How Do Members of the Duchenne and Becker Muscular Dystrophy Community Perceive a Discrete-Choice Experiment Incorporating Uncertain Treatment Benefit? An ... View Full Text


Ontology type: schema:ScholarlyArticle     


Article Info

DATE

2019-04

AUTHORS

John F. P. Bridges, Jui-Hua Tsai, Ellen Janssen, Norah L. Crossnohere, Ryan Fischer, Holly Peay

ABSTRACT

BACKGROUND: Best-worst scaling methods have been used in several Duchenne and Becker muscular dystrophy (DBMD) studies to quantify patient and caregiver priorities and preferences and promote patient-focused drug development (PFDD). We sought to assess the extent to which different members of the DBMD community would accept a discrete-choice experiment (DCE) that incorporates uncertainty regarding individual-level benefit. METHODS: A community advisory board encouraged the development and testing of a DCE to further examine treatment preferences in DBMD and to facilitate the inclusion of a policy-relevant uncertainty attribute. The DCE assessed preferences across a primary outcome (muscle strength) and several risks (uncertainty regarding treatment benefit, kidney damage risk, and fracture risk). The single instrument was tested among adult patients, caregivers, and professionals at the national Parent Project Muscular Dystrophy annual meeting. The DCE was analyzed using conditional logit. Instrument acceptability was evaluated using a previously developed set of questions assessing ease of understanding and answering, and if answers reflected the respondents' real preferences. We proposed a 75% agreement rate as a threshold of acceptability, and used a Z score to assess if this was met, exceeded, or rejected. RESULTS: A total of 161 people completed the survey including 9 patients, 87 caregivers, and 65 professionals. Patients reported high acceptability across all evaluation items (p values > 0.21). Caregivers and professionals exceeded the benchmark of acceptability on understanding and reflecting real preferences (p < 0.001). Professionals met the benchmark (p = 0.08) for ease of answering, but caregivers did not (p < 0.01). DCE results demonstrated that all groups made meaningful trade-offs, with patients being less tolerant of risks than either caregivers or professionals (p < 0.001), and with no significant difference between caregivers and professionals (p = 0.46). CONCLUSIONS: This study demonstrates the acceptable application of a single instrument across a multi-stakeholder population that used a complex preference method and included a policy-relevant uncertainty variable. Ease of answering was lowest among caregivers, but a post-hoc analysis revealed that it was most difficult for those with children under the age of 10, while those with older children met the threshold. The success of this study has laid the foundation for a global study of DBMD preferences using this method. More... »

PAGES

247-257

References to SciGraph publications

  • 2014-06. Risk as an Attribute in Discrete Choice Experiments: A Systematic Review of the Literature in THE PATIENT - PATIENT-CENTERED OUTCOMES RESEARCH
  • 2009-03. The treatment of hemophilia A: from protein replacement to AAV-mediated gene therapy in BIOTECHNOLOGY LETTERS
  • 2015-02. Caregiver Preferences for Emerging Duchenne Muscular Dystrophy Treatments: A Comparison of Best-Worst Scaling and Conjoint Analysis in THE PATIENT - PATIENT-CENTERED OUTCOMES RESEARCH
  • 2008-08. Conducting Discrete Choice Experiments to Inform Healthcare Decision Making in PHARMACOECONOMICS
  • 2000-07. The Relationship of Illness Uncertainty, Illness Intrusiveness, and Asthma Severity to Depression in Young Adults with Long-Standing Asthma in INTERNATIONAL JOURNAL OF REHABILITATION AND HEALTH
  • 2016-02. Preferences and Stated Adherence for Antibiotic Treatment of Cystic Fibrosis Pseudomonas Infections in THE PATIENT - PATIENT-CENTERED OUTCOMES RESEARCH
  • 2013-08. Quantifying Benefit–Risk Preferences for Medical Interventions: An Overview of a Growing Empirical Literature in APPLIED HEALTH ECONOMICS AND HEALTH POLICY
  • 2010-12. What you see may not be what you get: Asking consumers what matters may not reflect what they choose in MARKETING LETTERS
  • 2004-09. Discrete choice experiments in health economics in THE EUROPEAN JOURNAL OF HEALTH ECONOMICS
  • 2016-04. Prioritizing Parental Worry Associated with Duchenne Muscular Dystrophy Using Best-Worst Scaling in JOURNAL OF GENETIC COUNSELING
  • 2015-10. Incorporating patient-preference evidence into regulatory decision making in SURGICAL ENDOSCOPY
  • 2015-05. Conceptual and practical foundations of patient engagement in research at the patient-centered outcomes research institute in QUALITY OF LIFE RESEARCH
  • 2011-12. Can Patients Diagnosed with Schizophrenia Complete Choice-Based Conjoint Analysis Tasks? in THE PATIENT - PATIENT-CENTERED OUTCOMES RESEARCH
  • 2015-12. How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration in ORPHANET JOURNAL OF RARE DISEASES
  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1007/s40271-018-0330-8

    DOI

    http://dx.doi.org/10.1007/s40271-018-0330-8

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1107261580

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/30259384


    Indexing Status Check whether this publication has been indexed by Scopus and Web Of Science using the SN Indexing Status Tool
    Incoming Citations Browse incoming citations for this publication using opencitations.net

    JSON-LD is the canonical representation for SciGraph data.

    TIP: You can open this SciGraph record using an external JSON-LD service: JSON-LD Playground Google SDTT

    [
      {
        "@context": "https://springernature.github.io/scigraph/jsonld/sgcontext.json", 
        "about": [
          {
            "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/1402", 
            "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
            "name": "Applied Economics", 
            "type": "DefinedTerm"
          }, 
          {
            "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/14", 
            "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
            "name": "Economics", 
            "type": "DefinedTerm"
          }
        ], 
        "author": [
          {
            "affiliation": {
              "alternateName": "Johns Hopkins University", 
              "id": "https://www.grid.ac/institutes/grid.21107.35", 
              "name": [
                "Department of Health Policy and Management, The Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe St, 21205, Baltimore, MD, USA", 
                "Department of Health, Behavior, and Society, The Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe St, 21205, Baltimore, MD, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Bridges", 
            "givenName": "John F. P.", 
            "id": "sg:person.0744135726.94", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0744135726.94"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Johns Hopkins University", 
              "id": "https://www.grid.ac/institutes/grid.21107.35", 
              "name": [
                "Department of Health Policy and Management, The Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe St, 21205, Baltimore, MD, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Tsai", 
            "givenName": "Jui-Hua", 
            "id": "sg:person.010463531012.02", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.010463531012.02"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Johns Hopkins University", 
              "id": "https://www.grid.ac/institutes/grid.21107.35", 
              "name": [
                "Department of Health Policy and Management, The Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe St, 21205, Baltimore, MD, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Janssen", 
            "givenName": "Ellen", 
            "id": "sg:person.016640247455.87", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.016640247455.87"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Johns Hopkins University", 
              "id": "https://www.grid.ac/institutes/grid.21107.35", 
              "name": [
                "Department of Health, Behavior, and Society, The Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe St, 21205, Baltimore, MD, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Crossnohere", 
            "givenName": "Norah L.", 
            "id": "sg:person.015214720167.14", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.015214720167.14"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "Parent Project Muscular Dystrophy", 
              "id": "https://www.grid.ac/institutes/grid.437213.0", 
              "name": [
                "Parent Project Muscular Dystrophy, 401 Hackensack Avenue, 9th Floor, 07601, Hackensack, NJ, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Fischer", 
            "givenName": "Ryan", 
            "id": "sg:person.0646522326.37", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0646522326.37"
            ], 
            "type": "Person"
          }, 
          {
            "affiliation": {
              "alternateName": "RTI International", 
              "id": "https://www.grid.ac/institutes/grid.62562.35", 
              "name": [
                "Department of Health, Behavior, and Society, The Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe St, 21205, Baltimore, MD, USA", 
                "Parent Project Muscular Dystrophy, 401 Hackensack Avenue, 9th Floor, 07601, Hackensack, NJ, USA", 
                "RTI International, 701 13th St NW #750, 20005, Washington, DC, USA"
              ], 
              "type": "Organization"
            }, 
            "familyName": "Peay", 
            "givenName": "Holly", 
            "id": "sg:person.0654364140.12", 
            "sameAs": [
              "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0654364140.12"
            ], 
            "type": "Person"
          }
        ], 
        "citation": [
          {
            "id": "https://doi.org/10.1016/j.ncl.2014.05.002", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1001875634"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s40271-014-0104-x", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1002431225", 
              "https://doi.org/10.1007/s40271-014-0104-x"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/j.jval.2016.02.014", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1003756617"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s11136-014-0893-3", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1007355674", 
              "https://doi.org/10.1007/s11136-014-0893-3"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s11136-014-0893-3", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1007355674", 
              "https://doi.org/10.1007/s11136-014-0893-3"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/j.clinthera.2014.04.011", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1008312424"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.2165/00019053-200826080-00004", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1008806799", 
              "https://doi.org/10.2165/00019053-200826080-00004"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s10529-008-9869-0", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1011145467", 
              "https://doi.org/10.1007/s10529-008-9869-0"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s10529-008-9869-0", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1011145467", 
              "https://doi.org/10.1007/s10529-008-9869-0"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1002/hec.694", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1016958508"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/j.pec.2008.05.010", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1018740851"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/j.jbusres.2006.11.010", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1019257410"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s40258-013-0028-y", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1019614565", 
              "https://doi.org/10.1007/s40258-013-0028-y"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s00464-014-4044-2", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1020605777", 
              "https://doi.org/10.1007/s00464-014-4044-2"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s10897-015-9872-2", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1022339150", 
              "https://doi.org/10.1007/s10897-015-9872-2"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s40271-015-0124-1", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1022822227", 
              "https://doi.org/10.1007/s40271-015-0124-1"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1002/mus.25411", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1024568635"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1002/nur.4770070304", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1027159029"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1002/nur.4770070304", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1027159029"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/j.pec.2008.07.023", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1028413297"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s10198-004-0241-6", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1029354104", 
              "https://doi.org/10.1007/s10198-004-0241-6"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s10198-004-0241-6", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1029354104", 
              "https://doi.org/10.1007/s10198-004-0241-6"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1377/hlthaff.26.3.684", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1029974096"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.2165/11589190-000000000-00000", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1030717838", 
              "https://doi.org/10.2165/11589190-000000000-00000"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/j.jval.2010.11.013", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1030781921"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1186/s13023-015-0281-2", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1030905612", 
              "https://doi.org/10.1186/s13023-015-0281-2"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1186/s13023-015-0281-2", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1030905612", 
              "https://doi.org/10.1186/s13023-015-0281-2"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1177/0272989x05278931", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1031510921"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1177/0272989x05278931", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1031510921"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1002/cpt.332", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1031582725"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1192/apt.9.3.229", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1033190195"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1097/dbp.0000000000000234", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1035193797"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1097/dbp.0000000000000234", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1035193797"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/s1474-4422(09)70271-6", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1035959019"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/j.jval.2012.08.2223", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1037115565"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s11002-009-9098-x", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1044660722", 
              "https://doi.org/10.1007/s11002-009-9098-x"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s11002-009-9098-x", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1044660722", 
              "https://doi.org/10.1007/s11002-009-9098-x"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s11002-009-9098-x", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1044660722", 
              "https://doi.org/10.1007/s11002-009-9098-x"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1002/hec.1697", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1050265858"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1023/a:1012999205376", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1050752619", 
              "https://doi.org/10.1023/a:1012999205376"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "sg:pub.10.1007/s40271-014-0048-1", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1051946115", 
              "https://doi.org/10.1007/s40271-014-0048-1"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.2307/1910997", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1069639310"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://app.dimensions.ai/details/publication/pub.1076827614", 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1016/j.jval.2017.07.001", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1091083824"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.2147/ppa.s139471", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1092139353"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1080/03007995.2018.1456414", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1101679664"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1080/03007995.2018.1456414", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1101679664"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.2147/ppa.s153875", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1103759888"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1177/002224379303000303", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1110841776"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1177/002224379303000303", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1110841776"
            ], 
            "type": "CreativeWork"
          }, 
          {
            "id": "https://doi.org/10.1177/002224379303000303", 
            "sameAs": [
              "https://app.dimensions.ai/details/publication/pub.1110841776"
            ], 
            "type": "CreativeWork"
          }
        ], 
        "datePublished": "2019-04", 
        "datePublishedReg": "2019-04-01", 
        "description": "BACKGROUND: Best-worst scaling methods have been used in several Duchenne and Becker muscular dystrophy (DBMD) studies to quantify patient and caregiver priorities and preferences and promote patient-focused drug development (PFDD). We sought to assess the extent to which different members of the DBMD community would accept a discrete-choice experiment (DCE) that incorporates uncertainty regarding individual-level benefit.\nMETHODS: A community advisory board encouraged the development and testing of a DCE to further examine treatment preferences in DBMD and to facilitate the inclusion of a policy-relevant uncertainty attribute. The DCE assessed preferences across a primary outcome (muscle strength) and several risks (uncertainty regarding treatment benefit, kidney damage risk, and fracture risk). The single instrument was tested among adult patients, caregivers, and professionals at the national Parent Project Muscular Dystrophy annual meeting. The DCE was analyzed using conditional logit. Instrument acceptability was evaluated using a previously developed set of questions assessing ease of understanding and answering, and if answers reflected the respondents' real preferences. We proposed a 75% agreement rate as a threshold of acceptability, and used a Z score to assess if this was met, exceeded, or rejected.\nRESULTS: A total of 161 people completed the survey including 9 patients, 87 caregivers, and 65 professionals. Patients reported high acceptability across all evaluation items (p values\u2009>\u20090.21). Caregivers and professionals exceeded the benchmark of acceptability on understanding and reflecting real preferences (p\u2009<\u20090.001). Professionals met the benchmark (p\u2009=\u20090.08) for ease of answering, but caregivers did not (p\u2009<\u20090.01). DCE results demonstrated that\u00a0all groups made meaningful trade-offs, with patients being less tolerant of risks than either caregivers or professionals (p\u2009<\u20090.001), and with no significant difference between caregivers and professionals (p\u2009=\u20090.46).\nCONCLUSIONS: This study demonstrates the acceptable application of a single instrument across a multi-stakeholder population that used a complex preference method and included a policy-relevant uncertainty variable. Ease of answering was lowest among caregivers, but a post-hoc analysis revealed that it was most difficult for those with children under the age of 10, while those with older children met the threshold. The success of this study has laid the foundation for a global study of DBMD preferences using this method.", 
        "genre": "research_article", 
        "id": "sg:pub.10.1007/s40271-018-0330-8", 
        "inLanguage": [
          "en"
        ], 
        "isAccessibleForFree": false, 
        "isPartOf": [
          {
            "id": "sg:journal.1038760", 
            "issn": [
              "1178-1653", 
              "1178-1661"
            ], 
            "name": "The Patient - Patient-Centered Outcomes Research", 
            "type": "Periodical"
          }, 
          {
            "issueNumber": "2", 
            "type": "PublicationIssue"
          }, 
          {
            "type": "PublicationVolume", 
            "volumeNumber": "12"
          }
        ], 
        "name": "How Do Members of the Duchenne and Becker Muscular Dystrophy Community Perceive a Discrete-Choice Experiment Incorporating Uncertain Treatment Benefit? An Application of Research as an Event", 
        "pagination": "247-257", 
        "productId": [
          {
            "name": "readcube_id", 
            "type": "PropertyValue", 
            "value": [
              "bdcb229b119c544dfb6ffff82374bab635f18bca143183306278bb243b2cfeba"
            ]
          }, 
          {
            "name": "pubmed_id", 
            "type": "PropertyValue", 
            "value": [
              "30259384"
            ]
          }, 
          {
            "name": "nlm_unique_id", 
            "type": "PropertyValue", 
            "value": [
              "101309314"
            ]
          }, 
          {
            "name": "doi", 
            "type": "PropertyValue", 
            "value": [
              "10.1007/s40271-018-0330-8"
            ]
          }, 
          {
            "name": "dimensions_id", 
            "type": "PropertyValue", 
            "value": [
              "pub.1107261580"
            ]
          }
        ], 
        "sameAs": [
          "https://doi.org/10.1007/s40271-018-0330-8", 
          "https://app.dimensions.ai/details/publication/pub.1107261580"
        ], 
        "sdDataset": "articles", 
        "sdDatePublished": "2019-04-11T10:59", 
        "sdLicense": "https://scigraph.springernature.com/explorer/license/", 
        "sdPublisher": {
          "name": "Springer Nature - SN SciGraph project", 
          "type": "Organization"
        }, 
        "sdSource": "s3://com-uberresearch-data-dimensions-target-20181106-alternative/cleanup/v134/2549eaecd7973599484d7c17b260dba0a4ecb94b/merge/v9/a6c9fde33151104705d4d7ff012ea9563521a3ce/jats-lookup/v90/0000000352_0000000352/records_60335_00000003.jsonl", 
        "type": "ScholarlyArticle", 
        "url": "https://link.springer.com/10.1007%2Fs40271-018-0330-8"
      }
    ]
     

    Download the RDF metadata as:  json-ld nt turtle xml License info

    HOW TO GET THIS DATA PROGRAMMATICALLY:

    JSON-LD is a popular format for linked data which is fully compatible with JSON.

    curl -H 'Accept: application/ld+json' 'https://scigraph.springernature.com/pub.10.1007/s40271-018-0330-8'

    N-Triples is a line-based linked data format ideal for batch operations.

    curl -H 'Accept: application/n-triples' 'https://scigraph.springernature.com/pub.10.1007/s40271-018-0330-8'

    Turtle is a human-readable linked data format.

    curl -H 'Accept: text/turtle' 'https://scigraph.springernature.com/pub.10.1007/s40271-018-0330-8'

    RDF/XML is a standard XML format for linked data.

    curl -H 'Accept: application/rdf+xml' 'https://scigraph.springernature.com/pub.10.1007/s40271-018-0330-8'


     

    This table displays all metadata directly associated to this object as RDF triples.

    243 TRIPLES      21 PREDICATES      68 URIs      21 LITERALS      9 BLANK NODES

    Subject Predicate Object
    1 sg:pub.10.1007/s40271-018-0330-8 schema:about anzsrc-for:14
    2 anzsrc-for:1402
    3 schema:author N7552072d43c74f8e89be83e332f1f3da
    4 schema:citation sg:pub.10.1007/s00464-014-4044-2
    5 sg:pub.10.1007/s10198-004-0241-6
    6 sg:pub.10.1007/s10529-008-9869-0
    7 sg:pub.10.1007/s10897-015-9872-2
    8 sg:pub.10.1007/s11002-009-9098-x
    9 sg:pub.10.1007/s11136-014-0893-3
    10 sg:pub.10.1007/s40258-013-0028-y
    11 sg:pub.10.1007/s40271-014-0048-1
    12 sg:pub.10.1007/s40271-014-0104-x
    13 sg:pub.10.1007/s40271-015-0124-1
    14 sg:pub.10.1023/a:1012999205376
    15 sg:pub.10.1186/s13023-015-0281-2
    16 sg:pub.10.2165/00019053-200826080-00004
    17 sg:pub.10.2165/11589190-000000000-00000
    18 https://app.dimensions.ai/details/publication/pub.1076827614
    19 https://doi.org/10.1002/cpt.332
    20 https://doi.org/10.1002/hec.1697
    21 https://doi.org/10.1002/hec.694
    22 https://doi.org/10.1002/mus.25411
    23 https://doi.org/10.1002/nur.4770070304
    24 https://doi.org/10.1016/j.clinthera.2014.04.011
    25 https://doi.org/10.1016/j.jbusres.2006.11.010
    26 https://doi.org/10.1016/j.jval.2010.11.013
    27 https://doi.org/10.1016/j.jval.2012.08.2223
    28 https://doi.org/10.1016/j.jval.2016.02.014
    29 https://doi.org/10.1016/j.jval.2017.07.001
    30 https://doi.org/10.1016/j.ncl.2014.05.002
    31 https://doi.org/10.1016/j.pec.2008.05.010
    32 https://doi.org/10.1016/j.pec.2008.07.023
    33 https://doi.org/10.1016/s1474-4422(09)70271-6
    34 https://doi.org/10.1080/03007995.2018.1456414
    35 https://doi.org/10.1097/dbp.0000000000000234
    36 https://doi.org/10.1177/002224379303000303
    37 https://doi.org/10.1177/0272989x05278931
    38 https://doi.org/10.1192/apt.9.3.229
    39 https://doi.org/10.1377/hlthaff.26.3.684
    40 https://doi.org/10.2147/ppa.s139471
    41 https://doi.org/10.2147/ppa.s153875
    42 https://doi.org/10.2307/1910997
    43 schema:datePublished 2019-04
    44 schema:datePublishedReg 2019-04-01
    45 schema:description BACKGROUND: Best-worst scaling methods have been used in several Duchenne and Becker muscular dystrophy (DBMD) studies to quantify patient and caregiver priorities and preferences and promote patient-focused drug development (PFDD). We sought to assess the extent to which different members of the DBMD community would accept a discrete-choice experiment (DCE) that incorporates uncertainty regarding individual-level benefit. METHODS: A community advisory board encouraged the development and testing of a DCE to further examine treatment preferences in DBMD and to facilitate the inclusion of a policy-relevant uncertainty attribute. The DCE assessed preferences across a primary outcome (muscle strength) and several risks (uncertainty regarding treatment benefit, kidney damage risk, and fracture risk). The single instrument was tested among adult patients, caregivers, and professionals at the national Parent Project Muscular Dystrophy annual meeting. The DCE was analyzed using conditional logit. Instrument acceptability was evaluated using a previously developed set of questions assessing ease of understanding and answering, and if answers reflected the respondents' real preferences. We proposed a 75% agreement rate as a threshold of acceptability, and used a Z score to assess if this was met, exceeded, or rejected. RESULTS: A total of 161 people completed the survey including 9 patients, 87 caregivers, and 65 professionals. Patients reported high acceptability across all evaluation items (p values > 0.21). Caregivers and professionals exceeded the benchmark of acceptability on understanding and reflecting real preferences (p < 0.001). Professionals met the benchmark (p = 0.08) for ease of answering, but caregivers did not (p < 0.01). DCE results demonstrated that all groups made meaningful trade-offs, with patients being less tolerant of risks than either caregivers or professionals (p < 0.001), and with no significant difference between caregivers and professionals (p = 0.46). CONCLUSIONS: This study demonstrates the acceptable application of a single instrument across a multi-stakeholder population that used a complex preference method and included a policy-relevant uncertainty variable. Ease of answering was lowest among caregivers, but a post-hoc analysis revealed that it was most difficult for those with children under the age of 10, while those with older children met the threshold. The success of this study has laid the foundation for a global study of DBMD preferences using this method.
    46 schema:genre research_article
    47 schema:inLanguage en
    48 schema:isAccessibleForFree false
    49 schema:isPartOf N97f25a030c034e518c68eeba1bae6440
    50 Nc441cd51c23f4ba2814359eb46a240a4
    51 sg:journal.1038760
    52 schema:name How Do Members of the Duchenne and Becker Muscular Dystrophy Community Perceive a Discrete-Choice Experiment Incorporating Uncertain Treatment Benefit? An Application of Research as an Event
    53 schema:pagination 247-257
    54 schema:productId N1118d2e90edc48cbbda86443de16de20
    55 N3c1605d4ff72412cad7816d2f1aa940c
    56 N48d4951dc5ff4ff5b763eb9496ae0fd3
    57 Nab949c5fc9d44ca080dff2a5dd33acf9
    58 Nb2c2fee4d40845d497b67824b565d942
    59 schema:sameAs https://app.dimensions.ai/details/publication/pub.1107261580
    60 https://doi.org/10.1007/s40271-018-0330-8
    61 schema:sdDatePublished 2019-04-11T10:59
    62 schema:sdLicense https://scigraph.springernature.com/explorer/license/
    63 schema:sdPublisher N63c3cae7881943ec9c4e1828f75bbdf2
    64 schema:url https://link.springer.com/10.1007%2Fs40271-018-0330-8
    65 sgo:license sg:explorer/license/
    66 sgo:sdDataset articles
    67 rdf:type schema:ScholarlyArticle
    68 N1118d2e90edc48cbbda86443de16de20 schema:name dimensions_id
    69 schema:value pub.1107261580
    70 rdf:type schema:PropertyValue
    71 N195cb092f173466a896265459f0f0d19 rdf:first sg:person.015214720167.14
    72 rdf:rest N3f79a827c3ea408290785b4685fe5fd9
    73 N3c1605d4ff72412cad7816d2f1aa940c schema:name nlm_unique_id
    74 schema:value 101309314
    75 rdf:type schema:PropertyValue
    76 N3f79a827c3ea408290785b4685fe5fd9 rdf:first sg:person.0646522326.37
    77 rdf:rest Nb1dc2a7eec714222a2bbfa72f02e5a0e
    78 N48d4951dc5ff4ff5b763eb9496ae0fd3 schema:name doi
    79 schema:value 10.1007/s40271-018-0330-8
    80 rdf:type schema:PropertyValue
    81 N63c3cae7881943ec9c4e1828f75bbdf2 schema:name Springer Nature - SN SciGraph project
    82 rdf:type schema:Organization
    83 N7552072d43c74f8e89be83e332f1f3da rdf:first sg:person.0744135726.94
    84 rdf:rest Nfecf0872fb554e80aefd9c01d0b380bc
    85 N97f25a030c034e518c68eeba1bae6440 schema:volumeNumber 12
    86 rdf:type schema:PublicationVolume
    87 Nab949c5fc9d44ca080dff2a5dd33acf9 schema:name readcube_id
    88 schema:value bdcb229b119c544dfb6ffff82374bab635f18bca143183306278bb243b2cfeba
    89 rdf:type schema:PropertyValue
    90 Nb1dc2a7eec714222a2bbfa72f02e5a0e rdf:first sg:person.0654364140.12
    91 rdf:rest rdf:nil
    92 Nb2c2fee4d40845d497b67824b565d942 schema:name pubmed_id
    93 schema:value 30259384
    94 rdf:type schema:PropertyValue
    95 Nc441cd51c23f4ba2814359eb46a240a4 schema:issueNumber 2
    96 rdf:type schema:PublicationIssue
    97 Nf5a75d41ce3b44008417b875a3b31b84 rdf:first sg:person.016640247455.87
    98 rdf:rest N195cb092f173466a896265459f0f0d19
    99 Nfecf0872fb554e80aefd9c01d0b380bc rdf:first sg:person.010463531012.02
    100 rdf:rest Nf5a75d41ce3b44008417b875a3b31b84
    101 anzsrc-for:14 schema:inDefinedTermSet anzsrc-for:
    102 schema:name Economics
    103 rdf:type schema:DefinedTerm
    104 anzsrc-for:1402 schema:inDefinedTermSet anzsrc-for:
    105 schema:name Applied Economics
    106 rdf:type schema:DefinedTerm
    107 sg:journal.1038760 schema:issn 1178-1653
    108 1178-1661
    109 schema:name The Patient - Patient-Centered Outcomes Research
    110 rdf:type schema:Periodical
    111 sg:person.010463531012.02 schema:affiliation https://www.grid.ac/institutes/grid.21107.35
    112 schema:familyName Tsai
    113 schema:givenName Jui-Hua
    114 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.010463531012.02
    115 rdf:type schema:Person
    116 sg:person.015214720167.14 schema:affiliation https://www.grid.ac/institutes/grid.21107.35
    117 schema:familyName Crossnohere
    118 schema:givenName Norah L.
    119 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.015214720167.14
    120 rdf:type schema:Person
    121 sg:person.016640247455.87 schema:affiliation https://www.grid.ac/institutes/grid.21107.35
    122 schema:familyName Janssen
    123 schema:givenName Ellen
    124 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.016640247455.87
    125 rdf:type schema:Person
    126 sg:person.0646522326.37 schema:affiliation https://www.grid.ac/institutes/grid.437213.0
    127 schema:familyName Fischer
    128 schema:givenName Ryan
    129 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0646522326.37
    130 rdf:type schema:Person
    131 sg:person.0654364140.12 schema:affiliation https://www.grid.ac/institutes/grid.62562.35
    132 schema:familyName Peay
    133 schema:givenName Holly
    134 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0654364140.12
    135 rdf:type schema:Person
    136 sg:person.0744135726.94 schema:affiliation https://www.grid.ac/institutes/grid.21107.35
    137 schema:familyName Bridges
    138 schema:givenName John F. P.
    139 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0744135726.94
    140 rdf:type schema:Person
    141 sg:pub.10.1007/s00464-014-4044-2 schema:sameAs https://app.dimensions.ai/details/publication/pub.1020605777
    142 https://doi.org/10.1007/s00464-014-4044-2
    143 rdf:type schema:CreativeWork
    144 sg:pub.10.1007/s10198-004-0241-6 schema:sameAs https://app.dimensions.ai/details/publication/pub.1029354104
    145 https://doi.org/10.1007/s10198-004-0241-6
    146 rdf:type schema:CreativeWork
    147 sg:pub.10.1007/s10529-008-9869-0 schema:sameAs https://app.dimensions.ai/details/publication/pub.1011145467
    148 https://doi.org/10.1007/s10529-008-9869-0
    149 rdf:type schema:CreativeWork
    150 sg:pub.10.1007/s10897-015-9872-2 schema:sameAs https://app.dimensions.ai/details/publication/pub.1022339150
    151 https://doi.org/10.1007/s10897-015-9872-2
    152 rdf:type schema:CreativeWork
    153 sg:pub.10.1007/s11002-009-9098-x schema:sameAs https://app.dimensions.ai/details/publication/pub.1044660722
    154 https://doi.org/10.1007/s11002-009-9098-x
    155 rdf:type schema:CreativeWork
    156 sg:pub.10.1007/s11136-014-0893-3 schema:sameAs https://app.dimensions.ai/details/publication/pub.1007355674
    157 https://doi.org/10.1007/s11136-014-0893-3
    158 rdf:type schema:CreativeWork
    159 sg:pub.10.1007/s40258-013-0028-y schema:sameAs https://app.dimensions.ai/details/publication/pub.1019614565
    160 https://doi.org/10.1007/s40258-013-0028-y
    161 rdf:type schema:CreativeWork
    162 sg:pub.10.1007/s40271-014-0048-1 schema:sameAs https://app.dimensions.ai/details/publication/pub.1051946115
    163 https://doi.org/10.1007/s40271-014-0048-1
    164 rdf:type schema:CreativeWork
    165 sg:pub.10.1007/s40271-014-0104-x schema:sameAs https://app.dimensions.ai/details/publication/pub.1002431225
    166 https://doi.org/10.1007/s40271-014-0104-x
    167 rdf:type schema:CreativeWork
    168 sg:pub.10.1007/s40271-015-0124-1 schema:sameAs https://app.dimensions.ai/details/publication/pub.1022822227
    169 https://doi.org/10.1007/s40271-015-0124-1
    170 rdf:type schema:CreativeWork
    171 sg:pub.10.1023/a:1012999205376 schema:sameAs https://app.dimensions.ai/details/publication/pub.1050752619
    172 https://doi.org/10.1023/a:1012999205376
    173 rdf:type schema:CreativeWork
    174 sg:pub.10.1186/s13023-015-0281-2 schema:sameAs https://app.dimensions.ai/details/publication/pub.1030905612
    175 https://doi.org/10.1186/s13023-015-0281-2
    176 rdf:type schema:CreativeWork
    177 sg:pub.10.2165/00019053-200826080-00004 schema:sameAs https://app.dimensions.ai/details/publication/pub.1008806799
    178 https://doi.org/10.2165/00019053-200826080-00004
    179 rdf:type schema:CreativeWork
    180 sg:pub.10.2165/11589190-000000000-00000 schema:sameAs https://app.dimensions.ai/details/publication/pub.1030717838
    181 https://doi.org/10.2165/11589190-000000000-00000
    182 rdf:type schema:CreativeWork
    183 https://app.dimensions.ai/details/publication/pub.1076827614 schema:CreativeWork
    184 https://doi.org/10.1002/cpt.332 schema:sameAs https://app.dimensions.ai/details/publication/pub.1031582725
    185 rdf:type schema:CreativeWork
    186 https://doi.org/10.1002/hec.1697 schema:sameAs https://app.dimensions.ai/details/publication/pub.1050265858
    187 rdf:type schema:CreativeWork
    188 https://doi.org/10.1002/hec.694 schema:sameAs https://app.dimensions.ai/details/publication/pub.1016958508
    189 rdf:type schema:CreativeWork
    190 https://doi.org/10.1002/mus.25411 schema:sameAs https://app.dimensions.ai/details/publication/pub.1024568635
    191 rdf:type schema:CreativeWork
    192 https://doi.org/10.1002/nur.4770070304 schema:sameAs https://app.dimensions.ai/details/publication/pub.1027159029
    193 rdf:type schema:CreativeWork
    194 https://doi.org/10.1016/j.clinthera.2014.04.011 schema:sameAs https://app.dimensions.ai/details/publication/pub.1008312424
    195 rdf:type schema:CreativeWork
    196 https://doi.org/10.1016/j.jbusres.2006.11.010 schema:sameAs https://app.dimensions.ai/details/publication/pub.1019257410
    197 rdf:type schema:CreativeWork
    198 https://doi.org/10.1016/j.jval.2010.11.013 schema:sameAs https://app.dimensions.ai/details/publication/pub.1030781921
    199 rdf:type schema:CreativeWork
    200 https://doi.org/10.1016/j.jval.2012.08.2223 schema:sameAs https://app.dimensions.ai/details/publication/pub.1037115565
    201 rdf:type schema:CreativeWork
    202 https://doi.org/10.1016/j.jval.2016.02.014 schema:sameAs https://app.dimensions.ai/details/publication/pub.1003756617
    203 rdf:type schema:CreativeWork
    204 https://doi.org/10.1016/j.jval.2017.07.001 schema:sameAs https://app.dimensions.ai/details/publication/pub.1091083824
    205 rdf:type schema:CreativeWork
    206 https://doi.org/10.1016/j.ncl.2014.05.002 schema:sameAs https://app.dimensions.ai/details/publication/pub.1001875634
    207 rdf:type schema:CreativeWork
    208 https://doi.org/10.1016/j.pec.2008.05.010 schema:sameAs https://app.dimensions.ai/details/publication/pub.1018740851
    209 rdf:type schema:CreativeWork
    210 https://doi.org/10.1016/j.pec.2008.07.023 schema:sameAs https://app.dimensions.ai/details/publication/pub.1028413297
    211 rdf:type schema:CreativeWork
    212 https://doi.org/10.1016/s1474-4422(09)70271-6 schema:sameAs https://app.dimensions.ai/details/publication/pub.1035959019
    213 rdf:type schema:CreativeWork
    214 https://doi.org/10.1080/03007995.2018.1456414 schema:sameAs https://app.dimensions.ai/details/publication/pub.1101679664
    215 rdf:type schema:CreativeWork
    216 https://doi.org/10.1097/dbp.0000000000000234 schema:sameAs https://app.dimensions.ai/details/publication/pub.1035193797
    217 rdf:type schema:CreativeWork
    218 https://doi.org/10.1177/002224379303000303 schema:sameAs https://app.dimensions.ai/details/publication/pub.1110841776
    219 rdf:type schema:CreativeWork
    220 https://doi.org/10.1177/0272989x05278931 schema:sameAs https://app.dimensions.ai/details/publication/pub.1031510921
    221 rdf:type schema:CreativeWork
    222 https://doi.org/10.1192/apt.9.3.229 schema:sameAs https://app.dimensions.ai/details/publication/pub.1033190195
    223 rdf:type schema:CreativeWork
    224 https://doi.org/10.1377/hlthaff.26.3.684 schema:sameAs https://app.dimensions.ai/details/publication/pub.1029974096
    225 rdf:type schema:CreativeWork
    226 https://doi.org/10.2147/ppa.s139471 schema:sameAs https://app.dimensions.ai/details/publication/pub.1092139353
    227 rdf:type schema:CreativeWork
    228 https://doi.org/10.2147/ppa.s153875 schema:sameAs https://app.dimensions.ai/details/publication/pub.1103759888
    229 rdf:type schema:CreativeWork
    230 https://doi.org/10.2307/1910997 schema:sameAs https://app.dimensions.ai/details/publication/pub.1069639310
    231 rdf:type schema:CreativeWork
    232 https://www.grid.ac/institutes/grid.21107.35 schema:alternateName Johns Hopkins University
    233 schema:name Department of Health Policy and Management, The Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe St, 21205, Baltimore, MD, USA
    234 Department of Health, Behavior, and Society, The Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe St, 21205, Baltimore, MD, USA
    235 rdf:type schema:Organization
    236 https://www.grid.ac/institutes/grid.437213.0 schema:alternateName Parent Project Muscular Dystrophy
    237 schema:name Parent Project Muscular Dystrophy, 401 Hackensack Avenue, 9th Floor, 07601, Hackensack, NJ, USA
    238 rdf:type schema:Organization
    239 https://www.grid.ac/institutes/grid.62562.35 schema:alternateName RTI International
    240 schema:name Department of Health, Behavior, and Society, The Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe St, 21205, Baltimore, MD, USA
    241 Parent Project Muscular Dystrophy, 401 Hackensack Avenue, 9th Floor, 07601, Hackensack, NJ, USA
    242 RTI International, 701 13th St NW #750, 20005, Washington, DC, USA
    243 rdf:type schema:Organization
     




    Preview window. Press ESC to close (or click here)


    ...