{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,13]],"date-time":"2026-02-13T15:56:34Z","timestamp":1770998194040,"version":"3.50.1"},"reference-count":21,"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:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100004326","name":"Bayer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004326","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2022,3]]},"abstract":"<jats:sec><jats:title>KEY POINTS OF CONSIDERATION<\/jats:title><jats:p><jats:list list-type=\"bullet\">\n<jats:list-item><jats:p>To prepare for the introduction of gene therapies in haemophilia care, healthcare frameworks for evaluation and valuation will need to evolve to address the unique requirements of current and future innovations for treating this rare disease.<\/jats:p><\/jats:list-item>\n<jats:list-item><jats:p>The papers in this supplement provide an insightful and comprehensive state\u2010of\u2010the\u2010art assessment of these requirements and challenges.<\/jats:p><\/jats:list-item>\n<jats:list-item><jats:p>In terms of evaluation, the definition of a patient\u2010defined value framework that captures multi\u2010dimensional, patient\u2010centered outcomes is an important first step for determining the full benefit of gene therapy for persons with haemophilia.<\/jats:p><\/jats:list-item>\n<jats:list-item><jats:p>In terms of valuation and rewards for innovation, health systems will need to develop alternative payment models for risk\u2010sharing that will allow payers and society to address uncertainties about the ultimate clinical and economic value of these innovations. And health technology assessment authorities will need to exercise greater flexibility in evidence requirements given the unique features of data collection for a potentially curative therapy for a rare disease with long\u2010term uncertainties about durability of impact.<\/jats:p><\/jats:list-item>\n<jats:list-item><jats:p>Collaboration among stakeholders will be essential for developing the critical evidence requirements and providing the incentives needed to achieve sustainable budgets and broad access for persons with haemophilia worldwide.<\/jats:p><\/jats:list-item><\/jats:list><\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14439","type":"journal-article","created":{"date-parts":[[2022,3,23]],"date-time":"2022-03-23T06:53:13Z","timestamp":1648018393000},"page":"5-8","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Is the world ready for gene therapy?"],"prefix":"10.1111","volume":"28","author":[{"given":"Louis P.","family":"Garrison","sequence":"first","affiliation":[{"name":"The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute in the School of Pharmacy University of Washington  Seattle Washington USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Diane","family":"Kleinermans","sequence":"additional","affiliation":[{"name":"Commission of Drugs Reimbursement by National Institute for Health and Disability in Chez National Institute for Health and Disability Insurance (INAMI\u2010RIZIV)  Brussels 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