Access to Spinal Muscular Atrophy Medicines in Low- to Middle- Income Countries
Author(s)
Jorge Madrid Paredes, MSc1, Marijn Verhoef, MSc2, Hendrika van den Ham, PhD3.
1Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium, 2Access to Medicine Foundation, Amsterdam, Netherlands, 3Pharmacoepidemiology and Clinical Pharmacology, UU, Utrecht, Netherlands.
1Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium, 2Access to Medicine Foundation, Amsterdam, Netherlands, 3Pharmacoepidemiology and Clinical Pharmacology, UU, Utrecht, Netherlands.
OBJECTIVES: This study aimed to characterize access to spinal muscular atrophy medicines in low- and middle- income countries, and identify the barriers to access.
METHODS: A literature review with a systematic approach was performed to identify access to medicines and diagnostics in low- to middle income countries. Semi-structured interviews with patient organizations in Bangladesh, Bolivia, Indonesia, South Africa, Tunisia, and Ukraine was performed to delve into the main barriers to access.
RESULTS: Overall, there was a lack of access to spinal muscular atrophy medicines in low- and middle- income countries. Main reasons were associated with high costs, lack of reimbursement strategies, and lack of reach in the humanitarian programs of the pharmaceutical companies. Most families and patients from Bangladesh, Bolivia, Indonesia, South Africa, Tunisia, and Ukraine reported a high economic burden, a lack of understanding of the disease among clinicians, and fluent conversations with pharmaceutical companies but not with their respective governments.
CONCLUSIONS: 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.
METHODS: A literature review with a systematic approach was performed to identify access to medicines and diagnostics in low- to middle income countries. Semi-structured interviews with patient organizations in Bangladesh, Bolivia, Indonesia, South Africa, Tunisia, and Ukraine was performed to delve into the main barriers to access.
RESULTS: Overall, there was a lack of access to spinal muscular atrophy medicines in low- and middle- income countries. Main reasons were associated with high costs, lack of reimbursement strategies, and lack of reach in the humanitarian programs of the pharmaceutical companies. Most families and patients from Bangladesh, Bolivia, Indonesia, South Africa, Tunisia, and Ukraine reported a high economic burden, a lack of understanding of the disease among clinicians, and fluent conversations with pharmaceutical companies but not with their respective governments.
CONCLUSIONS: 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.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR15
Topic
Health Policy & Regulatory, Health Technology Assessment, Patient-Centered Research
Topic Subcategory
Approval & Labeling, Health Disparities & Equity, Insurance Systems & National Health Care, Reimbursement & Access Policy
Disease
Genetic, Regenerative & Curative Therapies, Neurological Disorders, Pediatrics, Rare & Orphan Diseases