Thursday, 26 September 2019

Questions (144)

Louise O'Reilly


144. Deputy Louise O'Reilly asked the Minister for Health if he plans to review the performance of the rare diseases technology review committee; and if he will make a statement on the matter. [39198/19]

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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 the Rare Diseases Technology Review Committee (RDTRC) in 2018. The Committee provides additional support for the HSE in its decisions around the reimbursement of 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.

I am satisfied with the work which has been undertaken by the RDTRC to date and I do not propose any review of the RDTRC’s performance at this time.

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.

In addition, the RDTRC has also been involved in processing applications for access to Nusinersen (Spinraza).