Abstract
BackgroundNusinersen is one of an increasing number of new, expensive orphan drugs to receive authorization. These drugs strain public healthcare budgets and challenge principles for resource allocation. Nusinersen was introduced in the Norwegian public healthcare system in 2018. A national expert group consisting of physicians was formed to oversee the introduction and continuation of treatment in light of specific start and stop criteria.MethodsWe have studied experiences within the expert group with a special emphasis on their application of the start and stop criteria, rationing of treatment, and experienced moral dilemmas. A research interview with six members of the national expert group was performed, then analysed with manifest content analysis. The analysis was supplemented with publically available sources on priority setting and the process leading up to the introduction of nusinersen and the establishment of the expert group.ResultsSixty-six patients have received treatment within the first 25 months since the national expert group’s establishment. Treatment has not been discontinued for any patient. No patients under 18 years of age have been denied treatment, as those who were referred at this age were all deemed to fulfill the start criteria. The expert group has, however, increased geographical treatment equity and facilitated important cooperation at the national level. Furthermore, it has enhanced open and critical discussions of both medical issues and new ethical dilemmas.ConclusionAlthough facilitating equal access to treatment for SMA patients, the national expert group has not discontinued treatment for any patient. It is suggested that in order for clinicians to be able to ration care for individual patients, they require both adequate support and sufficient formal authority. Start and stop criteria need to be re-evaluated as more knowledge and experience are gained regarding the treatment.
Highlights
Nusinersen is one of an increasing number of new, expensive orphan drugs to receive authorization
Much less attention has been given to rationing at the meso and, especially, the micro level; whether rationing at the micro/clinical level is possible or Magelssen et al BMC Med Ethics (2021) 22:54 desirable, and how clinicians should be aided in this task [2,3,4]
Twenty-five months after the introduction of nusinersen in Norway’s public health service, 66 patients in total have been treated, most of them having had the diagnosis for several years
Summary
Nusinersen is one of an increasing number of new, expensive orphan drugs to receive authorization. These drugs strain public healthcare budgets and challenge principles for resource allocation. It has been claimed that this is partly so because rationing involves modes of deliberation alien and perhaps harmful to the ethics of the physician–patient relationship [3]. In due course, it will become clear how the present case concerns clinical/bedside rationing
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