Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease characterized by progressive muscle weakness and atrophy. Uninterrupted access to diagnosis and care for SMA is vital.
As part of efforts to reduce diagnostic delays and enhance clinical trials, Cure SMA evaluated the impact of COVID-19 on SMA care and clinical trial management. The objective was to leverage understanding of challenges encountered during the pandemic to minimize the impact of similar future disruptions.
Two surveys were conducted. The provider impact survey, distributed via Alchemer between November 24, 2020 and March 8, 2021, assessed the pandemic’s effects on referral for evaluation of suspected SMA, barriers to access for care, and impact on clinical trials. The community impact survey was conducted in three segments from April 7, 2020 to July 19th, 2021 in tandem with Cure SMA COVID-19 support programs.
Of 48 complete provider survey responses, 22 came from SMA care sites and 26 from care-and-trial sites. Respondents reported a decrease in referrals for suspected SMA and a high rate of cancelled/delayed appointments. Care-and-trial sites reported a COVID-related reluctance for study participants to conduct in-person visits. One quarter reported pausing trial enrollment, and one third noted delays in study recruitment.
Community survey responses were received from 2,047 individuals with SMA, 47.6% with Type II, 29.5% with Type I, 21.5% with Type III, and 1.4% with Type IV. Due to COVID-19, 20% to 38% had a healthcare provider cancel/delay an appointment for SMA-related care.
This research provides insight on how the pandemic affected the SMA community, including some factors that interrupted care and research. This awareness might be used to mitigate future disruptions and increase preparedness for events which may affect access to care and research. While the research was conducted in the SMA community, the findings may have relevance for other patient communities.
Funding for this research was provided by members of the 2020 SMA-IC which included, Genentech/Roche, Novartis Gene Therapies, Biogen, Cytokinetics, and Scholar Rock.