Colm Burke (Fine Gael) | Oireachtas source

I thank the witnesses for the presentations. On the previous occasion I had a concern, which I raised at the September meeting, about the matter being revisited. The figures being given to me are to the effect that there are approximately 148 orphan drugs, with 133 approved and used in Germany. We are using either 53 or 55 and other countries around Europe seem to be way ahead of us in using new drugs. That is the reason I suggested in September that we revisit this. There is also the matter of the delay in the process. I have a note from one company, which made a submission in March 2015. It went through the process, and there was a decision in June 2017 that the drug would not be reimbursed. It filed an appeal and advised that the subject would be considered at a meeting of 14 September. As of today, the company is still not clear what has happened and has had no response. I got that from BioMarin about a specific drug. The case has been ongoing since March 2015, we are now in November 2017, and there has been no conclusion on whether the application is in or out. An initial decision was taken but the company filed an appeal against it.

The second matter is that we seem to be lumping orphan drugs into the same type of process as any other drugs that would be commonly used. We are talking about a very small cohort. There are some drugs that might only be available or suitable for approximately ten people in the country. I know it is a very difficult process but the argument has been put forward to me. I have dealt with rare diseases for quite a number of years. Last week I had representatives of a group of sufferers of epidermolysis bullosa into Leinster House. It is a genetic skin disorder and skin keeps peeling from people's bodies. There is no medication for it. I am fairly familiar with the rare diseases area. This is about orphan drugs being put through the same kind of process as drugs that would be commonly used. This seems to be a big concern of people involved in the rare diseases area.

Have we looked at how other jurisdictions process this? We have raised the German system and there is the question of how the German system operates. It allows the drug and there is a price negotiation process thereafter. I accept it would be quite difficult for us to implement that kind of system. I know we are talking about working with other countries to negotiate with drug companies. Our process seems to be extremely slow. Representatives of one company told me a drug has been accepted by the European Medicines Agency but it has had to file 300 or 400 pages of documents to support the application. I do not understand why we need to go through that kind of a complicated process.

There are cases where a drug is being used with the support of the drug company but we are told it will not get approval under the drugs refund scheme. Do we need to set up a mechanism to deal with the people who have now become reliant on that drug? In many cases they have been on the drug for anything up to 18 months, two years or even two and a half years while the process has gone on. Suddenly, they are told by the pharmaceutical company that it is no longer prepared to cover the cost and it will not be covered by the drugs refund scheme. Do we need to set up a process to deal with that? I have received numerous letters over recent weeks from people who are concerned about this. They are on medication but do not know what will happen from now on. Have we considered that? Orphan drugs are different from drugs used by wider numbers of people. Is there a need for a specific budget?

We use quality adjusted life years in the cost assessment. Should we consider using a different system when we deal with orphan drugs? I am not convinced that we are approaching our assessment correctly. It concerns a very small group of people in many different areas but we appear to be applying the same set of rules, and I do not know if that is appropriate in the area of orphan drugs.