{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,31]],"date-time":"2026-10-31T06:13:02Z","timestamp":1793427182496,"version":"4.5.0"},"reference-count":19,"publisher":"Springer Science and Business Media LLC","issue":"6","license":[{"start":{"date-parts":[[2025,7,21]],"date-time":"2025-07-21T00:00:00Z","timestamp":1753056000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2025,7,21]],"date-time":"2025-07-21T00:00:00Z","timestamp":1753056000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Ther Innov Regul Sci"],"published-print":{"date-parts":[[2025,11]]},"DOI":"10.1007\/s43441-025-00841-8","type":"journal-article","created":{"date-parts":[[2025,7,21]],"date-time":"2025-07-21T16:37:37Z","timestamp":1753115857000},"page":"1238-1244","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Transforming Objective Participatory Patient Advocacy"],"prefix":"10.1007","volume":"59","author":[{"given":"Pat","family":"Furlong","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Timothy R.","family":"Franson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter J.","family":"Pitts","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,7,21]]},"reference":[{"issue":"5","key":"841_CR1","doi-asserted-by":"publisher","first-page":"537","DOI":"10.1007\/s40271-019-00366-x","volume":"53","author":"DC Turk","year":"2019","unstructured":"Turk DC, Dworkin RH, Revicki D, Harding G, Burke LB, Cella D, Cowan P. Identifying important outcome domains for chronic pain clinical trials: An IMMPACT survey of people with pain. Therap Innov Regul Sci. 2019;53(5):537\u201346. https:\/\/doi.org\/10.1007\/s40271-019-00366-x.","journal-title":"Therap Innov Regul Sci"},{"key":"841_CR2","unstructured":"National Center for Biotechnology Information. (2018). Duchenne Muscular Dystrophy. In GeneReviews\u00ae. https:\/\/www.ncbi.nlm.nih.gov\/books\/NBK482346\/"},{"key":"841_CR3","unstructured":"Parent Project Muscular Dystrophy. https:\/\/www.parentprojectmd.org\/"},{"key":"841_CR4","unstructured":"H.R.717 \u2014 107th Congress (2001\u20132002). Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001. https:\/\/www.congress.gov\/bill\/107th-congress\/house-bill\/717"},{"key":"841_CR5","unstructured":"Parent Project Muscular Dystrophy. PPMD\u2019s Biomarkers Initiative. https:\/\/www.parentprojectmd.org\/research\/current-research\/our-strategy-impact\/ppmds-biomarkers-initiative\/"},{"key":"841_CR6","unstructured":"Parent Project Muscular Dystrophy. The Possibilities of Our Protein Mapping Project. https:\/\/www.parentprojectmd.org\/the-possibilities-of-our-protein-mapping-project\/"},{"key":"841_CR7","unstructured":"Critical Path Institute (C-Path). https:\/\/c-path.org\/"},{"key":"841_CR8","unstructured":"Parent Project Muscular Dystrophy. PPMD\u2019s Drug Development Roundtable. https:\/\/www.parentprojectmd.org\/research\/for-researchers-industry\/ppmds-drug-development-roundtable\/"},{"key":"841_CR9","unstructured":"U.S. Food and Drug Administration. (2014). Guidance for Industry: Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products. https:\/\/www.fda.gov\/media\/82644\/download"},{"key":"841_CR10","unstructured":"Parent Project Muscular Dystrophy. (2018). Compass Meeting Report. https:\/\/www.parentprojectmd.org\/wp-content\/uploads\/2018\/07\/PPMD_Compass_Meeting_Report.pdf"},{"key":"841_CR11","unstructured":"National Center for Biotechnology Information. (2011). Muscular Dystrophy Overview. In GeneReviews\u00ae. https:\/\/www.ncbi.nlm.nih.gov\/books\/NBK56187\/"},{"key":"841_CR12","unstructured":"U.S. Food and Drug Administration. (n.d.). 21st Century Cures Act. https:\/\/www.fda.gov\/regulatory-information\/selected-amendments-fdc-act\/21st-century-cures-act"},{"key":"841_CR13","unstructured":"Furlong, P. (2014). Testimony before the U.S. House of Representatives, Committee on Energy and Commerce. https:\/\/docs.house.gov\/meetings\/IF\/IF14\/20140711\/102451\/HMTG-113-IF14-Wstate-FurlongP-20140711.pdf"},{"key":"841_CR14","unstructured":"https:\/\/www.prnewswire.com\/news-releases\/first-ever-patient-initiated-guidance-for-industry-for-duchenne-muscular-dystrophy-submitted-to-fda-264581331.html"},{"key":"841_CR15","unstructured":"U.S. Food and Drug Administration. (2018). Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment \u2013 Guidance for Industry. https:\/\/www.fda.gov\/regulatory-information\/search-fda-guidance-documents\/duchenne-muscular-dystrophy-and-related-dystrophinopathies-developing-drugs-treatment-guidance"},{"issue":"5","key":"841_CR16","doi-asserted-by":"publisher","first-page":"531","DOI":"10.1177\/2168479018774556","volume":"52","author":"PJ Pitts","year":"2018","unstructured":"Pitts PJ, Brady P. From the valley of death to the crossroads of opportunity: a discussion of evolving benefit\/risk evaluation standards. Ther Innov Regul Sci. 2018;52(5):531\u20136. https:\/\/doi.org\/10.1177\/2168479018774556.","journal-title":"Ther Innov Regul Sci"},{"key":"841_CR17","unstructured":"U.S. Food and Drug Administration. Externally-Led Patient-Focused Drug Development Meetings. https:\/\/www.fda.gov\/industry\/prescription-drug-user-fee-amendments\/externally-led-patient-focused-drug-development-meetings"},{"key":"841_CR18","unstructured":"Parent Project Muscular Dystrophy. Benefit-Risk Assessment in Rare Disorders: A Blueprint for Engagement and Collaboration. April 2025. https:\/\/www.parentprojectmd.org\/wp-content\/uploads\/2025\/04\/PPMD-rare-disorder-benefit-risk-br_paper_v11__2_.pdf."},{"key":"841_CR19","unstructured":"Critical Path Institute. (n.d.). Rare Disease Cures Accelerators \u2013 Data and Analytics Platform. https:\/\/c-path.org\/program\/rare-disease-cures-accelerators-data-and-analytics-platform\/"}],"container-title":["Therapeutic Innovation &amp; Regulatory Science"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s43441-025-00841-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s43441-025-00841-8\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s43441-025-00841-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,11,1]],"date-time":"2025-11-01T20:17:48Z","timestamp":1762028268000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s43441-025-00841-8"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,7,21]]},"references-count":19,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2025,11]]}},"alternative-id":["841"],"URL":"https:\/\/doi.org\/10.1007\/s43441-025-00841-8","relation":{},"ISSN":["2168-4790","2168-4804"],"issn-type":[{"value":"2168-4790","type":"print"},{"value":"2168-4804","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,7,21]]},"assertion":[{"value":"1 May 2025","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"10 July 2025","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"21 July 2025","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Pat Furlong is President of Parent Project Muscular Dystrophy. Tim Franson and Peter Pitts report no conflicts of interest.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}},{"value":"This content has been made available to all.","name":"free","label":"Free to read"}]}}