Real-life stories
Meet Stefan
Stefan and the need for patient-relevant outcome measures
Stefan
When Stefan gained access to SMA medicines, his life changed. Growing up, he made a point of never allowing his disease to define him. Still, starting treatment motivated him to think about the future.
With this new mindset, Stefan could build the future of his web developer business and decided to get involved in patient advocacy to ensure that everyone living with SMA, like him, would have that opportunity.
It soon became clear to him that access to medicines, care, and medical equipment is highly inequitable throughout Europe. Even in his home country, the Netherlands, obstacles to access still prevent some people living with SMA from freely accessing the care that they want for themselves. For this reason, he joined Spierziekten Netherlands, the Dutch Neuromuscular Patient Association, in its efforts to expand access to medicines for all living with SMA.
The landscape of medicine access in the Netherlands is complex. One of the drugs is currently reimbursed for all people living with SMA on a conditional reimbursement program. When this medicine was first approved, clinical evidence of its efficacy on adults was not available. The government and manufacturer reached an agreement to make the medicine available for patients of all ages on a temporary basis, while collecting data from Dutch patients who are receiving the treatment. The temporary program will be reassessed based on this evidence. So far, the SMA clinical centre that collects the data is optimistic about the continuation of this program. Yet, for patients like Stefan, things can feel uncertain. Stefan, for instance, feels that current clinical assessments fail to capture the sizeable benefits that he experiences from treatment.
While access and reimbursement of this drug are still temporary, a second medicine was approved by the Dutch government. This medicine, however, is only reimbursed for adults up to 25 years of age. This decision was based on available evidence from clinical trials, which only included patients up to this age. No clear clinical concerns nor evidence exists that patients above 25 would respond to the treatment any differently, but the manufacturer was unable to provide data for efficacy above this age cut-off. In addition, despite the government’s efforts, negotiations with the manufacturer regarding the high pricing of the drug were unfruitful, and an agreement on price was not reached. Under these circumstances, the Dutch government is resistant to reimburse this drug for the older age groups, based on having an alternative treatment available.
Other than preventing adult patients from selecting their preferred medicine, this state of things also actively affects a sizeable group of people living with SMA who are over 25 years old but cannot receive the first medicine due to specific clinical conditions and the first medicine’s mode of administration. These patients are left with no access at all. This feels particularly cruel in a world where stabilizing treatments for SMA exist, and in a country that offers access to medicines to all other people living with SMA.
Spierziekten Netherlands is actively promoting new negotiations between the government and manufacturer. Yet, results from these conversations are hard and slow to yield. Currently, the Netherlands is in the process of forming a government after national elections, which may further delay decisions. Even if new evidence of efficacy was submitted promptly, the government’s review may take months or years. This is time that adults living with SMA who are currently excluded from treatment just do not have.
Patients are caught in the middle of political processes, market dynamics, and regulatory affairs that hinder their opportunities for access and, ultimately, their ability to live and even imagine the future. Stefan thinks keeping patients at the centre of all processes, starting from drug development, is vital to ultimately ensure access.
Stefan and Spierziekten Netherlands continue to lobby manufacturers and the government to come to an agreement, so no person living with SMA in the Netherlands is left behind.
Interview conducted on 25 March 2024. This story was published on 15 July 2024. In May 2024, an agreement that will change access to SMA medicines in the Netherlands was reached. At present, the details of this agreement are currently being worked on. People living with SMA are waiting for the decision to come into effect, with the potential to finally change the access landscape to the better.
Better outcome measures are needed to ensure that meaningful change can be measured in people living across the SMA spectrum.
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