ISOQOL 2004 Symposium
"Stating the Art: Advancing Outcomes Research Methodology and Clinical Applications"
June 27-29, 2004
Boston Park Plaza Hotel
Boston, MA, USA

Plenary Presentations

Click on the presenters name to view slides from each presentation.

Plenary Session II: Outcomes Research Applied
Presenters: Gordon Guyatt/Holger Schunemann, David Osoba and Robert J. Meyer
Chair: David Osoba

Clinical Research Challenges in HRQOL Assessment
David Osoba, MD
Consultant, West Vancouver, BC, Canada

What are meaningful changes in HRQOL scores? How should meaningful changes be presented in terms that resonate with the needs of various users? Is there a role for sensitivity analyses? How can HRQOL assessment be more widely taught and implemented? These items will be discussed.

Interpreting the Results of Quality of Life Measures in Clinical Trials: The Clinicians Perspective
Gordon Guyatt, MD
Department of Clinical Epidemiology, Biostatistics and Medicine, McMaster University Health Sciences Center, Hamilton, ON, Canada

Interpretation problems for clinicians looking at the results of clinical trials are not restricted to quality of life. For instance, large cardiology trials report absolute reductions in combined endpoints such as death and myocardial infarction. Clinicians seem willing to ignore the relative frequency of deaths versus infarcts, and large infarcts versus small. As long as they are convinced events are serious, the number prevented makes results interpretable. This provides a lesson to the optimal approach to making quality of life data interpretable to clinicians. First, establish the minimal important difference. Second, report mean differences (ideally in change, and in relation to the MID). Third, choose a threshold (ideally a threshold of change, and ideally the MID) and determine the proportion of patients in both groups who meet this threshold. Finally, use these data to determine the proportion of patients who have achieved important benefit from treatment, and the associated number needed to treat (NNT).

Regulatory Considerations for Outcomes Research
Robert J. Meyer, MD
Director, Office of Drug Evaluation II, Rockville, MD, USA

In the regulatory context of new drug approvals, "outcomes" research may have a number of meanings. Typically, the FDA does not have much experience with "outcomes" assessments as a part of the primary evidence of effectiveness, but this is not always the case. This talk will first seek to clarify the terminology from the regulatory perspective and then to reflect on the use of patient-reported outcomes and other types of outcomes research from the FDA perspective. Past examples will be shared as well as will be some thoughts on future directions.