{"id":292870,"date":"2024-06-11T00:00:00","date_gmt":"2024-06-11T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/acrehe0002-2024-biopharma-sickle-cell-disease-access-reimbursement-access-reimbursement-sickle\/"},"modified":"2026-03-31T10:29:44","modified_gmt":"2026-03-31T10:29:44","slug":"acrehe0002-2024-biopharma-sickle-cell-disease-access-reimbursement-access-reimbursement-sickle-cell-disease-us","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/acrehe0002-2024-biopharma-sickle-cell-disease-access-reimbursement-access-reimbursement-sickle-cell-disease-us\/","title":{"rendered":"Sickle Cell Disease &#8211; Access &#038; Reimbursement &#8211; Access &#038; Reimbursement &#8211; Sickle Cell Disease (US)"},"content":{"rendered":"<p>Sickle cell disease (<abbr title=\"sickle cell disease\">SCD<\/abbr>) is a rare genetic blood disorder characterized by polymerization of hemoglobin in red blood cells that distorts them into a sickle shape, leading to various complications (e.g., painful vaso-occlusive crisis). While hydroxyurea has been the <abbr title=\"standard of care\">SOC<\/abbr> for <abbr title=\"sickle cell disease\">SCD<\/abbr>, the <abbr title=\"Food and Drug Administration\">FDA<\/abbr>\u2019s approval of Endari, Oxbryta, and Adakveo has given patients targeted treatment options. Moreover, new gene therapies Lyfgenia (Bluebird Bio) and Casgevy (Vertex \/ CRISPR Therapeutics) offer curative potential. Two emerging symptomatic therapies\u2014inclacumab and GBT021601, both from Global Blood Therapeutics \/ Pfizer\u2014are promising targeted <abbr title=\"sickle cell disease\">SCD<\/abbr> treatments. Further, Pyrukynd (mitapivat; Agios Pharmaceuticals) is being developed for <abbr title=\"sickle cell disease\">SCD<\/abbr> (currently approved for hemolytic anemia in adults with <abbr title=\"pharmacokinetic\">PK<\/abbr> deficiency). This report explores hematologists\u2019 and payers\u2019 views and policies regarding approved <abbr title=\"sickle cell disease\">SCD<\/abbr> therapies and assesses expected use of new therapies in the context of potential coverage of such treatments.<\/p>\n<p><b>Questions answered<\/b><\/p>\n<ul class=\"round-bullets\">\n<li>How are approved <abbr title=\"sickle cell disease\">SCD<\/abbr> therapies covered? What restrictions do payers impose, and how do market access dynamics influence surveyed hematologists\u2019 prescribing?<\/li>\n<li>What price concessions and clinical data do payers want to favorably cover the gene therapies Lyfgenia and Casgevy and emerging symptomatic <abbr title=\"sickle cell disease\">SCD<\/abbr> therapies?<\/li>\n<li>How receptive are physicians to gene and emerging symptomatic therapies for <abbr title=\"sickle cell disease\">SCD<\/abbr>? What clinical and nonclinical factors will drive their use, and what payer controls will constrain prescribing?<\/li>\n<\/ul>\n<p><strong>Content highlights<\/strong><\/p>\n<p><strong>Geography:<\/strong> United States<\/p>\n<p><strong>Primary research:<\/strong> Survey of 100 U.S. hematologists; survey of 30 U.S. managed care organization (<abbr title=\"managed care organization\">MCO<\/abbr>) pharmacy and medical directors (<abbr title=\"pharmacy director\">PD<\/abbr>s\/<abbr title=\"medical director\">MD<\/abbr>s)<\/p>\n<p><strong>Key drugs covered: <\/strong>Adakveo (crizanlizumab), Endari (L-glutamine), Oxbryta (voxelotor), Lyfgenia (lovotibeglogene autotemcel; lovo-cel), Casgevy (exagamglogene autotemcel; exa-cel), inclacumab, GBT021601, Pyrukynd (mitapivat)<\/p>\n<p><strong>Production description<\/strong><\/p>\n<p>U.S. Access &#038; Reimbursement provides integrated brand- and disease-level insight on reimbursement dynamics and the impact of U.S. payer policy on physician prescribing behavior in the market access environment. Each report includes up-to-date analyses of drug coverage and restriction policies as well as payer and prescriber perspectives on key marketed therapies and their receptivity to emerging therapies.<\/p>\n<p><strong>Solution enhancement<\/strong><\/p>\n<p>Access &#038; Reimbursement includes key analyses from Clarivate Real-World Data Product\u2014comprehensive and timely data that provide clarity around the healthcare experiences and activities of hundreds of millions of patients, <abbr title=\"healthcare provider\">HCP<\/abbr>s, and payers. Analyses include payer mix, percentage of claim rejections for key therapies, and reasons for claim rejection.<\/p>\n","protected":false},"template":"","class_list":["post-292870","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-hematology","biopharma-therapy-areas-sickle-cell-disease","biopharma-geography-us","biopharma-date-985"],"acf":[],"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/292870","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report"}],"about":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/types\/report"}],"version-history":[{"count":10,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/292870\/revisions"}],"predecessor-version":[{"id":575952,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/292870\/revisions\/575952"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=292870"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}