As evidence continues to mount that some drugs metabolize differently based on age, sex, race, and other variables, the U.S. Food and Drug Administration (FDA) is recommending actions with implications for certain pharmaceuticals and medical devices.
Published in July, the FDA’s Population Pharmacokinetics Guidance for Industry draft aims to increase safety and efficacy for pharmaceutical products. The draft guidance calls for sponsors of new drug and biologics license applications to apply population PK analysis, which is frequently used to guide drug development and inform recommendations on therapeutic individualization. This guidance also includes common applications of population PK analysis, the FDA’s current thinking on the data and model requirements needed to support regulatory decisions, recommendations to sponsors on drug labeling based on population PK analysis, and general expectations.
The FDA guidance documents do not establish legally enforceable responsibilities. However, once a draft guidance is published, a new wave of industry best practices usually begins to take form. Companies that fail to follow suit risk losing competitive ground.
Impetus For Change
This population PK movement is largely the result of increased scientific knowledge, data collection capabilities, and industry insight, and does not intend to imply prior or present negligence on the part of drug and/or device manufacturers. Traditionally, studies often limited — and continue to limit — the breadth of trial subjects due to a variety of rational scientific and business factors. These include, but are not limited to, testing a desired result for a specific/relevant patient segment; the availability, vulnerability, and/or willingness of participants; and budgetary and time constraints.
Studying the effects of specific drugs and biologics on germane subpopulations could bring new information that leads to significant changes on drug dose and dosage frequency, use, and usage limitations for specific subpopulations, including a prominent boxed warning. Plus, genomics and proteomics are likely to drive even greater granularity of how drugs and biologics will be used in treatments. For example, as healthcare begins to incorporate personalized medicine into the mainstream, new discoveries will likely drive the need to account for treatment safety and efficacy for additional subpopulations.