Role Of Biomarkers In Phase Iii And Postmarketing Decisions

mechanism of action defined by animal studies? This question is answered by a clinical proof-of-concept study: often, a phase Ila study in a small number of patients. However, occasionally, clinical proof of concept can be addressed during phase I development if the mechanism of action can be demonstrated in healthy volunteers (e.g., effects on blood pressure, body weight, lipids). The changes in the data collected during this study do not achieve statistical significance; rather, there needs to be enough indication of potential efficacy to convince decision makers to open the gate and spend the resources to progress the drug further. Often, the minimal result for progression can be defined ahead of time, making the actual decision-making process much easier, more objective, and transparent.

Early in clinical development, multiple biomarker assays or technologies may be utilized in order to understand more fully the safety and actions of the drug in humans. One may use a collection of biomarker methods, most of which are not likely to be fully validated surrogate markers of effect. For early decision making, it is not necessary to have results that meet the same standards of accuracy and quality as those used for drug approval. Indeed, it is during these early trials that promising tests for use in later clinical development or marketing are first identified from among the pool of available experimental biomarkers. Resources can then be put to further developing the selected biomarker methodology to meet the quality standards expected for continued future use.

Knowledge of the biology of the disease may be evolving at the same time as the drug is being developed. Therefore, new biomarkers may need to be added during the development process as the science evolves. It should not be assumed that the "tried and true" biomarkers are the only ones worthy of consideration or that a biomarker that was used to obtain marketing approval in the past is the one that should be used today. Good science supported by convincing data can convince stakeholders that a novel biomarker is better than one already established.

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