News Alert: Orchard Therapeutics Agrees to Have Libmeldy Assessed via BeNeLuXa

PRICENTRIC BRIEF:

  • Orchard Therapeutics has confirmed plans to submit Libmeldy (atidarsagene autotemcel) via the BeNeLuXa initiative, sparking ongoing consultations between the group and the company. The end of the joint health technology assessment (HTA) procedure is scheduled for June 2022, but the timeline does not take into account possible clock stops at different times during the procedure.
  • According to BeNeLuXa, in December 2021, Orchard submitted a dossier of clinical and pharmacoeconomic evidence to support reimbursement in Belgium, Ireland and the Netherlands. As such, the three agencies are assessing the therapy under the initiative. Based on the outcome of the HTA, involved parties will determine if the joint assessment could be followed by a joint price negotiation.
  • The therapy is authorized in the European Union for children with late infantile or early juvenile forms of metachromatic leukodystrophy (MLD) who have not yet developed symptoms or for those with early juvenile MLD who have initial symptoms but can still walk independently and before the onset of cognitive decline. However, the nature of the therapy means that it holds a high price, and despite the European Commission’s approval, patient barriers still remain.

 THE DETAILS

BRUSSELS, Belgium — Orchard Therapeutics has confirmed plans to submit Libmeldy (atidarsagene autotemcel) via the BeNeLuXa initiative, sparking ongoing consultations between the group and the company.

The end of the joint health technology assessment (HTA) procedure is scheduled for June 2022, but the timeline does not take into account possible clock stops at different times during the procedure.

The autologous gene therapy is approved in the European Union for the treatment of metachromatic leukodystrophy (MLD) and has been since December 2020. However, the nature of the therapy means that it holds a high price, and despite the European Commission’s approval, patient barriers still remain. Specifically, the therapy is authorized in the region for children with late infantile or early juvenile forms of the disease who have not yet developed symptoms or for those with early juvenile MLD who have initial symptoms but can still walk independently and before the onset of cognitive decline.

According to BeNeLuXa, in December 2021, Orchard submitted a dossier of clinical and pharmacoeconomic evidence to support reimbursement in Belgium, Ireland and the Netherlands. As such, the three agencies are assessing the therapy under the initiative. Based on the outcome of the HTA, involved parties will determine if the joint assessment could be followed by a joint price negotiation.

BeNeLuxA Success

The cross-border pricing collaboration has previous successful experience assessing gene therapies, as it reached an agreement on the price of AveXis/Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) in October 2021. At the time the group determined that the spinal muscular atrophy (SMA) gene therapy will be reimbursed for patients with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of Type 1 and for presymptomatic patients with up to three copies of the SMN2 gene.

The outcome marked the first time that Belgium, Ireland and the Netherlands were able to jointly reach an agreement on the price of a drug.

The BeNeLuxA Initiative was initially formed in 2015 to promote cross-border collaboration in horizon scanning, information sharing, policy exchange and HTAs by Belgium and the Netherlands. That same year, Luxembourg joined the initiative, followed by Austria in 2016 and Ireland most recently, in June 2018.

Through BeNeLuxA, participating countries are aiming to increase efficiency of the assessment and pricing and reimbursement of medicines by exchanging expertise, as well as by mutual recognition of HTAs. Importantly in Zolgensma’s case, the group engaged in joint price negotiations for specific products in order to provide faster and broader patient access.

BeNeLuxA has completed around 16 HTAs to date and until Zolgensma had been most successful with pricing and reimbursement negotiations related to another SMA treatment, Biogen’s Spinraza (nusinersen), which came about in July 2018. Following negotiations, Belgium and the Netherlands successfully reached an agreement on the reimbursement of Biogen’s SMA treatment.

As one of Europe’s most mature cross-border initiatives, BeNeLuxA and its future success with negotiating access to innovative medicines, such as the final decision on Zolgensma, could be indicative of cross-border collaboration as future standard protocol in Europe. The Libmeldy outcome will be another good indicator on the future of these processes.

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