An early start to treatment is vital. The decision to reimburse this expensive medicine in the Netherlands was slow and remained limited, leading to ethical dilemmas. Doctor and researcher Féline Scheijmans (UMC Utrecht) obtained her PhD on this topic on 6 May.
The impact of this delay becomes clear in the context of the disease itself. SMA is a rare hereditary muscle disease. It causes muscles to become progressively weaker, leading to severe problems with movement, breathing and swallowing. Without treatment, children with SMA type 1 die young, life expectancy for type 2 is limited, and milder forms lead to progressive muscle weakness and disability despite a normal lifespan.
Spinraza was approved for Europe in May 2017, but reimbursement under the basic health insurance package for the first children in the Netherlands did not follow until 14 months later. At that time, there was little information on efficacy and safety in a broader population of (young) SMA patients. “Our research shows that it is a safe and effective treatment,” says Féline Scheijmans. Her research was funded by the Spieren voor Spieren foundation. “In most children, Spinraza helps to slow down the disease. If we manage to treat children at a very young age, even before they have symptoms, we see the best results.”
Despite its effectiveness, the high price plays a decisive role in access. Spinraza costs €522,000 per patient in the first year and €261,000 per year thereafter. The medicine therefore fell under the Coverage Lock scheme. The Coverage Lock is a measure introduced by the Dutch Healthcare Institute to temporarily exclude new, expensive medicines from the basic health insurance package. The Dutch Healthcare Institute first assesses effectiveness, cost-effectiveness, necessity and feasibility, followed by advice and price negotiations. Only after a positive decision by the minister is the medicine made available to patients.
This policy decision had immediate consequences for patients and their families. In the case of Spinraza, the Coverage Lock process took 14 months. Féline: “With a (rapidly) progressive muscle disease, a 14-month wait is a long time. During that period, some children’s motor skills deteriorated – something that might have been prevented had the drug been approved earlier.”
The outcome of the Coverage Lock was difficult in practice. Children up to 9.5 years of age were allowed to start treatment; older children and adults had to wait until January 2020 to participate in the conditional reimbursement scheme for Spinraza. The rationale behind this age limit was difficult for patients, parents and clinicians to understand and was based on a few clinical trials. According to Féline, the procedure had a significant emotional impact on patients and their families, causing feelings of uncertainty, powerlessness and unfairness. Those involved also found the process slow. Due to the waiting time, children with SMA lost motor skills that could not be regained and their physical condition deteriorated.
In addition to the personal impact, public support was also examined. A Dutch opinion poll shows that most citizens support the reimbursement of expensive medicines; only three percent are against it. At the same time, the public recognises that choices need to be made. Four in ten people support the Dutch Healthcare Institute’s Coverage Lock system, but five in ten people are in favour of medicines being made available to patients more quickly. This highlights the tension between costs, access and careful consideration of the basic healthcare package. The evaluation of the Coverage Lock was previously also shared in the research report “The Coverage Lock for expensive medicines – an ethical evaluation”.
To put the situation in the Netherlands into context, policies in neighbouring countries were examined. The ‘package gate’ measure meant that patients in the Netherlands had to wait longer for access to the medicine compared to patients in Germany, Belgium and France. This was often difficult for patients and their families to understand. Féline: “In our neighbouring countries, there are specific policies for new medicines for rare conditions, which makes it easier for them to keep things moving.” There are also some positive developments: since July 2022, SMA has been included in the newborn screening programme, meaning that new cases of SMA are usually detected before children show any symptoms. In addition to Spinraza, a one-time gene replacement therapy (Zolgensma) and the medicine Evrysdi are now also available.
Féline Scheijmans’ research draws on the expertise of the Centre of Expertise in Muscle Diseases at UMC Utrecht, and in particular the SMA Centre Netherlands. This nationally recognised centre provides specialist care to patients with muscle diseases, trains specialists and conducts research. We are ready to provide rapid diagnosis, support and treatment – often with a diagnosis and treatment advice within a single day. The Spieren voor Spieren Children’s Centre at the Wilhelmina Children’s Hospital provides this care for children. Here, specialists work together to ensure rapid diagnosis, appropriate treatment and support for the child and family. Our goal: to help children participate as fully as possible in daily life.
