a Practical Guide to Ethical Decision-Making Regarding Drug Coverage for Rare Diseases: Case Study of Nusinersen Therapy for Spinal Muscular Atrophy

Abstract

Nusinersen for the treatment of Spinal Muscular Atrophy was among the first definitive treatments for neurodegenerative conditions to come to market as of 2016. The drug’s rapid U.S. governmental approval came on the heels of the improvement in motor milestones observed in its two leading clinical trials, infusing newfound hope into a disease community that has long endured slow, often painful motor decline and early death. Genetic therapies have welcomed a tremendous amount of promise into the medical sphere – offering patients reductions in their disease burdens, and in some cases, the hope of cure. But the more individual and targeted the drugs become, the more important it is to consider their benefits carefully in a monetarily constrained atmosphere. In the case of Nusinersen, for example, the United Kingdom’s National Institute for Health and Care Excellence (NICE) has ruled as of October 2018 in favor of denying administration of the drug for patients in the United Kingdom, given the weighing of the drug’s existing clinical trial data against the need to preserve cost-effectiveness and fair allocation of resources of the National Health Services budget. With the Centers for Medicare and Medicaid Innovation already in the early stages of piloting global bundling pay structures, the possibility of similar constraints to the drug’s ongoing administration in the United States are coming to the fore, particularly for those patients that are unable to or prohibited from paying out-of-pocket or balancing paying by pharmaceutical firms. This is why, now, more than ever, the ethical frameworks surrounding coverage decisions must be critically investigated. This paper, therefore, will use Nusinersen therapy as a case study through which to examine and raise questions about the ethics utilized in decision-making contexts regarding drug coverage determinations for rare diseases. It will first provide a clinical overview of the drug’s function, relevant clinical trial data, and general ethical considerations in administration, and will then transition into a broader discussion on the ethics leveraged in drug coverage determinations using two ethical axes—those of cost-effectiveness analysis and medical futility. The hope, in doing so, will be to prepare ethicists, physicians, caretakers, and patients alike for what is bound to become part of the national policymaking agenda moving forward.