Thursday, 17 December 2020
Department of Health
Health Services Provision
611. 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. [44569/20]
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 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.
The additional funding of €50m provided for new medicines in Budget 2021 will allow the HSE to provide access to new and innovative medicines which have been recommended by the HSE Drugs Group, on the basis of efficacy and value for money in line with the 2013 Health Act.
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, 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 which was forwarded to the HSE Drugs Group for consideration.
The HSE has advised that Patisiran currently sits as the 3rd item for consideration on the draft January Drugs Group Agenda. The Statement from the Rare Diseases Technology Review Committee will be considered by the Drugs Group along with the Health Technology Assessment and the outcomes from commercial negotiations.
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. As of 16 December 2020, the applicant has not submitted a HTA dossier to the NCPE for assessment. A completed HTA is required to progress this application, as per the formal processes governing the pricing and reimbursement of medicines.
The decision-making body for the reimbursement of medicines under the Health Act 2013 is the HSE EMT. The EMT will, on receipt of the outcome of the respective Drugs Group deliberations, make the decision on whether a given medicine will be reimbursed.
The applications for Patisiran and Inotersen remain under consideration in line with the 2013 Health Act.