{"id":"https://openalex.org/W4389937583","doi":"https://doi.org/10.5694/mja2.52191","title":"Providing Australian children and adolescents with equitable access to new and emerging therapies through clinical trials: a call to action","display_name":"Providing Australian children and adolescents with equitable access to new and emerging therapies through clinical trials: a call to action","publication_year":2023,"publication_date":"2023-12-19","ids":{"openalex":"https://openalex.org/W4389937583","doi":"https://doi.org/10.5694/mja2.52191","pmid":"https://pubmed.ncbi.nlm.nih.gov/38112125"},"language":"en","primary_location":{"id":"doi:10.5694/mja2.52191","doi":"https://doi.org/10.5694/mja2.52191","is_oa":true,"landing_page_url":"http://dx.doi.org/10.5694/mja2.52191","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.5694/mja2.52191","source":{"id":"https://openalex.org/S13978405","display_name":"The Medical Journal of Australia","issn_l":"0025-729X","issn":["0025-729X","1326-5377"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Medical Journal of Australia","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.5694/mja2.52191","any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5064882732","display_name":"Michelle Lorentzos","orcid":"https://orcid.org/0009-0005-9608-6535","observed_orcids":["https://orcid.org/0009-0005-9608-6535"]},"institutions":[{"id":"https://openalex.org/I129604602","display_name":"The University of Sydney","ror":"https://ror.org/0384j8v12","country_code":"AU","type":"education","lineage":["https://openalex.org/I129604602"]},{"id":"https://openalex.org/I4210149013","display_name":"Sydney Children\u2019s Hospitals Network","ror":"https://ror.org/04d87y574","country_code":"AU","type":"healthcare","lineage":["https://openalex.org/I4210149013"]}],"countries":["AU"],"is_corresponding":true,"raw_author_name":"Michelle S Lorentzos","raw_affiliation_strings":["Kids Research, Sydney Children's Hospitals Network, Sydney, NSW","University of Sydney, Sydney, NSW","[email protected]"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Kids Research, Sydney Children's Hospitals Network, Sydney, NSW","institution_ids":["https://openalex.org/I4210149013"]},{"raw_affiliation_string":"University of Sydney, Sydney, NSW","institution_ids":["https://openalex.org/I129604602"]},{"raw_affiliation_string":"[email protected]","institution_ids":[]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5102758030","display_name":"David Metz","orcid":"https://orcid.org/0000-0003-4478-5308","observed_orcids":["https://orcid.org/0000-0003-4478-5308"]},"institutions":[{"id":"https://openalex.org/I4210099911","display_name":"Monash Children\u2019s Hospital","ror":"https://ror.org/016mx5748","country_code":"AU","type":"healthcare","lineage":["https://openalex.org/I4210099911","https://openalex.org/I4210121067"]},{"id":"https://openalex.org/I4210121067","display_name":"Monash Health","ror":"https://ror.org/02t1bej08","country_code":"AU","type":"healthcare","lineage":["https://openalex.org/I4210121067"]},{"id":"https://openalex.org/I56590836","display_name":"Monash University","ror":"https://ror.org/02bfwt286","country_code":"AU","type":"education","lineage":["https://openalex.org/I56590836"]}],"countries":["AU"],"is_corresponding":false,"raw_author_name":"David Metz","raw_affiliation_strings":["Monash Children's Clinical Trial Centre, Monash Children's Hospital, Melbourne, VIC","Monash University, Melbourne, VIC"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Monash Children's Clinical Trial Centre, Monash Children's Hospital, Melbourne, VIC","institution_ids":["https://openalex.org/I4210099911","https://openalex.org/I4210121067"]},{"raw_affiliation_string":"Monash University, Melbourne, VIC","institution_ids":["https://openalex.org/I56590836"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5035808651","display_name":"Andrew S. Moore","orcid":"https://orcid.org/0000-0001-8062-1779","observed_orcids":["https://orcid.org/0000-0001-8062-1779"]},"institutions":[{"id":"https://openalex.org/I165143802","display_name":"The University of Queensland","ror":"https://ror.org/00rqy9422","country_code":"AU","type":"education","lineage":["https://openalex.org/I165143802"]},{"id":"https://openalex.org/I4210167058","display_name":"Children's Health Queensland Hospital and Health Service","ror":"https://ror.org/02t3p7e85","country_code":"AU","type":"healthcare","lineage":["https://openalex.org/I1302211766","https://openalex.org/I2801244131","https://openalex.org/I4210167058"]}],"countries":["AU"],"is_corresponding":false,"raw_author_name":"Andrew S Moore","raw_affiliation_strings":["Child Health Research Centre, University of Queensland, Brisbane, QLD","Queensland Children's Hospital, Brisbane, QLD"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Child Health Research Centre, University of Queensland, Brisbane, QLD","institution_ids":["https://openalex.org/I165143802"]},{"raw_affiliation_string":"Queensland Children's Hospital, Brisbane, QLD","institution_ids":["https://openalex.org/I4210167058"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5004442035","display_name":"Laura Katherine Fawcett","orcid":"https://orcid.org/0000-0001-7069-6179","observed_orcids":["https://orcid.org/0000-0001-7069-6179"]},"institutions":[{"id":"https://openalex.org/I31746571","display_name":"UNSW Sydney","ror":"https://ror.org/03r8z3t63","country_code":"AU","type":"education","lineage":["https://openalex.org/I31746571"]},{"id":"https://openalex.org/I4210149013","display_name":"Sydney Children\u2019s Hospitals Network","ror":"https://ror.org/04d87y574","country_code":"AU","type":"healthcare","lineage":["https://openalex.org/I4210149013"]}],"countries":["AU"],"is_corresponding":false,"raw_author_name":"Laura K Fawcett","raw_affiliation_strings":["Kids Research, Sydney Children's Hospitals Network, Sydney, NSW","University of New South Wales, Sydney, NSW"],"raw_orcid":"https://orcid.org/0000-0001-7069-6179","affiliations":[{"raw_affiliation_string":"Kids Research, Sydney Children's Hospitals Network, Sydney, NSW","institution_ids":["https://openalex.org/I4210149013"]},{"raw_affiliation_string":"University of New South Wales, Sydney, NSW","institution_ids":["https://openalex.org/I31746571"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5005383208","display_name":"Paula Bray","orcid":"https://orcid.org/0000-0002-5874-1667","observed_orcids":["https://orcid.org/0000-0002-5874-1667"]},"institutions":[{"id":"https://openalex.org/I4210149013","display_name":"Sydney Children\u2019s Hospitals Network","ror":"https://ror.org/04d87y574","country_code":"AU","type":"healthcare","lineage":["https://openalex.org/I4210149013"]}],"countries":["AU"],"is_corresponding":false,"raw_author_name":"Paula Bray","raw_affiliation_strings":["Sydney Children's Hospitals Network, Sydney, NSW"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Sydney Children's Hospitals Network, Sydney, NSW","institution_ids":["https://openalex.org/I4210149013"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5093268649","display_name":"Lani Attwood","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210149013","display_name":"Sydney Children\u2019s Hospitals Network","ror":"https://ror.org/04d87y574","country_code":"AU","type":"healthcare","lineage":["https://openalex.org/I4210149013"]}],"countries":["AU"],"is_corresponding":false,"raw_author_name":"Lani Attwood","raw_affiliation_strings":["Kids Research, Sydney Children's Hospitals Network, Sydney, NSW"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Kids Research, Sydney Children's Hospitals Network, Sydney, NSW","institution_ids":["https://openalex.org/I4210149013"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5053659589","display_name":"Craig Frank Munns","orcid":"https://orcid.org/0000-0001-5898-5808","observed_orcids":["https://orcid.org/0000-0001-5898-5808"]},"institutions":[{"id":"https://openalex.org/I165143802","display_name":"The University of Queensland","ror":"https://ror.org/00rqy9422","country_code":"AU","type":"education","lineage":["https://openalex.org/I165143802"]}],"countries":["AU"],"is_corresponding":false,"raw_author_name":"Craig F Munns","raw_affiliation_strings":["Child Health Research Centre, University of Queensland, Brisbane, QLD"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Child Health Research Centre, University of Queensland, Brisbane, QLD","institution_ids":["https://openalex.org/I165143802"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5114205119","display_name":"Andrew Davidson","orcid":"https://orcid.org/0000-0002-7050-7419","observed_orcids":["https://orcid.org/0000-0002-7050-7419"]},"institutions":[{"id":"https://openalex.org/I1321649718","display_name":"Royal Children's Hospital","ror":"https://ror.org/02rktxt32","country_code":"AU","type":"healthcare","lineage":["https://openalex.org/I1321649718"]},{"id":"https://openalex.org/I4210150290","display_name":"Murdoch Children's Research Institute","ror":"https://ror.org/048fyec77","country_code":"AU","type":"nonprofit","lineage":["https://openalex.org/I4210150290"]}],"countries":["AU"],"is_corresponding":false,"raw_author_name":"Andrew Davidson","raw_affiliation_strings":["Melbourne Children's Trials Centre, Murdoch Children's Research Institute, Melbourne, VIC","Royal Children's Hospital, Melbourne, VIC"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Melbourne Children's Trials Centre, Murdoch Children's Research Institute, Melbourne, VIC","institution_ids":["https://openalex.org/I4210150290"]},{"raw_affiliation_string":"Royal Children's Hospital, Melbourne, VIC","institution_ids":["https://openalex.org/I1321649718"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":10,"corresponding_author_ids":["https://openalex.org/A5064882732"],"corresponding_institution_ids":["https://openalex.org/I129604602","https://openalex.org/I4210149013"],"apc_list":{"value":3090,"currency":"USD","value_usd":3090},"apc_paid":{"value":3090,"currency":"USD","value_usd":3090},"fwci":1.4669,"has_fulltext":true,"cited_by_count":4,"citation_normalized_percentile":{"value":0.82705092,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":89,"max":97},"biblio":{"volume":"220","issue":"3","first_page":"121","last_page":"125"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T12547","display_name":"Pharmaceutical studies and practices","score":0.8442000150680542,"subfield":{"id":"https://openalex.org/subfields/2735","display_name":"Pediatrics, Perinatology and Child Health"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},"topics":[{"id":"https://openalex.org/T12547","display_name":"Pharmaceutical studies and practices","score":0.8442000150680542,"subfield":{"id":"https://openalex.org/subfields/2735","display_name":"Pediatrics, Perinatology and Child Health"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T10582","display_name":"Ethics in Clinical Research","score":0.10050000250339508,"subfield":{"id":"https://openalex.org/subfields/2739","display_name":"Public Health, Environmental and Occupational Health"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T13417","display_name":"Biomedical Ethics and Regulation","score":0.020899999886751175,"subfield":{"id":"https://openalex.org/subfields/2737","display_name":"Physiology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/pediatric-clinical-trials","display_name":"pediatric clinical trials","score":0.9909999966621399},{"id":"https://openalex.org/keywords/rare-diseases","display_name":"rare diseases","score":0.5740000009536743},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.5170000195503235},{"id":"https://openalex.org/keywords/clinical-trial-governance","display_name":"clinical trial governance","score":0.49399998784065247},{"id":"https://openalex.org/keywords/clinical-trial-infrastructure","display_name":"clinical trial infrastructure","score":0.6449999809265137},{"id":"https://openalex.org/keywords/drug-pipeline","display_name":"drug development pipeline","score":0.4269999861717224},{"id":"https://openalex.org/keywords/advanced-therapies","display_name":"advanced therapies","score":0.32199999690055847},{"id":"https://openalex.org/keywords/australia","display_name":"Australia","score":0.30399999022483826},{"id":"https://openalex.org/keywords/children-and-adolescents","display_name":"children and adolescents","score":0.4020000100135803},{"id":"https://openalex.org/keywords/pediatric-oncology","display_name":"pediatric oncology","score":0.4350000023841858}],"concepts":[{"id":"https://openalex.org/C105152847","wikidata":"https://www.wikidata.org/wiki/Q5021685","display_name":"Call to action","level":2,"score":0.7465904951095581},{"id":"https://openalex.org/C2780791683","wikidata":"https://www.wikidata.org/wiki/Q846785","display_name":"Action (physics)","level":2,"score":0.5812487602233887},{"id":"https://openalex.org/C535046627","wikidata":"https://www.wikidata.org/wiki/Q30612","display_name":"Clinical trial","level":2,"score":0.57826167345047},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.4624163806438446},{"id":"https://openalex.org/C15744967","wikidata":"https://www.wikidata.org/wiki/Q9418","display_name":"Psychology","level":0,"score":0.3448656499385834},{"id":"https://openalex.org/C144133560","wikidata":"https://www.wikidata.org/wiki/Q4830453","display_name":"Business","level":0,"score":0.12781232595443726},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.07509073615074158},{"id":"https://openalex.org/C112698675","wikidata":"https://www.wikidata.org/wiki/Q37038","display_name":"Advertising","level":1,"score":0.06531825661659241},{"id":"https://openalex.org/C121332964","wikidata":"https://www.wikidata.org/wiki/Q413","display_name":"Physics","level":0,"score":0.0},{"id":"https://openalex.org/C62520636","wikidata":"https://www.wikidata.org/wiki/Q944","display_name":"Quantum mechanics","level":1,"score":0.0}],"mesh":[{"descriptor_ui":"D000293","descriptor_name":"Adolescent","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D001315","descriptor_name":"Australia","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002648","descriptor_name":"Child","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D011613","descriptor_name":"Psychotherapy","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D015928","descriptor_name":"Cognitive Behavioral Therapy","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true}],"locations_count":2,"locations":[{"id":"doi:10.5694/mja2.52191","doi":"https://doi.org/10.5694/mja2.52191","is_oa":true,"landing_page_url":"http://dx.doi.org/10.5694/mja2.52191","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.5694/mja2.52191","source":{"id":"https://openalex.org/S13978405","display_name":"The Medical Journal of Australia","issn_l":"0025-729X","issn":["0025-729X","1326-5377"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Medical Journal of Australia","raw_type":"journal-article"},{"id":"pmid:38112125","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/38112125","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"The Medical journal of Australia","raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.5694/mja2.52191","doi":"https://doi.org/10.5694/mja2.52191","is_oa":true,"landing_page_url":"http://dx.doi.org/10.5694/mja2.52191","pdf_url":"https://onlinelibrary.wiley.com/doi/pdfdirect/10.5694/mja2.52191","source":{"id":"https://openalex.org/S13978405","display_name":"The Medical Journal of Australia","issn_l":"0025-729X","issn":["0025-729X","1326-5377"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["cwts-core","dongbi-b","doyens","jufo-1","ki-jl-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc","license_id":"https://openalex.org/licenses/cc-by-nc","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Medical Journal of Australia","raw_type":"journal-article"},"sustainable_development_goals":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":0.9789000153541565}],"study_designs":[],"awards":[],"funders":[],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W4389937583.pdf","grobid_xml":"https://content.openalex.org/works/W4389937583.grobid-xml"},"referenced_works_count":33,"referenced_works":["https://openalex.org/W1592190775","https://openalex.org/W1641086794","https://openalex.org/W1831893120","https://openalex.org/W1868158647","https://openalex.org/W1929429364","https://openalex.org/W1969795860","https://openalex.org/W1992464859","https://openalex.org/W2000742403","https://openalex.org/W2015824105","https://openalex.org/W2067658642","https://openalex.org/W2148148407","https://openalex.org/W2263201754","https://openalex.org/W2758602702","https://openalex.org/W2787126858","https://openalex.org/W2789417097","https://openalex.org/W2904154481","https://openalex.org/W2911038251","https://openalex.org/W2914127236","https://openalex.org/W2985405196","https://openalex.org/W3090005936","https://openalex.org/W3097677008","https://openalex.org/W3128948078","https://openalex.org/W3152625822","https://openalex.org/W3158991679","https://openalex.org/W3213042299","https://openalex.org/W4210362647","https://openalex.org/W4210579489","https://openalex.org/W4224310817","https://openalex.org/W4282036924","https://openalex.org/W4283276298","https://openalex.org/W4283778388","https://openalex.org/W4295066965","https://openalex.org/W6693253322"],"related_works":["https://openalex.org/W2203987668","https://openalex.org/W1879140353","https://openalex.org/W1995015483","https://openalex.org/W1972576782","https://openalex.org/W2746672978","https://openalex.org/W4408936178","https://openalex.org/W4247374954","https://openalex.org/W4295693315","https://openalex.org/W2012566304","https://openalex.org/W4389216862"],"abstract_inverted_index":{"Opportunities":[0],"for":[1,144,171,207,217,232,346,353,544,575,623,645,733,813,835,853,969,1034,1142,1159,1244,1284,1319,1454,1650,1873,1995,2052,2105,2118,2137],"children":[2,39,233,271,414,486,1512,1606,1652,1711],"to":[3,18,36,65,82,84,220,280,303,389,407,425,460,532,538,580,584,588,705,723,749,774,797,927,951,1050,1129,1140,1155,1182,1262,1281,1293,1305,1312,1352,1382,1395,1422,1477,1484,1489,1510,1663,1682,1687,1734,1825,1838,1884,1921,1956,1961,1992,2014,2030,2035,2100],"benefit":[4,1012],"from":[5,458,469,597,978,1001,1302,1370,1513,1611,1655,2098],"novel":[6,221,401,707,1490,1841,1993,2003,2036],"therapies":[7,122,196,206,222,298,307,376,708,1286,1300,1842],"have":[8,123,300,423,585,619,747,894,1166,1700,2095],"increased":[9,1242],"substantially":[10],"over":[11],"the":[12,24,107,129,174,344,395,426,438,444,472,479,506,511,518,524,566,589,598,615,640,661,741,759,780,807,820,827,844,848,858,870,887,899,904,998,1060,1079,1143,1201,1251,1264,1288,1432,1452,1471,1479,1498,1514,1528,1562,1597,1600,1603,1612,1617,1620,1628,1643,1656,1658,1691,1728,1735,1738,1743,1758,1774,1792,1795,1801,1813,1854,1858,1863,1874,1916,1923,1951,1974,1979,1999,2070,2079],"past":[13],"decade.":[14],"Change":[15],"is":[16,46,226,435,487,523,840,877,1085,1177,1186,1259,1315,1349,1392,1810,1822,1843,1890],"needed":[17,35],"maximise":[19,1383],"these":[20,243,421,653,1299,1456,1567],"opportunities,":[21],"particularly":[22,510,1104],"in":[23,55,61,92,100,106,128,202,251,267,343,385,413,481,485,660,695,779,817,838,847,878,959,983,1013,1031,1099,1131,1145,1255,1260,1287,1428,1431,1470,1497,1536,1539,1542,1547,1550,1561,1599,1653,1679,1708,1737,1780,1812,1819,1852,1857,1886,1947,1964,1988,2012,2019,2055,2102,2108],"paediatric":[25,56,86,111,195,347,429,450,530,767,798,815,824,934,1022,1068,1189,1356,1474,1632,1667,1695,1965,2015,2021,2041],"trial":[26,936,1123,1148,1267,1272,1680,1721,1937,2017],"environment.":[27],"Investment":[28],"and":[29,52,68,77,95,97,114,120,158,164,178,187,192,211,237,312,398,415,446,471,474,496,501,514,517,528,535,541,604,637,649,667,690,698,715,718,721,727,755,772,777,782,787,801,810,826,876,909,921,943,953,961,972,1039,1045,1065,1087,1092,1097,1106,1113,1120,1135,1150,1152,1196,1205,1219,1225,1230,1234,1274,1278,1344,1368,1378,1437,1449,1481,1492,1552,1558,1607,1685,1689,1710,1773,1787,1797,1831,1835,1840,1850,1860,1895,1897,1902,1915,1925,1953,1978,1998,2090,2123],"a":[30,74,79,98,261,274,449,608,634,762,832,861,896,925,930,979,995,1067,1072,1178,1294,1317,1467,1573,1777,1788,1823,1827,1985,2010,2128],"coordinated":[31,883],"national":[32,763,795,833,862,931,1424,2053],"approach":[33,796,834,1181,1280,1297,2013],"are":[34,245,377,658,946,1048,1110,1192,1228,1249,1342,1446,1881,2025,2115],"prevent":[37],"Australian":[38,402,546,1021,1864,2082],"falling":[40],"behind":[41,2046],"their":[42,282,1798],"international":[43,47,745,1499],"peers.":[44],"There":[45],"recognition":[48],"that":[49,102,290,355,362,393,465,618,744,805,1084,1187,1206,1213,1269,1419,1443,1451,1631,1712,1741,1790,1900],"collaborative":[50,751,764,996],"approaches":[51,884],"strategic":[53],"investment":[54,632,1098,2054],"clinical":[57,66,87,104,224,331,337,411,430,483,602,605,610,624,629,662,682,699,785,799,935,954,1102,1164,1190,1246,1252,1266,1271,1289,1444,1475,1518,1696,1751,1754,1804,1929,1936,1966,2016,2022,2121,2138],"trials":[58,67,105,294,386,412,431,451,484,663,683,768,786,800,837,952,1023,1069,1165,1191,1290,1314,1357,1445,1457,1752,1805,1997,2023,2122,2139],"reap":[59],"benefits":[60,1483],"terms":[62],"of":[63,110,185,242,270,366,400,428,441,448,505,526,554,556,569,614,656,692,761,766,784,822,829,860,933,964,989,1020,1062,1081,1122,1137,1200,1208,1337,1389,1399,1434,1517,1572,1591,1619,1637,1646,1660,1694,1705,1767,1776,1794,1803,1829,1847,1862,1877,1927,1935,2002,2028,2065,2069,2075,2081],"access":[64,304,537,587,968,1488,1837,1991,2034,2059],"new":[69],"therapies.":[70,403,1307],"This":[71,563,594,737,790,842,1258,1276,1347,1402,1520,1641,1783],"article":[72],"exemplifies":[73],"multi-state":[75],"collaboration":[76,897,1019,1376],"presents":[78],"united":[80],"call":[81,1809,2051],"action":[83],"prioritise":[85],"trials,":[88,470,1103,1109,1476,1930],"by":[89,141,173,235,273,380,454,490,549,675,679,1090,1169,1406,1414,1441,1486,1493,1524,1893,1945,2062],"strategically":[90],"investing":[91],"effective":[93,199,358,1030],"governance":[94,1887],"infrastructure,":[96,1210],"shift":[99,595,1469],"culture":[101,1779],"embeds":[103],"core":[108,1046,1194,1321,1371,1390,1447],"business":[109,1195],"health":[112,592,1429,1771,1781,1865,2042],"care":[113,231,603,630,955,1203,1253,1430,1772,1849,1866,2043],"academic":[115],"institutions.":[116],"Recently,":[117],"numerous":[118,364],"life-changing":[119],"life-saving":[121],"emerged,":[124],"with":[125,260,492,565,643,710,880,924,1071,1125,1221,1323,1616,1905],"many":[126,823,1744],"more":[127,1295],"drug":[130,136,1575,1584,1613,1633,1714],"development":[131,427,760,1224,1576,1614,1634,1645],"pipeline":[132],"(Box":[133,418],"1).1-4":[134],"Phenotypic":[135],"discovery":[137],"has":[138,340,1028,1058,1505,1756],"been":[139,301,342,621,1029,1167,1507],"revolutionised":[140],"advanced":[142,189,292,375,669,788,802,942],"methods":[143],"pre-clinical":[145],"screening:":[146],"disease":[147,276,550,864,985,1088],"models":[148,617],"using":[149,1674],"induced":[150],"pluripotent":[151],"stem":[152],"cell":[153],"technologies,":[154],"organoids,":[155],"gene-editing":[156],"tools":[157],"imaging":[159],"assay":[160],"technologies.5":[161],"Between":[162],"2010":[163],"2020,":[165],"440":[166],"innovative":[167,1285],"drugs":[168,1593],"were":[169,1609],"approved":[170,1306,1720],"marketing":[172],"United":[175],"States":[176],"Food":[177,513],"Drug":[179,515],"Administration,2":[180],"including":[181,296],"an":[182,351,793,1153,1197,1385,1508,1647,1703,1844,1932],"increasing":[183],"number":[184],"gene":[186,205,297,980,991,2106],"other":[188,881,1335,2047],"therapeutics.1":[190],"New":[191],"truly":[193],"transformative":[194,306,1086],"include":[197],"highly":[198,1115],"modulator":[200],"therapy":[201,216,982,992,1006,2107],"cystic":[203],"fibrosis,6":[204],"spinal":[208],"muscular":[209],"atrophy,7":[210],"chimeric":[212,1002],"antigen":[213,1003],"receptor":[214,1004],"T-cell":[215,1005],"cancer.8":[218],"Access":[219],"through":[223,948,1232],"research":[225,332,391,606,1438,1654,1768,1778],"increasingly":[227,1665],"seen":[228],"as":[229,339,627,730,867,869,886,1298,1570,1670,1672,1910,1950,1973,2067],"optimal":[230],"affected":[234,272,548],"devastating":[236],"previously":[238,620],"untreatable":[239],"disease.":[240],"Many":[241],"diseases":[244,825,1127],"rare,":[246],"affecting":[247],"fewer":[248],"than":[249],"5":[250],"10":[252],"000":[253],"people.9":[254],"About":[255],"two":[256],"million":[257],"Australians":[258,583],"live":[259,279],"rare":[262,275,863,1126],"disease,":[263,557],"most":[264,374,1238],"first":[265],"experienced":[266,1000,1138],"childhood.":[268],"One-third":[269],"will":[277,791,986],"not":[278,1345,1350,1502,1569,1626,2133],"see":[281],"fifth":[283],"birthday.10,":[284],"11":[285],"Children":[286],"managed":[287],"at":[288,639,940,1007,1016,1211,1800,2026],"hospitals":[289,816,2112],"support":[291,686,706,743,1243,1313,1369,1423],"therapeutic":[293,803,1161,1592,1745],"\u2014":[295,299,642,652],"able":[302],"potentially":[305],"years":[308],"before":[309,437],"regulatory":[310,1585,1589],"approval":[311],"subsequent":[313],"reimbursement":[314,2126],"make":[315],"them":[316],"broadly":[317],"available.":[318],"For":[319,1237],"progressive":[320],"or":[321,551,560,1173,1334,1364,1408,1440,1581,2141],"life-limiting":[322],"conditions,":[323],"this":[324,1240,2056],"can":[325,356,687,1010,1941],"mean":[326],"major":[327,507],"direct":[328,1363,2135],"benefit.":[329],"Ideally,":[330],"should":[333],"be":[334,673,1404,1459,1522,1723,1943],"embedded":[335],"into":[336,607,1461,1753,1769,2005],"practice,":[338,1036],"long":[341,1506],"case":[345],"oncology.":[348],"Oncology":[349],"provides":[350,387],"exemplar":[352],"disciplines":[354],"provide":[357,533,792,1011],"overarching":[359,1209],"treatment":[360,369,2037],"paradigms":[361,1096],"incorporate":[363],"subsets":[365],"very":[367],"targeted":[368,944],"pathways.12,":[370],"13":[371],"Additionally,":[372],"while":[373,1868],"currently":[378,436,1420],"sponsored":[379],"overseas-based":[381],"pharmaceutical":[382,680,2119],"companies,":[383],"experience":[384,778],"opportunities":[388],"develop":[390,1826],"pipelines":[392,950],"enable":[394,581,1396],"development,":[396,648],"evaluation":[397],"translation":[399,2001],"Nevertheless,":[404],"considerable":[405],"challenges":[406,422,480],"delivering":[408,482,1250],"high":[409,701,734,1215],"quality":[410,702,713,735,1216,1226,1859],"adolescents":[416,1608],"remain":[417],"2).14-17":[419],"Internationally,":[420],"led":[424,1960],"networks.18":[432],"A":[433,665,1807],"Bill":[434],"US":[439,512,1957],"House":[440],"Representatives,":[442],"promoting":[443],"establishment":[445],"maintenance":[447],"network,":[452],"supported":[453],"substantial":[455,1073,1361],"grant":[456],"funding":[457,616,1047,1311,1340,1391,1418,2097,2136],"2024":[459],"2029.19":[461],"Mismatch":[462],"between":[463,499,601,1377],"costs":[464,1453],"study":[466],"sites":[467,2117],"recover":[468],"time":[473],"resources":[475,722,1038,1044],"required:":[476],"In":[477,1308,1968,2007],"Australia,":[478,1969,2020],"further":[488,1375],"compounded":[489],"distance,":[491],"less":[493],"concentrated":[494],"populations":[495],"large":[497],"distances":[498],"capitals,":[500],"our":[502,1309],"position":[503],"outside":[504,1582],"global":[508],"regulators,":[509],"Administration":[516,1977],"European":[519,1056],"Medicines":[520],"Agency.":[521],"It":[522,1624],"responsibility":[525],"tertiary":[527],"quaternary":[529],"centres":[531,1024,1358,1380,2024,2048],"equitable":[534,586,2032],"streamlined":[536,967],"early":[539,1107,1563],"phase":[540,1108],"emerging":[542,830,1839],"treatments":[543],"every":[545],"child":[547],"disability,":[552],"regardless":[553],"rarity":[555,821],"socio-economic":[558],"status,":[559],"geographical":[561],"location.":[562],"aligns":[564],"articulated":[567],"objective":[568],"Australia's":[570,1990,2040],"National":[571,849,871,888,1759,1814],"Strategic":[572,850],"Action":[573,851],"Plan":[574,852],"Rare":[576,854,1911],"Diseases,":[577],"which":[578,856,941,1764,1820,1959],"aims":[579,1381],"all":[582,814,1188,1355],"best":[590,1035],"available":[591],"technology.20":[593],"away":[596],"traditional":[599],"separations":[600],"seamless":[609,1296],"interface":[611],"requires":[612,956,1784],"reconsideration":[613],"utilised":[622],"trials.":[625,736,993,1257,1519],"Just":[626],"excellent":[628],"involves":[631,1241],"beyond":[633],"physical":[635],"bed":[636],"staff":[638,1139,1217],"bedside":[641],"resourcing":[644,1263],"education,":[646],"professional":[647,1223],"capacity":[650,2104,2140],"building":[651,2103,2143],"same":[654,742],"pillars":[655],"sustainability":[657],"essential":[659,1198,1845,1933],"sphere.21":[664],"thriving":[666],"expanding":[668],"therapeutics":[670,945,1994,2004],"service":[671],"cannot":[672,738],"established":[674,895],"haphazard":[676],"revenue":[677],"generated":[678],"company-funded":[681],"alone.":[684],"Increased":[685],"ensure:":[688],"recruitment":[689,1218],"retention":[691,1136],"expert":[693],"personnel":[694],"both":[696,970,1996],"paediatrics":[697,839],"trials;":[700],"local":[703],"infrastructure":[704,754,1158,1268,1400],"along":[709],"workflow":[711],"processes,":[712],"assurance":[714],"control":[716],"mechanisms;":[717],"adequate":[719],"staffing":[720],"meet":[724],"site":[725,753],"performance":[726],"data":[728],"standards,":[729],"recommended":[731],"prerequisites":[732],"occur":[739],"without":[740,2009],"networks":[746,1064],"received":[748,2096],"grow":[750],"networks,":[752],"resources.22":[756],"We":[757,893,1077,1465,2050],"recommend":[758,1078,1466],"community":[765],"centres,":[769],"enabling":[770],"clinicians":[771],"researchers":[773],"exchange":[775],"excellence":[776],"strategy":[781,866],"implementation":[783,988],"therapeutics.":[789],"efficient,":[794],"delivery":[804,1080,2018],"potentiates":[806],"health,":[808],"economic":[809],"social":[811],"impacts":[812],"Australia.":[818,1353,2109],"Given":[819],"complexity":[828],"therapies,":[831,1491],"complex":[836,1105,1147,1160,1256,1928],"imperative.":[841],"compliments":[843],"priorities":[845],"identified":[846],"Diseases,20":[855],"promotes":[857],"importance":[859,1061,1924],"workforce":[865],"well":[868,1671],"Clinical":[872,1760],"Trials":[873,1761],"Governance":[874,1762],"Framework21":[875],"line":[879],"nationally":[882],"such":[885,1063,1909,1949,1972],"Health":[889,1815],"Genomics":[890,1816],"Policy":[891,1817],"Framework.23":[892],"across":[898,1426],"Sydney":[900,2076,2091],"Children's":[901,906,911,916,919,2087,2092],"Hospitals":[902,2093],"Network,":[903],"Royal":[905],"Hospital":[907],"Melbourne":[908],"Murdoch":[910,2086],"Research":[912,2088],"Institute":[913,2089],"campus,":[914],"Monash":[915],"Hospital,":[917,920],"Queensland":[918],"university":[922],"partners,":[923],"view":[926],"eventually":[928],"creating":[929],"network":[932,1070],"centres.":[937],"The":[938,975,1055,1118,2063,2073,2085],"rate":[939],"moving":[947],"scientific":[949,1692],"rapid":[957,2000],"enhancements":[958],"knowledge":[960],"skillset.":[962],"Mapping":[963],"expertise":[965],"allows":[966],"industry":[971],"referring":[973],"clinicians.":[974,1878],"lessons":[976],"learnt":[977],"replacement":[981],"neuromuscular":[984],"inform":[987],"future":[990],"With":[994],"approach,":[997],"complications":[999,1015],"one":[1008],"centre":[1009,1325,1802],"managing":[1014],"another.":[1017],"Our":[1018,1184],"remains":[1025,1931],"informal":[1026],"but":[1027,1578,2131],"sharing":[1032],"ideas":[1033],"written":[1037],"educational":[1040],"material;":[1041],"however,":[1042,1566],"greater":[1043],"required":[1049,1254,1394],"achieve":[1051],"its":[1052,1726],"full":[1053],"potential.":[1054],"Union":[1057],"recognised":[1059],"funded":[1066,1168],"Horizon":[1074],"2020":[1075],"grant.24":[1076],"precision":[1082],"medicine":[1083],"specific,":[1089],"combining":[1091],"developing":[1093],"world":[1094,1598],"class":[1095],"infrastructure.":[1100,1183],"Paediatric":[1101],"resource":[1111,1133,1282],"intensive":[1112],"require":[1114],"skilled":[1116],"personnel.":[1117],"ebb":[1119],"flow":[1121],"activity":[1124,1448],"leads":[1128],"difficulty":[1130],"efficient":[1132,1397,1836,2033],"allocation":[1134],"cater":[1141],"complexities":[1144],"consent,":[1146],"protocols,":[1149],"engagement,15":[1151],"inability":[1154],"maintain":[1156,1214],"stable":[1157],"interventions.17":[1162],"Historically,":[1163],"specific":[1170],"investigator":[1171],"grants":[1172],"industry.":[1174],"What":[1175],"follows":[1176],"stop\u2013start,":[1179],"silo":[1180],"recommendation":[1185],"considered":[1193,1468],"part":[1199,1571,1636,2068],"patient":[1202,1796,1906],"journey,":[1204],"costings":[1207],"levels":[1212],"retention,":[1220],"ongoing":[1222,1386,1901],"improvement,":[1227],"quantified":[1229],"allocated":[1231],"sustainable":[1233],"prospective":[1235],"funding.":[1236,1338],"disciplines,":[1239],"multidisciplinary":[1245],"teams":[1247],"who":[1248],"addition":[1261],"fit-for-purpose":[1265],"streamlines":[1270],"set-up":[1273],"conduct.":[1275],"deliberate":[1277],"proactive":[1279],"mapping":[1283],"space":[1291],"contributes":[1292],"transition":[1301],"investigational":[1303],"products":[1304],"experience,":[1310],"rarely":[1316],"priority":[1318,1824],"hospital":[1320,1372],"funding,":[1322,1333,1367],"each":[1324],"relying":[1326],"heavily":[1327],"on":[1328,1360,1725,1749,2127],"philanthropy,":[1329],"program-specific":[1330],"state":[1331,1407],"government":[1332,1366,1410],"sources":[1336,1341],"These":[1339,1698],"intermittent":[1343],"guaranteed.":[1346],"problem":[1348],"unique":[1351],"Globally,":[1354],"rely":[1359],"philanthropic,":[1362],"indirect":[1365],"funds.":[1373],"While":[1374,1587,1716],"within":[1379],"efficiency,":[1384],"secure":[1387],"source":[1388],"ultimately":[1393],"use":[1398],"resources.":[1401],"could":[1403],"provided":[1405],"federal":[1409,1970],"directly":[1411],"(for":[1412],"example,":[1413],"utilising":[1415],"Commonwealth":[1416],"block":[1417],"exists":[1421],"standards":[1425],"innovation":[1427],"form":[1433],"teaching,":[1435],"training,":[1436],"funding)":[1439],"acknowledging":[1442],"accepting":[1450],"running":[1455],"must":[1458,1722],"incorporated":[1460],"hospitals\u2019":[1462],"operational":[1463],"budgets.":[1464],"conversation":[1472],"surrounding":[1473],"emphasise":[1478],"tangible":[1480],"immediate":[1482],"patients":[1485],"allowing":[1487],"Australia":[1494,1913],"remaining":[1495],"competitive":[1496],"arena.":[1500],"Although":[1501],"universal,":[1503],"there":[1504,1821],"impulse":[1509],"protect":[1511],"experimental":[1515],"nature":[1516],"may":[1521],"driven":[1523],"tragedies":[1525],"reported":[1526],"throughout":[1527],"20th":[1529,1739],"century":[1530,1740],"impacting":[1531],"young":[1532],"children:":[1533],"elixir":[1534],"sulfanilamide":[1535],"1937;25":[1537],"sulfisoxazole":[1538],"1956;26":[1540],"chloramphenicol":[1541],"1960;27":[1543],"synthetic":[1544],"vitamin":[1545],"K":[1546],"1961;28":[1548],"thalidomide":[1549,1604],"1962;29":[1551],"excipients":[1553],"benzyl":[1554],"alcohol,30":[1555],"propylene":[1556],"glycol31":[1557],"polysorbate":[1559],"8032":[1560],"1980s.":[1564],"Crucially,":[1565],"occurred":[1568],"regulated":[1574],"program,":[1577],"rather":[1579],"before,":[1580],"of,":[1583],"oversight.33":[1586],"robust":[1588],"oversight":[1590],"was":[1594,1625],"introduced":[1595],"around":[1596],"1960s":[1601],"following":[1602],"tragedy,13":[1605],"excluded":[1610],"process,":[1615],"coining":[1618],"term":[1621],"\u201ctherapeutic":[1622],"orphans\u201d.34":[1623],"until":[1627],"late":[1629],"1990s":[1630],"became":[1635],"standard":[1638],"care.33,":[1639],"35":[1640,1747],"followed":[1642],"necessary":[1644],"ethical":[1648],"framework":[1649],"involving":[1651],"1970s;36":[1657],"introduction":[1659],"pharmacometric":[1661],"techniques":[1662],"allow":[1664],"accurate":[1666],"dose":[1668],"prediction,":[1669],"confirmation":[1673],"limited":[1675],"blood":[1676],"samples;37":[1677],"advances":[1678],"design":[1681],"minimise":[1683],"risk":[1684,2027],"burden":[1686],"children;38":[1688],"crucially,":[1690],"revolution":[1693],"pharmacology.33":[1697],"developments":[1699],"mapped":[1701],"out":[1702],"understanding":[1704],"maturational":[1706],"processes":[1707],"infants":[1709],"impact":[1713],"handling.":[1715],"any":[1717],"given":[1718],"ethically":[1719],"judged":[1724],"merit,":[1727],"current":[1729],"landscape":[1730],"bears":[1731],"no":[1732],"relationship":[1733],"environment":[1736],"saw":[1742],"tragedies.28,":[1746],"Emphasis":[1748],"embedding":[1750],"practice":[1755],"informed":[1757,1892],"Framework,":[1763,1818],"recommends":[1765],"integration":[1766],"routine":[1770],"fostering":[1775],"organisations.21":[1782],"forward":[1785],"planning":[1786],"realignment":[1789],"refocuses":[1791],"needs":[1793],"family":[1799],"planning.":[1806,1938],"similar":[1808],"made":[1811],"model":[1828],"person-":[1830],"family-centred":[1832],"care.23":[1833],"Equitable":[1834],"component":[1846],"patient-centred":[1848],"aids":[1851],"maintaining":[1853],"family's":[1855],"trust":[1856],"competitiveness":[1861],"system":[1867,2044],"facilitating":[1869,1989],"cutting":[1870],"edge":[1871],"training":[1872],"next":[1875],"generation":[1876],"Cultural":[1879],"changes":[1880,1946,1955],"tangibly":[1882],"linked":[1883],"shifts":[1885],"priorities.":[1888],"Culture":[1889,1939],"also":[1891,1942],"experiences":[1894],"dialogue,":[1896],"we":[1898],"believe":[1899],"formalised":[1903],"partnerships":[1904],"advocacy":[1907],"groups,":[1908],"Voices":[1912],"(https://rarevoices.org.au)":[1914],"Childhood":[1917],"Dementia":[1918],"Initiative":[1919],"(https://www.childhooddementia.org),":[1920],"promote":[1922],"urgency":[1926],"aspect":[1934],"change":[1940,2011],"triggered":[1944],"legislation,":[1948],"2002":[1952],"2003":[1954],"law,":[1958],"widespread":[1962],"increase":[1963],"trials.33":[1967],"agencies":[1971],"Therapeutic":[1975],"Goods":[1976],"Pharmaceutical":[1980],"Benefits":[1981],"Advisory":[1982],"Committee":[1983],"play":[1984],"key":[1986],"role":[1987],"practice.":[2006],"conclusion,":[2008],"failing":[2029],"deliver":[2031],"options,":[2038],"leaving":[2039],"trailing":[2045],"internationally.":[2049],"space.":[2057],"Open":[2058],"publishing":[2060],"facilitated":[2061],"University":[2064,2074,2083],"Sydney,":[2066],"Wiley":[2071],"-":[2072],"agreement":[2077],"via":[2078],"Council":[2080],"Librarians.":[2084],"Network":[2094],"Roche":[2099],"aid":[2101],"All":[2110],"authors\u2019":[2111],"and/or":[2113],"institutions":[2114],"active":[2116],"company-sponsored":[2120],"receive":[2124,2134],"enrolment-based":[2125],"trial-by-trial":[2129],"basis,":[2130],"do":[2132],"capability":[2142],"unless":[2144],"stated":[2145],"above.":[2146],"Not":[2147],"commissioned;":[2148],"externally":[2149],"peer":[2150],"reviewed.":[2151]},"counts_by_year":[{"year":2026,"cited_by_count":2},{"year":2025,"cited_by_count":1},{"year":2024,"cited_by_count":1}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2023-12-20T00:00:00"}
