Pharmacogenomics: Role in Medicines Approval and Clinical Use

Abstract

Pharmacogenomics (PGx) and pharmacogenetics (PGt) are emerging interdisciplinary areas recently defined by the regulatory authorities at an international level as ‘the investigation of variations of DNA and RNA characteristics as related to drug response’ (PGx), and the study of ‘the influence of variations in DNA sequence on drug efficacy and toxicity’ (PGt). In recent years a number of studies have in fact produced growing evidence that, besides the effects of age, sex, diseases, and different drugs interactions, genetic factors play a role in the inter-individual variability of drugs response. The increasing genomic knowledge has also raised the profile and role of the so called ‘genomic biomarkers’ (GBs) in drug development, approval, and clinical use. The aims of this review are to (a) revisit the general understanding of the role of genomics and genetics in drug response, (b) provide an update on the definition and classification criteria of GBs as recently defined at a global level by regulatory agencies such as the European Medicines Agency and the Food and Drug Administration, and (c) illustrate with some examples current and potential applications of biomarkers in clinical practice and in drug development.

Pharmacogenomics of Osteoporosis

“…Pharmacogenomics and pharmacogenetics are emerging interdisciplinary areas recently defined by the regulatory authorities at an international level as ''the investigation of variations of DNA and RNA characteristics as related to drug response'' (pharmacogenomics), and the study of ''the influence of variations in DNA sequence on drug efficacy and toxicity'' (pharmacogenetics) [4,5]. Together they represent a rapidly developing field, aimed to improve efficacy, reduce toxicity and predict non-responders to treatments for different conditions, in a way that alternative therapy can be chosen or individual dose adjustments can be made.…”

Genetics of Human Sexual Behavior: Where We Are, Where We Are Going

“…Pharmacogenomics and pharmacogenetics, investigating the variations of DNA and RNA characteristics as related to (sexual) drug response and studying the influence of variations in DNA sequence on (prosexual and antisexual) drug efficacy and toxicity, will benefit of increased genetic knowledge in the field of sexual medicine [103]. Furthermore, a better understanding of the interactions between genes and lifestyle factors will help all sexual health caregivers with new therapeutic targets and insights, and most importantly, psychotherapeutic approaches to sexual dysfunctions could benefit from the concept that individual differences may have a genetic component.…”

Precision Medicine in Non-Communicable Diseases

“…This is why the pharmaceutical industry, academia, clinicians, and regulators have focused their attention on the genomic basis of individual response variability. This has led to a transition from population-based to more individualized treatment both in clinical drug development and clinical practice [17]. This concept is the basis of pharmacogenomics, a crucial pillar of "personalized medicine" that studies genomic and epigenomic factors that influence drug pharmacokinetics (PK) [18] and pharmacodynamics (PD), as well as drug-drug interactions (DDIs).…”