1 / 22

Progress Report on the Patient Reported Outcomes Harmonization Team

Progress Report on the Patient Reported Outcomes Harmonization Team. Jean Paul Gagnon, PhD Aventis Pharmaceuticals Inc. Objectives. Discuss Historical Development Describe Forum Outcomes Outline Future Activities. Objectives. Discuss Historical Developments.

thora
Download Presentation

Progress Report on the Patient Reported Outcomes Harmonization Team

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. Progress Report on the Patient Reported Outcomes Harmonization Team Jean Paul Gagnon, PhD Aventis Pharmaceuticals Inc.

  2. Objectives • Discuss Historical Development • Describe Forum Outcomes • Outline Future Activities

  3. Objectives • Discuss Historical Developments

  4. Who started the PRO Harmonization Group? • European Regulatory Issues on Quality-of-Life Assessment (ERIQA) • International Society of Pharmacoeconomic and Outcomes Research (ISPOR) • International Society of Quality of Life (ISOQOL) • Pharmaceutical Research and Manufacturers of America (PhRMA) Health Outcomes Committee (HOC)

  5. What is its mission? To harmonize Patient Reported Outcome issues used in drug development and Evaluation.

  6. What are its objectives? • Clarify areas of concern or confusion about PRO evaluation • Explain the added value of PRO outcomes among all key players, i.e., academics, regulators, industry researchers, prescribers and patients • Open and maintain communication between key players • Disseminate meeting outcomes, i.e., to publish papers, to participate in conferences

  7. Objectives • Discuss Historical Development • Describe Forum Outcomes

  8. Forums • Three Harmonization Forums held • March 31, 2000 • Sept. 14, 2000 • February 16, 2001 • Representatives from ERIQA, ISOQOL, ISPOR, PhRMA HOC and FDA observers

  9. March 2000 Meeting Objectives • Discuss problematic issues • Harmonize recommendations proposed by each organization • Recommend solutions when none exist • Proposed future steps to achieve consensus on problematic issues

  10. Meeting Outcomes • Consensus and areas of disagreement were identified in four areas: Concept, Study Design, Interpretation and Conditions for Claims* • Group agreed to continue discussion and form a coordination group with representatives from the four organizations to organize future meeting • Real issue “does outcomes research provide added value” *Report available on-line: www.mapi-research-inst/research/report.pdf

  11. Impact of Outcomes Data • Can the added-value of the outcome be demonstrated, i.e., what does the outcome provide beyond traditional measures? • Does the subjective impact of treatment provide value over observer-based assessments or simple symptom scales? • Does the measure capture information that is missed with traditional measures?

  12. September 2000 Meeting Outcomes • Conceptual framework was broadened to Patient Based Assessment (PBA) which was changed to Patient Reported Outcomes (PRO) for clarity • Decision made to continue discussions and schedule meeting with FDA and EMEA representatives

  13. February 2001 Meeting Objectives • Clarify aspects and components of PRO evaluation • Discuss where PRO can add value to clinical endpoints • Suggest use of this information within labeling and promotional claims

  14. Take Away Points • Patient has a unique voice and valuable perspective that should play a role in medical decision making • PROs can be measured in reliable and valid ways • PROs are increasingly used as efficacy endpoints in randomized controlled trials

  15. Patient Outcomes Assessment Sources and Examples Clinician - Reported Patient - Reported Physiological Caregiver - Reported For example, Global impressions Observation & tests of function For example, FEV1 HbA1c Tumor size For example, Dependency Functional status Global Impression Functional status Well-being Symptoms HRQL Satisfaction with TX Treatment adherence

  16. Areas of Agreement • Patient Reported Outcomes (PRO) evaluation is a valid concept • PRO can be operationalized • PRO claims should conform with the evidence and be pre-specified • PRO should be reported with fair balance in labeling and promotional claims

  17. “Objective” “Subjective” Exercise test versus physical functioning, r = 0.40 Wiklund I et al. Clin Cardiol 1991;14

  18. Objectives • Discuss Historical Development • Describe Forum Outcomes • Outline Future Activities

  19. Future Plans for Pro Team • Publish article on work to date, draft manuscript prepared • Develop slide presentations for conferences, e.g., ISPOR, ISOQOL, DIA, others • Supply content via website • Continue to hold forums until deemed unnecessary • Measure outcomes from Forums

  20. February 2002 Forum Agenda • Instrument development: what are the standards • Instrument selection: demonstrating hypothesis, relationships to measurement • Statistical issues: focus on handling missing data • Interpretation: Interpreting changes that are not consistent between outcomes

  21. Summary • Discussed Historical Developments • Described Meeting Outcomes • Outlined Future Activities

  22. QUESTIONSJean Paul Gagnonjean.gagnon@aventis.com 800-981-2491 Aventis Pharmaceuticals Inc.

More Related