Pharmacogenetics in drug development: Regulatory and clinical considerations

Pharmacogenetics (the study of inter-individual inherited variations in DNA sequence related to drug response) is increasingly gaining recognition as an integral part of modern drug development and health care because it provides significant opportunities to increase the safety and efficacy of medicines. The challenge for the pharmaceutical industry and the regulators is exactly how and when to integrate pharmacogenetics to deliver on this potential. There are numerous aspects to consider when determining the data requirements for a regulatory submission to support labeling claims based on genotype. Regulators are committed to fostering a collaborative approach with key stakeholders to address the issues and to develop guidelines as judiciously as possible. It is recognized that pharmacogenetics research must be integrated into an appropriate regulatory framework that does not stifle development of the science nor the applications that will benefit patients. Dialogue, together with a willingness to share experiences of pharmacogenetics in drug development, is critical to moving forward. Drug Dev. Res. 62:102–111, 2004. © 2004 Wiley-Liss, Inc.