Professor Michael Barry:

The first part of this document includes updates on some of the material that we presented on 6 June and considers developments elsewhere in Europe in terms of evaluations of medicines.

When we examine cost effectiveness, we consider the clinical outcome data that are available for the medication, including quality of life, and all relevant costs, including potential savings that a new treatment might afford, for example, through reducing hospitalisation. All of these factors are considered in our evaluation - it is not just about money. It is a standardised process and is in keeping with the guidelines set down by the Health Information and Quality Authority, HIQA, which were updated in 2014 and are being updated again. That process will be completed in a few weeks' time.

The process is well established. It entails an initial rapid review, in which we examine a product, usually over two to four weeks, to decide whether it needs a full evaluation. If it does not, it is usually reimbursed without delay. If it does, we move on to a full assessment, which means that the manufacturer has to submit a dossier to us outlining the benefits and so on of the medication and proving that it is cost effective. Orphan drugs are assessed in the same way as other drugs, although we are mindful of the differences and challenges in terms of patient numbers and so on, which we can discuss later.

The health technology assessment, HTA, submission process includes a facility for patient groups to make contributions. This is a new feature. Approximately 40% of assessments in 2016 included patient submissions, which were helpful to us.

The final decision on the reimbursement on any drug is not ours. That rests with the HSE. We give our unbiased, transparent opinion on the value for money proposition related to the medicine. I have provided the committee with examples of medications for rare diseases that we have considered. The majority have been reimbursed. We have not reimbursed Vimzim for Morquio A syndrome or Translarna for Duchenne muscular dystrophy.

I will highlight the recent development in the form of the rare diseases medicinal products-technology review committee, which is a mouthful, in recognition that there are differences with drugs for rare diseases. This committee will probably have a role in managed entry, in the sense that there may be entry or exit criteria that one would apply for rare diseases drugs. I hope that this will improve matters in terms of access to drugs for rare diseases.

The document I have supplied highlights the issue in Europe and joint HTAs, such as the European network for Health Technology Assessment, EUnetHTA, which aims to co-ordinate HTAs across European countries. Another development is the BeNeLuxA initiative, which involves Belgium, the Netherlands, Luxembourg and Austria. They are examining the prospect of undertaking combined HTAs. Recently, Belgium and the Netherlands undertook a joint HTA in respect of Orkambi, although it did not seem to improve access as such.

I will correct something in my submission. It reads: "As a result Orkambi is not reimbursed in either Belgium or the Netherlands today." That has just changed. The Netherlands will now make Orkambi available. It was factual at the time of writing. We have more than 300 citizens on Orkambi. All of this shows that we were not an outlier.

Under the Valletta declaration, Ministers from Cyprus, Greece, Italy, Malta, Portugal and Spain agreed to explore collaboration in this regard.

Ireland is also involved in that collaboration. Germany was mentioned in the invitation to this meeting. We must be clear that Germany remains the front-runner in Europe in terms of availability of new authorised medicines following market authorisation, but there is free pricing in the first year in Germany. Therefore a company will get immediate access and free pricing. That comes at a price. I think that has to be acknowledged. The vast majority of European countries will look for an assessment, following the market authorisation before the drug will be let into the market. That is a key point.

There was a very nice review last year, including 13 of the 14 countries from which we reference our drug prices and only two of them allow free entry. The other 13 countries have an assessment of the drug before it is let on to the market and the vast majority of cases relate to pricing. In contrast to Germany, reimbursement of medicines in many European countries including Ireland and other developed countries only occurs following a post marketing evaluation, which is what we do here.