{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T09:07:03Z","timestamp":1790154423501,"version":"4.0.1"},"reference-count":36,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2024,3,31]],"date-time":"2024-03-31T00:00:00Z","timestamp":1711843200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004331","name":"Johnson and Johnson","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004331","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pharmacoepidemiology and Drug"],"published-print":{"date-parts":[[2024,4]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Purpose<\/jats:title>\n                    <jats:p>In rare diseases, real\u2010world evidence (RWE) generation is often restricted due to small patient numbers and global geographic distribution. A federated data network (FDN) approach brings together multiple data sources harmonized for collaboration to increase the power of observational research. In this paper, we review how to increase reproducibility and transparency of RWE studies in rare diseases through disease\u2010specific FDNs.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Method<\/jats:title>\n                    <jats:p>To be successful, a multiple stakeholder scientific FDN collaboration requires a strong governance model in place. In such a model, each database owner remains in full control regarding the use of and access to patient\u2010level data and is responsible for data privacy, ethical, and legal compliance. Provided that all this is well documented and good database descriptions are in place, such a governance model results in increased transparency, while reproducibility is achieved through data curation and harmonization, and distributed analytical methods.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Leveraging the OHDSI community set of methods and tools, two rare disease\u2010specific FDNs are discussed in more detail. For multiple myeloma, HONEUR\u2014the Haematology Outcomes Network in Europe\u2014has built a strong community among the data partners dedicated to scientific exchange and research. To advance scientific knowledge in pulmonary hypertension (PH) an FDN, called PHederation, was established to form a partnership of research institutions with PH databases coming from diverse origins.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/pds.5778","type":"journal-article","created":{"date-parts":[[2024,4,1]],"date-time":"2024-04-01T01:57:02Z","timestamp":1711936622000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Increase transparency and reproducibility of\n                    <scp>real\u2010world<\/scp>\n                    evidence in rare diseases through disease\u2010specific Federated Data Networks"],"prefix":"10.1002","volume":"33","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-9021-0387","authenticated-orcid":false,"given":"Valerie","family":"van Baalen","sequence":"first","affiliation":[{"name":"Global Epidemiology Office of the Chief Medical Officer Johnson &amp; Johnson  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eva\u2010Maria","family":"Didden","sequence":"additional","affiliation":[{"name":"Global Epidemiology Office of the Chief Medical Officer Johnson &amp; Johnson  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Rosenberg","sequence":"additional","affiliation":[{"name":"Global Epidemiology Office of the Chief Medical Officer Johnson &amp; Johnson  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristina","family":"Bardenheuer","sequence":"additional","affiliation":[{"name":"Health Economics, Market Access and Reimbursement EMEA Real\u2010World Evidence and Value\u2010based Health Care Johnson &amp; Johnson  Neuss Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michel","family":"van Speybroeck","sequence":"additional","affiliation":[{"name":"Data Science IT EMEA Johnson &amp; Johnson Technology  Beerse Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monika","family":"Brand","sequence":"additional","affiliation":[{"name":"Global Epidemiology Office of the Chief Medical Officer Johnson &amp; Johnson  Basel Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,3,31]]},"reference":[{"key":"e_1_2_9_2_1","unstructured":"About Rare Diseases.Orphanet.2012https:\/\/www.orpha.net\/consor\/cgi-bin\/Education_AboutRareDiseases.php?lng=EN"},{"key":"e_1_2_9_3_1","unstructured":"FDA.Rare Diseases: Natural History Studies for Drug Development.2019."},{"key":"e_1_2_9_4_1","unstructured":"ENCePP.The European network of centres for pharmacoepidemiology and pharmacovigilance. Guide on Methodological Standards in Pharmacoepidemiology (Revision 11).EMA\/95098\/2010; 2010."},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/humu.22078"},{"key":"e_1_2_9_6_1","unstructured":"EUPAS4523.Disease characteristics and outcomes of pulmonary arterial hypertension in children and adolescents in real\u2010world clinical settings: systematic review of prospective observational registries.https:\/\/catalogues.ema.europa.eu\/node\/1697\/administrative-details"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2021.11.1357"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1145\/4229.4233"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.3389\/fpubh.2021.712569"},{"key":"e_1_2_9_10_1","unstructured":"Sentinel Distributed Database.https:\/\/www.sentinelinitiative.org\/about\/key-database-statistics"},{"key":"e_1_2_9_11_1","unstructured":"EHDEN.The European Health Data & Evidence Network.https:\/\/www.ehden.eu\/"},{"key":"e_1_2_9_12_1","unstructured":"Data Analysis and Real World Interrogation Network (DARWIN EU\u00ae).https:\/\/darwin-eu.org\/"},{"key":"e_1_2_9_13_1","unstructured":"Swiss Personalized Health Network (SPHN).https:\/\/sphn.ch\/"},{"key":"e_1_2_9_14_1","unstructured":"ATHENA (Augmenting THerapeutic Effectiveness through Novel Analytics).https:\/\/portal.athenafederation.org\/"},{"key":"e_1_2_9_15_1","unstructured":"EURACAN the European Reference Network for all rare adult solid cancers.https:\/\/euracan.eu\/"},{"key":"e_1_2_9_16_1","unstructured":"The OPTIMA consortium.https:\/\/www.optima-oncology.eu\/"},{"key":"e_1_2_9_17_1","unstructured":"BioGrid Australia Ltd.https:\/\/www.biogrid.org.au\/"},{"key":"e_1_2_9_18_1","unstructured":"Health care systems research network.http:\/\/www.hcsrn.org\/"},{"key":"e_1_2_9_19_1","unstructured":"Haematology outcomes network in Europe.https:\/\/portal.honeur.org\/"},{"key":"e_1_2_9_20_1","unstructured":"Pulmonary hypertension federated data network.https:\/\/portal.phederation.org\/"},{"key":"e_1_2_9_21_1","unstructured":"TriNetX international hospital EHR network.https:\/\/trinetx.com\/"},{"key":"e_1_2_9_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ijmedinf.2023.105322"},{"key":"e_1_2_9_23_1","unstructured":"OHDSI Observational Health Data Sciences and Informatics.https:\/\/www.ohdsi.org\/"},{"key":"e_1_2_9_24_1","doi-asserted-by":"publisher","DOI":"10.1155\/2017\/8327980"},{"key":"e_1_2_9_25_1","unstructured":"Sentinel common data model.https:\/\/www.sentinelinitiative.org\/methods-data-tools\/sentinel-common-data-model"},{"key":"e_1_2_9_26_1","unstructured":"OHDSI Observational Health Data Sciences and Informatics. Standardized Data: The OMOP Common Data Model.https:\/\/www.ohdsi.org\/data-standardization\/"},{"key":"e_1_2_9_27_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40273\u2010020\u201000981\u20109"},{"key":"e_1_2_9_28_1","unstructured":"ATLAS cohort builder.https:\/\/github.com\/OHDSI\/Atlas"},{"key":"e_1_2_9_29_1","unstructured":"OHDSI Workgroups.https:\/\/www.ohdsi.org\/workgroups\/"},{"key":"e_1_2_9_30_1","unstructured":"Handbook for PH registries to OMOP CDM conversion. GitHub.https:\/\/github.com\/OHDSI\/ETL-PulmonaryHypertensionRegistries"},{"key":"e_1_2_9_31_1","doi-asserted-by":"publisher","DOI":"10.1002\/cpt.2466"},{"key":"e_1_2_9_32_1","doi-asserted-by":"publisher","DOI":"10.1097\/01.HS9.0000563960.92802.d6"},{"key":"e_1_2_9_33_1","volume-title":"Patinet Similarity Analysis and Visualizaion in Multiple Myeloma","author":"Joane J","year":"2020"},{"key":"e_1_2_9_34_1","volume-title":"P944: Real\u2010World Patient Characteristics and Survival Outcomes of Lenalidomide Refractory Vs. Lenalidomide Exposed RRMM Patients in the HONEUR Federated Data Network","author":"Spicka I","year":"2023"},{"key":"e_1_2_9_35_1","unstructured":"Daratumumab with bortezomib and dexamethasone for previously treated multiple myeloma. Technology appraisal guidance. Reference number: TA897.2023https:\/\/www.nice.org.uk\/guidance\/ta897"},{"key":"e_1_2_9_36_1","doi-asserted-by":"publisher","DOI":"10.1183\/13993003.00879\u20102022"},{"key":"e_1_2_9_37_1","doi-asserted-by":"publisher","DOI":"10.1177\/09622802211060518"}],"container-title":["Pharmacoepidemiology and Drug Safety"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pds.5778","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,10,2]],"date-time":"2024-10-02T04:36:20Z","timestamp":1727843780000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pds.5778"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,3,31]]},"references-count":36,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2024,4]]}},"alternative-id":["10.1002\/pds.5778"],"URL":"https:\/\/doi.org\/10.1002\/pds.5778","archive":["Portico"],"relation":{},"ISSN":["1053-8569","1099-1557"],"issn-type":[{"value":"1053-8569","type":"print"},{"value":"1099-1557","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,3,31]]},"assertion":[{"value":"2023-11-30","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-03-04","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-03-31","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e5778"}}