Metreleptin for treating lipodystrophy (HST14)
Evidence-based recommendations on metreleptin (Myalepta) for treating lipodystrophy in children and adults
Search results
1410 results
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Migalastat for treating Fabry disease (HST4)
Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16
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Eliglustat for treating type 1 Gaucher disease (HST5)
Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults
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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
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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
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Maralixibat for treating type 2 progressive familial intrahepatic cholestasis ID3818
Proposed [GID-HST10040] Expected publication date: TBC
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Palovarotene for treating flare ups of heterotopic ossification associated with fibrodysplasia ossificans progressiva [ID2721]
In development [GID-HST10033] Expected publication date: TBC
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Palovarotene for preventing heterotopic ossification associated with fibrodysplasia ossificans progressiva [ID3739]
In development [GID-HST10032] Expected publication date: TBC
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VTS-270 for treating Niemann-Pick type C1 (ID1267)
Proposed [GID-HST10020] Expected publication date: TBC
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Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223
Proposed [GID-HST10019] Expected publication date: TBC
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OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]
Proposed [GID-HST10022] Expected publication date: TBC
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Human alpha1-proteinase inhibitor for treating emphysema ID856
In development [GID-HST10017] Expected publication date: TBC
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Givosiran for treating acute hepatic porphyria [ID1549]
In development [GID-HST10035] Expected publication date: TBC
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Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (review of HST3) ID1642
In development [GID-HST10044] Expected publication date: TBC
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Avalglucosidase alfa for treating Pompe disease [ID3737]
Proposed [GID-HST10030] Expected publication date: TBC
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Afamelanotide for treating erythropoietic protoporphyria [ID927]
In development [GID-HST10009] Expected publication date: TBC
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Velmanase alfa for treating alpha-mannosidosis [ID800]
In development [GID-HST10010] Expected publication date: TBC
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Lonafarnib for treating progeria and progeroid laminopathies [ID3867]
Proposed [GID-HST10039] Expected publication date: TBC
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Arimoclomol for treating Niemann-Pick disease Type C [ID1312]
Proposed [GID-HST10037] Expected publication date: TBC
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Asfotase alfa for treating paediatric-onset hypophosphatasia (review of HST6) [ID3927]
Proposed [GID-HST10046] Expected publication date: TBC
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OTL-200 for treating metachromatic leukodystrophy [ID1666]: evaluation consultation
We are listening to your views on this highly specialised technology. Comments close 30 July 2021.
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Odevixibat for progressive familial intrahepatic cholestasis [ID1570]
In development [GID-HST10043] Expected publication date: 26 January 2022
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Lysosomal acid lipase deficiency - sebelipase alfa [ID737]
In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] Expected publication date: TBC
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Lenti-D for treating cerebral adrenoleukodystrophy [ID1284]
In development [GID-HST10038] Expected publication date: TBC
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Elosulfase alfa for treating mucopolysaccharidosis type IVa (review of HST2) [ID1643]
In development [GID-HST10027] Expected publication date: TBC
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Setmelanotide for treating obesity caused by LEPR or POMC deficiency [ID3764]
In development [GID-HST10034] Expected publication date: 23 March 2022
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Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 years and over [ID1590]
In development [GID-HST10045] Expected publication date: 05 May 2022
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OTL-200 for treating metachromatic leukodystrophy [ID1666]
In development [GID-HST10028] Expected publication date: TBC
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Advocacy services for adults with health and social care needs
In development [GID-NG10156] Expected publication date: 30 March 2022
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Adults with complex needs: social work interventions including assessment, care management and support
In development [GID-NG10145] Expected publication date: 27 April 2022
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Disabled children and young people up to 25 with severe complex needs: integrated service delivery and organisation across health, social care and education
In development [GID-NG10113] Expected publication date: 19 January 2022
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Drisapersen for the first-line treatment of Duchenne's muscular dystrophy [ID911]
In development [GID-HST10004] Expected publication date: TBC
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Eteplirsen for treating Duchenne muscular dystrophy [ID1003]
In development [GID-HST10007] Expected publication date: TBC
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Trifluridine–tipiracil for treating metastatic gastric cancer or gastro-oesophageal junction adenocarcinoma after 2 or more therapies (TA669)
Evidence-based recommendations on trifluridine–tipiracil (Lonsurf) for treating metastatic gastric cancer or gastro-oesophageal junction adenocarcinoma in adult
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Brigatinib for ALK-positive advanced non-small-cell lung cancer that has not been previously treated with an ALK inhibitor (TA670)
Evidence-based recommendations on brigatinib (Alunbrig) for anaplastic lymphoma kinase (ALK)-positive advanced non-small-cell lung cancer that has not been
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Mepolizumab for treating severe eosinophilic asthma (TA671)
Evidence-based recommendations on mepolizumab (Nucala) for treating severe eosinophilic asthma in adults
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Brolucizumab for treating wet age-related macular degeneration (TA672)
Evidence-based recommendations on brolucizumab (Beovu) for treating wet age-related macular degeneration in adults
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Niraparib for maintenance treatment of advanced ovarian, fallopian tube and peritoneal cancer after response to first-line platinum-based chemotherapy (TA673)
Evidence-based recommendations on niraparib for maintenance treatment of advanced (FIGO stages 3 and 4) high-grade epithelial ovarian, fallopian tube or primary
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Pembrolizumab for untreated PD-L1-positive, locally advanced or metastatic urothelial cancer when cisplatin is unsuitable (terminated appraisal) (TA674)
NICE is unable to make a recommendation on pembrolizumab (Keytruda) for untreated PD-L1-positive, locally advanced or metastatic urothelial cancer when
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Vernakalant for the rapid conversion of recent onset atrial fibrillation to sinus rhythm (terminated appraisal) (TA675)
NICE is unable to make a recommendation on vernakalant (Brinavess) for the rapid conversion of recent onset atrial fibrillation to sinus rhythm in adults. This
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Filgotinib for treating moderate to severe rheumatoid arthritis (TA676)
Evidence-based recommendations on filgotinib (Jyseleca) for moderate to severe rheumatoid arthritis in adults
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Autologous anti-CD19-transduced CD3+ cells for treating relapsed or refractory mantle cell lymphoma (TA677)
Evidence-based recommendations on autologous anti-CD19-transduced CD3+ cells for treating relapsed or refractory mantle cell lymphoma in adults who have
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Omalizumab for treating chronic rhinosinusitis with nasal polyps (terminated appraisal) (TA678)
NICE is unable to make a recommendation on omalizumab (Xolair) for treating chronic rhinosinusitis with nasal polyps in adults because Novartis Pharmaceuticals
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Dapagliflozin for treating chronic heart failure with reduced ejection fraction (TA679)
Evidence-based recommendations on dapagliflozin (Forxiga) for symptomatic chronic heart failure with reduced ejection fraction in adults
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Olaparib for maintenance treatment of relapsed platinum-sensitive ovarian, fallopian tube or peritoneal cancer (TA620)
Evidence-based recommendations on olaparib (Lynparza) for maintenance treatment of relapsed platinum-sensitive ovarian, fallopian tube or primary peritoneal
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Sotagliflozin with insulin for treating type 1 diabetes (TA622)
Evidence-based recommendations on sotagliflozin with insulin for treating type 1 diabetes in adults with a body mass index (BMI) of at least 27 kg/m2
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Patiromer for treating hyperkalaemia (TA623)
Evidence based recommendations on patiromer (Veltassa) for treating hyperkalaemia in adults
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Peginterferon beta-1a for treating relapsing–remitting multiple sclerosis (TA624)
Evidence-based recommendations on peginterferon beta-1a (Plegridy) for treating relapsing–remitting multiple sclerosis in adults
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Recombinant human parathyroid hormone for treating hypoparathyroidism (terminated appraisal) (TA625)
NICE is unable to make a recommendation on recombinant human parathyroid hormone for treating hypoparathyroidism because Shire Pharmaceuticals
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Avatrombopag for treating thrombocytopenia in people with chronic liver disease needing a planned invasive procedure (TA626)
Evidence-based recommendations on avatrombopag (Doptelet) for treating severe thrombocytopenia in adults with chronic liver disease needing a planned invasive