Written answers

Thursday, 21 January 2021

Department of Health

Medicinal Products

Photo of Colm BurkeColm Burke (Cork North Central, Fine Gael)
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310. To ask the Minister for Health if consideration will be given to the reimbursement of the cost of the gene therapy treatment Luxturna for persons suffering with inherited retinal diseases such as retinitis pigmentosa and Leber's congenital amaurosis, which are rare, serious and life-limiting diseases; and if he will make a statement on the matter. [2984/21]

Photo of Colm BurkeColm Burke (Cork North Central, Fine Gael)
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311. To ask the Minister for Health if he will correspond with the National Centre for Pharmacoeconomics to reconsider its decision regarding the reimbursement of the cost of the gene therapy treatment Luxturna in that early administration of this medicine can lead to greater, life-enhancing benefits for those suffering with degenerative retinal disease; and if he will make a statement on the matter. [2985/21]

Photo of Colm BurkeColm Burke (Cork North Central, Fine Gael)
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312. To ask the Minister for Health if consultations have taken place to improve the cost effectiveness of the gene therapy treatment Luxturna, a life-changing gene therapy medicine that, upon early administration, leads to better, life-enhancing benefits for those suffering with inherited retinal diseases; and if he will make a statement on the matter. [2986/21]

Photo of Colm BurkeColm Burke (Cork North Central, Fine Gael)
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313. To ask the Minister for Health if consideration will be given to the reimbursement of gene therapy treatment luxturna which complements national policy objectives as set out in the programme for Government to update the rare diseases plan and the promotion of research particularly clinical trials which are key to improving the prevention, diagnosis and treatment of rare diseases; and if he will make a statement on the matter. [2987/21]

Photo of Stephen DonnellyStephen Donnelly (Wicklow, Fianna Fail)
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I propose to take Questions Nos. 310 to 313, inclusive, together.

The Oireachtas put in place a robust legal framework, 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 reimbursement of medicines, taking account of a range of objective factors and expert opinion as appropriate.

In line with the 2013 Health Act and the national framework agreed with industry, a company must submit an application to the HSE to have a new medicine added to the reimbursement list.

Reimbursement is for licensed indications which have been granted market authorisation by the European Medicines Agency or the Health Products Regulatory Authority.

In making a relevant reimbursement decision, the HSE is required under the Act to have regard to a number of criteria including efficacy, the health needs of the public, cost effectiveness and potential or actual budget impact.

HSE decisions on which medicines are reimbursed by the taxpayer are made on objective, scientific and economic grounds, on the advice of the National Centre for Pharmacoeconomics (NCPE).

The HSE has advised that on 23 September 2019 an application was received for the reimbursement of Voretigene neparvovec (Luxturna) for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

On 29 October 2019, following the completion by the NCPE of a rapid review, the HSE commissioned a full pharmacoeconomic assessment with respect to this indication.

On 3 April 2020, the NCPE received the applicant’s submission for this assessment. On 18 September 2020, the NCPE completed its assessment and recommended that Voretigene neparvovec not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatments.

The final HTA report will be reviewed by the HSE Drugs Group, along with the outputs of commercial negotiations which took place in November 2020 and the patient group submission received during the HTA process. The HSE advises that Voretigene neparvovec is expected to be included on a Drugs Group agenda in early 2021.

The HSE Executive Management Team is the decision-making body for the reimbursement of medicines under the Health Act 2013 and it will, following receipt of the outcome of the Drugs Group's deliberations, make the decision on whether Voretigene neparvovec will be reimbursed.

A National Rare Disease Plan for Ireland (2014 – 2018) was launched by the Minister for Health in July 2014. Many of the major recommendations of the plan have already been implemented.

In December 2013 the National Clinical Programme for Rare Diseases was established as a joint initiative between the HSE and the Royal College of Physicians of Ireland. The opening of the National Rare Disease Office, (NRDO) in 2015 was a significant milestone for people living with a rare disease, their families and their healthcare professionals.

On foot of the HSE Chief Clinical Officer’s (CCO) review of all the national clinical programmes in April 2019, it was recommended that the National Clinical Programme for Rare Diseases be operationalised and incorporated into the National Rare Disease Office. From 1 September 2020, the NRDO has become the main contact point and driver for national HSE rare disease projects and initiatives. The work of the NRDO will be overseen and governed by HSE Acute Operations. This means that the National Clinical Programme for Rare Diseases is being wound down and any outstanding projects transferred to the NRDO.

Building on the significant progress to date, a number of themes for inclusion in a roadmap for the coming period have been agreed with the Rare Disease Task Force, which comprises the main rare disease advocacy groups; Rare Disease Ireland, the Medical Research Charities Group (MRCP), and the Irish Platform for Patient Organisations, Science and Industry (IPPPOSI).

In addition, a number of key themes have been identified for future progress, including: Patient Awareness; European Reference Networks; Research & Registries; Access to Services; Access to Medicines; Diagnosis; Education and Training and Legislation and Policy.

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