{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T04:57:04Z","timestamp":1789016224420,"version":"build-2803163510"},"reference-count":29,"publisher":"Wiley","issue":"S2","license":[{"start":{"date-parts":[[2022,3,22]],"date-time":"2022-03-22T00:00:00Z","timestamp":1647907200000},"content-version":"vor","delay-in-days":21,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2022,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Emergence of new therapies are anticipated to improve clinical outcomes and quality of life of persons with haemophilia. Challenges in conducting randomized clinical trials in rare diseases have resulted in a lack of direct head\u2010to\u2010head comparisons to support value\u2010based decision\u2010making between different treatments.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We conducted a literature review for new and emerging haemophilia A and B therapies (extended half\u2010life [EHL] replacement factor, non\u2010replacement therapies [NRT], and gene therapies [GT]) to identify differentiating patient\u2010centred outcomes defined previously in a haemophilia value framework. Since the literature included all publication types (e.g., surveys, modelling studies, commentaries\/reviews), collected data were assigned level of evidence scores.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Across different classes of therapies, bleeding was determined as the most frequently reported differentiating outcome, with EHL, NRT, and GT each demonstrating an advantage over comparator replacement therapies. EHL therapies for haemophilia A and B and NRT for haemophilia A showed good representation across Tier 1 outcomes (health status achieved\/retained), while more publications were identified with Tier 2 (process of recovery) outcomes for NRT than EHL or GT. In Tier 3 (sustainability of health), frequency of breakthrough bleeds represented a differentiating outcome for EHL (both haemophilia A and B), NRT (haemophilia A only), and GT (haemophilia B only), whereas sustained good health was differentiating for most comparisons.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>We demonstrate the utility of the haemophilia value framework as a common core outcome set for effectively comparing therapies. Application of this framework will serve as a useful decision\u2010making tool for patients, clinicians, and within health technology assessments.<\/jats:p><\/jats:sec><jats:sec><jats:title>KEY POINTS OF CONSIDERATION<\/jats:title><jats:p><jats:list list-type=\"bullet\">\n<jats:list-item><jats:p>With the emergence of high\u2010cost, paradigm changing treatments across multiple areas of medicine, we, the haemophilia community, need to be equipped to meet the growing demands for more rigorous evidence\u2010based value assessments using the tools expected by assessors.<\/jats:p><\/jats:list-item>\n<jats:list-item><jats:p>The traditional access toolbox needs to evolve to meet the paradigm shift in treatment options. Value can no longer be defined by annualized bleed rates alone. To realize the full impact of new therapies, we need to utilize tools, such as a value framework, to organize evidence, identify data gaps, and assess patient\u2010defined, meaningful outcomes across a multi\u2010faceted dimension.<\/jats:p><\/jats:list-item>\n<jats:list-item><jats:p>The haemophilia value framework is an effective tool for organizing the available evidence and identifying gaps in the evidence. This can be used for assessing the value of emerging therapies in haemophilia utilizing data generated through randomized clinical trials and real world evidence generation.<\/jats:p><\/jats:list-item>\n<jats:list-item><jats:p>This is a call for incorporating the Value Framework into official submissions to authorities, as it captures a broader range of outcomes, including patient meaningful outcomes, in ways that better assess the potential benefits of new therapies.<\/jats:p><\/jats:list-item>\n<\/jats:list><\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14511","type":"journal-article","created":{"date-parts":[[2022,3,23]],"date-time":"2022-03-23T06:53:13Z","timestamp":1648018393000},"page":"9-18","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":13,"title":["A preliminary application of a haemophilia value framework to emerging therapies in haemophilia"],"prefix":"10.1111","volume":"28","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0934-0680","authenticated-orcid":false,"given":"Mark W.","family":"Skinner","sequence":"first","affiliation":[{"name":"Institute for Policy Advancement, Ltd.  Washington DC USA"},{"name":"McMaster University  Hamilton Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3270-6932","authenticated-orcid":false,"given":"Gerry","family":"Dolan","sequence":"additional","affiliation":[{"name":"Guy's and St Thomas\u2019 NHS Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1372-0619","authenticated-orcid":false,"given":"Hermann","family":"Eichler","sequence":"additional","affiliation":[{"name":"Institute of Clinical Haemostaseology and Transfusion Medicine Saarland University  Homburg Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9780-6972","authenticated-orcid":false,"given":"Brian","family":"O'Mahony","sequence":"additional","affiliation":[{"name":"Irish Haemophilia Society  Dublin Ireland"},{"name":"Trinity College  Dublin 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