08 October 2021 - Outcome of joint negotiations for Zolgensma
Belgium, Ireland and the Netherlands have reached an agreement on the pricing of Zolgensma, a gene therapy for the treatment of Spinal Muscular Atrophy (SMA). Zolgensma will be reimbursed for two specific groups of young patients in all three countries.
The aim of the Beneluxa Initiative is to enhance the access of patients to high quality and affordable treatment. The joint reimbursement process for Zolgensma started with a Health Technology Assessment by Belgium, Ireland and the Netherlands, with Austria as external reviewer in the Belgian procedure. In July 2021 the three directly participating countries initiated price negotiations.
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. As a result, the gene therapy will be available in all three countries for SMA 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.
Frank Vandenbroucke, the Belgian Minister of Health and Social Affairs:
“Beneluxa is still called an Initiative, but is in the meanwhile a strong ‘brand’ and gold standard for voluntary collaborations between Member States. Our ability to make Zolgensma affordable for SMA patients in our three countries simultaneously shows that Beneluxa’s stated aim to ensure sustainable access to innovative medicines can be translated into tangible and shared results.”
Paul Blokhuis, the Dutch State Secretary for Health, Welfare and Sport:
“Great that the collaboration between the countries is such a success. This partnership makes medicine more accessible and this is great news for the patients who now get access to this medicine. We are committed to continuing this successful collaboration in the future.”
Stephen Donnelly, the Irish Minister for Health:
“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 wish to take the opportunity to recognise and thank the HSE, including the National Centre for Pharmacoeconomics, and the other participating countries for their significant role in this process.”
Paul Reid, the Irish HSE Chief Executive Officer:
“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. 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.”
Beneluxa achievements
In 2015 the Beneluxa cooperation was initiated with the aim of sustainable access to and appropriate use of medicines. Together, Belgium, the Netherlands, Luxembourg, Austria and Ireland want to enhance patients’ access to high quality and affordable pharmaceutical treatments. Besides undertaking joint price negotiations, the partnership works together on horizon scanning, health technology assessments and sharing of knowledge and expertise on policies and challenges yet to come.
Recently, the International Horizon Scanning Initiative (IHSI) has been launched, having originated within the Beneluxa collaboration. Its goal is to build a permanent horizon scanning system that can support countries and institutions in policy planning and their decision making regarding the reimbursement of new pharmaceuticals. A wide group of countries is currently invested in the IHSI.
01 October 2021 - Assessment of COVID-19 monoclonal antibodies is needed
The Beneluxa Initiative considers value assessments of medicines prior to reimbursement in their respective countries, to be essential in quantifying the potential added benefit of treatments relative to the price requested. This concept was reaffirmed in a recent joint statement with the Nordic Pharmaceutical Forum.1
The urgency of the pandemic had required some changes to normal medicine regulatory processes to tackle the global pressure on health systems; for the EMA this has resulted in the introduction of rolling reviews. Rolling reviews involve a real time examination of evidence to establish if sufficient information is available to support a marketing authorization. This allowed countries to prepare early on for joint procurement of e.g. vaccines at an exceptional time.
More recently the EMA has begun rolling reviews on monoclonal antibodies to treat some patients with COVID-19 (September 2021)2. However the Beneluxa considers it appropriate at this stage of the pandemic that these therapies3 are assessed for value within established national processes, before decisions on reimbursement or procurement are made. Informed decision making in this matter includes exchange of available HTA information within Beneluxa.
1 https://amgros.dk/4889/
2 https://www.ema.europa.eu/en/human-regulatory/overview/public-health-threats/coronavirus-disease-covid-19/treatments-vaccines/treatments-covid-19/covid-19-treatments-under-evaluation
3 https://www.bmj.com/content/374/bmj.n2231
29 June 2021 - Short Statement on Alzheimer’s HTA
The Beneluxa group is following with attention the therapies relative to Alzheimer’s disease coming to the market. Recently the FDA in the US approved an anti-amyloid antibody amid some controversy; a decision from EMA is not yet available.
Beneluxa wishes to emphasize that clinical endpoints such as changes in cognitive scores or dementia scales are considered as the endpoints relevant for HTA agencies in Alzheimer’s clinical trials. Biomarker data are not considered sufficient for demonstration of benefit for HTA agencies.
04 June 2021 - Joint statement of the Beneluxa Initiative and the Nordic Pharmaceutical Forum
In recent years, efforts to enhance international payer collaboration and the willingness to exchange expertise and knowledge have increased substantially. The need for international exchange intensifies due to the challenges that authorities, including payers face concerning pricing, reimbursement and procurement of medicines and therapies. In order to ensure access to new medicines for patients these issues are essential to solve in an effective and rational way. The efforts have materialised in the form of international platforms such as the Nordic Pharmaceutical Forum (NLF); constituting of Norway, Sweden, Finland, Denmark and Iceland, and the Beneluxa Initiative; constituting of Belgium, the Netherlands, Austria, Ireland and Luxembourg. Concrete outcomes of these platforms include the organisation of the international horizon scanning initiative (IHSI) as well as an increased interaction between these platforms. A number of strategic meetings have taken place where other international payers also joined these discussions. Recently, the European Commission has launched a new Pharmaceutical Strategy for Europe highlighting similar issues with regards to ensuring access to affordable medicines. The Strategy specifically refers to the strengthening of existing and potential new cross-country collaborations.
Our joint efforts have focused on a number of issues related to the pricing and reimbursement of medicines. The Beneluxa Initiative and the Nordic Pharmaceutical Forum will initially aim to examine the following matters together:
The importance of cost effectiveness in the assessment of value of medicines. The appropriate use of cost effectiveness offers a framework, which allows decision makers to maximise health gain within a limited budget. It also allows decision makers to estimate the price which is likely to achieve value. Therefore, the certainty of cost-effectiveness should be considered in any negotiations. i.e. the (un)certainty about the effectiveness of the medicine balanced with the high monetary risk.
Availability of robust clinical evidence for decision makers in a timely manner. The Beneluxa Initiative and Nordic Pharmaceutical Forum agree that timely access to effective medicines for patients is important. However, a number of recent developments within the process of market authorisation have been designed to facilitate earlier access to medicines. With this, the challenge arises that there may be a lack of data demonstrating clear benefit at earlier time-points. This makes value assessments increasingly difficult. Therefore, the availability of clinical evidence (including studies where the endpoints are not met) is of vital importance to allow decision makers to adequately assess benefit and value. We aim to mainly examine the following: 1) the timing of the (cost) effectiveness assessment, and 2) the value-framework for assessing medicines in general, with a focus on those medicines for which there is limited or no data to support a decision based on the (cost) effectiveness.
The need to collaborate on the challenges facing decision makers in relation to achieving value from medicines is abundantly clear. We will address some of these issues together through continuous dialogue and an intensification of the collaboration between countries aiming to achieve more alignment in our value assessments.
See also: https://amgros.dk/4889/
28 May 2021 - Beneluxa Statement on high cost SMA treatments
Pharmacological treatments of Spinal Muscular Atrophy (SMA) have expanded across the full spectrum of the disease. Onasemnogene abeparvovec targets disease in patients with SMA who are presymptomatic or who are type 1 symptomatic, and less than 6 months of age. Nusinersen has a broad licence encompassing type 1 to type 3 SMA and includes adulthood. Risdiplam, which received CHMP positive opinion in February 2021, is the first oral treatment aimed at treating patients from 2 months of age into adulthood (Type 1 to 3).
It is possible that patients would receive at least 2 different of these treatments over their lifetime. Further, there are additional treatments in the pipeline. Currently the evidence base at point of regulatory approval mainly considers these treatments as stand alone. However, sequential use of at least two of these therapies, is not outside the scope of the licences, and has been observed in the clinical setting. Moreover, evidence of the long-term efficacy and safety of these treatments is lacking.
Given the considerable uncertainty associated with the long term effect of these treatments, health technology assessors and payers should incorporate the potential sequential costs of these high cost treatments. The (financial) risk associated with the lack of long term data and potential use of sequential treatments should not be carried by payers alone.
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.