{"id":"https://openalex.org/W7225797968","doi":"https://doi.org/10.21203/rs.3.rs-10777360/v1","title":"R&D imbalance in rare\u2011disease medicines in China: A cross\u2011sectional analysis of clinical trial registrations and coverage by the national rare\u2011disease catalogue","display_name":"R&D imbalance in rare\u2011disease medicines in China: A cross\u2011sectional analysis of clinical trial registrations and coverage by the national rare\u2011disease catalogue","publication_year":2026,"publication_date":"2026-11-11","ids":{"openalex":"https://openalex.org/W7225797968","doi":"https://doi.org/10.21203/rs.3.rs-10777360/v1"},"language":"en","primary_location":{"id":"doi:10.21203/rs.3.rs-10777360/v1","doi":"https://doi.org/10.21203/rs.3.rs-10777360/v1","is_oa":true,"landing_page_url":"https://doi.org/10.21203/rs.3.rs-10777360/v1","pdf_url":null,"source":{"id":"https://openalex.org/S4306525896","display_name":"Research Square","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Research Square","raw_type":"posted-content"},"volume":null,"type":"preprint","is_traditional":true,"indexed_in":["crossref"],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"https://doi.org/10.21203/rs.3.rs-10777360/v1","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5161427261","display_name":"Ying Zhao","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210091236","display_name":"Jinan Maternity And Care Hospital","ror":"https://ror.org/00d2wxy31","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210091236"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Ying Zhao","raw_affiliation_strings":["Jinan Maternity And Care Hospital"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Jinan Maternity And Care Hospital","institution_ids":["https://openalex.org/I4210091236"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5161486955","display_name":"Feiyan Duan","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210163399","display_name":"Shandong First Medical University","ror":"https://ror.org/05jb9pq57","country_code":"CN","type":"education","lineage":["https://openalex.org/I4210163399"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Feiyan Duan","raw_affiliation_strings":["Shandong First Medical University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Shandong First Medical University","institution_ids":["https://openalex.org/I4210163399"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5101983593","display_name":"Yuge Li","orcid":"https://orcid.org/0000-0003-2388-7655","observed_orcids":["https://orcid.org/0000-0003-2388-7655"]},"institutions":[{"id":"https://openalex.org/I4210163399","display_name":"Shandong First Medical University","ror":"https://ror.org/05jb9pq57","country_code":"CN","type":"education","lineage":["https://openalex.org/I4210163399"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Yuge Li","raw_affiliation_strings":["Shandong First Medical University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Shandong First Medical University","institution_ids":["https://openalex.org/I4210163399"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5115562347","display_name":"Jialin Mu","orcid":"https://orcid.org/0000-0002-0072-0249","observed_orcids":["https://orcid.org/0000-0002-0072-0249"]},"institutions":[{"id":"https://openalex.org/I4210091236","display_name":"Jinan Maternity And Care Hospital","ror":"https://ror.org/00d2wxy31","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210091236"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Jialin Mu","raw_affiliation_strings":["Jinan Maternity And Care Hospital"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Jinan Maternity And Care Hospital","institution_ids":["https://openalex.org/I4210091236"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5151634499","display_name":"Jinhan Huo","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I192578869","display_name":"Chengde Medical University","ror":"https://ror.org/02bzkv281","country_code":"CN","type":"education","lineage":["https://openalex.org/I192578869"]}],"countries":["CN"],"is_corresponding":false,"raw_author_name":"Jinhan Huo","raw_affiliation_strings":["Chengde Medical University"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Chengde Medical University","institution_ids":["https://openalex.org/I192578869"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5041616363","display_name":"Hui Zou","orcid":"https://orcid.org/0000-0002-5336-8656","observed_orcids":["https://orcid.org/0000-0002-5336-8656"]},"institutions":[{"id":"https://openalex.org/I4210091236","display_name":"Jinan Maternity And Care Hospital","ror":"https://ror.org/00d2wxy31","country_code":"CN","type":"healthcare","lineage":["https://openalex.org/I4210091236"]}],"countries":["CN"],"is_corresponding":true,"raw_author_name":"Hui Zou","raw_affiliation_strings":["Jinan Maternity And Care Hospital"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Jinan Maternity And Care Hospital","institution_ids":["https://openalex.org/I4210091236"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":3,"corresponding_author_ids":["https://openalex.org/A5041616363"],"corresponding_institution_ids":["https://openalex.org/I4210091236"],"apc_list":null,"apc_paid":null,"fwci":null,"has_fulltext":false,"cited_by_count":0,"citation_normalized_percentile":null,"cited_by_percentile_year":null,"biblio":{"volume":null,"issue":null,"first_page":null,"last_page":null},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11792","display_name":"Pharmaceutical Economics and Policy","score":0.5746999979019165,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},"topics":[{"id":"https://openalex.org/T11792","display_name":"Pharmaceutical Economics and Policy","score":0.5746999979019165,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},{"id":"https://openalex.org/T11649","display_name":"Pharmaceutical industry and healthcare","score":0.18629999458789825,"subfield":{"id":"https://openalex.org/subfields/2910","display_name":"Issues, ethics and legal aspects"},"field":{"id":"https://openalex.org/fields/29","display_name":"Nursing"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T11235","display_name":"Statistical Methods in Clinical Trials","score":0.121799997985363,"subfield":{"id":"https://openalex.org/subfields/2613","display_name":"Statistics and Probability"},"field":{"id":"https://openalex.org/fields/26","display_name":"Mathematics"},"domain":{"id":"https://openalex.org/domains/3","display_name":"Physical Sciences"}}],"keywords":[],"concepts":[{"id":"https://openalex.org/C535046627","wikidata":"https://www.wikidata.org/wiki/Q30612","display_name":"Clinical trial","level":2,"score":0.8553000092506409},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.8216000199317932},{"id":"https://openalex.org/C512399662","wikidata":"https://www.wikidata.org/wiki/Q3505712","display_name":"Family medicine","level":1,"score":0.5220999717712402},{"id":"https://openalex.org/C204787440","wikidata":"https://www.wikidata.org/wiki/Q188504","display_name":"Alternative medicine","level":2,"score":0.49239999055862427},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.4805999994277954},{"id":"https://openalex.org/C2777267654","wikidata":"https://www.wikidata.org/wiki/Q3519023","display_name":"Test (biology)","level":2,"score":0.4278999865055084},{"id":"https://openalex.org/C2992672634","wikidata":"https://www.wikidata.org/wiki/Q30612","display_name":"Drug trial","level":3,"score":0.4041000008583069},{"id":"https://openalex.org/C39896193","wikidata":"https://www.wikidata.org/wiki/Q380344","display_name":"Descriptive statistics","level":2,"score":0.3952000141143799},{"id":"https://openalex.org/C19527891","wikidata":"https://www.wikidata.org/wiki/Q1120908","display_name":"Medical physics","level":1,"score":0.33649998903274536},{"id":"https://openalex.org/C2778653282","wikidata":"https://www.wikidata.org/wiki/Q60752967","display_name":"Trial registration","level":3,"score":0.3224000036716461},{"id":"https://openalex.org/C206345919","wikidata":"https://www.wikidata.org/wiki/Q20380951","display_name":"Resource (disambiguation)","level":2,"score":0.2922999858856201},{"id":"https://openalex.org/C195910791","wikidata":"https://www.wikidata.org/wiki/Q1324077","display_name":"Medical record","level":2,"score":0.2897999882698059},{"id":"https://openalex.org/C2779473830","wikidata":"https://www.wikidata.org/wiki/Q1540899","display_name":"MEDLINE","level":2,"score":0.28859999775886536},{"id":"https://openalex.org/C3018146709","wikidata":"https://www.wikidata.org/wiki/Q974135","display_name":"Anticancer drug","level":3,"score":0.2870999872684479},{"id":"https://openalex.org/C502991105","wikidata":"https://www.wikidata.org/wiki/Q5133849","display_name":"Clinical research","level":2,"score":0.2825999855995178},{"id":"https://openalex.org/C2992293740","wikidata":"https://www.wikidata.org/wiki/Q4781752","display_name":"Drug approval","level":3,"score":0.2822999954223633},{"id":"https://openalex.org/C534262118","wikidata":"https://www.wikidata.org/wiki/Q177719","display_name":"Medical diagnosis","level":2,"score":0.271699994802475},{"id":"https://openalex.org/C1862650","wikidata":"https://www.wikidata.org/wiki/Q186005","display_name":"Physical therapy","level":1,"score":0.26930001378059387},{"id":"https://openalex.org/C187212893","wikidata":"https://www.wikidata.org/wiki/Q123028","display_name":"Pediatrics","level":1,"score":0.2687999904155731},{"id":"https://openalex.org/C177713679","wikidata":"https://www.wikidata.org/wiki/Q679690","display_name":"Intensive care medicine","level":1,"score":0.25200000405311584},{"id":"https://openalex.org/C199475168","wikidata":"https://www.wikidata.org/wiki/Q1539689","display_name":"MedDRA","level":4,"score":0.25099998712539673}],"mesh":[],"locations_count":1,"locations":[{"id":"doi:10.21203/rs.3.rs-10777360/v1","doi":"https://doi.org/10.21203/rs.3.rs-10777360/v1","is_oa":true,"landing_page_url":"https://doi.org/10.21203/rs.3.rs-10777360/v1","pdf_url":null,"source":{"id":"https://openalex.org/S4306525896","display_name":"Research Square","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Research Square","raw_type":"posted-content"}],"best_oa_location":{"id":"doi:10.21203/rs.3.rs-10777360/v1","doi":"https://doi.org/10.21203/rs.3.rs-10777360/v1","is_oa":true,"landing_page_url":"https://doi.org/10.21203/rs.3.rs-10777360/v1","pdf_url":null,"source":{"id":"https://openalex.org/S4306525896","display_name":"Research Square","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Research Square","raw_type":"posted-content"},"sustainable_development_goals":[],"study_designs":[],"awards":[],"funders":[],"has_content":{"pdf":false,"grobid_xml":false},"content_urls":null,"referenced_works_count":4,"referenced_works":["https://openalex.org/W4288723778","https://openalex.org/W4381839562","https://openalex.org/W4389682172","https://openalex.org/W7163572461"],"related_works":[],"abstract_inverted_index":{"Abstract":[0],"Objective":[1],"From":[2],"the":[3,13,25,33,54,80,85,217,229,234,268],"perspective":[4],"of":[5,16,29,36,79,142,157,187,201,299,306,328],"R&D":[6,44,319,336,342,364],"resource":[7],"equity,":[8],"this":[9],"study":[10],"systematically":[11],"describes":[12],"registration":[14,111],"landscape":[15],"clinical":[17,26,100,286,308],"trials":[18,101,146,158,255,287,309],"for":[19,136,176,181,310,362],"rare\u2011disease":[20,311,363],"drugs":[21,174,312],"in":[22,32,84,162,166,216,228,264,313,354,366],"China,":[23],"evaluates":[24],"development":[27],"coverage":[28,224],"diseases":[30,193,215,282,294,333],"included":[31],"two":[34,269],"batches":[35],"China\u2019s":[37],"Rare":[38,87],"Disease":[39,88],"Catalogue,":[40],"and":[41,46,61,76,96,119,130,178,210,292,369],"identifies":[42],"major":[43],"gaps":[45],"structural":[47],"problems.":[48],"Methods":[49],"We":[50],"retrieved":[51],"data":[52],"from":[53,160],"National":[55],"Medical":[56],"Drug":[57],"Clinical":[58],"Trial":[59],"Registration":[60],"Information":[62,108],"Disclosure":[63],"Platform":[64],"up":[65,297],"to":[66,105,164,199],"15":[67],"August":[68],"2026.":[69],"Search":[70],"terms":[71],"comprised":[72],"official":[73],"standard":[74],"names":[75],"common":[77],"aliases":[78],"207":[81],"disorders":[82],"listed":[83],"two\u2011batch":[86],"Catalogue.":[89],"Two":[90],"independent":[91],"trained":[92],"investigators":[93],"performed":[94],"screening":[95],"verification.":[97],"Drug\u2011based":[98],"interventional":[99],"were":[102,134,147],"enrolled":[103],"according":[104],"pre\u2011defined":[106],"criteria.":[107],"including":[109],"trial":[110,113,195,223],"number,":[112],"phase,":[114],"drug":[115],"category,":[116],"sponsor,":[117],"indication,":[118],"first":[120,230],"public":[121],"release":[122],"date":[123],"was":[124,236],"extracted.":[125],"Descriptive":[126],"statistics,":[127],"chi\u2011square":[128],"test":[129,133],"Fisher\u2019s":[131],"exact":[132],"applied":[135],"between\u2011group":[137],"comparisons.":[138],"Results":[139],"A":[140],"total":[141,202],"1":[143],"256":[144],"eligible":[145],"included,":[148],"covering":[149],"107":[150],"(51.7%)":[151],"catalogue\u2011listed":[152,281],"diseases.":[153],"The":[154,190,304],"annual":[155],"number":[156,327],"rose":[159],"6":[161],"2013":[163],"192":[165],"2025,":[167],"with":[168,259,334],"marked":[169],"acceleration":[170],"after":[171],"2018.":[172],"Chemical":[173],"accounted":[175],"54.5%":[177],"biological":[179],"products":[180],"44.8%;":[182],"domestic":[183],"enterprises":[184],"sponsored":[185],"68.2%":[186],"all":[188],"trials.":[189],"top":[191],"10":[192],"by":[194],"volume":[196,305],"cumulatively":[197],"contributed":[198],"53.4%":[200],"trials,":[203],"among":[204],"which":[205,338],"haemophilia,":[206],"idiopathic":[207],"pulmonary":[208],"fibrosis":[209],"melanoma":[211],"ranked":[212],"highest.":[213],"Numerically,":[214],"second":[218],"catalogue":[219],"batch":[220,231],"showed":[221],"higher":[222],"(58.1%)":[225],"than":[226,256],"those":[227],"(47.1%),":[232],"though":[233],"difference":[235,263],"not":[237],"statistically":[238,261],"significant":[239,262],"(\u03c7\u00b2":[240,271],"=":[241,244,247,272,275],"2.45,":[242],"df":[243,274],"1,":[245,276],"P":[246,277],"0.12).":[248],"Domestic":[249],"sponsors":[250],"conducted":[251],"significantly":[252],"more":[253],"early\u2011phase":[254],"foreign":[257],"sponsors,":[258],"a":[260,325],"phase":[265],"distribution":[266],"between":[267],"groups":[270],"241.25,":[273],"<":[278],"0.001).":[279],"One\u2011hundred":[280],"had":[283],"no":[284,335],"registered":[285,307],"at":[288],"all;":[289],"inherited":[290],"metabolic":[291],"endocrine":[293],"together":[295],"made":[296],"42%":[298],"these":[300],"uncovered":[301],"conditions.":[302],"Conclusions":[303],"China":[314],"has":[315],"increased":[316],"substantially.":[317],"Nevertheless,":[318],"resources":[320],"are":[321],"heavily":[322],"concentrated":[323],"on":[324],"small":[326],"diseases,":[329],"leaving":[330],"numerous":[331],"rare":[332],"activity,":[337],"indicates":[339],"prominent":[340],"inter\u2011disease":[341],"imbalance.":[343],"Policy":[344],"incentives":[345],"should":[346],"be":[347],"further":[348],"tilted":[349],"toward":[350],"unmet":[351],"medical":[352],"needs":[353],"under\u2011explored":[355],"areas.":[356],"Our":[357],"findings":[358],"may":[359],"provide":[360],"references":[361],"governance":[365],"other":[367],"low\u2011":[368],"middle\u2011income":[370],"countries":[371],"worldwide.":[372]},"counts_by_year":[],"updated_date":"2026-11-12T12:28:57.059296","created_date":"2026-11-12T00:00:00"}