Original Title: Patisiran e inotersen en amiloidosis familiar mediada por transtiretina

Authors' recommendations: Moderate-quality evidence shows that the use of patisiran in patients with polyneuropathy (Stage 1 and Stage 2) due to hereditary transrthyretin-mediated amyloidosis, might show a significant improvement in polyneuropathy and quality of life versus the standard of care. Although patisiran showed mild to moderate adverse effects, these were well tolerated. No significant differences were observed in terms of mortality between patisiran and the standard of care. Moderate-quality evidence shows that the use of inotersen in patients with polyneuropathy (Stage 1 and Stage 2) due to hereditary transrthyretin-mediated amyloidosis, might show a significant improvement in polyneuropathy and quality of life versus not using it. The use of inotersen, when compared with the standard of care plus placebo showed significant adverse effects, such as glomerulonephritis and thrombocytopenia. No evidence directly comparing patisiran with inotersen or with any other active drugs for the treatment of hereditary transrthyretin-mediated amyloidosis has been found. Consequently, the interpretation of the magnitude of the net benefit of each one was indirect and based on the research team judgement. Hence, these “comparative” effects should be careful assessed due to the significant differences among the studies in terms of population, length of treatment and minimal differences in the scales used to assess the polyneuropathy severity. This evidence would suggest that the use of patisiran might promote a slowdown in polyneuropathy progression and an improvement in quality of life when compared with inotersen. To date, there are no studies assessing the safety and the efficacy of these drugs in the subgroup of patients with cardiac amyloidosis. There is just information based on the subgroup analyses from the clinical trials available, which did not yield conclusive results. Patisiran and inotersen have not been approved in Argentina yet; consequently their use is compassionate or in the context of clinical trials. Both drugs have been approved by the European Medicines Agency and the United States Food and Drug Administration.There is consensus among the clinical practice guidelines consulted on recommending the use of patisiran or inotersen to treat patients with polyneuropathy (Stage 1 and Stage 2) in hereditary transrthyretin-mediated amyloidosis considering that the disease is devastating both for the patient and for the relatives and that the results of the studies, although not at long-term, were significant in preventing polyneuropathy progression. There is no economic information available regarding these drugs in Argentina. The information has been obtained mainly from international health systems such as the United Kingdom health system, which after reaching a confidential commercial agreement- whose agreed price is unknown - considered that both drugs were cost-effective for the payment threshold in their health system for very rare severe diseases, where cost-effectiveness thresholds up to ten times higher than standard are allowed. However, it is not possible to apply this to the Argentinean system.

Project Status: Completed

Year Published: 2021

URL for published report: https://www.iecs.org.ar/publicacion/?id=21216

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Argentina

Organisation Name: Institute for Clinical Effectiveness and Health Policy

Contact Address: Dr. Emilio Ravignani 2024, Buenos Aires - Argentina, C1414 CABA

Contact Name: info@iecs.org.ar

Contact Email: info@iecs.org.ar

Copyright: Institute for Clinical Effectiveness and Health Policy (IECS)