Updating results

1143 results

Sort: Relevance | Date

Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations (HST11)

Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherit

Highly specialised technologies guidance Published October 2019

Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST12)

Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children

Highly specialised technologies guidance Published November 2019

Elosulfase alfa for treating mucopolysaccharidosis type IVa (HST2)

Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa in adults and children

Highly specialised technologies guidance Published December 2015

Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children

Highly specialised technologies guidance Published January 2015

Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6)

Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children

Highly specialised technologies guidance Published August 2017

Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency (HST7)

Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency

Highly specialised technologies guidance Published February 2018

Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)

Evidence-based recommendations on burosumab (Crysvita) for treating X-linked hypophosphataemia (XLH) in children and young people

Highly specialised technologies guidance Published October 2018

Inotersen for treating hereditary transthyretin amyloidosis (HST9)

Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis

Highly specialised technologies guidance Published May 2019

Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (HST3)

Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people aged

Highly specialised technologies guidance Published July 2016

Migalastat for treating Fabry disease (HST4)

Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16

Highly specialised technologies guidance Published February 2017

Eliglustat for treating type 1 Gaucher disease (HST5)

Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults

Highly specialised technologies guidance Published June 2017

Patisiran for treating hereditary transthyretin amyloidosis (HST10)

Evidence-based recommendations on patisiran (Onpattro) for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy

Highly specialised technologies guidance Published August 2019

Ravulizumab for treating paroxysmal nocturnal haemoglobinuria ID1457

Proposed [GID-HST10023] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Afamelanotide for treating erythropoietic protoporphyria [ID927]

In development [GID-HST10009] Expected publication date: TBC

Highly specialised technologies guidance In development

Lysosomal acid lipase deficiency - sebelipase alfa [ID737]

In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] Expected publication date: TBC

Highly specialised technologies guidance In development

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] Expected publication date: TBC

Highly specialised technologies guidance In development

OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]

Proposed [GID-HST10022] Expected publication date: TBC

Highly specialised technologies guidance Proposed

OTL-200 for treating metachromatic leukodystrophy [ID1666]

Proposed [GID-HST10028] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Human alpha1-proteinase inhibitor for treating emphysema ID856

In development [GID-HST10017] Expected publication date: TBC

Highly specialised technologies guidance In development

Elosulfase alfa for treating mucopolysaccharidosis type IVa (review of HST2) [ID1643]

In development [GID-HST10027] Expected publication date: 11 November 2020

Highly specialised technologies guidance In development

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Onasemnogene abeparvovec for treating spinal muscular atrophy type 1 [ID1473]

In development [GID-HST10026] Expected publication date: TBC

Highly specialised technologies guidance In development

VTS-270 for treating Niemann-Pick type C1 (ID1267)

Proposed [GID-HST10020] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Metreleptin for treating lipodystrophy (ID861)

In development [GID-HST10011] Expected publication date: 13 January 2021

Highly specialised technologies guidance In development

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]

In development [GID-HST10015] Expected publication date: 27 May 2020

Highly specialised technologies guidance In development

Drisapersen for the first-line treatment of Duchenne's muscular dystrophy [ID911]

In development [GID-HST10004] Expected publication date: TBC

Highly specialised technologies guidance In development

Eteplirsen for treating Duchenne muscular dystrophy [ID1003]

In development [GID-HST10007] Expected publication date: TBC

Highly specialised technologies guidance In development

Crisaborole for treating mild to moderate atopic dermatitis in people aged 2 years and older (ID1195)

In development [GID-TA10435] Expected publication date: 03 June 2020

Technology appraisal guidance In development

Panobinostat for treating multiple myeloma after at least 2 previous treatments (TA380)

Evidence-based recommendations on panobinostat (Farydak) for treating multiple myeloma after at least 2 previous treatments.

Technology appraisal guidance Published January 2016

Inhaler devices for routine treatment of chronic asthma in older children (aged 5–15 years) (TA38)

Evidence-based recommendations on inhaler devices for routine treatment of chronic asthma in older children (aged 5–15 years)

Technology appraisal guidance Published March 2002

Nintedanib for treating idiopathic pulmonary fibrosis (TA379)

Evidence-based recommendations on nintedanib (Ofev) for people with idiopathic pulmonary fibrosis.

Technology appraisal guidance Published January 2016

Ramucirumab for treating advanced gastric cancer or gastro–oesophageal junction adenocarcinoma previously treated with chemotherapy (TA378)

Evidence-based recommendations on ramucirumab (Cyramza)

Technology appraisal guidance Published January 2016

Enzalutamide for treating metastatic hormone-relapsed prostate cancer before chemotherapy is indicated (TA377)

Evidence-based recommendations on enzalutamide (Xtandi) for treating metastatic, hormone-relapsed prostate cancer before chemotherapy is indicated

Technology appraisal guidance Published January 2016

Adalimumab, etanercept, infliximab, certolizumab pegol, golimumab, tocilizumab and abatacept for rheumatoid arthritis not previously treated with DMARDs or after conventional DMARDs only have failed (TA375)

Evidence-based recommendations on adalimumab (Humira), etanercept (Enbrel), infliximab (Remicade, Remsima, Inflectra), certolizumab pegol (Cimzia), golimumab…

Technology appraisal guidance Published January 2016

Abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis (TA373)

Evidence-based recommendations on abatacept, adalimumab, etanercept and tocilizumab for treating polyarticular juvenile idiopathic arthritis (JIA)

Technology appraisal guidance Published December 2015

Bortezomib for previously untreated mantle cell lymphoma (TA370)

Evidence-based recommendations on bortezomib (Velcade) for treating previously untreated mantle cell lymphoma in adults

Technology appraisal guidance Published December 2015

Ciclosporin for treating dry eye disease that has not improved despite treatment with artificial tears (TA369)

Evidence-based recommendations on ciclosporin (Ikervis) for treating dry eye disease that has not improved despite treatment with artificial tears

Technology appraisal guidance Published December 2015

Vortioxetine for treating major depressive episodes (TA367)

Evidence-based recommendations on vortioxetine (Brintellix) for treating major depressive episodes in adults

Technology appraisal guidance Published November 2015

Pembrolizumab for advanced melanoma not previously treated with ipilimumab (TA366)

Evidence-based recommendations on pembrolizumab (Keytruda) for treating advanced melanoma in adults not previously treated with ipilimumab

Technology appraisal guidance Published November 2015 Last updated September 2017

Naltrexone–bupropion for managing overweight and obesity (TA494)

Evidence-based recommendations on naltrexone–bupropion (Mysimba) for managing overweight and obesity in adults

Technology appraisal guidance Published December 2017

Atezolizumab for untreated PD-L1-positive locally advanced or metastatic urothelial cancer when cisplatin is unsuitable (TA492)

Evidence-based recommendations on atezolizumab (Tecentriq) for untreated PD-L1-positive locally advanced or metastatic urothelial carcinoma in adults

Technology appraisal guidance Published December 2017 Last updated July 2018

Ibrutinib for treating Waldenstrom's macroglobulinaemia (TA491)

Evidence-based recommendations on ibrutinib (Imbruvica) for treating Waldenstrom's macroglobulinaemia (non-Hodgkin lymphoma) in adults after at least 1 therapy

Technology appraisal guidance Published November 2017

Nivolumab for treating squamous cell carcinoma of the head and neck after platinum-based chemotherapy (TA490)

Evidence-based recommendations on nivolumab (Opdivo) for squamous cell carcinoma (SCC) of the head and neck in adults who have had platinum-based chemotherapy

Technology appraisal guidance Published November 2017

Guidance on the use of ultrasound locating devices for placing central venous catheters (TA49)

Evidence-based recommendations on ultrasound locating devices for placing central venous catheters into the internal jugular vein

Technology appraisal guidance Published October 2002

Vismodegib for treating basal cell carcinoma (TA489)

Evidence-based recommendations on vismodegib (Erivedge) for treating basal cell carcinoma (BCC; non-melanoma skin cancer; rodent ulcers) in adults

Technology appraisal guidance Published November 2017

Regorafenib for previously treated unresectable or metastatic gastrointestinal stromal tumours (TA488)

Evidence-based recommendations on on regorafenib (Stivarga) for previously treated gastrointestinal stromal tumours in adults

Technology appraisal guidance Published November 2017

Venetoclax for treating chronic lymphocytic leukaemia (TA487)

Evidence-based recommendations on venetoclax (Venclyxto) for treating chronic lymphocytic leukaemia in adults

Technology appraisal guidance Published November 2017

Aflibercept for treating choroidal neovascularisation (TA486)

Evidence-based recommendations on aflibercept (Eylea) for treating myopic choroidal neovascularisation in adults

Technology appraisal guidance Published November 2017

Sarilumab for moderate to severe rheumatoid arthritis (TA485)

Evidence-based recommendations on sarilumab (Kevzara) for treating moderate to severe rheumatoid arthritis in adults

Technology appraisal guidance Published November 2017

Nivolumab for previously treated non-squamous non-small-cell lung cancer (TA484)

Evidence-based recommendations on nivolumab (Opdivo) for locally advanced or metastatic (secondary) non-squamous non-small-cell lung cancer in adults after

Technology appraisal guidance Published November 2017