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These challenges can result in long diagnostic journeys and inadequate care. Over 30\u00a0years ago, the Rare Disease Registries for Gaucher, Fabry, Mucopolysaccharidosis type I and Pompe diseases were established to address knowledge gaps in disease natural history, clinical manifestations of disease and treatment outcomes. Evidence generated from the real-world data collected in these registries supports multiple stakeholders, including patients, healthcare providers, drug developers, researchers and regulators. To maximise the impact of real-world evidence from these registries, engagement and collaboration with the patient communities is essential. To this end, the Rare Disease Registries Patient Council was established in 2019 as a partnership between the Rare Disease Registries and global and local patient advocacy groups to share perspectives on how registry data are used and disseminated. The Patient Council has resulted in a number of patient initiatives including patient representation at Rare Disease Registries advisory boards; development of plain language summaries of registry publications to increase availability of real-world evidence to patient communities; and implementation of digital innovations such as electronic patient-reported outcomes, and patient-facing registry reports and electronic consent (in development), all to enhance patient engagement. The Patient Council is building on the foundations of industry\u2013patient advocacy group collaboration to fully integrate patient communities in decision-making and co-create solutions for the rare disease community.<\/jats:p>\n                <jats:p><jats:bold>Graphical Abstract<\/jats:bold><\/jats:p>","DOI":"10.1186\/s13023-024-03262-2","type":"journal-article","created":{"date-parts":[[2024,7,10]],"date-time":"2024-07-10T08:02:29Z","timestamp":1720598549000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":17,"title":["A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem"],"prefix":"10.1186","volume":"19","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7154-4342","authenticated-orcid":false,"given":"T. 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TK is President and CEO of National MPS Society. JB is an employee of Sanofi. SB received travel grants from Amicus Therapeutics, Sangamo Therapeutics and Biomarin, and received travel grants and honorarium from Sanofi. TCH is the CEO of IGA, with which she participated in advisory boards for Avrobio. IGA received grants from Sanofi, Prevail, Pfizer, Avrobio, Takeda, M6P and Gain. IGA received consulting fees from UK Gaucher Association and Gaucher Outcome Survey. TCH received payment for expert testimony from NICE. AD received grants and royalties from Casa Hunter, Federa\u00e7\u00e3o Brasileira das Associa\u00e7\u00f5es de Doen\u00e7as Raras (FEBRARARAS) and Casa dos Raros; consulting fees, speaking honoraria, payment for expert testimony, meeting attendance support, advisory board from Casa Hunter; meeting support attendance from FEBRARARAS, and patents issued with Casa dos Raros. DD is an employee of Sanofi and is a member of the PCORI Rare Disease Advisory Panel. JH participated in advisory boards for Zevra Therapeutics, Azafaros and the International Niemann-Pick Disease Registry; board member of Uplifting Athletes and National Niemann-Pick Disease. JJ is an employee and executive director of FSIG; FSIG received grants from 4D Molecular Therapeutics, Amicus Therapeutics, Avrobio, Chiesi Global Rare Diseases, Codexis, Freeline Therapeutics, Idorsia Pharmaceuticals, Sangamo Therapeutics, Sanofi, Siglion Therapeutics and Spark Therapeutics; received honoraria from Chiesi Global Rare Diseases, Idorsia Pharmaceuticals, Sanofi, Spark Therapeutics, Takeda and Protalix; received meeting attendance support from Chiesi Global Rare Diseases and Sanofi. JJ is a voluntary Vice-President Americas and Global of FIN. TL is the Vice-Chairperson of Hong Kong MPS & Rare Genetic Diseases Mutual Aid Group. MP is the voluntary President of FIN and the Greek Lysosomal Organization; FIN received grants from Amicus Therapeutics, Chiesi Pharmaceuticals, Sangamo Therapeutics, Sanofi, Spark Therapeutics and Takeda; FIN received payment for expert testimony from Sanofi and Takeda. TS is a board member of IPA and Chairman of Pompe Deutschland. IPA received grants and Pompe Deutschland received charitable donations from Sanofi. TS received meeting attendance support from Sanofi. I\u017d is a board member of IGA and Vice-Chair of the Slovenian Gaucher Association; received meeting and advisory board attendance support from Sanofi. LDB declares no conflict of interest.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"262"}}