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, 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.
The RDTRC finalised a statement summarising the clinician and patient engagement on Patisiran which was forwarded to the HSE Drugs Group the week ending 27 November. The HSE has advised that the Drugs Group is expected to consider Patisiran again to include the output of the Committee early in 2021.
The application for Patisiran remains under consideration with the HSE and is being assessed in line with the 2013 Health Act.
