En el National Institute for Health and Care Excelence (NICE) inglés, que tiene juridiscción además en Gales, Escocia e Irlanda del Norte, también tienen un procedimiento de evaluación de medicamentos de tecnologías muy especializadas dirigidos a enfermedades muy raras: Highly specialised technologies guidance.
De momento son ocho los productos evaluados bajo este mecanismo:
| Title | Reference number | Published | Last updated |
| Burosumab for treating X-linked hypophosphataemia in children and young people | HST8 | October 2018 | October 2018 |
| Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency | HST7 | February 2018 | February 2018 |
| Asfotase alfa for treating paediatric-onset hypophosphatasia | HST6 | August 2017 | August 2017 |
| Eliglustat for treating type 1 Gaucher disease | HST5 | June 2017 | June 2017 |
| Migalastat for treating Fabry disease | HST4 | February 2017 | February 2017 |
| Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene | HST3 | July 2016 | July 2016 |
| Elosulfase alfa for treating mucopolysaccharidosis type IVa | HST2 | December 2015 | December 2015 |
| Eculizumab for treating atypical haemolytic uraemic syndrome | HST1 | January 2015 | January 2015 |
Comentarios
Publicar un comentario