Patricia Ryan (Kildare South, Sinn Fein)
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1575. To ask the Minister for Health if he will take steps to expedite funding for gene-silencing treatment for amyloidosis such as Patisiran and equivalent drugs; and if he will make a statement on the matter. [14716/21]

Stephen Donnelly (Wicklow, Fianna Fail)
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The HSE has statutory responsibility for decisions on pricing and reimbursement of medicines under the community drugs schemes, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013.

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.

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 it received an application in December 2018 for the reimbursement of Patisiran (Onpattro) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.

In January 2019, a full health technology assessment (HTA) with respect to this indication was commissioned by the HSE. This assessment was completed in February 2020, with the NCPE recommending that Patisiran not be considered for reimbursement unless cost-effectiveness could be improved relative to existing treatments.

The HTA report with respect to Patisiran was reviewed by the HSE Drugs Group in July 2020, along with the outputs of commercial discussions with the applicant which took place in May 2020, and the patient group submission received during the HTA process.  The HSE Drugs Group requested patient and clinician input via the Rare Diseases Technology Review Committee (RDTRC) to assist the group in making its recommendation to the HSE Executive Management Team (EMT) regarding reimbursement of Patisiran.

The RDTRC convened on 24 September and reviewed the clinical data previously submitted as part of the established pricing and reimbursement process. The Committee also heard from Consultants involved in the specialist management of patients with hATTR amyloidosis.

The RDTRC continued its review of Patisiran at its subsequent meeting on 5 November which included patient input on the impact of hATTR amyloidosis.

Following these meetings, the Committee finalised a statement summarising the clinician and patient engagement on Patisiran. This was received by the HSE Drugs Group on 26 November.

The HSE has advised that it met with the applicant company in December 2020 and in early January 2021 for further discussions and deliberations on the pricing position.

At its January 2021 meeting, the HSE Drugs Group reviewed all the relevant documentation concerning the application, including information provided by the RDTRC. The HSE has advised that, having considered all of the criteria of which is obliged to take account, the HSE Drugs Group was unable to recommend in favour of reimbursement.

The decision-making authority in the HSE is the HSE EMT. The HSE EMT decides on the basis of all the demands with which it is faced (across all services) whether it can fund a new medicine, or new uses of an existing medicine, from the resources that have been provided to it in line with the 2013 Health Act.

The HSE has confirmed that it issued notice to the applicant company of the proposed decision of the EMT not to support reimbursement of Patisiran on 9 February.

Where the HSE EMT has considered a recommendation of non-reimbursement from the HSE Drugs Group and proposes to accept such a recommendation, the HSE is legally required (in line with the 2013 Health Act) to set out in detail a notice of any proposed decision to an applicant company.

The HSE is also legally required in such circumstances to provide at least a 28-day period (from the formal written notice of proposal) to enable an applicant company to consider the proposal not to reimburse and to make representations to the HSE. The HSE is legally required to consider any such representations in advance of a formal decision.

The HSE advises that the applicant company submitted representations on the 5 March 2021 in response to the EMT’s proposed decision. The HSE Drugs Group will review these representations before making a recommendation to the HSE EMT on the basis of all of the available evidence, in line with the 2013 Health Act.

As decision-making authority, the HSE EMT will, following receipt of the outcome of the HSE Drugs Group's deliberations, make a decision on whether Patisiran will be reimbursed.

I am further advised by the HSE that it received an application in August 2019 for the reimbursement of Inotersen (Tegsedi) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).

On 26 August 2019, the HSE commissioned a rapid review with respect to this indication. Following receipt of a rapid review dossier, the NCPE advised the HSE on 7 October 2019 that a full HTA was required for this medicine to assess the clinical effectiveness and cost effectiveness of Inotersen compared with the current standard of care.

The HSE commissioned a full HTA on 9 October 2019 as per agreed processes. The NCPE is currently awaiting the submission of a HTA dossier by the applicant company. A completed HTA is required to progress this application, as per the formal processes governing the pricing and reimbursement of medicines.

The applications for Patisiran and Inotersen remain under consideration in line with the 2013 Health Act.