Chapter 6 120 • England - Great Ormond Street Hospital – 21 patients aged 8.3 – 113.1 months March – October 2017 Tillmann, A et al. (2018) • Germany – 61 patients aged 1 – 93 months in seven neuromuscular centres November 2016 – June 2017 Pechmann, A et al. (2018) • Italy – 104 patients – aged 3 months – 19 years 9 months - first six months of EAP Pane, Pane M et al. (2018) • Hoy, S (2018)‘ The committee responded: ‘The committee have taken into account the consultation comments including the views of patients, carers and clinical experts alongside the updated economic model and proposed MAA. Nusinersen is now recommended for pre-symptomatic and types 1, 2, and 3 SMA in the context of a MAA. The company stated that they did not consider these data ‘because the results were consistent with the clinical data that it had presented and, in comparison, the data were immature, would be from non-UK sources and would only include SMA type 1’. The committee stated ‘that it would have liked the company to identify supportive real world evidence, given the clinical uncertainties identified.’ - but also acknowledged that the company already included several types of data. In the end, Nusinersen became available through a managed access agreement‘, including the collection of more data to address the uncertainties.‘ HST7 Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency In 2018, NICE assessed the cost-effectiveness of strimvelis, used to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). Patients with ADA-SCID have a dysfunctional gene, needed for the production of the enzyme adenosine deaminase (ADA), leading to defective lymphocytes and thereby severe immunodeficiency. Strimvelis consists of genetically modified bone marrow cells of the patient, reactivating ADA production. Strimvelis is used in patients who are ineligible for allogeneic bone-marrow transplantation. Since strimvelis is a gene therapy product, its cost-effectiveness is evaluated through a ‘highly specialised technology guidance’ (HST). GlaxoSmithKline was the submitting company, and the Centre for Reviews and Dissemination and Centre for Health Economics in York prepared the Evidence Review Group (ERG) report.
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