{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,22]],"date-time":"2026-07-22T08:38:53Z","timestamp":1784709533489,"version":"3.55.0"},"reference-count":19,"publisher":"Elsevier BV","issue":"5","license":[{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,6,25]],"date-time":"2025-06-25T00:00:00Z","timestamp":1750809600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","cysticfibrosisjournal.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Journal of Cystic Fibrosis"],"published-print":{"date-parts":[[2025,9]]},"DOI":"10.1016\/j.jcf.2025.06.006","type":"journal-article","created":{"date-parts":[[2025,7,4]],"date-time":"2025-07-04T21:26:37Z","timestamp":1751664397000},"page":"920-922","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":4,"title":["Challenges and opportunities of adding CFTR modulators for cystic fibrosis to the WHO essential medicines list"],"prefix":"10.1016","volume":"24","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2616-994X","authenticated-orcid":false,"given":"Enrico","family":"Costa","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1339-9818","authenticated-orcid":false,"given":"Hendrika A","family":"van den Ham","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7175-1499","authenticated-orcid":false,"given":"Hubert G.M.","family":"Leufkens","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.jcf.2025.06.006_bib0001","unstructured":"Proposal for the addition of ivacaftor\/tezacaftor\/elexacaftor to the who model list of essential medicines for the treatment of adults and children with cystic fibrosis n.d. https:\/\/cdn.who.int\/media\/docs\/default-source\/2025-eml-expert-committee\/addition-of-new-medicines\/a.11_elexacaftor-ivacaftor-tezacaftor.pdf?sfvrsn=5133bfda_1 (accessed March 27, 2025)."},{"key":"10.1016\/j.jcf.2025.06.006_bib0002","doi-asserted-by":"crossref","first-page":"188","DOI":"10.1186\/s13023-022-02350-5","article-title":"The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis","volume":"17","author":"Costa","year":"2022","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.jcf.2025.06.006_bib0003","first-page":"37","article-title":"Cystic fibrosis in low and middle-income countries (LMIC): a view from four different regions of the world","volume":"38","author":"da","year":"2021","journal-title":"Paediatr Respir Rev"},{"key":"10.1016\/j.jcf.2025.06.006_bib0004","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1016\/S0140-6736(16)31599-9","article-title":"Essential medicines for universal health coverage","volume":"389","author":"Wirtz","year":"2017","journal-title":"Lancet"},{"key":"10.1016\/j.jcf.2025.06.006_bib0005","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1016\/S2213-2600(19)30337-6","article-title":"The future of cystic fibrosis care: a global perspective","volume":"8","author":"Bell","year":"2020","journal-title":"Lancet Respir Med"},{"key":"10.1016\/j.jcf.2025.06.006_bib0006","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1016\/S2213-2600(15)00507-X","article-title":"Treatment of cystic fibrosis in low-income countries","volume":"4","author":"Cohen-Cymberknoh","year":"2016","journal-title":"Lancet Respir Med"},{"key":"10.1016\/j.jcf.2025.06.006_bib0007","doi-asserted-by":"crossref","first-page":"308","DOI":"10.1186\/s13023-021-01923-0","article-title":"Essential list of medicinal products for rare diseases: recommendations from the IRDiRC Rare Disease Treatment Access Working Group","volume":"16","author":"Gahl","year":"2021","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.jcf.2025.06.006_bib0008","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1002\/ajh.27525","article-title":"Globalization in clinical drug development for sickle cell disease","volume":"100","author":"Costa","year":"2025","journal-title":"Am J Hematol"},{"key":"10.1016\/j.jcf.2025.06.006_bib0009","article-title":"Medicines not recommended for inclusion in the who essential medicines list: a retrospective observational study","volume":"12","author":"Costa","year":"2025","journal-title":"Front Med (Lausanne)"},{"key":"10.1016\/j.jcf.2025.06.006_bib0010","doi-asserted-by":"crossref","first-page":"1701","DOI":"10.1016\/S0140-6736(24)02414-0","article-title":"Hope for rare diseases","volume":"404","year":"2024","journal-title":"Lancet"},{"key":"10.1016\/j.jcf.2025.06.006_bib0011","doi-asserted-by":"crossref","first-page":"22","DOI":"10.2471\/BLT.23.289731","article-title":"Uptake of orphan drugs in the WHO essential medicines lists","volume":"102","author":"Costa","year":"2024","journal-title":"Bull World Health Organ"},{"key":"10.1016\/j.jcf.2025.06.006_bib0012","doi-asserted-by":"crossref","first-page":"e597","DOI":"10.1016\/S2352-3026(19)30238-8","article-title":"Essential medicines: a balancing act","volume":"6","author":"Haematology","year":"2019","journal-title":"Lancet Haematol"},{"key":"10.1016\/j.jcf.2025.06.006_bib0013","doi-asserted-by":"crossref","DOI":"10.1136\/bmj-2023-077776","article-title":"Refocusing the World Health Organization\u2019s model List of Essential Medicines on the needs of low and middle income countries","author":"Wirtz","year":"2024","journal-title":"BMJ"},{"key":"10.1016\/j.jcf.2025.06.006_bib0014","doi-asserted-by":"crossref","first-page":"949","DOI":"10.1016\/j.jcf.2023.07.006","article-title":"Traits, trends and hits of orphan drug designations in cystic fibrosis","volume":"22","author":"Costa","year":"2023","journal-title":"J Cyst Fibros"},{"key":"10.1016\/j.jcf.2025.06.006_bib0015","article-title":"What does it mean to put new hepatitis C drugs on a list of essential medicines?","volume":"353","author":"Urrutia","year":"2016","journal-title":"BMJ"},{"key":"10.1016\/j.jcf.2025.06.006_bib0016","doi-asserted-by":"crossref","first-page":"1622","DOI":"10.1002\/ppul.26954","article-title":"International disparities in diagnosis and treatment access for cystic fibrosis","volume":"59","author":"Guo","year":"2024","journal-title":"Pediatr Pulmonol"},{"key":"10.1016\/j.jcf.2025.06.006_bib0017","doi-asserted-by":"crossref","first-page":"e676","DOI":"10.1016\/S2214-109X(15)00156-4","article-title":"Disparity in market prices for hepatitis C virus direct-acting drugs","volume":"3","author":"Andrieux-Meyer","year":"2015","journal-title":"Lancet Glob Health"},{"key":"10.1016\/j.jcf.2025.06.006_bib0018","doi-asserted-by":"crossref","first-page":"866","DOI":"10.1016\/j.jcf.2022.04.007","article-title":"Current prices versus minimum costs of production for CFTR modulators","volume":"21","author":"Guo","year":"2022","journal-title":"J Cyst Fibros"},{"key":"10.1016\/j.jcf.2025.06.006_bib0019","doi-asserted-by":"crossref","first-page":"932","DOI":"10.1016\/S0140-6736(15)01025-9","article-title":"The ethics of expanding access to cheaper, less effective treatments","volume":"388","author":"Persad","year":"2016","journal-title":"Lancet"}],"container-title":["Journal of Cystic Fibrosis"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1569199325015206?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1569199325015206?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,5,28]],"date-time":"2026-05-28T07:35:14Z","timestamp":1779953714000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1569199325015206"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,9]]},"references-count":19,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2025,9]]}},"alternative-id":["S1569199325015206"],"URL":"https:\/\/doi.org\/10.1016\/j.jcf.2025.06.006","relation":{},"ISSN":["1569-1993"],"issn-type":[{"value":"1569-1993","type":"print"}],"subject":[],"published":{"date-parts":[[2025,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Challenges and opportunities of adding CFTR modulators for cystic fibrosis to the WHO essential medicines list","name":"articletitle","label":"Article Title"},{"value":"Journal of Cystic Fibrosis","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jcf.2025.06.006","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Author(s). Published by Elsevier B.V. on behalf of European Cystic Fibrosis Society.","name":"copyright","label":"Copyright"}]}}