Spelling Out Context and Structure
Under FDA’s new biomarker guidance, descriptions of the context for using a biomarker should include: (1) the general area, (2) specific biomarker use, and (3) critical parameters that define when and how the biomarker should be used. General area includes but isn’t limited to pharmacology, toxicology, efficacy, safety, and disease.
Specific biomarker uses can cover factors ranging from patient selection and dose optimization to drug response monitoring. Critical parameters include specificity of use by class or product, disease diagnosis and phenotypes, sample collection, assay specifications, tissue or physiological/pathological process, species, demographics, and environmental factors.
The context should be supported, the guidance noted, by data available in the biomarker’s initial qualification dossier submission. Should that data prove inconsistent with the proposed context, drug developers could be asked to furnish additional data during the process of qualifying their biomarker.
According to the guidance, the structure of qualification submissions should include an overview of biomarker qualification, with a justification for its proposed context of use, followed by summaries of analytical assay data, nonclinical biomarker data, and clinical biomarker data. Once a biomarker is qualified, researchers need not generate additional data to justify its use.
“These kinds of things help in the determination of whether or not a patient is going to appropriately respond,” David L. Rosen, a partner and co-chair of the life sciences industry team at the law firm Foley & Lardner LLP, told GEN.
“That’s what we’re looking at: How well can the diagnostic agent help people assess whether or not they have particular characteristics that would respond? Can they differentiate between people who have a particular marker or not? How susceptible is the biomarker to false positive readings? I think all those play into that determination,” said Rosen, who spent 14 years at the FDA.