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.
For a medicine to be considered for reimbursement by the HSE, it must first have a marketing authorisation from the European Medicines Agency (EMA) or the Health Products Regulatory Authority (HPRA), before being assessed under the 2013 Act.
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 only treatments that are clinical and cost effective are reimbursed.
The HSE strives to reach a decision in as timely a manner as possible. However, because of the significant monies involved, it must ensure that the best price is achieved, as these commitments are often multi-million euro investments on an on-going basis. This can lead to protracted deliberative processes which are usually bound by strict confidentially clauses at the insistence of companies.
Nusinersen (Spinraza) is indicated for the treatment of 5q spinal muscular atrophy (SMA), a disorder characterised by progressive muscle atrophy and weakness.
In May 2017, the EMA granted market authorisation for nusinersen and in October 2017, the HSE received a reimbursement application for nusinersen.
In December 2017, the NCPE completed a HTA of nusinersen and did not recommend reimbursement at the price submitted. No decision has been arrived as yet on the application for the reimbursement of nusinersen as the statutory process is still on-going.
Two members of the Beneluxa Initiative (Belgium and the Netherlands) completed a joint negotiation for the reimbursement of nusinersen in July 2018. This process began before Ireland joined the collaboration and Ireland was not party to these negotiations.
In February I received the Report on Evaluating Orphan Drugs from the Joint Committee on Health. Currently my Department is examining the Committee's Report and its recommendations. The report raises some significant policy issues which require careful consideration by my Department, the HSE and the National Centre for Pharmacoeconomics.