The HSE has statutory responsibility for medicine pricing and reimbursement decisions, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013. The Act specifies the criteria for decisions on the reimbursement of medicines. As Minister for Health, I do not have any statutory power or function in relation to reimbursement of medicines.
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. This process ensures that treatments that are clinical and cost effective are reimbursed. In circumstances where a company has failed to demonstrate the clinical benefit to support the price that it is seeking for its treatment, the HSE reaches a decision not to reimburse the medicine.
Kuvan (sapropterin) is a medicinal product for the treatment of phenylketonuria (PKU). It is an expensive medicine and treatment, once initiated, would be expected to be lifelong.
The HTA of sapropterin was completed on 15 September 2017. The NCPE did not recommend reimbursement as the product was not deemed cost effective.
The application was considered by the HSE Drugs Group in January 2018 and it was referred to the Rare Diseases Technology Review Committee to engage with prescribers in specialist centres to being clear reimbursement guidelines to target sapropterin use in patient cohorts for which it maybe a cost and clinically effective intervention.
Upon consideration of the Rare Diseases Technology Review Committee’s recommendations, HSE leadership will issue a decision on the reimbursement of Kuvan.
It is expected that this will occur in the near future in line with the 2013 Act.