BRUSSELS, Jan 30th, 2020.
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.
Last update: 30 January 2020