Could nusinersen be used in a different age range of those of the pivotal trials CHERISH and ENDEAR (till 14 15 years), say a patient 31 years old with SMA (severe stage)?Hammersmith 6 FIL 64 Barthel 40 severe dependence
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- Answered 13 Feb 2020 Conflict of interest declaration: None In 2017 the European Medicines Agency published an EPAR on Nusineran  which reported "No trial has been performed including adults at study start". However, later in the document it states: "Clinical trial data are not available in patients with very severe, inborn symptoms and in patients with a mild, adult onset course. However, these patients are part of the continuum in phenotypes of one genetically defined but clinically heterogeneous disease. Based on the efficacy shown in the other, most prevalent phenotypes and the established mechanism of action of Spinraza, the therapeutic indication recommended by the CHMP includes all 5q13.2 SMA patients. The decision to treat should be based on an individualised expert evaluation of the expected benefits balanced against the potential risks for that individual. In addition, the need for continuation of therapy should be reviewed regularly on an individual basis, especially as long-term data are not available at this point." Neurology published two articles in April 2019, with these concluding: "The degree of impact on strength and function in adult SMA treated with Nusinersen remains unclear, and complications secondary to its intrathecal administration can be serious. More data is needed to better understand the role of Nusinersen in adult SMA patients."  And "Nusinersen has been well tolerated. Improvement trends are emerging at all disability levels by multiple outcome measures, demonstrating nusinersen efficacy for adults with SMA."  Finally, also published in 2019 was "Safety and Treatment Effects of Nusinersen in Longstanding Adult 5q-SMA Type 3 - A Prospective Observational Study."  which concluded (based on small numbers of patients): "This prospective observational study indicates a mild treatment effect in adults with long-standing SMA3 after 10 months of treatment with Nusinersen, which had never occurred in the natural history of the disease. In our cohort, the most significant outcome measures were the 6MWT with statistically significant changes after day 180 and day 300, RULM after day 300 and peak cough flow after day 180." References 1) https://www.ema.europa.eu/en/documents/assessment-report/spinraza-epar-public-assessment-report_en-0.pdf 2) https://n.neurology.org/content/92/15_Supplement/P4.4-015 3) https://n.neurology.org/content/92/15_Supplement/S58.007 4) https://www.ncbi.nlm.nih.gov/pubmed/31594243
- Answered 13 Feb 2020 Conflict of interest declaration: None Thanks for the reply JB. Perhaps I should have added to my consultation "in developing countries" due to the "catastrophic" cost of the drug, which puts global health system financing at risk. In Argentina, for example, it is estimated that there are around 300 400 truly eligible cases. The previous minister of health declared that, if all the patients mentioned were treated, the resulting cost would be the same as that of the entire immunization program (1) (2). Thus, a clear and evidence-based indication is crucial, as well as devising effective strategies that allow for sustainable financing. It is also said that nusinersen is, in our context (poorly regulated), up to 40% more expensive than in Europe and Canada. I appreciate again the documentary support. (1)http://www.nogracias.org/2019/06/21/spinraza-aprobado-en-argentina-con-un-precio-de-venta-catastrofico-por-lorena-dos-santos-sebastian-genero-y-ana-cristina-sanchez/ (2) López V. Spinraza: el medicamento sin precio que reclaman cientos de familias. 17/04/2019. Diario Clarín. Disponible en: https://www.clarin.com/sociedad/spinraza-medicamento-precio-reclaman-cientos-familias_0_RXx–Qs0Q.html
- Answered 13 Feb 2020 Conflict of interest declaration: None I guess there are two issues - the effectiveness of the intervention AND the cost of the intervention which then leads to the cost-effectiveness (or cost benefit) I'm based in the UK and NICE prohibits a number of drugs due to issues of cost versus benefit. I suspect the issues and the rationing are based on roughly the same tensions (cost versus benefit) irrespective of the size of the budget!