Ms Avril Daly:

The most recent national plan for rare diseases recommended the establishment of a review committee with patients on it that would look at orphan therapies that failed the process at the drugs committee and that needed more information, where we could look, for example, at real world evidence and the reality of what it would mean to a person to get access to this therapy as opposed to not, and what the costs would be if one did not administer these therapies. Often, if someone waits a long time for these therapies to be administered because he or she is looking to alleviate cost, then this is a false economy because this patient is going through this odyssey of different types of what are perhaps often inappropriate interventions or ones that are no longer suitable for them because they are now immune to those therapies. There are many different complexities around this. I draw the attention of the Deputy in the context of again being practical about this to looking at where we can potentially work with our European partners to address an issue that is common to many countries. The eighth recommendation of the Rare 2030: Foresight in Rare Disease Policy study, states that we need to improve the three "As", availability, accessibility, and affordability, of rare disease treatments by attracting investment, fostering innovation and collaboration across countries. We are not going to solve this problem on our own and we need to work together. There are initiatives coming out of Europe and looking at this right now.

Once a therapy is approved by the EMA for safety and efficacy, it comes to 70 different agencies across Europe, including our own, where we have to reassess based on health technology and value assessments . Perhaps there are ways that we can work together with our European partners, instead of having all of these individual processes, which are very different from each other, towards having a uniform process that could allow us to get better and cheaper access to these therapies. These are incentives that we need to be involved with as a small country and from which we could benefit. It is within those recommendations and it is something that the Government very much need to review, in the context of and going back to previous points about the national plan that was published in 2014. We have moved on and we need to review these with a new lens based upon where we are right now. We are in a State where we have curative therapies for children and for adults and we are missing out.