OBJECTIVES: Living with a rare disease such as SMA can be extremely arduous not only for patients but also for their families and caregivers and, from an economic perspective, for the society as a whole. In order to address the increasing prices of emerging innovative therapies for SMA, it is essential to determine the burden of the disease in Belgium.

METHODS: We are evaluating the disease cost through a detailed assessment of the direct and indirect costs of illness (COI) of SMA, using standardized questionnaires. Current funding recommendations from most Health Technology Agencies (HTA) are still mainly based on the incremental cost-effectiveness ratio (ICER). However, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) established recent good practices to implement the principles of Multi-Criteria Decision Analysis (MCDA) for assessing potential public reimbursement of orphan drugs (ODs). MCDA is indeed considered as a more holistic approach to assess the overall value of medicines under a wider spectrum of stakeholder participation (e.g., patients, clinicians, researchers), with the aim of balancing different and potentially opposing interests.

RESULTS: In the context of SMA, we reviewed how HTAs and health authorities backed up their reimbursement decisions regarding Nusinersen (Spinraza®, Biogen), approved by FDA in 2016 and by EMA in 2017, across European countries and more recently Zolgensma® (Novartis). In order to achieve this, we evaluated the review process by conducting in-depth interviews at the level of several European regulatory agencies, including the Benelux countries. Interestingly, Nusinersen was the first medication to be evaluated in the context of a crossborder assessment in the BeneluxA. Barriers and critical success factors for its implementation are presented.

CONCLUSIONS: The key lessons learned from the case of SMA may be applied as a more general model for other rare diseases.