Regulatory Aspects of Quality of Life
We discuss some issues in using quality of life endpoints in studies that will lead to an application to license a product or add an indication to an already licensed product. Studies of this sort should be double blinded, randomized and use validated questionnaires. The duration of the study should be appropriate for the indication. Missing values can be a serious problem and plans for handling them should be included. Sensitivity analyses are important in this context. An example of a sensitivity analysis shows how substitutions for missing values can offer insight into the effect of these missing values. An alternative model is given to analyze data of this sort. Other analytic methods are discussed in the second part of the paper. The views and opinions expressed in this paper are those of the authors and do not necessarily represent those of the Food and Drug Administration.
KeywordsLast Observation Carry Forward Miss Data Mechanism Missingness Mechanism Miss Data Imputation Pattern Mixture Model
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