Access to Innovative Medicines Nusinersen, Onasemnogene Abeparvovec, and Risdiplam in Low- to Middle- Income Countries

Abstract

The recent marketing approval of nusinersen, risdiplam, and onasemnogene abeparvovec by the Food and Drug Administration and the European Medicines Agency has revolutionized access to care for spinal muscular atrophy patients. However, their high prices have raised concerns in several countries about patients and governments’ ability to pay for these medicines, especially in low- and middle- income countries. Since on-time access to these medicines has a significant impact on quality of life, and psychosocial health, this study aimed to identify access to medicines, epidemiology, diagnosis, onset, and regulatory and health technology assessment frameworks for spinal muscular atrophy in low- and middle- income countries. An analysis of publicly available literature and interviews with key spinal muscular atrophy stakeholders, including patient and families’ organizations and a pharmaceutical companies’ consultant, was performed. Overall, there was a lack of access to spinal muscular atrophy medicines in low- and middle- income countries. Main reasons were high cost of spinal muscular atrophy medicines, lack of efforts from companies and governments, lack of reimbursement for these medicines, and lack of reach in companies’ humanitarian programs. Most families and patients from Bangladesh, Bolivia, Indonesia, South Africa, Tunisia, and Ukraine reported a high economic burden, a lack of understanding among clinicians of the disease, and fluent conversations with pharmaceutical companies but not with their respective governments. In conclusion, there was an overall lack of access to spinal muscular atrophy in low- and middle- income countries, although regional differences were observed. Capacity building, cross-national training programs, and global health partnerships could help clinicians in low- and middle- income countries to identify early spinal muscular atrophy symptomatology. Moreover, the creation of an Essential Medicines List for rare diseases could allow the democratization of orphan medicines.