cannot get a medication because of price. That needs to be looked at and medications like that need to be accessible. Is it any easier now to gain access to orphan drugs? My feeling is that it is not. What are our witnesses' views on the National Centre for Pharmacoeconomics’s criteria for the pricing of orphan drugs?