Patient and public engagement in health-related quality of life and patient-reported outcomes research: what is important and why should we care? Findings from the first ISOQOL patient engagement symposium

Haywood, Kirstie L.; Brett, Jo; Salek, Sam; Marlett, Nancy; Penman, Colin; Shklarov, Svetlana; Norris, Colleen M.; Santana, Maria; Staniszewska, Sophie

doi:10.1007/s11136-014-0796-3

Cited by 65 publications

(80 citation statements)

References 23 publications

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“…Haywood et al [4] present results of a study of QOL investigators which suggests that many of us are at a loss as to how to effectively engage with patients throughout the research process. Accordingly, a set of articles address methods that were developed or modified to facilitate patient engagement research.…”

Section: Novel Methods For Engaging Under-represented Patientsmentioning

confidence: 99%

Introduction to special section on patient engagement

Schwartz

Revicki

2015

Qual Life Res

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Section: Novel Methods For Engaging Under-represented Patientsmentioning

confidence: 99%

Introduction to special section on patient engagement

Schwartz

Revicki

2015

Qual Life Res

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“…The active involvement of patients and the public across the research process has emerged as a formal requirement for many programmes and funding agencies, including the Patient-Centred Outcomes Research Institute (PCORI) in the USA, the Canadian Institutes of Health Research (CIHR) in Canada and the National Institute for Health Research (NIHR) in the UK, as well as the EU Innovative Medicines Initiative [44]. In general, PE offers an opportunity to improve the science of PRO measure development, and to reinforce the patient centeredness of the process [44,45].…”

Section: Patient Engagementmentioning

confidence: 99%

“…In general, PE offers an opportunity to improve the science of PRO measure development, and to reinforce the patient centeredness of the process [44,45]. For example, PE may facilitate identification of topics/areas for future research, development of optimal study design, selection of appropriate outcomes/domains to include in a PRO instrument, and clarification of patients' description of symptoms.…”

Section: Patient Engagementmentioning

confidence: 99%

Streamlining the Validation of Patient Reported Outcome (PRO) Measures in Drug Regulatory Processes

Salek

Kamudoni²

2015

Pharm Med

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The context and setting in which patient-reported outcome (PRO) measures applied in drug development programmes are developed and applied are undergoing rapid changes. The emergence of social media, coupled with advancement of patient-centred methodologies, has led to more connected and empowered patients than before. Drug regulatory agencies are beginning to embrace a new role as collaborators in the drug development process, and new models for defining core outcomes in various disease areas are emerging. Technological advances and social changes have led to the wide use of personal gadgets with greater internet connectivity and computing power than most computers had a generation ago. To explore how progress in the measurement of PROs might be achieved amidst such changes, lessons were drawn from the PRO labelling claims reviewed by the US FDA following the publication of the PRO guidance in 2009. In addition, semi-structured interviews were carried out with seven experts working with drug regulatory agencies and in the pharmaceutical industry to identify major issues and possible solutions. The following recommendations are proposed: (1) that comprehensive hypotheses about the PRO concept be developed; (2) that PRO concepts/measures maintain substantive and clinical validity; (3) that the use of advanced psychometric methods be considered as the new gold standard in the development of PRO measures; (4) that psychometric properties of measures established through ''validation by application'' be enhanced; (5) that the generalizability of PRO measures be enhanced; (6) that the assessment of ability to detect change, and the interpretability of scores, be improved; and (7) that PRO guidelines by major regulatory authorities be aligned. These entail a greater transparency on the decisions taken during the PRO measure development process and greater understanding of the integration between qualitative aspects of the PRO measure and the score/measurement attributes. Key PointsMoving the science of patient-reported outcome (PRO) measurement within drug regulatory settings forward requires greater transparency on the decisions taken during the PRO measure development process and greater understanding of the integration between qualitative aspects of the PRO measure and the score/measurement attributes.Enhancing the validation of PROs will require ensuring that PRO measures have a strong conceptual foundation, that their clinical meaningfulness and relevance is adequately demonstrated, that a high degree of precision is established and that measurement equivalence across groups is determined. Furthermore, there is an urgent need for alignment in guidelines on PROs across medicines regulatory agencies supporting generalizability of measures.Electronic supplementary material The online version of this article (

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“…The theme of Patient Engagement has exploded over the last several years, as numerous global initiatives have sought to engage patients not just as research participants but as partners in the design and implementation of the research itself [6,7]. ISOQOL has embraced the inclusion of patients as partners in research and has advanced this topic through the activities of the Patient Engagement Special Interest Group [8]. Patient Engagement is not a new concept, as it is at the foundation of patient-reported outcomes and community-based participatory research.…”

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confidence: 99%