The Meier Advocacy & Support Center for Spinal Muscular Atrophy (SMA) and the Shenzhen Research Institute of the Chinese University of Hong Kong jointly released a report titled “Research on the Quality of Life of Chinese SMA Patients.” The report indicates that among patients treated with Biogen’s Spinraza (nusinersen), 76.4% started the treatment only after the drug was included in the National Reimbursement Drug List (NRDL).
Report Details
The report’s follow-up research is based on data from the Meier SMA patient registration database, with a total of 1,175 valid questionnaires collected. Among them, 214 patients used Spinraza before NRDL coverage, 694 patients used it after coverage, 46 patients used Roche’s Evrysdi (risdiplam, not covered by NRDL), and 164 patients never used any medication.
Medical Expenses
In terms of medical expenses, the median personal out-of-pocket payments in the first three patient groups were RMB9,292 (USD1,367), RMB5,350 (USD787), and RMB20,097 (USD2,958), respectively. These amounts account for 92.9%, 76.4%, and 182.7% of the average monthly household income. The report points out that after NRDL inclusion, the number of patients starting Spinraza increased significantly. The disease burden is expected to continue decreasing as the first year of treatment ends.
Quality of Life
In terms of quality of life, patients who took medication, especially those on long-term treatment, showed significantly improved autonomy in daily activities and higher average quality of life scores compared with non-medication patients.
SMA and Treatment Options
Spinal muscular atrophy (SMA) is an inherited, progressive rare disease characterized by muscle atrophy and weakness. China has 30,000 to 50,000 SMA sufferers, with 1,500 to 2,000 new cases annually. Biogen’s Spinraza gained market approval in China in February 2019 at a price of RMB699,700 (USD102,990) per shot. In November 2021, the price was cut by 95% to RMB33,000 (USD4,857) per shot to gain NRDL inclusion.
Other SMA Treatments
Roche’s Evrysdi and Novartis’ Zolgensma are also available globally for SMA patients. Evrysdi entered the US and China in August 2020 and June 2021, respectively. Roche reduced its online procurement price to RMB14,500 (USD2,134) per bottle in June 2023. For patients with the maximum dose (2 years old and above, weighing over 20 kg), the annual treatment cost is less than RMB450,000 (USD66,200). Zolgensma, a gene therapy approved in the US in May 2019, offers a potential cure for SMA at a cost of USD2.125 million (RMB14 million) per shot. The drug received a clinical trial nod in China in January 2023.-Fineline Info & Tech
