As Minister for Health, I am determined to support our screening programmes, which are a valuable part of our health service, enabling early treatment and care for many people, and improving the overall health of our population.
Currently, all babies (between 3 and 5 days old) are offered newborn bloodspot screening (generally known as the ‘heel prick’ test), which tests for nine rare but serious conditions that are treatable if detected early in life. The Programme for Government commits to continually reviewing the number of conditions babies are screened for.
Incremental progress continues to be made on the expansion of the National Newborn Bloodspot Screening Programme (NNBSP). In 2023, two recommendations from the National Screening Advisory Committee (NSAC) on the addition of Severe Combined Immunodeficiency (SCID) and Spinal Muscular Atrophy (SMA) to the NNBSP were approved.
In Budget 2024, an additional €1.4 million of new development funding was provided to support the SMA/SCID implementation process. Once both conditions have been fully implemented, the number of conditions screened for in Ireland will increase to 11.
In terms of the current implementation status, the HSE has advised that the equipment needed to enable the roll-out testing for SMA and SCID has been purchased, delivered and successfully installed at the newborn screening laboratory and significant progress continues with regards to the laboratory verification process. The recruitment process to hire the additional staff required to support the expansion of the NNBSP has been completed and work on developing screening and treatment pathways for both conditions is in its final stages. It is expected that screening for both SCID and SMA will commence in early Q2 this year.
In relation to concerns raised around the length of the implementation process, it should be emphasised that Ireland is not taking longer than other comparable countries to assess and implement additions to the NNBSP.
The Health Council of the Netherlands first recommended screening for SMA in 2019 and implementation was completed three years later in October 2022. In the United States of America, it took six years to fully implement SMA screening after it was added to the Recommended Uniform Screening Panel (RUSP) in 2018. In the United Kingdom, screening for SCID and SMA have been under consideration by the UK National Screening Committee since 2017 and 2018 respectively.
Nevertheless, I am acutely aware of how difficult it is for parents, families and children who have received a diagnosis of a rare disease, and how challenging daily life can be for them. This is why I remain committed to the further expansion of screening in Ireland in accordance with internationally accepted criteria and best practice.
In response to the query on the Beneluxa Initiative, this a voluntary collaboration between Ireland, Belgium, the Netherlands, Luxembourg and Austria. Beneluxa plays a role in member states’ shared priority of securing faster access to high cost, innovative treatments at affordable prices, while respecting the national competencies of pricing and reimbursement. Since joining in 2018, Ireland has worked closely with members of the Initiative on horizon scanning, health technology assessment, information sharing and policy exchange, and joint price negotiations, to create opportunities for greater access to new medicines.
Onasemnogene abeparvovec (Zolgensma®) was selected by Beneluxa member states as a new medicine which would be suitable for a joint assessment and negotiation process. It was approved for reimbursement in Ireland following joint assessment and negotiations under the Beneluxa Initiative, involving Ireland, Belgium and the Netherlands, for the treatment of patients with spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or pre-symptomatic patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
As a condition of reimbursement, an individual patient approval system has been put in place by the HSE, to enable reimbursement for patients who meet the pre-defined criteria as per a HSE devised managed access protocol.
Medicines pricing and reimbursement is a national competency. 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, if a company would like a medicine to be reimbursed by the HSE, the company must submit an application to the HSE to have the 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.
In making a relevant reimbursement decision, the HSE is required under the Act to have regard to a number of criteria, including efficacy, the health needs of the public, cost effectiveness and potential or actual budget impact.
The HSE has advised that it is committed to providing access to as many medicines as possible, in as timely a fashion as possible, from the resources available (provided) to it.
The HSE must robustly assess applications for pricing and reimbursement to make sure that it can stretch available resources as far as possible and to deliver the best value in relation to each medicine and ultimately more medicines to Irish citizens and patients.
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 once a medicine is approved by the European Medicines Agency, the HSE is dependent on a pharmaceutical company making an application to Ireland for its drug or new indication to be assessed for reimbursement. All medicines are considered for pricing of reimbursement in order of application.
While many companies engage proactively with the HSE, Ireland, by virtue of its size and market share, may not always be prioritised by some companies in the first stages of marketing a new medicine. Reports describing timelines for reimbursement from EMA approval to HSE reimbursement approval do not consider this important factor, as statutorily the HSE are not able to add a new medicine to the reimbursement list until an application is received.