Criteria for monitoring the effectiveness of pharmacotherapy in patients with spinal muscular atrophy and progressive Duchenne muscular dystrophy receiving treatment through the Krug Dobra Foundation

Background. Long-term clinical efficacy data are typically limited when medicines are added to the procurement lists of the Krug Dobra Foundation. Moreover, the absence of standardized criteria for evaluating treatment effectiveness complicates longitudinal assessment of patients in real-world clinical practice. This issue is particularly important in spinal muscular atrophy (SMA) and progressive Duchenne muscular dystrophy (DMD), two severe disabling diseases for which the Foundation provides several disease-modifying therapies. Objective: To develop a set of criteria for monitoring the effectiveness of pharmacotherapy in patients with SMA and DMD receiving treatment through the Krug Dobra Foundation. Materials and methods. The study was conducted in two stages. The first stage comprised a systematic review of comparative studies of medicines provided through the Krug Dobra Foundation for the treatment of SMA and DMD to identify a comprehensive set of potential criteria. This was followed by a series of expert meetings involving physicians responsible for prescribing and monitoring disease-modifying therapy for these conditions. Results. The study produced sets of criteria for monitoring treatment effectiveness in patients with SMA and DMD, covering motor, respiratory, and cardiovascular function. For patients with SMA, the criteria included various motor function scales selected according to the patient's age and motor abilities, as well as parameters related to noninvasive ventilation and swallowing function. For patients with DMD, criteria were developed to assess motor abilities according to ambulatory status, together with respiratory and cardiovascular parameters. Conclusion. The proposed sets of criteria are intended to standardize the evaluation of treatment effectiveness in patients with SMA and DMD across healthcare institutions in the Russian Federation and provide a basis for informed decisions regarding the continued provision of treatment through the Krug Dobra Foundation.