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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

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

Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 (CLN2) in children

Highly specialised technologies guidance Published November 2019

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

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

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

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

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

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

OTL-200 for treating metachromatic leukodystrophy [ID1666]

Proposed [GID-HST10028] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Velmanase alfa for treating alpha-mannosidosis [ID800]

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

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

Afamelanotide for treating erythropoietic protoporphyria [ID927]

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

Highly specialised technologies guidance In development

Metreleptin for treating lipodystrophy (ID861)

In development [GID-HST10011] 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

Ravulizumab for treating paroxysmal nocturnal haemoglobinuria ID1457

Proposed [GID-HST10023] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Lysosomal acid lipase deficiency - sebelipase alfa [ID737]

In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] 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

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

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]

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

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

Lenalidomide for previously treated follicular lymphoma and marginal zone lymphoma [ID1374]

In development [GID-TA10323] Expected publication date: 01 April 2020

Technology appraisal guidance In development

Guidance on the use of coronary artery stents (TA71)

Evidence-based recommendations on coronary artery stents for treating coronary artery disease (CAD)

Technology appraisal guidance Published October 2003 Last updated July 2008

Guidance on the use of imatinib for chronic myeloid leukaemia (TA70)

Evidence-based recommendations on imatinib (Glivec) for treating chronic myeloid leukaemia (CML)

Technology appraisal guidance Published October 2003 Last updated January 2016

Guidance on the use of liquid-based cytology for cervical screening (TA69)

Evidence-based recommendations on liquid-based cytology (LBC) for cervical screening

Technology appraisal guidance Published October 2003

Human growth hormone (somatropin) in adults with growth hormone deficiency (TA64)

Evidence-based recommendations on human growth hormone (somatropin; Genotropin, Humantrope, Norditropin, Saizen) in adults with growth hormone deficiency

Technology appraisal guidance Published August 2003

Alectinib for previously treated anaplastic lymphoma kinase-positive advanced non-small-cell lung cancer (terminated appraisal) (TA438)

NICE is unable to make a recommendation about the use in the NHS of alectinib for anaplastic lymphoma kinase-positive advanced non-small-cell lung cancer

Technology appraisal guidance Published March 2017

Bevacizumab for treating relapsed, platinum‑resistant epithelial ovarian, fallopian tube or primary peritoneal cancer (terminated appraisal) (TA353)

NICE is unable to make recommendations about the use of bevacizumab for treating relapsed, platinum-resistant epithelial ovarian, fallopian tube or…

Technology appraisal guidance Published August 2015

Paclitaxel as albumin-bound nanoparticles with carboplatin for untreated non-small-cell lung cancer (terminated appraisal) (TA362)

NICE is unable to make a recommendation about the use in the NHS of paclitaxel as albumin-bound nanoparticles with carboplatin for untreated..

Technology appraisal guidance Published October 2015

Cangrelor for reducing atherothrombotic events in people undergoing percutaneous coronary intervention or awaiting surgery requiring interruption of anti‑platelet therapy (terminated appraisal) (TA351)

NICE is unable to make recommendations about the use of cangrelor for reducing atherothrombotic events in people undergoing percutaneous coronary…

Technology appraisal guidance Published July 2015

Ruxolitinib for treating polycythaemia vera (terminated appraisal) (TA356)

NICE is unable to make a recommendation about the use in the NHS of ruxolitinib for treating polycythaemia vera...

Technology appraisal guidance Published September 2015

Ramucirumab for treating unresectable hepatocellular carcinoma after sorafenib (terminated appraisal) (TA609)

NICE is unable to make a recommendation about the use in the NHS of ramucirumab (Cyramza) for treating unresectable hepatocellular carcinoma after sorafenib

Technology appraisal guidance Published October 2019

Rucaparib for maintenance treatment of relapsed platinum-sensitive ovarian, fallopian tube or peritoneal cancer (TA611)

Evidence-based recommendations on rucaparib (Rubraca) for relapsed platinum-sensitive ovarian, fallopian tube or primary peritoneal cancer that has responded to

Technology appraisal guidance Published November 2019

Pentosan polysulfate sodium for treating bladder pain syndrome (TA610)

Evidence-based recommendations on pentosan polysulfate sodium (Elmiron) for bladder pain syndrome in adults

Technology appraisal guidance Published November 2019

Ibrutinib with rituximab for treating Waldenstrom's macroglobulinaemia (terminated appraisal) (TA608)

NICE is unable to make a recommendation about the use in the NHS of ibrutinib (Imbruvica) with rituximab for treating Waldenstrom’s macroglobulinaemia in adults

Technology appraisal guidance Published October 2019

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

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

Holoclar for treating limbal stem cell deficiency after eye burns (TA467)

Evidence-based recommendations on Holoclar for treating limbal stem cell deficiency in adults after eye burns

Technology appraisal guidance Published August 2017

Percutaneous vertebroplasty and percutaneous balloon kyphoplasty for treating osteoporotic vertebral compression fractures (TA279)

Evidence-based recommendations on percutaneous vertebroplasty and percutaneous balloon kyphoplasty for osteoporotic vertebral compression fractures

Technology appraisal guidance Published April 2013

Methylnaltrexone for treating opioid-induced bowel dysfunction in people with advanced illness receiving palliative care (terminated appraisal) (TA277)

NICE is unable to recommend the use in the NHS of methylnaltrexone for treating opioid-induced bowel dysfunction in people with advanced illness...

Technology appraisal guidance Published March 2013

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