Updating results

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

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

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

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

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

Managing medicines for adults receiving social care in the community (NG67)

This guideline covers medicines support for adults (aged 18 and over) who are receiving social care in the community. It aims to ensure that people who receive social care are supported to take and look after their medicines effectively and safely at home. It gives advice on assessing if people need help with managing their medicines, who should provide medicines support and how health and social care staff should work together.

NICE guideline Published March 2017

Patient group directions (MPG2)

This guideline covers good practice for developing, authorising, using and updating patient group directions. It also offers advice on deciding whether a patient group direction is needed.

Medicines practice guideline Published August 2013 Last updated March 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

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

Controlled drugs: safe use and management (NG46)

This guideline covers systems and processes for using and managing controlled drugs safely in all NHS settings except care homes. It aims to improve working practices to comply with legislation and have robust governance arrangements. It also aims to reduce the safety risks associated with controlled drugs.

NICE guideline Published April 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

Developing and updating local formularies (MPG1)

This guideline covers good practice for developing and updating local formularies in line with statutory requirements. It supports developing formularies that reflect local needs, reduce variation in prescribing, and allow rapid adoption of new medicines and treatments.

Medicines practice guideline Published March 2014 Last updated October 2015

Antimicrobial stewardship: systems and processes for effective antimicrobial medicine use (NG15)

This guideline covers the effective use of antimicrobials (including antibiotics) in children, young people and adults. It aims to change prescribing practice to help slow the emergence of antimicrobial resistance and ensure that antimicrobials remain an effective treatment for infection.

NICE guideline Published August 2015

Safe midwifery staffing for maternity settings (NG4)

This guideline covers safe midwifery staffing in all maternity settings, including at home, in the community, in day assessment units, in obstetric units, and in units led by midwives (both alongside hospitals and free-standing). It aims to improve maternity care by giving advice on monitoring staffing levels and actions to take if there are not enough midwives to meet the needs of women and babies in the service.

NICE guideline Published February 2015

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

Safe staffing for nursing in adult inpatient wards in acute hospitals (SG1)

This guideline covers organisational and managerial approaches to safe nurse staffing of inpatient wards for people aged 18 and over in acute hospitals. It aims to ensure that patients receive the nursing care they need, regardless of the ward to which they are allocated, the time of the day, or the day of the week.

Safe staffing guideline Published July 2014

Lysosomal acid lipase deficiency - sebelipase alfa [ID737]

In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] 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

Inpatient mental health settings

In development [GID-SGWAVE0701] Expected publication date: TBC

Safe staffing guideline In development

Accident and emergency departments

In development [GID-SGWAVE0762] Expected publication date: TBC

Safe staffing guideline In development

Management and organisational approaches to safe staffing

In development [GID-SGWAVE0761] Expected publication date: TBC

Safe staffing guideline 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

Afamelanotide for treating erythropoietic protoporphyria [ID927]

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

Highly specialised technologies guidance In development

Mental health community settings

In development [GID-SGWAVE0702] Expected publication date: TBC

Safe staffing guideline 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

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

Proposed [GID-HST10020] 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

Community nursing care settings

In development [GID-SGWAVE0700] Expected publication date: TBC

Safe staffing guideline 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

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] 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: 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