Thursday, 26 September 2019

Questions (143)

Louise O'Reilly


143. Deputy Louise O'Reilly asked the Minister for Health the number of sittings the rare diseases technology review committee has held since its establishment in tabular form; the dates of all future meetings scheduled; and if he will make a statement on the matter. [39197/19]

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Written answers (Question to Health)

In line with the recommendations of the National Rare Diseases Plan 2014-2018, the HSE established a Rare Diseases Technology Review Committee (RDTRC) in 2018. The Committee considers Orphan Medicinal Products that are referred to it from the HSE Drugs Group, particularly where there are issues relating to access to drugs for rare diseases.

The RDTRC is responsible for:

- reviewing proposals received from industry or expert groups in Ireland for funding of new products for rare diseases or expanded indications for existing products for rare diseases; and

- providing contributions to the development of clinical guidelines for relevant Orphan Medicinal Products (OMPs) and supporting the implementation of guidelines in conjunction with the National Drugs Management Programme Office where applicable.

The Committee’s recommendations for reimbursement of OMPs are not intended to replace any part of the existing medicines appraisal or reimbursement process but rather to complement it.

Since the establishment of the RDTRC, it has held five face-to face meetings:


Number of meetings

15 October 2018


31 October 2018


8 November 2018


26 March 2019




As part of their work programme the RDTRC have considered a number of medicines to date including:

- Sapropterin (Kuvan) for the treatment of Phenylketonuria: this drug was approved for reimbursement following the development of prescribing guidance by the RDTRC;

- Nusinersen (Spinraza) for the treatment of Spinal Muscular Atrophy: this drug was approved for reimbursement following referral to the RDTRC for the development of prescribing guidance;

- Sebelipase alfa (Kanuma) for the treatment of Lysosomal acid lipase deficiency;

- Teduglutide (Revestive) for the treatment of patients aged 1 year and above with short bowel syndrome.

Future meetings will be scheduled as referrals are made to the Rare Disease Technology Review Committee by the HSE Drugs Group.