Biostatistics in Experimental

Pharmacology

What is Biostatistics?
Using the tools of statistics, biostatisticians help answer pressing research questions in medicine,
biology and public health, such as whether a new drug works, what causes cancer and other
diseases, and how long a person with a certain illness is likely to survive.

Biostatistics is the application of statistical principles to questions and problems in medicine, public health or
biology. One can imagine that it might be of interest to characterize a given population (e.g., adults in
Boston or all children in the United States) with respect to the proportion of subjects who are overweight or
the proportion who have asthma, and it would also be important to estimate the magnitude of these
problems over time or perhaps in different locations. In other circumstances in would be important to make
comparisons among groups of subjects in order to determine whether certain behaviors (e.g., smoking,
exercise, etc.) are associated with a greater risk of certain health outcomes. It would, of course, be
impossible to answer all such questions by collecting information (data) from all subjects in the populations
of interest. A more realistic approach is to study samples or subsets of a population. The discipline of
biostatistics provides tools and techniques for collecting data and then summarizing, analyzing, and
interpreting it. If the samples one takes are representative of the population of interest, they will provide
good estimates regarding the population overall. Consequently, in biostatistics one analyzes samples in
order to make inferences about the population. This module introduces fundamental concepts and
definitions for biostatistics.

Biostatistics in Pharmacology
Biostatistics has an important role in both designing a pharmaceutical experiment and evaluating
its result. Randomization techniques are essentially important in designing an experiment. The
goal of randomization is transforming systematic errors into random errors and confirming
comparability among experimental groups. Randomization also provides a rationale for applying
statistical tests. Combining randomization techniques with blinding and local control enables us to
construct a scientifically reliable and effective experiment. An appropriate statistical analysis
absolutely depends on the method of randomization. In order for a pharmacological study to be
successful, it is very important to consider statistical aspects in the designing stage.
Applications
- To find action of drugs
- To compare two different drugs
- To compare a new drug with reference to a new drug.

2
Hypothesis Testing and ANOVA
One of the main goals of statistical hypothesis testing is to estimate the P value, which is the
probability of obtaining the observed results, or something more extreme, if the null hypothesis
were true. If the observed results are unlikely under the null hypothesis, your reject the null
hypothesis. Alternatives to this "frequentist" approach to statistics include Bayesian statistics and
estimation of effect sizes and confidence intervals.

There are different ways of doing statistics. The technique used by the vast majority of
biologists, and the technique that most of this handbook describes, is sometimes called
"frequentist" or "classical" statistics. It involves testing a null hypothesis by comparing the data you
observe in your experiment with the predictions of a null hypothesis. You estimate what the
probability would be of obtaining the observed results, or something more extreme, if the null

3
hypothesis were true. If this estimated probability (the P value) is small enough (below the
significance value), then you conclude that it is unlikely that the null hypothesis is true; you reject
the null hypothesis and accept an alternative hypothesis.
Many statisticians harshly criticize frequentist statistics, but their criticisms haven't had much
effect on the way most biologists do statistics. Here I will outline some of the key concepts used in
frequentist statistics, then briefly describe some of the alternatives.