ACTIVITIES

Activities:

ISPOR 12th Annual International Meeting Forum Presentation May 21, 2007
Putting the ‘O’ Back in ISPOR: Patient-Reported Outcomes Good Research

Description: Recently, in the US, the FDA issued a draft Guidance for the Industry on Patient-Reported Outcome (PRO) Measures and, in Europe, EMEA issued a Reflection Paper on the Regulatory Guidance for the Use of Health Related Quality of Life Measures in the Evaluation of Medicinal Products. ISPOR members have identified a need for clarity on PRO measurements. In response, ISPOR initiated 3 PRO Task Forces (TFs) on the use and modification of existing instruments and instrument language change and administration change. The ISPOR PRO Task Force Chairs will discuss issues and concerns in the development of PRO good research practices documents.
Moderators: Donald Patrick PhD , ISPOR Health Science Policy Council Member & Professor, University of Washington, Seattle, WA, USA & Patrick Marquis PhD Chair, ISPOR Patient Reported Outcomes SIG’s PRO / QOL Information in Regulatory & Health Care Decisions Working Group & Managing Director, MAPI Values, Boston, MA, USA
Use of Existing PRO Instruments and their Modification
Speaker: Margaret Rothman, PhD, Chair: Use of Existing PRO Instruments & their Modification TF and Executive Director, Health Economics, Johnson & Johnson, Raritan, NJ
Changing Culture or Language of PRO Application
Speaker: Diane Wild MSc, Chair: Changing Culture/Language of PRO Instruments TF & Director, Oxford Outcomes, Cassington, Oxford, UK
Changing Mode of Administration of PRO Instruments, including ePRO
Stephen J. Coons PhD Chair, Changing Mode of PRO Instrument Administration/ePRO TF & Professor of Pharmacy and Public Health, University of Arizona, Tucson, AZ, USA
http://www.ispor.org/research_initiatives/PROForum052007.pdf

ISPOR Ninth European Congress Forum October 30, 2006
FDA Draft Guidelines on the Development, Validation and Implementation for PRO Studies ∙ Development of ePRO Good Research Practices ∙ Cross-Cultural Translational Adaptation Working Group’s Next Steps
Moderator: Judith Barr MEd, ScD, Chair, ISPOR PRO SIG & Associate Professor and Director, National Education and Research Center for Outcomes Assessment, Northeastern University, Boston, MA, USA
Speakers: Patrick Marquis MBA, MD, Chair, ISPOR PRO / QOL Information in Regulatory and Health Decisions Working Group, Managing Director, Mapi Values, Boston, MA, USA ; Diane Wild MSc, Chair, ISPOR Cross-Cultural & Translational Adaptation Working Group & Partner, Oxford Outcomes, Oxford, UK; Isabelle Mear, Leadership Group member, ePRO Working Group and Managing Director, Director of Linguistic Validation Department, Mapi Research Institute, Lyon, FR
Description: The patients’ perspective has never been more important in medical evaluations and interventions. The FDA recently issued draft guidelines on development, validation, and implementation for PRO studies, which will have an effect on all PRO studies. During this forum the ISPOR PRO SIG -Information in Regulatory and Health Decisions Working Group will comment on the proposed FDA PRO Guidelines, the e-PRO will give an update on their development of ePRO good research practices, and the Cross-Cultural Translational Adaptation Working Group will describe their next steps.

ISPOR 11th Annual International Meeting Forum Tuesday, May 23, 2006
Patient-reported Outcomes Draft FDA Guidance
Speakers: Patrick Marquis, MD, MBA ISPOR PRO SIG PRO/QOL Information in Regulatory & Health Care Decisions Working Group Chair and Managing Director, Mapi Values, Boston, MA, USA Diane Wild MSc, Cross-Cultural & Translational Adaptation (CTA) Working Group Chair and Partner, Oxford Outcomes, Oxford, UK
Description: The ISPOR PRO SIG PRO/QOL Information in Regulatory & Health Care Decisions Working Group developed the ISPOR response to the Patient-reported Outcomes Draft FDA Guidance. The response was based on comments and suggestions received from over 50 ISPOR members. This Forum will discuss the Draft FDA Guidance’s pros and cons and our comments to address these.

ISPOR Eighth European Congress Issue Panel Presentation November 7, 2005
IP3: Submission of Patient Reported Outcomes to the EMEA and the FDA: Areas of Convergence or Divergence
Moderator: Patrick Marquis MBA, MD, Managing Director, Mapi Values, Boston, MA, USA
Panelists: Laurie Burke RPh, MPH, Director, Study Endpoints and Label Development, Food and Drug Administration, Rockville, MD, US; Giovanni Apolone MD, Head of Translational and Outcomes Research Lab, Mario Negri Institute, Milan, Italy
Issue: Discussion on FDA and EMEA patient reported outcome (PRO) submissions and reviews. Areas of convergence and divergence will be identified and discussed.
Overview: The creation of the Study Endpoints and Label Development group (FDA/CDER/OND/SEALD) three years ago and the development of a draft PRO guidance document illustrate the US regulators' commitment towards developing and maintaining consistent criteria review for PRO and other study endpoint evaluations. In the meantime, the EMEA has developed a reflection paper on health related quality of life.

The FDA and EMEA have gained experience and examples of industry-reviewer interactions which will be shared in an effort to encourage quality and consistency in the study endpoint review process. During the panel discussion, an emphasis will be put on practical experiences when submitting and assessing PRO data at different stages of the clinical program with a focus on early phases (e.g., development of hypotheses, development and validation of PRO questionnaires, strategy for analysis and interpretation). Convergences and divergences will be underlined. Although most of the recommendations when implementing PROs in global clinical development programs can be agreed upon by different regulators, issues related to definition of concepts, PRO paradigm, PRO as primary end-point, role in evaluation of treatment risk/benefit, cross-cultural issues, and clinical significance, deserve further discussion. A specific focus will be put on oncology.

ISPOR 10th Annual International Meeting Forum Tuesday, May 23, 2005
Benefits of Using ‘Real World’ Quality of Life/Patient Reported Outcomes Challenges and Limitations of ‘Real World’ Quality of Life/Patient Reported Outcomes
Moderator: Pennifer Erickson PhD, Chair PRO WG & QOL SIG and Co-founder, O.L.G.A., State College, PA, USA Speaker: Mary Cifaldi RPh, PhD Assistant Director, Abbott Laboratories, Abbott Park, IL, USA
Speaker: Richard Chapman PhD, Director, Health Economics, ValueMedics Research, Alexandria, VA
Description: Health Status and Patient Reported Outcomes/Quality of Life Measures in the ‘Real World’ Health status and quality of life measures are becoming widely used in not only in clinical research but also in studying health care delivery systems, for monitoring population health and for allocating resources. The PRO Working Group within the ISPOR's Task Force on Real World Data is beginning a dialogue on the use of PRO data for decision making by a wide range of stakeholders.