BeNeLuxA initiative supports the reimbursement of Zolgensma® in Belgium, Ireland and the Netherlands
Following the news that Zolgensma® (onasemnogene abeparvovec) has been reimbursed in Belgium, Ireland and the Netherlands, Yasmina Zegaoui, Consultant at Lightning Health, looks at how this managed entry agreement initiative can enable the reimbursement of a much-needed therapy for patients.
After successful managed entry agreements were negotiated in the EU4 and the UK, Zolgensma®, a gene therapy for the treatment of spinal muscular atrophy (SMA), has been successful in achieving pricing and reimbursement in Belgium, Ireland, and the Netherlands. These negotiations are part of the BeNeLuxA initiative, which is aimed at enhancing patient access to high quality and affordable treatment.
The joint HTA review of Zolgensma® began in April 2020, initiated by Belgium, Ireland and the Netherlands, with Austria as the external reviewer in the Belgian procedure. In July 2021, the three directly participating countries (i.e., Belgium, Ireland and the Netherlands) initiated price negotiations which led earlier this month, to an agreement with AveXis/Novartis regarding the reimbursement of Zolgensma® for two specific groups of young patients in all three countries.
As a result, the gene therapy will be available in all three countries that participated in the review (i.e., Belgium, Ireland and the Netherlands) for SMA patients with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of Type 1 and for pre-symptomatic patients with up to three copies of the SMN2 gene.
Zolgensma® will qualify for reimbursement in Ireland with immediate effect. In the Netherlands, the medicine will be reimbursed from 1 November 2021, while Belgium will reportedly follow suit on 1 December 2021.
The cost effectiveness of Zolgensma® was informed by a cost utility model constructed around three aspects of the disease: functional motor milestones, need for permanent ventilation and mortality. The model assumes that the benefit of Zolgensma® is maintained for life, whereas the benefit of Spinrasa (nusinersen), an ongoing treatment, is only for the duration of the treatment.
Tghe net budget impact of Zolgensma® for the reimbursed population is expected to be €47.9M, €26.2M, and €74.4M over the first 5 years, respectively in Belgium, Ireland and the Netherlands.
It is the first time that Belgium, Ireland and the Netherlands have jointly reached an agreement on the price of a drug. While previous BeNeLuxA pilots have already proven that joint negotiations with two countries can be effective, the process for Zolgensma® shows that negotiations by three countries can also be successful.
 BeNeLuxA statements. October 2021. https://beneluxa.org/statements [Accessed October 2021]
 Belgium, Ireland, and The Netherlands Jointly Agree under Aegis of Beneluxa Initiative to Reimburse Zolgensma®. October 2021. Online: https://www.vbb.com/insights/corporate-commercial-regulatory/belgium-ireland-and-the-netherlands-jointly-agree-under-aegis-of-beneluxa-initiative-to-reimburse-zolgensma [Accessed October 2021]
Article published 20 October 2021