Beneluxa Cooperation for Medicines - Belgium and The Netherlands Jointly Decide not to Reimburse Cystic Fibrosis Medicine of Vertex

• 26/05/2017
• Articles

On 23 May 2017 Belgian Minister for Social Affairs and Public Health Maggie De Block and Dutch State Secretary responsible for Health, Welfare and Sport Martin van Rijn each issued statements indicating that they had jointly conducted negotiations with US-based pharmaceutical firm Vertex regarding the reimbursement of Orkambi but that these talks had failed. Orkambi is indicated for the treatment of a specific form of cystic fibrosis, a rare inherited disease that has severe effects on the lungs and the digestive system (“CF”).
 
While both officials recognised that Orkambi offers a therapeutical added value over the existing standard of care, they also reached the conclusion that Orkambi’s allegedly excessive cost does not justify the benefits on offer, namely a limited improvement of the lung function and a decrease of the exacerbations and associated hospital treatments. On this basis, the officials considered that investing in Orkambi would cause them to forego other innovative and more cost-efficient medicines. Still, they expressed the hope that Vertex would at some point come back with an improved pricing offer.
 
The press releases issued by Ms. De Block and Mr. van Rijn can be found below.

• Ms. De Block's press release: Dutch / French;
• Mr. van Rijn's press release: Dutch. 

In addition, Mr. van Rijn explained his decision in more detail in a letter addressed to the second chamber of the Dutch Parliament (see attached).
 
Yesterday’s announcement offers a stark reminder of the risks for pharmaceutical firms associated with starting reimbursement negotiations with 2 or more governments simultaneously under the Beneluxa umbrella. While Vertex recently managed to secure reimbursement for two CF medicines in countries such as Ireland, it ran into insurmountable opposition when facing the Belgian and Dutch governments. For the time being, that leaves all sides empty-handed, as Belgian and Dutch CF patients will not obtain the best possible care, while Vertex is denied access to the market.
 
The Beneluxa cooperation is still a work in progress with few official pronouncements so far on the nature, scope and practical details of the cooperation. That should change in July 2017 when a dedicated, government-run website is expected to go live.
 
Nonetheless, during a general introduction to the principles and practices governing the reimbursement of medicines in early May 2017, staff members of the National Institute for Health and Disability Insurance had this to say regarding the Beneluxa cooperation (“BC”):

1. BC has 4 components, namely (1) Horizon scanning; (2) Health Technology Assessment; (3) the exchange of information; and (4) pricing and reimbursement, including market access discussions and even in specific cases managed entry agreements. By contrast, Beneluxa does not involve the joint procurement of medicines and its primary aim is not to cause a downward pressure on the prices of medicines but rather to create access to innovative medicines at affordable prices (However, this week’s announcement regarding Orkambi would seem to suggest that price will be at the forefront of the various governments’ minds).
    
2. BC does not only apply to orphan medicines, as was initially suggested. BC rather covers innovative and expensive medicines.
    
3. BC will not change the national reimbursement rules. Whatever outcome produced by joint action under the aegis of Beneluxa will have to be fitted in the existing national rules, procedures and institutional architecture.
    
4. Horizon scanning – The countries will probe jointly for significant expected developments in both the short and long run. The Belgian Healthcare Knowledge Centre (“BHKC”) published on 20 April 2017 a proposal for Beneluxa Collaboration on the subject. It recommends the creation of a central horizon scanning unit in one of many possible forms, including an entity under the umbrella of an existing agency of one of the participating countries; an extended current horizon scanning entity; or a designated third party. There are at present discussions on how results of horizon scanning will be shared with stakeholders, including patients. It is, for example, crucial to manage patient expectations carefully. 
    
5. Health Technology Assessment -  The joint Health Technology Assessment should not be a duplication of efforts already made at the European level under the umbrella of EUnetHTA. By contrast, there will be scope for operational cooperation in the form of the mutual recognition of assessments and the joint drafting of assessment reports. This aspect of Beneluxa will also involve the sharing of expertise.
    
6. Exchange of information – The different countries will exchange information on medicine consumption; best practices in health budget management; post marketing evidence gathering through instruments such as registries; and policy issues.
    
7. Pricing and reimbursement – managed entry agreements – This is the most spectacular aspect of the Beneluxa cooperation, but it has not yet generated noteworthy results. The creation of a joint reimbursement process in a specific case will always result from a voluntary decision by both the governments and the marketing authorisation holder. The outcome of the joint discussions has to be fitted in the existing reimbursement procedures in the participating countries, as there is no international reimbursement procedure. This means, for example, that in the Netherlands the outcome has to be run past the patient organisations. Similarly, the financial details of a managed entry agreement will remain confidential, as is the case in Belgium today.