{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T14:35:01Z","timestamp":1793370901221,"version":"4.5.0"},"reference-count":21,"publisher":"Springer Science and Business Media LLC","issue":"6","license":[{"start":{"date-parts":[[2026,8,8]],"date-time":"2026-08-08T00:00:00Z","timestamp":1786147200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2026,8,8]],"date-time":"2026-08-08T00:00:00Z","timestamp":1786147200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"name":"This study was sponsored by National Pharmaceutical Council.","id":[{"id":"https:\/\/ror.org\/027608a92","id-type":"ROR","asserted-by":"crossref"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Ther Innov Regul Sci"],"published-print":{"date-parts":[[2026,11]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Some US payers delay coverage for non-oncology treatments approved via the Food and Drug Administration (FDA)\u2019s accelerated approval (AA) pathway.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives<\/jats:title>\n                    <jats:p>To estimate the economic burden and characteristics of diseases with therapies approved via the AA pathway compared to diseases with no AA therapies.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>The study identified the initial FDA-approved AA indications for all non-oncology therapies from January 2015 to February 2025. For comparison, 100 non-oncology diseases with the most recent FDA-approved non-AA therapies were identified. Annual economic burden and disease characteristics were extracted from peer-reviewed literature, with costs inflated to 2025 USD. Economic burden studies that used a societal perspective were prioritized. Comparative analyses were conducted using the Mann\u2013Whitney U Test.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Thirty-two non-oncology AA treatments were identified representing 16 unique diseases. Average annual incremental societal costs for AA diseases were 1.4 times higher than for non-AA diseases ($91,857 vs. $65,468,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.012). Additionally, the annual average productivity loss and caregiver burden were 1.8 times ($17,635 vs. $9,620,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.092) and 2.4 times ($20,268 vs. $8,294,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.055) greater in the AA diseases, respectively. Diseases with newly-approved AA treatments were characterized by shorter life expectancy (59.3 vs 65.1\u00a0years), lower average quality of life (0.64 vs 0.69), and more likely to be rare diseases (68.8% vs 53.1%) compared to non-AA diseases.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Non-oncology diseases with treatments approved via the AA pathway imposed a higher economic burden and had more severe disease characteristics compared to non-AA diseases. These findings should be considered alongside AA coverage policy decision-making.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1007\/s43441-026-01030-x","type":"journal-article","created":{"date-parts":[[2026,8,8]],"date-time":"2026-08-08T13:22:14Z","timestamp":1786195334000},"page":"1840-1847","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Clinical and Economic Burden of Diseases with Accelerated Approval Therapies: Evidence of Unmet Patient Need"],"prefix":"10.1007","volume":"60","author":[{"given":"Hanke","family":"Zheng","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shanshan","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jason","family":"Shafrin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shravani","family":"Bheema","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn","family":"Spurrier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan D.","family":"Campbell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2026,8,8]]},"reference":[{"issue":"4","key":"1030_CR1","doi-asserted-by":"publisher","first-page":"1047","DOI":"10.1111\/1468-0009.12670","volume":"101","author":"PJ Neumann","year":"2023","unstructured":"Neumann PJ, Crummer E, Chambers JD, Tunis SR. 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SW, SB, KS and JS are employees of FTI Consulting, Inc, a company that provides consulting services to the health care, life sciences, government and non-governmental entities among others. JS is also an adjunct professor at the University of Southern California and serves on the Scientific Advisory Committee of the Schizophrenia and Psychosis Action Alliance. No other potential conflicts of interest were reported by the authors. This study was funded by NPC. NPC provided input into the initial study concept design, study execution, and the decision to publish.","order":1,"name":"Ethics","label":"Conflict of interest","group":{"name":"EthicsHeading","label":"Declarations"}}]}}