{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T08:13:17Z","timestamp":1777882397087,"version":"3.51.4"},"reference-count":37,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2026,3,1]],"date-time":"2026-03-01T00:00:00Z","timestamp":1772323200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,3,1]],"date-time":"2026-03-01T00:00:00Z","timestamp":1772323200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"name":"Real-World Evidence and Outcomes-Based Agreements Working Group"},{"DOI":"10.13039\/100006483","name":"abbvie","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006483","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100002429","name":"amgen","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100002429","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004325","name":"astrazeneca","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004325","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Janssen"},{"DOI":"10.13039\/100004319","name":"pfizer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004319","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004337","name":"roche","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004337","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Therapeutic Advances in Rare Disease"],"published-print":{"date-parts":[[2026,3]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Outcome-based agreements (OBAs) may facilitate earlier patient access to promising therapies, particularly when evidence is limited. The authors of this paper investigated how to operationalize an OBA using real-world data (RWD) from the Canadian Neuromuscular Disease Registry (CNDR).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>The first objective of this research was to determine which spinal muscular atrophy (SMA) health outcomes in the CNDR are suitable for an OBA. The second objective was to evaluate the current process of data collection in the CNDR and explore how to operationalize data processes to support an OBA, including identifying gaps and proposing solutions.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design:<\/jats:title>\n                    <jats:p>This qualitative expert-led assessment was conducted through a series of focus group discussions with selected experts.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>A selected group of experts participated in 17 focus group meetings. The ability of the CNDR to generate real-world evidence (RWE) for an OBA was evaluated for eight SMA health outcomes. The criteria used were data readiness, interpretability, and timeframe within the CNDR. Next, the processes involved were evaluated, specifically to determine how data tracking within the CNDR could be operationalized for an OBA, including identifying gaps and possible solutions. Based on the findings, the group proposed a future state for an OBA process using the CNDR. The group followed up with stakeholder feedback interviews to validate the findings of this research and to gather insights.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Five SMA outcomes within the CNDR were identified as potentially suitable outcomes for OBA. Three process gaps were identified in the current state of the CNDR, and corresponding solutions were proposed. A proposed future-state process flow for CNDR was developed to support RWE generation for OBA.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>Expert consultations suggest that operationalizing an OBA using CNDR RWD is feasible.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/26330040261433035","type":"journal-article","created":{"date-parts":[[2026,3,25]],"date-time":"2026-03-25T11:41:13Z","timestamp":1774438873000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Could an outcome-based agreement be operationalized using real-world data from the Canadian Neuromuscular Disease Registry? Perspectives from an expert-led assessment in spinal muscular atrophy"],"prefix":"10.1177","volume":"7","author":[{"ORCID":"https:\/\/orcid.org\/0009-0007-9165-5342","authenticated-orcid":false,"given":"Arif","family":"Mitha","sequence":"first","affiliation":[{"name":"20Sense, Toronto, ON, Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Victoria","family":"Hodgkinson","sequence":"additional","affiliation":[{"name":"Department of Clinical Neurosciences, University of Calgary, Calgary, AB, Canada"},{"name":"Hotchkiss Brain Institute, University of Calgary, Calgary, AB, Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Susi","family":"Vander Wyk","sequence":"additional","affiliation":[{"name":"Cure SMA Canada Society, Chilliwack, BC, Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chris","family":"Cameron","sequence":"additional","affiliation":[{"name":"EVERSANA Life Science Services LLC, Chicago, IL, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-9124-5278","authenticated-orcid":false,"given":"Allison","family":"Wills","sequence":"additional","affiliation":[{"name":"20Sense, Toronto, ON, Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2026,3,25]]},"reference":[{"key":"e_1_3_4_2_2","unstructured":"Health Canada. About drugs and rare diseases in Canada https:\/\/www.canada.ca\/en\/health-canada\/services\/licences-authorizations-registrations-drug-health-products\/regulatory-approach-drugs-rare-diseases\/about-drugs-rare-diseases.html (2018 accessed 5 February 2026)."},{"key":"e_1_3_4_3_2","unstructured":"Casey B. Canadians affected by rare diseases and disorders: Improving access to treatment. Report of the Standing Committee on Health 42nd Parliament 1st Session https:\/\/www.ourcommons.ca\/Content\/Committee\/421\/HESA\/Reports\/RP10349306\/hesarp22\/hesarp22-e.pdf (2019 accessed 5 February 2026)."},{"key":"e_1_3_4_4_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.healthpol.2024.105044"},{"key":"e_1_3_4_5_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2023.12.011"},{"key":"e_1_3_4_6_2","unstructured":"Government of Canada. Building a national strategy for high-cost drugs for rare diseases. A discussion paper for engaging Canadians [Internet] https:\/\/www.canada.ca\/content\/dam\/hc-sc\/documents\/services\/health-related-consultation\/National-Strategy-High-Cost-Drugs-eng.pdf (2021 accessed 5 February 2026)."},{"key":"e_1_3_4_7_2","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2020.594446"},{"issue":"1","key":"e_1_3_4_8_2","first-page":"5","article-title":"Building infrastructure for outcomes-based agreements in Canada: can administrative health data be used to support an outcomes-based agreement in oncology?","volume":"31","author":"Cheung WY","year":"2023","unstructured":"Cheung WY, Cameron C, Mitha A, et al. Building infrastructure for outcomes-based agreements in Canada: can administrative health data be used to support an outcomes-based agreement in oncology? 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Rare disease-based registries https:\/\/www.cda-amc.ca\/drugs-rare-diseases-rare-disease-based-registries#:~:text=Key%20Activities%20and%20Published%20Reports throughout%20the%20drug%20life%20cycle (2026 accessed 5 February 2026)."},{"key":"e_1_3_4_12_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-200538"},{"key":"e_1_3_4_13_2","doi-asserted-by":"publisher","DOI":"10.1093\/pch\/pxx125"},{"key":"e_1_3_4_14_2","unstructured":"Canadian Rare Disease Network. Brain Canada invests in expanded Neuromuscular Disease Registry https:\/\/canadianrdn.ca\/brain-canada-invests-in-expanded-neuromuscular-disease-registry\/ (2025 accessed 5 February 2026)."},{"key":"e_1_3_4_15_2","doi-asserted-by":"publisher","DOI":"10.1017\/cjn.2020.111"},{"key":"e_1_3_4_16_2","doi-asserted-by":"publisher","DOI":"10.1177\/22143602251336076"},{"key":"e_1_3_4_17_2","first-page":"1","article-title":"Equitable access to disease-modifying therapies for Canadian children with SMA and four SMN2 copies","volume":"52","author":"McMillan HJ","year":"2024","unstructured":"McMillan HJ, Gonorazky H, Campbell C, et al. Equitable access to disease-modifying therapies for Canadian children with SMA and four SMN2 copies. Can J Neurol Sci 2024; 52: 1\u20133.","journal-title":"Can J Neurol Sci"},{"key":"e_1_3_4_18_2","doi-asserted-by":"publisher","DOI":"10.1017\/cjn.2020.161"},{"key":"e_1_3_4_19_2","unstructured":"Biogen Canada news release on PR Newswire. 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Quebec treatments https:\/\/curesma.ca\/quebec-treatments\/ (2024 accessed 5 February 2026)."},{"key":"e_1_3_4_31_2","doi-asserted-by":"crossref","unstructured":"Varni JW Limbers CA Burwinkle TM. How young can children reliably and validly self-report their health-related quality of life? An analysis of 8 591 children across age subgroups with the PedsQLTM 4.0 Generic Core Scales. Health Qual Life Outcomes 2007; 5 https:\/\/hqlo.biomedcentral.com\/articles\/10.1186\/1477-7525-5-1 (2007 accessed 5 February 2026).","DOI":"10.1186\/1477-7525-5-1"},{"key":"e_1_3_4_32_2","doi-asserted-by":"publisher","DOI":"10.1002\/ana.20473"},{"key":"e_1_3_4_33_2","doi-asserted-by":"publisher","DOI":"10.1007\/s40120-022-00411-2"},{"key":"e_1_3_4_34_2","unstructured":"Government of Canada. 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