Professor Michael Barry:

I thank the committee for inviting me here today. With regard to access to medicines and technologies for people with rare diseases, recommendation No. 30 on page 54 from the National Rare Disease Plan for Ireland 2014-2018 suggested the establishment of an assessment system similar to that for cancer therapies established under the national cancer control programme. The HSE has implemented this recommendation in setting up the rare diseases technology review committee, RDTRC. The aim of the committee is to facilitate the incorporation of the patient and clinician perspectives into an agreed statement, which will be presented to the HSE drugs group to aid the group's reimbursement recommendations. It is important to remember the rare diseases technology review committee, is not a voting or decision-making forum but it merely provides advice to the drugs group.

I was asked by the HSE to help establish and to chair the rare diseases technology review committee and I was very happy to take on that task. Membership of the committee includes the chair, the clinical lead for the HSE rare diseases programme, five consultant physicians with expertise in rare diseases, two pharmacists, one health technology assessor, one representative from the Health Information and Quality Authority and two patient representatives. It is a total of 13 members. The first meeting of the committee was held on 15 October 2018 and a second meeting was held on 8 November 2018. Meetings were also held with patient representatives on 8 November and 30 October. We also held a meeting with the manufacturer of nusinersen on 30 October 2018.