Department of Health of Ireland

10/08/2021 | Press release | Distributed by Public on 10/08/2021 05:13

Minister for Health announces HSE approval to reimburse Zolgensma (onasemnogene abeparvovec) as part of a joint BeNeLuxA collaboration

Following the first joint agreement on the price of a drug between Belgium, Ireland and the Netherlands, the HSE has decided to approve the gene therapy drug Zolgensma (onasemnogene abeparvovec) for reimbursement in the treatment of Spinal Muscular Atrophy (SMA) for patients who meet the specified criteria.

The Minister for Health, Stephen Donnelly, TD has welcomed the HSEs decision, which represents a very significant development for the State as it is the first time Ireland has agreed to fund a drug for rare diseases in partnership with other countries in the BeNeLuxA Initiative.

The BeNeLuxA Initiative is a partnership approach to pharmaceutical policy with Belgium, the Netherlands, Luxembourg and Austria that aims to secure sustainable access to high quality and affordable medicines in the participating countries. Ireland became a member of the BeNeLuxA Initiative in June 2018, alongside existing members Belgium, the Netherlands, Luxembourg and Austria.

SMA is a rare, genetic neuromuscular condition causing progressive muscle wasting and weakness leading to loss of movement. This may affect walking and upper body movement, breathing and swallowing.

The new innovative gene therapy treatment Zolgensma was granted EU marketing authorisation for treating SMA in May 2020. The joint process for Zolgensma consisted of a Health Technology Assessment, followed by a price negotiation which commenced in July 2021. Considerable engagement and negotiation with the manufacturer Novartis Gene Therapies has allowed Belgium, Ireland and the Netherlands to jointly reach an agreement on the price of a drug for the first time.

Speaking today, Minister Donnelly said:

"The reimbursement of Zolgensma by the HSE represents a great achievement in ensuring access to this gene therapy for two groups of young SMA patients. This is a welcome decision for their families, carers and friends.

"The success of the joint process for Zolgensma represents a tangible output of the BeNeLuxA partnership and I look forward to more successes like this in the future. I wish to take the opportunity to recognise and thank the HSE and the National Centre for Pharmacoeconomics for their significant role in this process."

HSE Chief Executive Officer Paul Reid said:

"The HSE, as its standard, engages in commercial negotiations with drug manufacturers to ensure we obtain the best possible value for new medicines. This enables the HSE to reimburse as many medicines as possible, for as many patients as possible, within its resources.

"HSE decisions on which medicines are reimbursed by the taxpayer must be made on objective, scientific and economic grounds. Providing funding for new high-cost innovative medicines alongside the many demands of the health service presents a continual challenge and the BeNeLuxA engagement with international colleagues aims to collectively address this challenge."

Mr. Reid added:

"This is the first time that Belgium, Ireland and the Netherlands have jointly conducted such a negotiation and reached an agreement on the price of a medicine. From today, the HSE will provide reimbursement of Zolgensma for a subgroup of patients who meet the specified criteria on which the BeNeLuxA assessment and negotiation was based."

ENDS

Notes to the Editor

What is Spinal Muscular Atrophy?

SMA is a rare genetic disease which causes life-limiting, progressive muscle disease leading to reduced mobility, curvature of the spine, loss of the arm and hand functions and paralysis of the respiratory muscles.

It is caused by the lack a protein called 'survival motor neuron' (SMN), which is essential for the normal functioning and survival of motor neurons. Without this protein, the motor neurons deteriorate and die eventually leading to muscle wasting and weakness.

There are different forms of SMA and a wide spectrum of how severely children and adults are affected. The most common form is known as 5q SMA. 5q refers to the chromosome containing the genetic cause of the disease.

What has Zolgensma (onasemnogene abeparvovec) been approved for?

The gene therapy Zolgensma (onasemnogene abeparvovec) has been approved by the HSE for reimbursement in the treatment of patients with SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type I, 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.

What is the BeNeLuxA Initiative?

The BeNeLuxA Initative is a collaboration between Belgium, The Netherlands, Luxembourg, Austria and Ireland in pharmaceutical policy. Through collaboration, the Initiative aims to ensure sustainable access to innovative medicine at affordable cost for patients.

More information can be found online at: https://beneluxa.org/