Suggested Remit: To evaluate the benefits and costs of emapalumab within its marketing authorisation for treating primary haemophagocytic lymphohistiocytosis for national commissioning by NHS England.
Status Proposed
Process TAG
ID number 1438

Project Team

Project lead Gemma Barnacle

Email enquiries

Evidence Review Group / Assessment Group BMJ Technology Assessment Group (BMJ-TAG), BMJ


Companies sponsors Swedish Orphan Biovitrum (emapalumab)
Others Department of Health and Social Care
  NHS England
  NHS Medway CCG
  NHS Wokingham CCG
  Welsh Goverment
Patient carer groups Action for Sick Children
  Anthony Nolan
  Genetic Alliance UK
  Genetic Disorders UK
  Histio UK
  Metabolic Support UK
  Muslim Council of Britain
  Primary Immunodeficiency UK
  South Asian Health Foundation
  Specialised Healthcare Alliance
  Together for Shorter Lives
Professional groups Association of British Neurologists
  Association of Genetic Nurses & Counsellors
  Association of Paediatric Emergency Medicine
  British Inherited Metabolic Disease Group
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  British Society for Human Genetics
  British Society for Rheumatology
  British Transplantation Society
  Genomics England
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics & Child Health
  Royal College of Pathologists
  Royal College of Physicians
  Royal Pharmaceutical Society
  Royal Society of Medicine
  Society for the Study of Inborn Errors of Metabolism
  UK Clinical Pharmacy Association
  UK Genetic Testing Network


Associated public health groups Public Health England
  Public Health Wales
Comparator companies Accord Healthcare (etoposide)
  Allergan (prednisolone)
  AMCo (methotrexate, prednisolone)
  Aspen Pharma Trading (dexamethasone)
  Auden McKenzie (dexamethasone, prednisolone)
  Beacon Pharmaceuticals (prednisolone)
  Boston Healthcare (prednisolone)
  Bristol-Myers Squibb (etoposide)
  Cardinal Health Martindale Products (dexamethasone)
  Chemidex Pharma (dexamethasone)
  Concordia International (dexamethasone)
  Colorama Pharmaceuticals (ciclosporin)
  Cubic Pharmaceuticals (ciclosporin)
  DE Pharmaceuticals (ciclosporin, dexamethasone, methotrexate, prednisolone)
  Dexcel (ciclosporin)
  Hameln (methotrexate, dexamethasone)
  Hospira UK (dexamethasone, methotrexate)
  Icarus Pharmaceuticals (ciclosporin)
  Medac (methotrexate, etoposide)
  Merck Sharp & Dohme (dexamethasone)
  Mylan UK (ciclosporin)
  Niche Pharma (ciclosporin)
  Novartis (ciclosporin)
  Orion (methotrexate)
  Pfizer (methotrexate, prednisolone)
  Rosemont Pharmaceuticals Ltd (dexamethasone, methotrexate)
  Sandoz (methotrexate)
  Sanofi (alemtuzumab)
  Teva UK (ciclosporin, methotrexate, prednisolone)
  Wockhardt (methotrexate, prednisolone)
  Zentiva UK (prednisolone)
General commentators All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  Board of Community Health Councils in Wales
  British National Formulary
  British Society of Blood and Marrow Transplantation
  Care Quality Commission
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Inherited Metabolic and Lysosomal Disease Service, Cardiff and Vale UHB
  Medicines and Healthcare Products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  National Services Division (specialised services in Scotland)
  NHS Alliance
  NHS Blood and Transplant
  NHS Confederation
  Scottish Medicines Consortium
  United Kingdom National Screening Committee
  Welsh Blood Service
  Welsh Health Specialised Services Committee
Relevant research groups Cell and Gene Therapy Catapult
  Cancer Research UK
  Cochrane Cystic Fibrosis and Genetic Disorders Group
  Gene and Cell Therapy Group - UCL
  Medical Research Council - Human Genetics Unit
  MRC Clinical Trials Unit
  National Institute for Health Research


Key events during the development of the guidance:

Date Update
30 August 2019 Invitation to participate
21 August 2019 The Department for Health and Social Care has asked us to carry out a Single Technology Appraisal of Emapalumab for treating primary haemophagocytic lymphohistiocytosis [ID1438]. The appraisal is expected to start in approximately late August 2019 when we will write to you about how you can get involved. The deadline for submissions is expected in approximately late October 2019.
14 September 2018 (14:00) Scoping workshop (London)
31 July 2018 - 21 August 2018 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators

For further information on how we develop guidance, please see our page about NICE technology appraisal guidance