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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

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

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

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

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

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

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

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

Human alpha1-proteinase inhibitor for treating emphysema ID856

In development [GID-HST10017] 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

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

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

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] 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

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

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

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

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

Denosumab for preventing skeletal-related events in multiple myeloma (terminated appraisal) (TA549)

NICE is unable to make a recommendation about the use in the NHS of denosumab for preventing skeletal-related events in multiple myeloma because no evidence

Technology appraisal guidance Published December 2018

Decitabine for untreated acute myeloid leukaemia (terminated appraisal) (TA548)

NICE is unable to make a recommendation about the use in the NHS of decitabine for untreated acute myeloid leukaemia because no evidence submission was

Technology appraisal guidance Published December 2018

Abatacept for treating psoriatic arthritis after DMARDs (terminated appraisal) (TA568)

NICE is unable to make a recommendation on abatacept (Orencia) for treating psoriatic arthritis after DMARDs in adults because of no evidence submission from

Technology appraisal guidance Published March 2019

Erythropoiesis‑stimulating agents (epoetin and darbepoetin) for treating anaemia in people with cancer having chemotherapy (TA323)

Evidence-recommendations on erythropoiesis-stimulation agents (epoetin alfa, beta, theta and zeta and darbepoetin alfa) for anaemia in people with cancer..

Technology appraisal guidance Published November 2014

Denosumab for the prevention of skeletal-related events in adults with bone metastases from solid tumours (TA265)

Evidence-based recommendations on denosumab (XGEVA) for the prevention of skeletal-related events in adults with bone metastases from solid tumours

Technology appraisal guidance Published October 2012

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

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

Lanadelumab for preventing recurrent attacks of hereditary angioedema (TA606)

Evidence-based recommendations on lanadelumab (Takhzyro) for preventing recurrent attacks of hereditary angioedema in people aged 12 and over

Technology appraisal guidance Published October 2019

Sunitinib for the first-line treatment of advanced and/or metastatic renal cell carcinoma (TA169)

Evidence-based recommendations on sunitinib (Sutent) for the first-line treatment of advanced and/or metastatic renal cell carcinoma

Technology appraisal guidance Published March 2009

Endovascular stent–grafts for the treatment of abdominal aortic aneurysms (TA167)

Evidence-based recommendations on endovascular stent–grafts for the treatment of abdominal aortic aneurysms

Technology appraisal guidance Published February 2009

Inhaled corticosteroids for the treatment of chronic asthma in adults and in children aged 12 years and over (TA138)

Evidence-based recommendations on onhaled corticosteroids for treating chronic asthma in adults and children aged 12 years and over

Technology appraisal guidance Published March 2008

Bevacizumab and cetuximab for the treatment of metastatic colorectal cancer (TA118)

Evidence-based recommendations on bevacizumab (Avastin) and cetuximab (Erbitux) for treating metastatic colorectal cancer

Technology appraisal guidance Published January 2007 Last updated January 2012

Stapled haemorrhoidopexy for the treatment of haemorrhoids (TA128)

Evidence-based recommendations on stapled haemorrhoidopexy for treating haemorrhoids

Technology appraisal guidance Published September 2007

Dual‑chamber pacemakers for symptomatic bradycardia due to sick sinus syndrome without atrioventricular block (TA324)

Evidence-based recommendations on dual-chamber pacemakers for symptomatic bradycardia due to sick sinus syndrome without atrioventricular block

Technology appraisal guidance Published November 2014

Human growth hormone (somatropin) for the treatment of growth failure in children (TA188)

Evidence-based recommendations on human growth hormone (somatropin) for treating growth failure in children

Technology appraisal guidance Published May 2010

Infliximab and adalimumab for the treatment of Crohn's disease (TA187)

Evidence-based recommendations on infliximab (Remicade) and adalimumab (Humira) for treating Crohn's disease in adults

Technology appraisal guidance Published May 2010

Bevacizumab (first-line), sorafenib (first- and second-line), sunitinib (second-line) and temsirolimus (first-line) for the treatment of advanced and/or metastatic renal cell carcinoma (TA178)

Evidence-based recommendations on evacizumab (Avastin), soradenib (Nexavar), sunitinib (Sutent) and temsirolimus (Torisel) for renal cell carcinoma

Technology appraisal guidance Published August 2009

Guidance on the use of Riluzole (Rilutek) for the treatment of Motor Neurone Disease (TA20)

Evidence-based recommendations on the use of Riluzole (Rilutek) for treating motor neurone disease (MND)

Technology appraisal guidance Published January 2001

Imatinib for the treatment of unresectable and/or metastatic gastrointestinal stromal tumours (TA209)

Evidence-based recommendations on imatinib (Glivec) for treating unresectable (inoperable) and/or metastatic gastrointestinal stromal tumours (GISTs)

Technology appraisal guidance Published November 2010

Temsirolimus for the treatment of relapsed or refractory mantle cell lymphoma (terminated appraisal) (TA207)

NICE is unable to make a recommendation about the use in the NHS of temsirolimus for treating relapsed or refractory mantle cell lymphoma because no evidence

Technology appraisal guidance Published October 2010

Peginterferon alfa and ribavirin for the treatment of chronic hepatitis C (TA200)

Evidence-based recommendations on peginterferon alfa (2a [Pegasys] or 2b [ViraferonPeg]) and ribavirin (Copegus or Rebetol) for treating chronic hepatitis C

Technology appraisal guidance Published September 2010