Learning outcomes

Introduction to Patient reported outcomes

Goals

  • Gaining a global understanding of the importance, development, implementation, interpretation ofPatient Reported Outcomes.

Content

Introduction to Patient-reported outcomes (PRO)

PRO development

PRO validation

PRO implementation

PRO interpretation

Exercices

NA

Assessment method

  • Course participation, presentation and discussion of a peer-reviewed manuscript, homework (~ 5-10 pages of reflection on a theme discussed during the course)

    The exact modalities of the evaluation are likely to be modified during the preparation of the examination schedules, depending on the practical constraints with which the faculty administration may be confronted, or in the event of illness / force majeure / encroachment with an internship, or because of the health situation related to the coronavirus.

Sources, references and any support material

  • USA. Food and Drug Administration. (2009) Guidance for Industry. Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Available at: https://www.fda.gov/media/77832/download
  • USA.  Food and Drug Administration. (2020 ) Patient-Focused Drug Development: Collecting Comprehensive and Representative Input. Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drug-development-collecting-comprehensive-and-representative-input
  • USA. Food and Drug Administration. (2018) Methods to Identify What is Important to Patients & Select, Develop or Modify Fit-for-Purpose Clinical Outcomes Assessments. Available at: https://www.fda.gov/media/116277/download
  •  USA. Food and Drug Administration. (2019) Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision-Making. Available at: https://www.fda.gov/media/132505/download
  • USA. Food and Drug Administration. (2019) Clinical Outcome Assessment Compendium. Available at: https://www.fda.gov/drugs/development-resources/clinical-outcome-assessment-compendium
  • European Medicines Agency. (2016) The use of patient-reported outcome (PRO) measures in oncology studies. Available at: https://www.ema.europa.eu/en/documents/other/appendix-2-guideline-evalu…
  • International Society for Quality of Life Research. (2015) User’s Guide to Implementing Patient-Reported Outcomes Assessment in Clinical Practice. Available at: https://www.isoqol.org/wp-content/uploads/2019/09/2015UsersGuide-Version2.pdf
  • Walton MK, Powers JH III, Hobart J, et al. Clinical outcome assessments: a conceptual foundation – report of the ISPOR Clinical Outcomes Assessment Emerging Good Practices Task Force. Value Health. 2015;18(6):741-752.
  • Anatchkova, M., Donelson, S. M., Skalicky, A. M., McHorney, C. A., Jagun, D., & Whiteley, J. (2018). Exploring the implementation of patient-reported outcome measures in cancer care: need for more real-world evidence results in the peer reviewed literature. Journal of patient-reported outcomes, 2(1), 64. https://doi.org/10.1186/s41687-018-0091-0
  • Deane, K., Delbecque, L., Gorbenko, O. et al. on behalf of the PFMD Patient Engagement Meta-framework Co-creation Team. Co-creation of patient engagement quality guidance for medicines development: an international multi stakeholder initiative. BMJ Innovations 2019;5:43-55.
  • Patrick DL, Burke L, Gwaltney CJ, et al. Content validity—establishing and reporting the evidence in newly-developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: Part I – eliciting concepts for a new PRO instrument. Value Health 2011;14:967–77
  • Donald L. Patrick, et al. Content Validity—Establishing and Reporting the Evidence in Newly Developed Patient-Reported Outcomes (PRO) Instruments for Medical Product Evaluation: ISPOR PRO Good Research Practices Task Force Report: Part 2—Assessing Respondent Understanding VALUE IN HEALTH 14 (2011) 978 –988
  •  Clinician-Reported Outcome (ClinRO) Assessments of Treatment Benefit: Report of the ISPOR Clinical Outcome Assessment Emerging Good Practices Task Force, January 2017 Value in Health 20(1)
  • Recommendations on Evidence Needed to Support Measurement Equivalence between Electronic and Paper-Based Patient-Reported Outcome (PRO) Measures: ISPOR ePRO Good Research Practices Task Force Report Stephen Joel Coons, PhD,1 Chad J. Gwaltney, PhD,2 Ron D. Hays, PhD,3 J. Jason Lundy, PhD,4 Jeff A. Sloan, PhD,5 Dennis A. Revicki, PhD,6 William R. Lenderking, PhD,7 David Cella, PhD,8 Ethan Basch, MD, MSc,9 on behalf of the ISPOR ePRO Task Force. VA LUE IN HEA LTH 2009
  • Calvaert et al. Reporting of Patient-Reported Outcomes in Randomized Trials The CONSORT PRO Extension. JAMA. 2013;309(8):814-822
  • Matthew Reaney, Elizabeth N. Bush, Bradley Curtis, Peter Black, Kate van Brunt, Chad Gwaltney. One Programme, Four Stakeholders: An Overview of the Utilisation of Patient-Reported Outcomes in Intervention Development to Meet the Needs of Regulators, Payers, Healthcare Professionals and Patients Pharm Med (2015) 29:69–78

Language of instruction

French