19 May 2020: Joint HTA assessment of Zolgensma
On May 19 the European Commission granted conditional approval for Zolgensma (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.
The Beneluxa Initiative welcomes a dialogue with the company AveXis/Novartis on access and affordability of the product in each country. Belgium, Ireland and the Netherlands aim to undertake a joint HTA assessment of Zolgensma as part of a reimbursement application by the manufacturer, with Austria acting as the reviewing expert in the procedure. The company AveXis/Novartis has stated its willingness to collaborate on this joint assessment and dialogue on reimbursement. As a consequence, HTA agencies in the three countries will align on the timing and content of the local HTA procedures. Joint price negotiations within the Beneluxa Initiative always start with a joint HTA procedure. Based on the outcome of the HTA assessment,countries will determine if the joint assessment will be followed by a joint price negotiation.
25 February 2020: Beneluxa Position Statement on CAR-T
Because of considerable upfront costs, CAR-T(°) treatments are in the process of being reviewed by a number of countries to determine whether they represent value for money. Many countries have determined that the early evidence indicates a benefit, but there is uncertainty due to the single-arm nature of the trials and the lack of data in the longer term. The impact of hematopoietic stem cell transplantation in patients treated with CAR-T further adds to the uncertainty as to whether the CAR-T is responsible for the possible prolongation of survival or whether the stem cell transplant primarily contributes to this.
Clinicians have indicated that a proportion of patients in paediatric acute lymphoblastic leukaemia who receive CAR-T are likely to continue to receive stem cell transplants. In determining comparative and cost effectiveness, the influence of this choice of treatment pathway should be carefully considered.
(°) Chimeric antigen receptor-engineered T-lymphocytes
30 Jan 2020: No “lottery for life” - Statement by Beneluxa Health Ministers addressing the global managed access program designed by Novartis and Avexis
The Health Ministers of Belgium, the Netherlands, Luxembourg, Austria and Ireland have strong reservations in relation to the plans of Novartis and AveXis to let chance decide which seriously ill children will receive medication before it reaches the European market.
The approach set by the global managed access program is unprecedented and differs significantly from established early access schemes.
The Ministers – united in the Beneluxa-initiative – urge pharmaceutical companies to use objective medical criteria when they allow early access to innovative medicines.
The pharmaceutical company Novartis has recently applied for Market Authorization at the European Medicines Authority (EMA) for its product Zolgensma ®, for the treatment of Spinal Muscular Atrophy. By announcing this approach, Novartis has raised hope throughout patient communities but has remained silent on the exact plan and the legal challenges of such a system.
In their joint statement, the Ministers express strong concern at the organisation of a lottery-like approach for patients who are eagerly awaiting potential treatment: "The high level of uncertainty and the non-transparent approach is unacceptable. It proves no sincere commitment to patients and only increases the distress of the families concerned. They are given false hope. If one equals the fate of a patient to a lottery ballot, human dignity and moral values get out of sight. Lotteries are by their nature a form of gambling and this is absolutely the wrong model to bring to healthcare.”
Companies are certainly free to provide their products to patients before they are reimbursed. But the ministers involved believe that this should always involve close cooperation with physicians on the basis of impartial medical criteria and medical need.
The Beneluxa Ministers of Health call upon Novartis and AveXis to revise their system for providing early treatment for European patients pending marketing authorisation by the European Medicines Agency (EMA).
Commitment to patients
The Public Health Ministers of the Beneluxa Initiative appreciate the company's efforts to develop a drug for this vulnerable patient group. Alongside patients and prescribers, they await the final marketing authorisation for the product.
The Ministers fully understand the motivations of desperate parents fighting for the life and well-being of their children and their physicians to secure a life-saving treatment and are fully supportive of a properly implemented early access programme that could benefit individual patients.
The Ministers from the Beneluxa initiative have a commitment to patients to fight for sustainable access to medicines in their countries. They strive to increase patients’ access to high quality and affordable treatments. In a fair and accountable manner.
If the drug is approved in Europe, this treatment for spinal muscular atrophy will be the most expensive drug to date with an estimated cost of 2 million EUR per patient.
The Ministers therefore urge Novartis and AveXis to enter into a genuine dialogue with the Beneluxa initiative to explore sustainable, clinically and ethically appropriate ways to provide this medication to all young patients who need it after obtaining marketing authorization.
16 May 2019: Transparency of prices
The members of the Beneluxa Initiative highly value transparency as a key contributor to achieving sustainability of access to medicines. Transparency will assist in improving insight into the inner workings of the pharmaceutical value chain. We strongly support access to data generated by clinical research, including negative and inconclusive outcomes. We welcome a wide debate on these topics and further discussion at international level. The first concrete step should be to create price transparency among countries.