For example, by utilizing knowledge about genetic polymorphism, scientists are developing information needed to better predict who will be responding favorably and with less toxicity to a given therapy. Much of our knowledge at present results from chance observations. By using surrogate endpoints as tools to predict outcomes, we hope to increase the odds of finding effective, but less toxic therapies more accurately and efficiently. Therefore, the research community should consider investigation in the area of surrogate markers for both efficacy and toxicity. Another critical area as we work to advance this field is to standardize nomenclature.
Surrogates clearly serve as valuable tools in late stage clinical trials.
While we do not expect validation of a marker in preliminary trials, it can sometimes provide valuable information about drug activity. By clarifying the utility of such markers, using a common nomenclature, we will able to keep a consistent approach to use of these tools. I fully recognize that as we move ahead, it is critical that there be both active communication and collaboration among those working in the field in the industry, academic community and federal scientific agencies. Because we at FDA have a critical role in this process, we need to be involved as science is emerging. Ideally, we must interact frequently and meaningfully as the clinical research proceeds.