Measuring treatment related side effect burden in myasthenia gravis

Treatment options for patients with myasthenia gravis (MG) are rapidly increasing. Seven new MG therapies across 2 novel classes have been approved by the FDA to treat MG; a robust treatment pipeline spans at least 7 novel therapeutic classes. International MG Consensus Guidance define successful treatments as those that result in minimal disease manifestations or remission with no more than minimal adverse events (AE). The MG community has a number of disease specific outcome measures to evaluate treatment response and patient quality of life. Treatment efficacy only represents one important way to assess treatment value; side effects, burden of therapy, and cost all reduce treatment value. Indeed, patients and clinicians both rate treatment safety as a key consideration when choosing among treatment options. Despite this concern, we have not established the best way to measure side effect burden in MG clinical trials or clinics. To date, the MG community has been focused on the negative side effects associated with corticosteroids, despite well documented, unique side effect profiles that are associated with both traditional and new generation non-steroid MG treatments, some of which can be serious and life threatening. Careful measurement of side effect burden and comparison of this burden among MG therapies will be critical as we attempt to optimize treatment for our patients. This chapter focuses on the importance of measuring MG treatment side effect burden and three measures that may facilitate measuring this burden in future comparative effectiveness trials and in the clinic.