Jeff A. Sloan, Ph.D. Mayo Clinic, Rochester, MN, USA. Clinical Significance for Quality of Life Endpoints in Clinical Trials. FDA/Industry Statistics Workshop Washington, September 16, 2005. Primary goal: advance the state of the science to help cancer patient QOL soar.
Jeff A. Sloan, Ph.D.
Mayo Clinic, Rochester, MN, USA
Clinical Significance for Quality of Life Endpoints in Clinical Trials
FDA/Industry Statistics Workshop
Washington, September 16, 2005
Primary goal: advance the state of the science to help cancer patient QOL soar
It takes a certain amount of bravery to work in QOL research
Science is a candle in the dark
- Carl Sagan
We will use the candle of science to improve the QOL of cancer patients
How do you determine
the clinical significance
of QOL assessments?
What is a clinically meaningful QOL burden?
No single statistical decision rule or procedure can take the place of well-reasoned consideration of all aspects of the data by a group of concerned, competent, and experienced persons with a wide range of scientific backgrounds and points of view.
If it looks like a duck, sounds like a duck, and walks like a duck, the odds of it being a worm or an elephant in a clever disguise are small in the extreme.
(Sloan, J Chronic Obs. Pul. Dis. 2: 57-62, 2005.)
Presenting global solutions is always interesting
The MID method in one slide
= 16.7% of theoretical range
small, moderate, large effect(0.2, 0.5, 0.8 SD shift)
Norman GR, Sloan JA, Wyrwich KW. Expert Review of Pharmacoeconomics and Outcomes Research Sept 2004; 4(5): 515 – 519
Sloan JA, Cella D, Hays R. J Clin Epidemiol (in press).
New ideas have enabled us to make advances in QOL science