Lawsuits for medicines for the treatment of spinal muscular atrophy in light of the criteria and therapeutic indications for marketing authorization and reimbursement of technologies in health systems
1
National Health Council, Ministry of health of Brazil, Brasília, Brazil
2
Department of Administration and Planning in Health, Fundação Oswaldo Cruz (Fiocruz), Rio de Janeiro, Brazil
Popul. Med. 2026;8(Supplement Supplement 1):A1460
ABSTRACT
INTRODUCTION:
Rare diseases and their treatment pose challenges for families and healthcare systems, especially when it comes to defining medical and health criteria for access, and lawsuits have been used to enforce provision.
METHODS:
This study, through document analysis, investigated lawsuits filed against the Brazilian Federal Government for medicines for the treatment of spinal muscular atrophy, and the marketing authorization and incorporation/reimbursement criteria for these technologies in Brazil and in selected countries, namely England, Colombia, United States of America and Europe.
RESULTS:
For nusinersen and risdiplam, the main marketing authorization criteria included therapeutic indication, administration by experienced professionals, and clinical and laboratory monitoring. For onasemnogene abeparvovec, age below 2 years, dosage of AAV9 antibodies and use of systemic corticosteroid. The reimbursement bodies defined clinical and genetic diagnosis, monitoring of use, absence of invasive ventilation, use of other medicines and age. For nusinersen, absence of scoliosis, contractures, previous surgeries, and other diseases. Medicines were delivered in 65.3% of the cases. 99.3% of the requests were made after marketing authorization and 54.2% after incorporation to the system. 43.8% of the claims for the supply of onasemnogene indicated previous use of nusinersen or risdiplam. An important part of the demands for nusinersen did not meet the criteria for scoliosis (40.2%) and onasemnogene (81.6%) and risdiplam (67.9%) did not meet the maximum age recommended. Most of the claims had missing information: invasive ventilation (54.9%), spinal surgery (83.3%), other conditions (95.1%), and administration by a healthcare professional (74.5%). Motor function assessment was performed in 24.5%.
CONCLUSIONS:
There was high convergence between the criteria established by health agencies and technology assessment bodies. Most claims were missing information or did not meet the criteria, which can pose health risks to the claimant and highlights the need for greater care in the analysis for granting the medicine.
Rare diseases and their treatment pose challenges for families and healthcare systems, especially when it comes to defining medical and health criteria for access, and lawsuits have been used to enforce provision.
METHODS:
This study, through document analysis, investigated lawsuits filed against the Brazilian Federal Government for medicines for the treatment of spinal muscular atrophy, and the marketing authorization and incorporation/reimbursement criteria for these technologies in Brazil and in selected countries, namely England, Colombia, United States of America and Europe.
RESULTS:
For nusinersen and risdiplam, the main marketing authorization criteria included therapeutic indication, administration by experienced professionals, and clinical and laboratory monitoring. For onasemnogene abeparvovec, age below 2 years, dosage of AAV9 antibodies and use of systemic corticosteroid. The reimbursement bodies defined clinical and genetic diagnosis, monitoring of use, absence of invasive ventilation, use of other medicines and age. For nusinersen, absence of scoliosis, contractures, previous surgeries, and other diseases. Medicines were delivered in 65.3% of the cases. 99.3% of the requests were made after marketing authorization and 54.2% after incorporation to the system. 43.8% of the claims for the supply of onasemnogene indicated previous use of nusinersen or risdiplam. An important part of the demands for nusinersen did not meet the criteria for scoliosis (40.2%) and onasemnogene (81.6%) and risdiplam (67.9%) did not meet the maximum age recommended. Most of the claims had missing information: invasive ventilation (54.9%), spinal surgery (83.3%), other conditions (95.1%), and administration by a healthcare professional (74.5%). Motor function assessment was performed in 24.5%.
CONCLUSIONS:
There was high convergence between the criteria established by health agencies and technology assessment bodies. Most claims were missing information or did not meet the criteria, which can pose health risks to the claimant and highlights the need for greater care in the analysis for granting the medicine.
| eISSN: | 2654-1459 |
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