{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T13:17:16Z","timestamp":1790255836660,"version":"4.1.0"},"reference-count":101,"publisher":"Informa UK Limited","issue":"1","funder":[{"DOI":"10.13039\/100005693","name":"Parent Project Muscular Dystrophy","doi-asserted-by":"publisher","award":["60068612"],"award-info":[{"award-number":["60068612"]}],"id":[{"id":"10.13039\/100005693","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["www.tandfonline.com"],"crossmark-restriction":true},"short-container-title":["Expert Review of Pharmacoeconomics &amp; Outcomes Research"],"published-print":{"date-parts":[[2020,1,2]]},"DOI":"10.1080\/14737167.2020.1734454","type":"journal-article","created":{"date-parts":[[2020,2,26]],"date-time":"2020-02-26T09:54:31Z","timestamp":1582710871000},"page":"57-68","update-policy":"https:\/\/doi.org\/10.1080\/tandf_crossmark_01","source":"Crossref","is-referenced-by-count":26,"title":["The evolution of patient-focused drug development and Duchenne muscular dystrophy"],"prefix":"10.1080","volume":"20","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2811-1330","authenticated-orcid":false,"given":"Norah L.","family":"Crossnohere","sequence":"first","affiliation":[{"name":"Department of Health, Behavior and Society, Johns Hopkins School of Public Health, Baltimore, MD, USA"},{"name":"Department of Biomedical Informatics, The Ohio State University Wexner Medical Center, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ryan","family":"Fischer","sequence":"additional","affiliation":[{"name":"Parent Project Muscular Dystrophy, Hackensack, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emily","family":"Crossley","sequence":"additional","affiliation":[{"name":"Duchenne UK, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth","family":"Vroom","sequence":"additional","affiliation":[{"name":"Duchenne Parent Project Muscular Dystrophys, Amsterdam, NL, Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John FP","family":"Bridges","sequence":"additional","affiliation":[{"name":"Department of Health, Behavior and Society, Johns Hopkins School of Public Health, Baltimore, MD, USA"},{"name":"Department of Biomedical Informatics, The Ohio State University Wexner Medical Center, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"301","published-online":{"date-parts":[[2020,3,6]]},"reference":[{"key":"cit0001","unstructured":"FDA mission. [cited 2020 Mar 2]. Available from: https:\/\/www.fda.gov\/aboutfda\/whatwedo\/default.htm-mission."},{"issue":"3","key":"cit0002","first-page":"170","volume":"1","author":"Van Norman GA.","year":"2016","journal-title":"JACC"},{"key":"cit0003","doi-asserted-by":"publisher","DOI":"10.1377\/hlthaff.23.1.52"},{"key":"cit0004","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2017.0410"},{"key":"cit0005","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aac6023"},{"key":"cit0006","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23528"},{"key":"cit0007","doi-asserted-by":"publisher","DOI":"10.1016\/0960-8966(91)90039-U"},{"key":"cit0008","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2014.03.008"},{"key":"cit0009","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-015-0281-2"},{"key":"cit0010","doi-asserted-by":"publisher","DOI":"10.1007\/978-90-481-9485-8_28"},{"key":"cit0011","unstructured":"Patient-focused drug development: collecting comprehensive and representative input, indraft guidance for industry, food and drug administration staff, and other stakeholders. \u00a0Silver Spring, MD: US Department of Health and Human Services; 2018."},{"key":"cit0012","unstructured":"Medical Device Innovation Consortium (MDIC). Patient centered benefit-risk project report: a framework for incorporating information on patient preferences regarding benefit and risk into regulatory assessments of new medical technology. Minneapolis, MN: Medical Device Innovation Consortium; 2015."},{"key":"cit0013","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2015.15818"},{"key":"cit0014","doi-asserted-by":"publisher","DOI":"10.1111\/dote.12437"},{"key":"cit0015","volume-title":"\u201cRational Medicine\u201d Initiative","author":"Kondo T","year":"2017"},{"key":"cit0016","unstructured":"PDUFA Reauthorization performance goals and procedures fiscal years 2013 trough 2017. Silver Spring, MD: Dept. of Health and Human Services, US Food and Drug Administration; 2012."},{"key":"cit0017","doi-asserted-by":"publisher","DOI":"10.1016\/S0899-3467(07)60142-6"},{"key":"cit0018","doi-asserted-by":"publisher","DOI":"10.1093\/humupd\/dmh058"},{"key":"cit0019","doi-asserted-by":"publisher","DOI":"10.1007\/s40271-014-0046-3"},{"key":"cit0020","unstructured":"Smith JA, Flowers P, Osborn M. Interpretative phenomenological analysis and the psychology of health and illness. In: L. Yardley, editor.\u00a0Material discourses of health and illness. London, Uk: Taylor & Frances\/Routledge. 1997;68\u201391."},{"key":"cit0021","doi-asserted-by":"publisher","DOI":"10.1191\/1478088706qp062oa"},{"key":"cit0022","volume-title":"Framework for FDA\u2019s real-world evidence program","year":"2018"},{"key":"cit0023","volume-title":"Guidance for industry patient-reported outcome measures: use in medical product development to support labeling claims","year":"2009"},{"key":"cit0024","doi-asserted-by":"publisher","DOI":"10.1126\/science.6836280"},{"key":"cit0025","doi-asserted-by":"publisher","DOI":"10.1126\/science.3399889"},{"key":"cit0026","unstructured":"Federal Food, Drug, and Cosmetic Act, in21 U.S.C. ch. 9 \u00a7301 et seq, t.U.S. Congress, Editor. 1938."},{"key":"cit0027","unstructured":"Drug Ammendments Act, in21 U.S.C. ch. 9 \u00a7301 et seq, t.U.S. Congress, Editor. 1962."},{"key":"cit0028","doi-asserted-by":"publisher","DOI":"10.1002\/cpt1978242133"},{"key":"cit0029","doi-asserted-by":"publisher","DOI":"10.1002\/cpt1978245499"},{"key":"cit0030","doi-asserted-by":"publisher","DOI":"10.1038\/clpt.1989.116"},{"key":"cit0031","first-page":"177","volume":"5","author":"Gieringer DH","year":"1985","journal-title":"Cato J"},{"key":"cit0032","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pmed.1000316"},{"key":"cit0033","unstructured":"The Orphan Drug Act, in21 U.S.C. ch. 9, subch. V \u00a7 360aa-360ee. 1983."},{"key":"cit0034","doi-asserted-by":"publisher","DOI":"10.4000\/quaderni.262"},{"key":"cit0035","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0140002"},{"key":"cit0036","volume-title":"National healthcare quality report","author":"Agency for Healthcare Research and Quality","year":"2006"},{"key":"cit0037","unstructured":"FDA Backgrounder on FDAMA. 1997 [cited 2019 Mar 25]. Available from: https:\/\/www.fda.gov\/RegulatoryInformation\/LawsEnforcedbyFDA\/SignificantAmendmentstotheFDCAct\/FDAMA\/ucm089179.htm."},{"key":"cit0038","volume-title":"Institute of Medicine. Institute of Medicine committee on quality of health care in America, in Crossing the quality chasm: a new health system for the 21st century","year":"2001"},{"key":"cit0039","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2012.500"},{"key":"cit0040","doi-asserted-by":"publisher","DOI":"10.1046\/j.1524-4733.2003.65309.x"},{"key":"cit0041","volume-title":"DIA Workshop on Pharmacoeconomic and Quality of Life Labeling and Marketing Claims, New Orleans, LA","author":"Burke L","year":"2000"},{"key":"cit0042","unstructured":"Colapinto J,Mother Courage, inThe New Yorker. 2010."},{"key":"cit0043","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"cit0044","volume-title":"Former FDA chief margaret hamburg speaks out about califf, cruz, and congress","author":"Berke R","year":"2016"},{"key":"cit0045","unstructured":"Food and Drug Safety and Innovation Act, t.U. Congress, Editor. 2012."},{"key":"cit0046","doi-asserted-by":"publisher","DOI":"10.1111\/hex.12353"},{"key":"cit0047","volume-title":"PDUFA V: medical innovation, jobs, and patients, in committee on energy and commerce"},{"key":"cit0048","doi-asserted-by":"publisher","DOI":"10.1093\/jmp\/jhn021"},{"key":"cit0049","volume-title":"Structured approach to benefit-risk assessment in drug regulatory decision-making: draft PDUFA V implementation plan\u2014February 2013: fiscal years 2013\u20132017","author":"Food, U. and D. Administration","year":"2013"},{"key":"cit0050","doi-asserted-by":"publisher","DOI":"10.1038\/nrd4104"},{"key":"cit0051","doi-asserted-by":"publisher","DOI":"10.1016\/j.jbusres.2006.11.010"},{"key":"cit0052","volume-title":"Guidance for Industry: Factors to consider regarding benefitrisk in medical device product availability, compliance, and enforcement decisions","year":"2017"},{"key":"cit0053","unstructured":"Prescription Drug User Fee Act reauthorization (PDUFA VI, Medical Device User Fee Act reauthorization (MDUFA IV), Generic Drug User Fee Act reauthorization (GDUFA II), and Biosimilar User Fee Act reauthorization (BsUFA II), inSenate Committee on Health, Education, Labor, and Pensions."},{"key":"cit0054","doi-asserted-by":"publisher","DOI":"10.1016\/S0738-3991(99)00086-5"},{"key":"cit0055","unstructured":"21st Century Cures ActinH.R. 34, 114th Congress. 2016: United States."},{"key":"cit0056","volume-title":"Guidance for industry and food and drug administration staff: policy for device software functions and mobile medical applications","year":"2019"},{"key":"cit0057","volume-title":"Patient-Focused Drug Development: Methods to Identify What Is Important to Patients Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders. Draft Guidance.","year":"2018"},{"key":"cit0058","volume-title":"Patient-focused drug development guidance public workshop: methods to identify what is important to patients","year":"2018"},{"key":"cit0059","volume-title":"Patient-focused drug development discussion document: incorporating clinical outcome assessments into endpoints for regulatory decision-making","year":"2019"},{"key":"cit0060","volume-title":"Benefit-Risk Methodology Project","year":"2009"},{"key":"cit0061","doi-asserted-by":"publisher","DOI":"10.1192\/apt.9.3.229"},{"key":"cit0062","volume-title":"Outcome report on pilot to involve patients in benefit\/risk discussions at CHMP meetings","year":"2017"},{"key":"cit0063","doi-asserted-by":"publisher","DOI":"10.1016\/j.socscimed.2005.02.001"},{"key":"cit0064","doi-asserted-by":"publisher","DOI":"10.1377\/hlthaff.26.3.684"},{"key":"cit0065","doi-asserted-by":"publisher","DOI":"10.1377\/hlthaff.28.4.w555"},{"key":"cit0066","volume-title":"Doctors talking with patients\/patients talking with doctors: improving communication in medical visits","author":"Roter D","year":"2006"},{"key":"cit0067","unstructured":"Stakeholder Engagement Report. European Medicines Agency; 2017."},{"key":"cit0068","doi-asserted-by":"crossref","unstructured":"Putting Patients First. Parent Project Muscular Dystrophy; 2013.","DOI":"10.12968\/indn.2013.15.4.98105"},{"key":"cit0069","unstructured":"Patients are Waiting. Parent Project Muscular Dystrophy; 2014."},{"key":"cit0070","doi-asserted-by":"publisher","DOI":"10.1016\/j.clinthera.2014.04.011"},{"key":"cit0071","doi-asserted-by":"publisher","DOI":"10.1007\/s40271-014-0104-x"},{"key":"cit0072","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2016.02.014"},{"key":"cit0073","doi-asserted-by":"crossref","unstructured":"Hollin I, Peay H, Fischer R, et al. Engaging patients and caregivers in prioritizing symptoms impacting quality of life for Duchenne and Becker muscular dystrophy.\u00a0Quality of Life Research. 2018;27(9):2261-2273.","DOI":"10.1007\/s11136-018-1891-7"},{"key":"cit0074","volume-title":"Duchenne muscular dystrophy and related dystrophinopathies: developing drugs for treatment guidance for industry","author":"US Department of Health and Human Services, F.D.A.","year":"2015"},{"key":"cit0075","volume-title":"Duchenne muscular dystrophy and related dystrophinopathies: developing drugs for treatment guidance for industry","year":"2018"},{"key":"cit0076","volume-title":"A new voice in science: patient participation in decision-making on biomedical research","author":"Caron-Flinterman JF","year":"2005"},{"key":"cit0077","unstructured":"Key Considerations in Developing & Integrating Patient Perspectives in Drug Development. Parent Project Muscular Dystrophy & Biotechnology Innovation Organization; 2016."},{"key":"cit0078","author":"Bridges JF","year":"2018","journal-title":"Patient-Patient-Centered Outcomes Res"},{"key":"cit0079","volume-title":"FDA turns down santhera pharmaceuticals\u2019 request for accelerated approval for idebenone to treat DMD","year":"2016"},{"key":"cit0080","volume-title":"Scientific dispute regarding accelerated approval of Sarepta therapeutics\u2019 eteplirsen - commissioner\u2019s decision","author":"Califf R","year":"2016"},{"key":"cit0081","volume-title":"A look at deflazacourt and exon-skipping therapies for Duchenne muscular dystrophy","year":"2019"},{"key":"cit0082","unstructured":"DMD: Public Comment on ICER Draft Report. 2019."},{"issue":"2","key":"cit0083","first-page":"200","volume":"92","author":"Burkell J","year":"2004","journal-title":"J Med Libr Assoc"},{"key":"cit0084","volume-title":"Benefit-risk assessment in drug regulatory decision making; draft PDUFA VI implementation plan","year":"2018"},{"key":"cit0085","unstructured":"Memorandum of Understanding Between the U.S. Department of Health and Human Services, Food and Drug Administration, and the National Organization For Rare Disorders. 2018."},{"key":"cit0086","unstructured":"FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. 2019."},{"key":"cit0087","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-019-1069-6"},{"key":"cit0088","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0213649"},{"key":"cit0089","volume-title":"Benefit-risk assessment in rare disorders","year":"2013"},{"key":"cit0090","volume-title":"Cures 2.0","author":"DeGette D","year":"2019"},{"key":"cit0091","unstructured":"BENEFIT Act S.1052. 2017, 115th Congress of the United States."},{"key":"cit0092","doi-asserted-by":"publisher","DOI":"10.1016\/j.socscimed.2013.07.026"},{"key":"cit0093","volume-title":"Rare diseases: natural history studies for drug development guidance for industry","year":"2019"},{"key":"cit0094","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aab2404"},{"key":"cit0095","doi-asserted-by":"publisher","DOI":"10.1179\/2047480615Z.000000000329"},{"key":"cit0096","unstructured":"PDUFA REAUTHORIZATION PERFORMANCE GOALS AND PROCEDURES FISCAL YEARS 2018 THROUGH 2022. 2016., U.S.F.D. Administration, Editor. 2016."},{"key":"cit0097","doi-asserted-by":"publisher","DOI":"10.1080\/03007995.2019.1631149"},{"key":"cit0098","volume-title":"Patient preference information\u2013voluntary submission, review in premarket approval applications, humanitarian device exemption applications, and de novo requests, and inclusion in decision summaries and device labeling","author":"CDRH F","year":"2016"},{"key":"cit0099","unstructured":"Health spending. Organisation for Economic Co-operation and Development; 2018. [cited 2020 Mar 2]. Available from: https:\/\/data.oecd.org\/healthres\/health-spending.htm"},{"key":"cit0100","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-7-74"},{"key":"cit0101","volume-title":"Quality-adjusted life years and the devaluation of life with disability","year":"2019"}],"container-title":["Expert Review of Pharmacoeconomics &amp; Outcomes Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.tandfonline.com\/doi\/pdf\/10.1080\/14737167.2020.1734454","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,27]],"date-time":"2023-09-27T17:38:24Z","timestamp":1695836304000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.tandfonline.com\/doi\/full\/10.1080\/14737167.2020.1734454"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,1,2]]},"references-count":101,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2020,1,2]]}},"alternative-id":["10.1080\/14737167.2020.1734454"],"URL":"https:\/\/doi.org\/10.1080\/14737167.2020.1734454","relation":{},"ISSN":["1473-7167","1744-8379"],"issn-type":[{"value":"1473-7167","type":"print"},{"value":"1744-8379","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,1,2]]},"assertion":[{"value":"The publishing and review policy for this title is described in its Aims & Scope.","order":1,"name":"peerreview_statement","label":"Peer Review Statement"},{"value":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=ierp20","URL":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=ierp20","order":2,"name":"aims_and_scope_url","label":"Aim & Scope"},{"value":"2019-12-10","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-02-21","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-03-06","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}