Professor Michael Barry:

As regards the assessment of orphan drugs, in some cases there is a challenge in respect of submitting data to demonstrate their cost-effectiveness because of patient numbers. That is particularly so for ultra-orphan drugs. As regards Orkambi, the manufacturer submitted a very good dossier and was able to put forward a case for cost-effectiveness even though we said "No". Mindful of that and in the context of the rare diseases plan, the rare diseases medicinal products technology review committee is being set up. It can be quite complex in the sense that possibly 10% or 15% of people in a particular cohort will respond to the drug. The new committee will be of benefit in that regard as it may be able to devise managed access for patients who respond to the drug and ensure they get access to the drug. That is a reasonable response to the current situation and very similar to what we do for cancer drugs. The national cancer control programme technology review group provides its opinion on top of the data we send to the HSE drugs group. The rare diseases medicinal products technology review committee is modelled on that and may go a little further.

We are getting better at facilitating stakeholder involvement and we need to do continue that. Some 40% of the HTAs we looked at in 2016 included stakeholder engagement and we have been working with groups such as the Irish Platform for Patient Organisations, Science and Industry, IPOSI. We have been working with IPOSI to provide patient groups with more information in respect of the HTA process and that is ongoing and educational. We are very grateful for its collaboration in that regard.

Deputy Murphy O'Mahony asked if the process is broken. I do not believe it is. It is a fair, open, transparent and rigorous assessment. It is unquestionably rigorous. We are lucky to have an expert team who are able to go through all the clinical and economic data. In terms of what transpires from the process, when we make a negative recommendation, it does not finish there, but rather goes to the HSE to negotiate a better price with the pharmaceutical industry. Everyone wins with that because we are now obtaining discounts in the order of 30% to 50% for products. If we said we had no problem taking the asking price, for 2016 that would have added €1 billion over five years. That we can enable the HSE, using our data, to obtain such discounts is a good thing because the money saved can go to other aspects of health care. It does not tell me the system is broken, but rather that the prices being asked for by the pharmaceutical industry in many cases are completely over the top.