Efficacy Data Evaluation Tools

The basic framework for the design and analysis of efficacy evaluation trials is provided by EPPO standard PP 1/152(4). The focus of this standard is to enable a reliable, scientifically and statistically sound data collection and evaluation.

In practise, the choice of the efficacy data evaluation tools is often influenced by a variety of factors, such as the availability of suitable software or data sets and even economic considerations, such as the investment to be made for the data evaluation. The latter, however, can offer an added value, since investing in the use of statistically suitable make-to-measure tools and methods  positively effects the quality of the results of an analysis, prevents unneccessary study costs as much as possible, and consequently helps to present convincing data with the highest efficacy rates possible in a BAD or dRR.

Naturally, the reduction in the number of efficacy trial series and the decline of investments in studies does not start with data evaluation. The starting point is the layout of a study program and the design of the individual study. Already at this level, appropriate statistical tools play a very important role. For example, “spare treatments” of dose justification trials, required by statistical needs, may be used to test additional products of the portfolio or IPM application schemes, fulfilling statistical and economic requirements simultaneously.