{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T00:27:42Z","timestamp":1791246462061,"version":"4.1.0"},"reference-count":32,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2024,4,1]],"date-time":"2024-04-01T00:00:00Z","timestamp":1711929600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Pulmonology"],"published-print":{"date-parts":[[2024,6]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Elexacaftor\/tezacaftor\/ivacaftor (ETI) has revolutionized cystic fibrosis (CF) treatment. However, previous research has demonstrated profound global disparities in diagnosis and treatment access. If unaddressed, these threaten to widen existing health inequities. Therefore, in this analysis we aimed to reappraise gaps and evaluate progress in diagnosis and treatment equity in high\u2010income (HIC) versus low\u2010 and middle\u2010income countries (LMICs).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Estimates of the global CF population were made in 158 countries using patient registries, systematic literature searches, and an international survey of 14 CF experts. Estimates of the global burden of undiagnosed CF were made using epidemiological studies identified in literature searches and registry coverage data. The proportion of people receiving ETI was estimated using publicly available revenue data and a survey of 23 national drug pricing databases.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>188,336 (163,421\u2013209,204) people are estimated to have CF in 96 countries. Of these, 111,767 (59%) were diagnosed and 51,322 (27%) received ETI. The undiagnosed patient burden is estimated to be 76,569 people, with 82% in LMICs. ETI is reimbursed in 35 HICs, but only one LMIC. Four years after approval, there are 13,723 people diagnosed with CF who live in a country where ETI is inaccessible. This increases to 76,199 when including the estimated undiagnosed population.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Equitable access to CFTR modulators must become a top priority for the international CF community. ETI costs up to $322,000 per year but could be manufactured for $5000 to allow access under a voluntary license. Given the extent of disparities, other mechanisms to improve access that circumvent the manufacturer should also be considered.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/ppul.26954","type":"journal-article","created":{"date-parts":[[2024,4,1]],"date-time":"2024-04-01T09:34:35Z","timestamp":1711964075000},"page":"1622-1630","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":83,"title":["International disparities in diagnosis and treatment access for cystic fibrosis"],"prefix":"10.1002","volume":"59","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8007-1076","authenticated-orcid":false,"given":"Jonathan","family":"Guo","sequence":"first","affiliation":[{"name":"School of Public Health, Faculty of Medicine Imperial College London London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ibukunoluwa","family":"King","sequence":"additional","affiliation":[{"name":"School of Public Health, Faculty of Medicine Imperial College London London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew","family":"Hill","sequence":"additional","affiliation":[{"name":"Department of Pharmacology and Therapeutics University of Liverpool Liverpool UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,4]]},"reference":[{"key":"e_1_2_12_2_1","unstructured":"SilvermanE. Battle between Vertex insurers leads to crushing CF drug costs. STATNews. 2023. Accessed November 26 2023.https:\/\/www.statnews.com\/pharmalot\/2023\/02\/20\/cystic\u2010fibrosis\u2010drug\u2010costs\u2010copays\u2010vertex\/"},{"key":"e_1_2_12_3_1","unstructured":"GuzmanG KollarM. U.S. Census Bureau. Income in the United States: 2022. Current Population Reports. 2023(September):60\u2010279."},{"key":"e_1_2_12_4_1","unstructured":"Turkish Statistical Institute. T\u00dc\u0130K Kurumsal. Accessed February 17 2024.https:\/\/data.tuik.gov.tr\/Bulten\/Index?p=Income\u2010Distribution\u2010Statistics\u20102023\u201053711"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(20)30055-2"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.1136\/bmj.g1445"},{"key":"e_1_2_12_7_1","unstructured":"National Institute for Health and Care Excellence. Draft Guidance Consultation: Ivacaftor\u2013Tezacaftor\u2013Elexacaftor Tezacaftorivacaftor and Lumacaftor\u2013ivacaftor for Treating Cystic Fibrosis. 2023."},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2023.1163391"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(19)30337-6"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2022.01.009"},{"key":"e_1_2_12_11_1","unstructured":"SEC Filings. Vertex Pharmaceuticals. Accessed June 5 2023.https:\/\/investors.vrtx.com\/financial\u2010information\/sec\u2010filings"},{"key":"e_1_2_12_12_1","unstructured":"Vertex Announces Reimbursement Agreement in Australia for TRIKAFTA\u00ae (Elexacaftor\/Tezacaftor\/Ivacaftor and Ivacaftor) for Patients With Cystic Fibrosis Ages 12 Years and Older With at Least One F508del Mutation in the CFTR Gene. Vertex Pharmaceuticals Newsroom. Accessed June 5 2023.https:\/\/news.vrtx.com\/news\u2010releases\/news\u2010release\u2010details\/vertex\u2010announces\u2010reimbursement\u2010agreement\u2010australia\u2010trikaftar"},{"key":"e_1_2_12_13_1","unstructured":"UNAIDS. Seizing the Moment\u2014Tackling Entrenched Inequalities to End Epidemics. 2020.https:\/\/www.unaids.org\/sites\/default\/files\/media_asset\/2020_global\u2010aids\u2010report_en.pdf"},{"key":"e_1_2_12_14_1","unstructured":"New World Bank Country Classifications by Income Level: 2022\u20102023. Accessed January 17 2024.https:\/\/blogs.worldbank.org\/opendata\/new\u2010world\u2010bank\u2010country\u2010classifications\u2010income\u2010level\u20102022\u20102023"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2017.11.013"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2006.07.004"},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.1111\/resp.12656"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.26041"},{"key":"e_1_2_12_19_1","unstructured":"ECFS European Twinning Project. European Cystic Fibrosis Society (ECFS). Accessed November 27 2023.https:\/\/www.ecfs.eu\/ecfs\u2010standards\u2010care\/twinning"},{"key":"e_1_2_12_20_1","unstructured":"NolenS RobbinsR. This cystic fibrosis drug Is a \u2018Miracle\u2019 but These Families Can't Get It. The New York Times. 2023. Accessed June 5 2023.https:\/\/www.nytimes.com\/2023\/02\/07\/health\/cystic\u2010fibrosis\u2010drug\u2010trikafta.html"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2022.04.007"},{"key":"e_1_2_12_22_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202307-1200VP"},{"key":"e_1_2_12_23_1","unstructured":"The Pharmaceutical Benefits Scheme. Elexacaftor 100 mg\/Tezacaftor 50 mg\/Ivacaftor 75 mg Film\u2010coated Tablets Co\u2010packaged with Ivacaftor 150 mg Film\u2010Coated Tablets. Vertex Pharmaceuticals (Australia) Pty Ltd. 2021."},{"key":"e_1_2_12_24_1","unstructured":"Vertex Pharmaceuticals. Medicines. Expanded Access. Accessed June 5 2023.https:\/\/www.vrtx.com\/en\u2010us\/medicines\/expanded\u2010access\/"},{"key":"e_1_2_12_25_1","unstructured":"Cystic Fibrosis Australia. Trikafta Compassionate Access Update. 2022. Accessed November 28 2023.https:\/\/cysticfibrosis.org.au\/wp\u2010content\/uploads\/2023\/05\/Communique\u2010384\u2010TRIKAFTA\u2010COMPASSIONATE\u2010ACCESS\u2010UPDATE.pdf"},{"key":"e_1_2_12_26_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(23)00297-7"},{"key":"e_1_2_12_27_1","unstructured":"KarumbaA. Medicines Patent Pool 2021 Annual Report: Turning the Tide on Inequity in Access 26 Billion Doses of Treatment in 11 Years. 2021. Accessed June 5 2023.https:\/\/medicinespatentpool.org\/uploads\/2022\/08\/MPP\u20102021\u2010annual\u2010report\u2010web.pdf"},{"key":"e_1_2_12_28_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.26335"},{"key":"e_1_2_12_29_1","unstructured":"TRIPS Database\u2014Medicines Law & Policy. Accessed June 5 2023.http:\/\/tripsflexibilities.medicineslawandpolicy.org\/"},{"key":"e_1_2_12_30_1","unstructured":"CONITEC. Elexacaftor\/Tezacaftor\/Ivacaftor for the Treatment of Cystic Fibrosis Patients Aged 6 Years and Older with at Least One f508del Mutation in the Cystic Fibrosis Transmembrane Conduction Regulator Gene. 2023;(September):117."},{"key":"e_1_2_12_31_1","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.01241-2023"},{"key":"e_1_2_12_32_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jcf.2023.02.004"},{"key":"e_1_2_12_33_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1569-1993(08)60436-1"}],"container-title":["Pediatric Pulmonology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ppul.26954","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,1,17]],"date-time":"2025-01-17T09:32:19Z","timestamp":1737106339000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ppul.26954"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,4]]},"references-count":32,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2024,6]]}},"alternative-id":["10.1002\/ppul.26954"],"URL":"https:\/\/doi.org\/10.1002\/ppul.26954","archive":["Portico"],"relation":{},"ISSN":["8755-6863","1099-0496"],"issn-type":[{"value":"8755-6863","type":"print"},{"value":"1099-0496","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,4]]},"assertion":[{"value":"2024-02-10","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-02-27","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-04-01","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}