{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,24]],"date-time":"2026-04-24T12:21:45Z","timestamp":1777033305628,"version":"3.51.4"},"reference-count":28,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2022,4,19]],"date-time":"2022-04-19T00:00:00Z","timestamp":1650326400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004319","name":"Pfizer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004319","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2022,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>With the development of gene therapy for people with haemophilia (PWH), it is important to understand how people impacted by haemophilia (PIH) and clinicians prioritise haemophilia treatment attributes to support informed treatment decisions.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objective<\/jats:title><jats:p>To examine the treatment attribute preferences of PIH and clinical experts in the United Kingdom (UK) and to develop a profile of gene therapy characteristics fit for use in future discrete choice experiments (DCEs).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Semi\u2010structured interviews were conducted with PIH (<jats:italic>n<\/jats:italic>= 14) and clinical experts (<jats:italic>n<\/jats:italic>= 6) who ranked pre\u2010defined treatment attributes by importance. Framework analysis was conducted to identify key themes and treatment attributes; points were allocated based on the rankings. Synthesis of results by a multidisciplinary group informed development of a profile of gene therapy characteristics for use in future research.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Key themes identified by PIH and clinical experts included patient relevant features and the importance of \u2018informed decision making'. The six top\u2010ranked treatment attributes were \u2018effect on factor level\u2019 (79 points), \u2018uncertainty regarding long\u2010term risks\u2019 (57 points), \u2018impact on daily life\u2019 (41 points), \u2018frequency of monitoring\u2019 (33 points), \u2018impact on ability to participate in physical activity\u2019 (29 points), and \u2018uncertainty regarding long\u2010term benefits\u2019 (28 points). The final treatment characteristics were categorised as therapeutic option, treatment effectiveness, safety concerns, impact on self\u2010management and quality of life (role limitations).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>We identified several gene therapy characteristics important to PIH and clinicians in the UK. These characteristics will be used in a future DCE to further investigate patient preferences for gene therapy.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14572","type":"journal-article","created":{"date-parts":[[2022,4,19]],"date-time":"2022-04-19T17:38:59Z","timestamp":1650389939000},"page":"588-609","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":14,"title":["Examining patient and professional perspectives in the UK for gene therapy in haemophilia"],"prefix":"10.1111","volume":"28","author":[{"given":"Ione","family":"Woollacott","sequence":"first","affiliation":[{"name":"Pfizer Ltd. 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