John Brassil (Kerry, Fianna Fail) | Oireachtas source

I thank the representatives of the HSE for coming in to address the committee.

I will get straight to the nub of the issue as I see it. A total of 148 orphan drugs are available to deal with rare diseases and ultra-rare diseases. Of those, 53 qualify for reimbursement in Ireland, which is 36%. While I do not want to get into other countries' models and how they deal with it, if we compare with other countries, the UK reimburses 68 of the 148; Spain reimburses 75; France reimburses 84; and Germany reimburses 133. By comparison with those EU countries we are at the bottom end of the scale.

Deputy Murphy O'Mahony stated she believed the system was broken, which the HSE witnesses rejected. If it is not broken, it is certainly not operating satisfactorily from a patient and a public representative point of view. I am sure it is also true from the HSE's point of view because Mr. Flanagan said he would love to be in a position to approve every drug that could be of benefit. At 36% and 53 out of 148, we are not performing to as high a level as we should. That is factual and we need to do something about it.

In its assessment the HSE uses a quantitative approach. It follows the 2013 Act. While that Act mentions cost effectiveness, it is not the bible. It does not have to use cost effectiveness and other parameters could be used. The HSE could take a qualitative approach to certain drugs if it wanted to. Rare diseases affect less than five in 10,000 which is 0.05% and ultra-rare diseases affect 0.002%. The patient cohort is tiny. It is extremely difficult to acquire clinical data on those patients. Using a quantitative approach in such cases will never work and will always result in "No" as the answer.

The witnesses can correct me if I am wrong in this opinion. We are not bound to the quantitative assessment. If we want to use a different approach, we can. We can determine that the health technology assessment guidelines are not relevant to a particular drug if we want to. We can take a particular drug and decide that we will not use the HTA guidelines because in this particular case they are not relevant and then come up with a different methodology that gives it some chance.

When dealing with a very specific disease we will never help those two, five, seven or eight people in a population unless we take a different approach. We need to take that on board as part of the HSE's review or otherwise we will be back in here every six months having the same debate, be it Respreeza, Translarna, Kuvan or whatever drug we are battling for on behalf of our constituents.

I ask the witnesses to take that into consideration. We need to broaden our approach to give these drugs some chance of getting over the line.

Let me mention specifically a few drugs. One currently under review is Kuvan for phenylketonuria, PKU. There was a time when we were world leaders in dealing with this particular disease. We came up with a dietary concept and we changed the lives of the people who suffer from this very rare condition. My colleague, Deputy Kenny brought in people who suffer from this particular disease to the AV room and we had a very good presentation. We are at a very critical stage of that. Mr. Shaun Flanagan mentioned about the ability to argue cost. My information, and it might be wrong - I hope it is - is that the HSE has not gone back to the company looking for a negotiated cost on this.