Accurate accounting of caplacizumab cost effectiveness

We have substantial concerns about the final input parameters in cost-effectiveness analysis by Sanofi that was used to support National Institute for Health and Care Excellence (NICE) recommendation caplacizumab acute acquired thrombotic thrombocytopenic purpura, 1 ExcellenceTechnology appraisal guidance TA667: with plasma exchange immunosuppression treating purpura. https://www.nice.org.uk/guidance/ta667Date: Dec 16, 2020 Date accessed: December 20, Google Scholar as described Mary Hughes colleagues. 2 M Prescott C Elliott N Adler AI NICE on thrombocytopenia Lancet Haematol. 2021; 8: e14-e15 Summary Full Text PDF PubMed Scopus (1) With a list price of US$270 000 per course, warrants close scrutiny whether it is truly cost-effective use Service (NHS) resources, even after incorporating undisclosed discounts made available Sanofi. Unlike current highly effective standard care, does not alter underlying pathophysiology purpura associated an increased risk haemorrhage disease relapse. 3 Scully Cataland SR Peyvandi F et al. Caplacizumab treatment Engl J Med. 2019; 380: 335-346 Crossref (340) purpuraOn 2020, published guidance1 recommending combined immunosuppressive therapy option episodes adults young patients aged 12 years over. The states physicians experienced managing microangiopathies should start supervise treatment, company provides according agreed commercial arrangement. Full-Text Accurate accounting cost effectiveness – Authors' replyGeorge Goshua colleagues interest decision making process technology purpura.1 They question given their own analyses. As describes, rare, acutely life-threatening condition. Data presented were limited two trials, which did measure death or quality life outcomes, there no comparative data longer term outcomes stops.