NEW YORK (GenomeWeb News) – The US Food and Drug Administration has issued a new draft guidance aimed at helping drug developers evaluate how human genomic variations can affect the way drugs function in different people and how they can cause varying clinical responses.
The guidance document, for which FDA currently is taking comments, includes non-binding recommendations covering how and when genomic information should be considered as a way of dealing with questions that arise during the drug development process and during regulatory review. The specific goals of this document, “Clinical Pharmacogenomics: Premarketing Evaluation in Early Phase Clinical Studies" is to provide advice related to general principles of study design, data collection, and data analysis when using pharmacogenomic approaches in pharmaceutical development.
FDA did not draft the recommendations to address statistical considerations used in later phases of randomized clinical trials that are intended to draw definitive conclusions from genomic subgroup effects. Rather, the considerations provided in the guidance, FDA states, are relevant for "exploratory and observational studies," such as investigation into how early phase data on genomic-dependent dosing can be used to guide later studies or the collection of genetic and biomarker data.
FDA hopes that using genomic information for all subjects early in the drug development process will allow for the discovery of important genomic differences, and it has aimed its recommendations at improving the quality of the data collected and the ability of investigators to assess genomic relationships.
Early genomic studies can be used in several ways, FDA noted, such as to identify populations that should receive lower or higher doses of drugs because of excretory or metabolic differences; to identify responder populations based on phenotypic or genetic characteristics; to help define the dose range for later trials by identifying dose response for pertinent biomarkers and identify specific subsets of subjects with different dose-response relationships; and to identify high-risk groups based on genetic or metabolic factors that potentially could be managed in clinical trials and support the approval of some drugs.
FDA will accept public comments on the draft guidance until April 19.