Spinal Muscular Atrophy: The High Costs of Innovative Therapies for Rare Diseases

Abstract Background: Despite numbers of prescriptions filled remaining constant, expenditures for drugs are rising. Among others, this is caused by high prices for orphan drugs and advanced therapy medicinal products (ATMPs). Despite attempts by policymakers to intervene, the increasing use of these therapies poses numerous challenges on the health care system.Results: Using data from the University Hospital Heidelberg, we found that the division of pediatric neurology is experiencing a strong increase in drug costs, caused by two pharmaceuticals for the treatment of spinal muscular atrophy (SMA), Nusinersen and Onasemnogene Abeparvovec. To put this finding in a broader context, we conducted a survey of 41 German SMA treatment centers revealing a general lack of human and infrastructure resources for therapy and follow-up care, which demonstrates insufficient reimbursement of treatment. To improve structural conditions, we propose that disease-independent registries for medication surveillance that comply with the rules of the European Union are needed as well as the development of a fair funding model.Conclusion: Innovative forms of therapy require a critical discussion and concise regulation of treatment application and follow-up reimbursement as well as international industry-independent registry work. Based on the previously described model of “evidence-based dynamic pricing” by the Techniker Krankenkasse, we propose a revised model which offers an internationally scalable solution to meet these challenges.