To appraise the clinical and cost effectiveness of caplacizumab within its marketing authorisation for treating adults experiencing an episode of acquired thrombotic thrombocytopenic purpura.
 
Status In progress
Process STA 2018
ID number 1185

Project Team

Project lead Jo Ekeledo

Email enquiries

Evidence Review Group / Assessment Group Peninsula Technology Assessment Group (PenTAG), University of Exeter

Consultees

Companies sponsors Sanofi (caplacizumab)
Others Department of Health
  NHS England
  Welsh Government
Patient carer groups AOFAC Foundation
  Genetic Alliance UK
  TTP Network
Professional groups British Committee for Standards in Haematology
  Royal College of Physicians
  Royal College of Radiologists

Commentators

Associated public health groups None
Comparator companies Baxter healthcare (cyclophosphamide) (Confidentiality Agreement not signed, not participating)
  Roche (rituximab) Confidentiality Agreement not signed, not participating)
  Hospira UK (vincristine) Confidentiality Agreement not signed, not participating)
  Sandoz (cyclophosphamide, rituximab) Confidentiality Agreement not signed, not participating)
General commentators All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  British National Formulary
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare Products Regulatory Agency
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee
Relevant research groups None

Timeline

Key events during the development of the guidance:

Date Update
12 June 2020 - 03 July 2020 Appraisal consultation: 1
12 June 2020 For information on 30 April 2020, the European Medicines Agency’s Committee for Medicinal Products for Human Use adopted a positive opinion recommending that a marketing authorisation extension is granted for caplacizumab for ‘adolescents of 12 years of age and older weighing at least 40 kg. NICE have updated the Final scope to reflect this licence extension.
09 August 2019 Invitation to participate
28 December 2018 As you will be aware the Department for Health and Social Care has asked NICE to carry out a Single Technology Appraisal of caplacizumab within its marketing authorisation for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression. Please note that following on from advice received from the company this appraisal has now been rescheduled back into the work programme. The appraisal is now anticipated to being in early August 2019.
14 June 2018 In progress. Topic referred
14 June 2018 As you will be aware the Department for Health and Social Care has asked NICE to carry out a Single Technology Appraisal of caplacizumab within its marketing authorisation for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression. The timelines for this appraisal are still to be confirmed following a recent update from Ablynx related to Sanofi’s previously announced tender offers to acquire all of the outstanding shares (including shares represented by American Depositary Shares, warrants and convertible bonds) of Ablynx. Please refer to link for further information http://www.ablynx.com/uploads/data/files/press%20release_sanofi%20squeez%20out_14%20may%202018_eng_final.pdf NICE is monitoring the situation and will update interested parties as and when further information is available.
05 December 2017 Referral

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