Billy Kelleher (Cork North Central, Fianna Fail)
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134. To ask the Minister for Health if the recent application submitted for Translarna came before the technology review committee for rare diseases. [48698/18]

Simon Harris (Wicklow, Fine Gael)
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The HSE has statutory responsibility for decisions on pricing and reimbursement of medicines, in accordance with the provisions of the Health (Pricing and Supply of Medical Goods) Act 2013; therefore, the matter has been referred to the HSE for reply to the Deputy.

Billy Kelleher (Cork North Central, Fianna Fail)
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135. To ask the Minister for Health if there will be engagement with or inclusion of patient representation as part of the assessment process for Translarna. [48699/18]

Simon Harris (Wicklow, Fine Gael)
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The HSE has statutory responsibility for decisions on pricing and reimbursement of medicines, in accordance with the provisions of the Health (Pricing and Supply of Medical Goods) Act 2013; therefore, the matter has been referred to the HSE for reply to the Deputy.

Billy Kelleher (Cork North Central, Fianna Fail)
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136. To ask the Minister for Health if the assessment process for orphan drugs has changed. [48700/18]

Billy Kelleher (Cork North Central, Fianna Fail)
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137. To ask the Minister for Health his plans to revise the Health (Pricing and Supply of Medical Goods) Act 2013 to allow for a different and unique rule set to specifically apply when assessing orphan drugs in view of the fact that the current assessment process for orphan drugs is not fit for purpose. [48701/18]

Simon Harris (Wicklow, Fine Gael)
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I propose to take Questions Nos. 136 and 137 together.

The Oireachtas has put in place a robust legal framework for medicines reimbursement in the Health (Pricing and Supply of Medical Goods) Act 2013, which gives full statutory powers to the HSE to assess and make decisions on the reimbursement of medicines. The Act sets out criteria for medicine reimbursement decisions.  

The 2013 Act requires the HSE to have regard to both clinical benefits and cost effectiveness, but it does not provide for a different rule set when assessing orphan drugs. It would take an act of the Oireachtas to change the current assessment process for medicines. However, both the National Centre for Pharmacoeconomics (NCPE) and the HSE are mindful of the differences and challenges in terms of patient numbers when assessing this type of medicine.

The NCPE conducts health technology assessments (HTAs) for the HSE and makes recommendations on reimbursement to assist HSE decisions. The NCPE uses a decision framework to systematically assess whether a drug is cost-effective as a health intervention.

There has been substantial engagement between the Department, the HSE and the NCPE over the last number of months, which has resulted in a number of key changes in the HSE medicines assessment process, within the confines of the 2013 Health Act. 

In June 2018, the HSE leadership appointed a Technology Review Committee for Rare Diseases, which is responsible for:

1. 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 making recommendations on the implementation of the relevant recommendations from the National Rare Diseases Plan 2011-2018; and

2. Contributing to the development of clinical guidelines for relevant orphan medicinal products (OMPs) and supporting the implementation of guidelines 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 process, but rather to complement it.

The Technology Review Committee for Rare Diseases is intended to provide greater balance and transparency to the assessment process. The inclusion of two public or patient representatives, from a selected panel of three, recognises the value of having patient representation on the Committee. As well as examining the methodologies for assessing orphan drugs, the Committee will also consider the views of patients, caregivers and the wider issues that go into health technology assessments.

This Committee is operational and has already completed work in relation to orphan products.

The composition of the HSE Drugs Group has recently been expanded to include two representatives from the National Patients Forum and more clinical expertise in the area of rare diseases.

In light of these developments, I do not propose to bring forward proposals to change the assessment criteria for orphan drugs, which would require the approval of the Oireachtas.