{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,30]],"date-time":"2026-07-30T09:29:10Z","timestamp":1785403750075,"version":"3.56.0"},"reference-count":50,"publisher":"Cambridge University Press (CUP)","issue":"1","license":[{"start":{"date-parts":[[2026,3,9]],"date-time":"2026-03-09T00:00:00Z","timestamp":1773014400000},"content-version":"unspecified","delay-in-days":67,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Clin. Trans. Sci."],"published-print":{"date-parts":[[2026]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec id=\"S2059866126107195_as1\">\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Evidence suggests that partnerships between researchers and patient communities result in clinical trials that better reflect the patient experience, but there are few documented and successful models of such partnerships. Within the clinical research landscape, the Duchenne Muscular Dystrophy (DMD) community has emerged as a positive outlier in this regard, having engaged in a research partnership over multiple years and in the approval of several disease-modifying therapies where there were previously none.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec id=\"S2059866126107195_as2\">\n                    <jats:title>Methodology:<\/jats:title>\n                    <jats:p>The successful partnership factors observed in the DMD community were mapped onto the three levels of the Behavior Change Wheel framework. This mapping created an academic model of patient community readiness for research partnerships. The academic model was further translated into a user-friendly, pragmatic model and blueprint.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec id=\"S2059866126107195_as3\">\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>The FOCUS Model outlines the development of a research partnership from the ground up, where each theme represents an essential component of the partnership structure. Accompanying this model is a discussion guide that communities can use in meetings or workshops to assess and enhance their readiness for research partnerships.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec id=\"S2059866126107195_as4\">\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>The FOCUS model and its blueprint offer generalizable approaches that other rare disease patient communities can leverage to foster effective and lasting partnerships with scientific, medical, and regulatory stakeholders. The approach proposed in this study has the potential to help both the clinical research community and rare disease patient communities overcome typical barriers to rare disease clinical development.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1017\/cts.2026.10719","type":"journal-article","created":{"date-parts":[[2026,3,9]],"date-time":"2026-03-09T09:01:39Z","timestamp":1773046899000},"source":"Crossref","is-referenced-by-count":0,"title":["The FOCUS model: A guide to promote patient partnership in clinical trial designs"],"prefix":"10.1017","volume":"10","author":[{"given":"Jennifer Catena","family":"Davis","sequence":"first","affiliation":[{"id":[{"id":"https:\/\/ror.org\/00y4zzh67","id-type":"ROR","asserted-by":"publisher"}],"name":"The George Washington University"},{"name":"Worldwide Clinical Trials"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick","family":"Corr","sequence":"additional","affiliation":[{"id":[{"id":"https:\/\/ror.org\/00y4zzh67","id-type":"ROR","asserted-by":"publisher"}],"name":"The George Washington University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy","family":"Raymond","sequence":"additional","affiliation":[{"name":"Worldwide Clinical Trials"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3155-8130","authenticated-orcid":false,"given":"Sabrina","family":"Figueiredo","sequence":"additional","affiliation":[{"id":[{"id":"https:\/\/ror.org\/00y4zzh67","id-type":"ROR","asserted-by":"publisher"}],"name":"The George Washington University"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"56","published-online":{"date-parts":[[2026,3,9]]},"reference":[{"key":"S2059866126107195_ref39","doi-asserted-by":"publisher","DOI":"10.1007\/978-1-4614-4998-0"},{"key":"S2059866126107195_ref14","doi-asserted-by":"publisher","DOI":"10.1016\/j.clon.2015.06.020"},{"key":"S2059866126107195_ref6","doi-asserted-by":"publisher","DOI":"10.1016\/j.clinthera.2014.04.011"},{"key":"S2059866126107195_ref12","doi-asserted-by":"publisher","DOI":"10.1245\/s10434-016-5309-x"},{"key":"S2059866126107195_ref50","first-page":"57","article-title":"The evolution of patient-focused drug development and Duchenne muscular dystrophy","volume":"20","author":"Crossnohere","year":"2020","journal-title":"Expert Rev Pharm Out"},{"key":"S2059866126107195_ref1","doi-asserted-by":"publisher","DOI":"10.1602\/neurorx.1.3.307"},{"key":"S2059866126107195_ref13","first-page":"e231809","article-title":"Physician and nurse well-being and preferred interventions to address burnout in hospital practice: factors associated with turnover, outcomes, and patient safety","volume":"4","author":"Aiken","year":"2023","journal-title":"JAMA"},{"key":"S2059866126107195_ref10","first-page":"571","volume-title":"Clinical Trials, Disparities, and Financial Burden: Its Time to Intervene","author":"Moy","year":"2015"},{"key":"S2059866126107195_ref30","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.13977"},{"key":"S2059866126107195_ref9","doi-asserted-by":"publisher","DOI":"10.1016\/j.clinthera.2020.06.015"},{"key":"S2059866126107195_ref11","doi-asserted-by":"publisher","DOI":"10.1177\/2168479018801566"},{"key":"S2059866126107195_ref33","unstructured":"33. 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Demonstrating substantial evidence of effectiveness for human drug and biological products: draft guidance for industry, (https:\/\/www.fda.gov\/regulatory-information\/search-fda-guidance-documents\/demonstrating-substantial-evidence-effectiveness-human-drug-and-biological-products) Accessed October 26, 2022."},{"key":"S2059866126107195_ref28","doi-asserted-by":"publisher","DOI":"10.1111\/j.1469-8749.2011.03939.x"},{"key":"S2059866126107195_ref46","doi-asserted-by":"publisher","DOI":"10.1111\/hex.13909"},{"key":"S2059866126107195_ref22","doi-asserted-by":"publisher","DOI":"10.1093\/jnci\/djy064"},{"key":"S2059866126107195_ref18","doi-asserted-by":"publisher","DOI":"10.1186\/s40900-024-00622-x"},{"key":"S2059866126107195_ref31","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(16)30035-7"},{"key":"S2059866126107195_ref3","unstructured":"3. U.S. Food and Drug Administration. Demonstrating substantial evidence of effectiveness with one adequate and well-controlled clinical investigation and confirmatory evidence: Draft guidance for industry, (https:\/\/www.fda.gov\/regulatory-information\/search-fda-guidance-documents\/demonstrating-substantial-evidence-effectiveness-one-adequate-and-well-controlled-clinical) Accessed October 26, 2022."},{"key":"S2059866126107195_ref40","doi-asserted-by":"publisher","DOI":"10.7748\/nr.2021.e1786"},{"key":"S2059866126107195_ref44","unstructured":"44. Canadian Institute of Health Research (CIHR). Strategy for Patient-Oriented Research (SPOR), (https:\/\/www.cihr-irsc.gc.ca\/e\/41204.html) Accessed March 24, 2025."},{"key":"S2059866126107195_ref29","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.12213"},{"key":"S2059866126107195_ref16","doi-asserted-by":"publisher","DOI":"10.1007\/s43441-019-00083-5"},{"key":"S2059866126107195_ref47","doi-asserted-by":"publisher","DOI":"10.35680\/2372-0247.1812"},{"key":"S2059866126107195_ref24","unstructured":"24. U.S. Food Drug Administration. E6(R2) good clinical practice: integrated addendum to ICH E6(R1): guidance for industry. Silver Spring, MD: U.S. Department of Health and Human Services, (https:\/\/www.fda.gov\/files\/drugs\/published\/E6%28R2%29-Good-Clinical-Practice-Integrated-Addendum-to-ICH-E6%28R1%29.pdf) Accessed October 26, 2022."},{"key":"S2059866126107195_ref49","doi-asserted-by":"publisher","DOI":"10.1186\/s40900-023-00527-1"},{"key":"S2059866126107195_ref7","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0171114"},{"key":"S2059866126107195_ref19","doi-asserted-by":"publisher","DOI":"10.31219\/osf.io\/txzp8"},{"key":"S2059866126107195_ref34","volume-title":"Toward a Patient-Partnered Model of Clinical Trial Design: A Qualitative Descriptive Case Study of the Duchenne Muscular Dystrophy Clinical Research Partnership","author":"Davis","year":"2024"},{"key":"S2059866126107195_ref20","doi-asserted-by":"publisher","DOI":"10.2147\/PPA.S445399"},{"key":"S2059866126107195_ref38","unstructured":"38. U.S. Food and Drug Administration. FDA patient-focused drug development guidance series for enhancing the incorporation of the patient\u2019s voice in medical product development and regulatory decision making, (https:\/\/www.fda.gov\/drugs\/development-approval-process-drugs\/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical) Accessed October 10, 2022."},{"key":"S2059866126107195_ref32","unstructured":"32. Administration USFaD. Duchenne muscular dystrophy and related dystrophinopathies: developing drugs for treatment; guidance for industry, (https:\/\/search-proquest-com.proxygw.wrlc.org\/docview\/2002427418) Accessed December 12, 2022."},{"key":"S2059866126107195_ref5","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3181dbb884"},{"key":"S2059866126107195_ref15","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-017-1577-6"},{"key":"S2059866126107195_ref23","doi-asserted-by":"publisher","DOI":"10.1007\/s11606-021-07104-w"},{"key":"S2059866126107195_ref25","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.13277"},{"key":"S2059866126107195_ref8","doi-asserted-by":"publisher","DOI":"10.1177\/2168479018788053"},{"key":"S2059866126107195_ref21","doi-asserted-by":"publisher","DOI":"10.1111\/hex.12888"},{"key":"S2059866126107195_ref41","doi-asserted-by":"publisher","DOI":"10.1111\/jep.12805"},{"key":"S2059866126107195_ref42","unstructured":"42. Patient-Centered Outcomes Research Institute (PCORI). PCORI engagement rubric. PCORI (Patient-Centered Outcomes Research Institute) website., (http:\/\/www.pcori.org\/sites\/default\/files\/Engagement-Rubric.pdf) Accessed November 5, 2023."},{"key":"S2059866126107195_ref48","doi-asserted-by":"publisher","DOI":"10.1016\/S0277-9536(96)00339-5"},{"key":"S2059866126107195_ref17","volume-title":"Principles and Practice of Clinical Trials","author":"Sigal","year":"2020"},{"key":"S2059866126107195_ref35","doi-asserted-by":"publisher","DOI":"10.1186\/1748-5908-6-42"},{"key":"S2059866126107195_ref36","volume-title":"The Behaviour Change Wheel. A Guide to Designing Interventions","author":"Michie","year":"2014"},{"key":"S2059866126107195_ref37","doi-asserted-by":"publisher","DOI":"10.1097\/MLR.0000000000000273"},{"key":"S2059866126107195_ref43","unstructured":"43. Patient-Centered Outcomes Research Institute (PCORI). 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