Suggested Remit: To appraise the clinical and cost effectiveness of tafamidis within its marketing authorisation for treating transthyretin amyloid cardiomyopathy for national commissioning by NHS England.
Status Proposed
Process STA 2018
ID number 1531

Provisional Schedule

Committee meeting 18 March 2020
Expected publication 10 June 2020

Project Team

Project lead Gemma Barnacle

Email enquiries

Evidence Review Group / Assessment Group School of Health and Related Research (ScHARR), University of Sheffield


Companies sponsors Pfizer (tafamidis)
Others Department of Health & Social Care
  NHS England
  NHS Leicester City CCG
  NHS Walsall CCG
  Welsh Government
Patient carer groups Action on Pain
  British Heart Foundation
  British Liver Trust
  British Society for Heart failure
  Cardiomyopathy UK
  Genetic Alliance UK
  Muslim Council of Britain
  Pain Concern
  Pain Relief Foundation
  Pain UK
  South Asian Health Foundation
  Specialised Healthcare Alliance
  UK ATTR Amyloidosis Patients’ Association
Professional groups Association of British Neurologists
  Association of Genetic Nurses and Counsellors
  British Association for the Study of the Liver
  British Cardiovascular Society
  British Peripheral Nerve Society
  British Society for Gene and Cell therapy
  British Society for Genetic Medicine
  British Society for Human Genetics
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Pathologists
  Royal College of Physicians
  Royal Society of Medicine
  UK Clinical Pharmacy Association
  UK Genetic Testing Network


Associated public health groups Public Health England
  Public Health Wales
Comparator companies Accord Healthcare (furosemide)
  Advanz Pharma (furosemide)
  Akcea Therapeutics UK (inotersen)
  Alnylam Pharmaceuticals (patisiran)
  Aurobindo Pharma (furosemide)
  Generics UK (bumetanide)
  Hameln Pharmaceuticals (furosemide)
  M & A Pharmachem (furosemide)
  Orion Pharma (furosemide)
  Pinewood Healthcare (furosemide)
  Rosemont Pharmaceuticals (furosemide)
  Sanofi (furosemide)
  Thame Laboratories (furosemide)
  Wockhardt UK (furosemide)
General commentators All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  Board of Community Health Councils in Wales
  British National Formulary
  Care Quality Commission
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare Products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  NHS Alliance
  NHS Confederation
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee
Relevant research groups British Society for Cardiovascular Research
  Cochrane Heart Group
  Cochrane Neuromuscular
  Foundation for Liver Research
  Genomics England
  Heart Research UK
  MRC Clinical Trials Unit
  National Amyloidosis Centre, at UCL
  National Institute for Health Research


Key events during the development of the guidance:

Date Update
15 July 2019 Invitation to participate
15 July 2019 The Department for Health and Social Care has asked NICE to carry out a Single Technology Appraisal of tafamidis for treating transthyretin amyloid cardiomyopathy. Consultees and commentators will be provided with details of the timelines when the appraisal starts in mid-July. The timings are based on a request from the company to reschedule the initial date set by NICE, in order to facilitate a suitably comprehensive and robust evidence submission. As a result, NICE cannot deliver on its commitment to publish guidance within 3 months of marketing authorisation for a product that was subject to the Early Access to Medicines Scheme (EAMS).
12 March 2019 (14:00) Scoping workshop (London)
23 January 2019 - 20 February 2019 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