On the 26th July myself and Joanne from Melanoma Support Ireland attended a HTA training day at EU house in Dublin. The main purpose of the training was to inform patient advocates on how they can become involved in the submission process for the approval of new medicines in Ireland. This couldn’t have come at a better time as far as I was concerned because I was in the middle of putting together a submission for the approval of Nivo/Ippi for the treatment of advanced metastatic melanoma.

We were welcomed by Derek Mitchell of IPPOSI who outlined their role in helping patient advocate groups to become engaged with the different stakeholders within the healthcare system they do this by facilitating the training of advocates.

Professor Michael Barry of the National Center for Pharmacoeconomics explained their role in the process of drug approval and gave an overview of the difficulties that can arise for the approval of new therapies in today’s economic climate especially with many new drugs costing tens of thousands of Euro per patient per year. There is huge pressure from growing patient numbers on health systems for the  provision of these new drugs whilst also ensuring the budget is fairly allocated for to meet the needs of all who find themselves in need of treatment. From a patient’s perspective this is a very difficult position to find oneself in, if you know there is a new drug that has been approved in another country and you can’t access it in yours especially if it is a matter of life and death for the patient, because of the cost of the drug to your HTA. It is a problem that has become a worldwide one in recent years with the development of new high tech medications, and one that seemingly there are no quick easy answers to. Pharma sets a price at what they think a HTA is willing to pay and then negotiations begin, but in the mean time patients lives hang in the balance while they wait. One solution may be more countries grouping together to increase their buying negotiation power. At present there is very little transparency about who is paying how much for what.

Ms Claire Gorry, from NCPE went through the patient submission template and explained the importance of the submission process. Once a new drug goes for approval their is a 90 day period when a interested party can make a submission. On the NCPE website there is a patient submission template that can be downloaded and the NCPE staff are available to answer any questions about the process. Once the submission is made it goes forward to the HSE drugs approval committee for consideration with the Pharma submission, it is them up to them to decide if the medication is safe, of benefit to Irish patients and if it meets their cost = benefit criteria.

Jennifer Dickson of the Scottish Medicines Consortium ( SMC ) explained the system there which is very patient centered, they actively go and seek out patient groups to make submissions and support them through the process. In Ireland there has only been four previous submissions for new medications from patient advocate groups to date a figure I feel is surprisingly low.  Also in Scotland something that is quiet different is the appeals process called PACE, if a drug gets an initial NO from the approval committee it can go through the PACE redress system, a further submission is made by the patient advocacy group and they get to put their argument for the new drug in person to the approval committee at an oral hearing this of course still does not guarantee that the drug will gain approval but it does give the patients a very fair process of engagement in the approval system of new medications. Hopefully this is a system that could be adapted in Ireland in time to come it is a very transparent system, you know who is on the approval committee and there are event two public patient representatives on the committee.

Yvonne Hughes of the Cystic Fibrosis Trust UK gave us her insight into the Scottish approval process from the patients perspective. Yvonne is a very inspiring lady, who is a CF patient herself and was able to explain in great detail what was involved in making a submission. Her energy and drive was remarkable, she was very honest about the difficulties in engaging in the process and her own lack of knowledge when she started.

Philip Wyatt from CF Ireland who have been in operation with many years also gave a talk on the patient perspective in Ireland. He was able to explain how things have changed over the years in terms of patient engagement.

One thing I was delighted to have come from the HTA training day was the chance to engage with NCPE on the submission process and because of feedback they received that day instead of putting just the five key points from the submission to the approval committee the whole submission will go to them as an appendix on the submission, this is a much better system as the patient submission is a long document and to have it condensed down to five key points seemed to me to be an unfair reflection of the amount of time, effort and information contained in the full document submitted, also the patient template form will be changed so that it can be downloaded as a word document and filled in on line instead of its current PDF format which cannot be amended.