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Pharmacogenetics is a subset of personalized medicine that applies knowledge about genetic variation in gene–drug pairs to help guide optimal dosing. There is a lack of data, however, about pharmacogenetic variation in underserved populations. One strategy for increasing participation of underserved populations in pharmacogenetic research is to include communities in the research process. We have established academic–community partnerships with American Indian and Alaska Native people living in Alaska and Montana to study pharmacogenetics. Key features of the partnership include community oversight of the project, research objectives that address community health priorities, and bidirectional learning that builds capacity in both the community and the research team. Engaging the community as coresearchers can help build trust to advance pharmacogenetic research objectives.

The 2010 US FDA–Drug Industry Association (DIA) Pharmacogenomics (PGx) Workshop follows a series that began in 2002 bringing together multidisciplinary experts spanning regulatory authorities, medical research, healthcare and industry. This report summarizes the ‘Building PGx into Labels’ sessions from the workshop, which discussed the critical elements in developing PGx outcomes leading to product labels that inform efficacy and/or safety. Examples were drawn from US prescribing information, which integrated PGx knowledge into medical decisions (e.g., panitumumab, warfarin and clopidogrel). Attendees indicated the need for broader dialog and for guidelines on evidentiary considerations for PGx to be included into product labels. Also discussed was the understanding of appropriate PGx placement on labels; how to encourage adoption by medical communities of label recommendations on PGx tests; and, given the global nature of drug development, worldwide considerations including European Summary of Product Characteristics.

The 4th US FDA/Industry workshop, in a series on Pharmacogenomics, was on ‘Biomarkers and Pharmacogenomics in Drug Development and Regulatory Decision Making’ and was held on December 10–12, 2007 in Bethesda, MD, USA, with clear objectives to continue the dialogue that began in 2002 for enabling the use of biomarkers and pharmacogenomics in drug development and regulatory decision-making. This brief commentary will highlight the major topics and outcomes discussed at this meeting that was jointly sponsored by the FDA, The Pharmacogenomics Working Group (PWG), The Pharmaceutical Research and Manufacturers of America (PhRMA), The Biotechnology Industry Organization (BIO) and The Drug Information Association (DIA).

Over the past four years, the annual US FDA–DIA pharmacogenomic workshops have brought together attendees with wide-ranging expertise spanning industry, regulatory authorities and academia. This special report summarizes a breakout session using a novel, interactive case format as a way to engage participants, raise awareness and share diverse learnings via ‘real life’ decisions that project teams might face in developing a new medicine. This case was situated just prior to approval by a Regulatory Authority as a project team is finalizing a new medicine label. To effectively integrate new biomarkers such as pharmacogenomics into developing new medicines, this session highlighted the importance in considering medical practice implications as relevant (or not) to information or actions by a prescriber; progressing validation beyond assay to clinical; and fitting pharmacogenomics into context with other evidence often built over decades during a drug’s development. All converge onto a label that must communicate evidence–based use of a new medicine that is effective and safe.

2004 © Future Medicine Ltd IS The FDA/DIA/PWG/PhRMA/BIO workshop on ‘Pharmacogenomics in Drug Development and Regulatory Decision-making: the Genomic Data Submission (GDS) Proposal’ was held in Washington DC in November last year to focus on the principles of the new draft guidance, and their application to non-clinical, clinical pharmacology and clinical case studies that yield pharmacologic data that may or may not be required for submission. A summary of the workshop is available in a previous issue of Pharmacogenomics [1]. Pharmacogenomic data that are required to be submitted follow the existing regulations when data are used for regulatory decision making, provide supportive information, and are derived from valid or probably valid tests. On the other hand, exploratory data are required only as a synopsis once an application for approval is submitted. In order to promote and enable the utility of pharmacogenomics in drug development, the FDA is encouraging submission of full reports of exploratory pharmacogenomic data during the investigational new drug (IND) phase, and is also encouraging full pharmacogenomic reports when only abbreviated or synopsis reports are required for the later phases of drug approval. Highlights of the workshop case discussions for each of the three areas of drug development (non-clinical, clinical pharmacology, and clinical) are described in greater detail than previously summarized in the following three special reports [2-4]. The basis for the discussion centers on the three algorithms for submission reports as

Over the past 10 years, the US FDA has become a strong pharmacogenomics advocate as part of its mission to both protect and advance public health by enabling innovations that make medicines safer to use and more effective. The agency has evolved its advocacy cautiously on a foundation of science-based information from novel programs, such as the Voluntary Genomics Data Submission initiative, and on careful regulatory assessment of the extraordinary advances in clinical pharmacogenomics that have supported the update of drug labels with genetic information. This commentary goes into detail on the evolution of these achievements. However, many challenges remain for pharmacogenomics, and they will continue to evolve, and all stakeholders must work together. As the decade draws to a close, we have presented four major areas that need to be addressed collectively to assure that pharmacogenomics continues to mature over the next 10 years into a science that is essential to the practice of medicine.