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Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

Evidence-based recommendations on culizumab (Soliris) for treating atypical haemolytic uraemic syndrome (aHUS)

Highly specialised technologies guidance Published January 2015

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..

Highly specialised technologies guidance Published July 2016

Elosulfase alfa for treating mucopolysaccharidosis type IVa (HST2)

Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa (MPS IVA)

Highly specialised technologies guidance Published December 2015

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

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

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

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 (confirmed biallelic RPE65 gene mutations)

Highly specialised technologies guidance Published October 2019

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

Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency (ADA–SCID)

Highly specialised technologies guidance Published February 2018

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

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

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

Metreleptin for treating lipodystrophy (ID861)

In development [GID-HST10011] Expected publication date: TBC

Highly specialised technologies guidance In development

Afamelanotide for treating erythropoietic protoporphyria [ID927]

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

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

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

Proposed [GID-HST10020] Expected publication date: TBC

Highly specialised technologies guidance Proposed

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

Proposed [GID-HST10022] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Ravulizumab for treating paroxysmal nocturnal haemoglobinuria ID1457

Proposed [GID-HST10023] 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: 08 September 2020

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

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]

In development [GID-HST10015] Expected publication date: 03 June 2020

Highly specialised technologies guidance In development

Human alpha1-proteinase inhibitor for treating emphysema ID856

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

Highly specialised technologies guidance In development

Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 [ID943]

In development [GID-HST10008] Expected publication date: 27 November 2019

Highly specialised technologies guidance In development

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

In development [GID-HST10027] Expected publication date: TBC

Highly specialised technologies guidance In development

Atezolizumab with carboplatin and etoposide for untreated extensive-stage small-cell lung cancer ID1504

In development [GID-TA10400] Expected publication date: 11 December 2019

Technology appraisal 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

Afatinib for treating advanced squamous non-small-cell lung cancer after platinum-based chemotherapy (terminated appraisal) (TA444)

NICE is unable to make a recommendation about the use in the NHS of afatinib for advanced squamous non-small-cell lung cancer after platinum-based chemotherapy

Technology appraisal guidance Published May 2017

Ocrelizumab for treating relapsing–remitting multiple sclerosis (TA533)

Evidence-based recommendations on ocrelizumab (Ocrevus) for treating relapsing–remitting multiple sclerosis (MS) in adults

Technology appraisal guidance Published July 2018

Lutetium (177Lu) oxodotreotide for treating unresectable or metastatic neuroendocrine tumours (TA539)

Evidence-based recommendations on lutetium (177Lu) oxodotreotide (Lutathera) for treating unresectable or metastatic neuroendocrine tumours in adults

Technology appraisal guidance Published August 2018

Intrabeam radiotherapy system for adjuvant treatment of early breast cancer (TA501)

Evidence-based recommendations on using Intrabeam radiotherapy during breast-conserving surgery for adults with early breast cancer

Technology appraisal guidance Published January 2018

Sofosbuvir–velpatasvir–voxilaprevir for treating chronic hepatitis C (TA507)

Evidence-based recommendations on sofosbuvir–velpatasvir–voxilaprevir (Vosevi) for treating chronic hepatitis C (HCV) in adults

Technology appraisal guidance Published February 2018

Omalizumab for previously treated chronic spontaneous urticaria (TA339)

Evidence-based recommendations on omalizumab (Xolair) for treating previously treated severe chronic spontaneous urticaria (hives)

Technology appraisal guidance Published June 2015

Rifaximin for preventing episodes of overt hepatic encephalopathy (TA337)

Evidence-based recommendations on rifaximin (Targaxan) for preventing episodes of overt hepatic encephalopathy

Technology appraisal guidance Published March 2015

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

Ezetimibe for treating primary heterozygous-familial and non-familial hypercholesterolaemia (TA385)

Evidence-based recommendations on ezetimibe (Ezetrol) for treating primary heterozygous-familial and non-familial hypercholesterolaemia.

Technology appraisal guidance Published February 2016

TNF-alpha inhibitors for ankylosing spondylitis and non-radiographic axial spondyloarthritis (TA383)

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

Technology appraisal guidance Published February 2016

Ombitasvir–paritaprevir–ritonavir with or without dasabuvir for treating chronic hepatitis C (TA365)

Evidence-based recommendations on ombitasvir–paritaprevir–ritonavir (Viekirax) with or without dasabuvir (Exviera) for some types of chronic hepatitis C

Technology appraisal guidance Published November 2015

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

Tacrolimus and pimecrolimus for atopic eczema (TA82)

Evidence-based recommendations on non-steroidal tacrolimus and pimecrolimus for treating atopic eczema (atopic dermatitis)

Technology appraisal guidance Published August 2004

Adefovir dipivoxil and peginterferon alfa-2a for the treatment of chronic hepatitis B (TA96)

Evidence-based recommendations on adefovir dipivoxil (Hepsera) and peginterferon alfa-2a (Pegasys) for the treatment of chronic hepatitis B

Technology appraisal guidance Published February 2006

Osimertinib for treating locally advanced or metastatic EGFR T790M mutation-positive non-small-cell lung cancer (TA416)

Evidence-based recommendations on osimertinib (Tagresso) for treating locally advanced or metastatic EGFR T790M mutation-positive non-small-cell lung cancer

Technology appraisal guidance Published October 2016

Guidance on the use of capecitabine and tegafur with uracil for metastatic colorectal cancer (TA61)

Evidence-based recommendations on capecitabine for treating metastatic colorectal cancer in adults

Technology appraisal guidance Published May 2003

Pre-hospital initiation of fluid replacement therapy in trauma (TA74)

Evidence-based recommendations on giving intravenous (IV) fluid replacement therapy for people with serious injuries before reaching hospital

Technology appraisal guidance Published January 2004

Fluid-filled thermal balloon and microwave endometrial ablation techniques for heavy menstrual bleeding (TA78)

Evidence-based recommendations on fluid-filled thermal balloon and microwave endometrial ablation techniques for treating heavy menstrual bleeding (HMB)

Technology appraisal guidance Published April 2004

Obeticholic acid for treating primary biliary cholangitis (TA443)

Evidence-based recommendations on obeticholic acid (Ocaliva) for treating primary biliary cholangitis (CBC / cirrhosis) in adults

Technology appraisal guidance Published April 2017

Guidance on the use of paclitaxel in the treatment of ovarian cancer (TA55)

Evidence-based recommendations on paclitaxel (Taxol) for treating ovarian cancer

Technology appraisal guidance Published January 2003 Last updated May 2005

Guidance on the use of electroconvulsive therapy (TA59)

Evidence-based recommendations on electroconvulsive therapy (ECT) for treating severe depressive illness, mania and catatonia

Technology appraisal guidance Published April 2003 Last updated October 2009

Elbasvir–grazoprevir for treating chronic hepatitis C (TA413)

Evidence-based recommendations on elbasvir–grazoprevir (Zepatier) for treating genotype 1 or 4 chronic hepatitis C (HCV) in adults

Technology appraisal guidance Published October 2016

Bevacizumab for treating EGFR mutation-positive non-small-cell lung cancer (terminated appraisal) (TA436)

NICE was unable to make a recommendation about the use in the NHS of bevacizumab for epidermal growth factor receptor mutation-positive non-small-cell lung

Technology appraisal guidance Published March 2017