Abstract
Heterogeneity in the underlying mechanisms of disease processes and inter-patient variability in drug responses are major challenges in drug development. To address these challenges, biomarker strategies based on a range of platforms, such as microarray gene-expression technologies, are increasingly being applied to elucidate these sources of variability and thereby potentially increase drug development success rates. With the aim of enhancing understanding of the regulatory significance of such biomarker data by regulators and sponsors, the US Food and Drug Administration initiated a programme in 2004 to allow sponsors to submit exploratory genomic data voluntarily, without immediate regulatory impact. In this article, a selection of case studies from the first 5 years of this programme — which is now known as the voluntary exploratory data submission programme, and also involves collaboration with the European Medicines Agency — are discussed, and general lessons are highlighted.
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Federico M. Goodsaid, Shashi Amur, Jiri Aubrecht, Jennifer Catalano, Rosane Charlab, Albert J. Fornace Jr, Huixiao Hong, David Jacobson-Kram, Ansar Jawaid, Lawrence Lesko, Heng-Hong Li, Klaus Lindpainter, Marisa Papaluca-Amati, Timothy W. Robison, Ina Schuppe-Koistinen, Leming Shi, Weida Tong, Li Zhang, and Issam Zineh declare no competing financial interests.
Michael E. Burczynski has none beyond previous employment by Wyeth and current employment by F. Hoffmann-La Roche.
Kevin Carl is an employee and shareholder of Novartis Pharmaceuticals Corporation.
Sandra Close is an employee and shareholder of Eli Lilly and Company.
Catherine Cornu-Artis is a full-time employee of Novartis Pharma AG, Switzerland.
Laurent Essioux is a full-time employee of F. Hoffmann-La Roche.
Lois Hinman is an employee of Novartis and shareholder of Novartis stock.
Ian Hunt is an employee of AstraZeneca.
David Laurie is a full-time employee of Novartis Pharma AG, Switzerland and a holder of shares in Novartis.
James Mayne has stocks in Pfizer, which participates in business activities that are regulated by the US FDA and the EMA.
Peter Morrow is an employee and shareholder of Eli Lilly and Company.
John Roth is a full-time employee of F. Hoffmann-La Roche.
Olivia Spleiss is a full-time employee of F. Hoffmann-La Roche.
Sharada L. Truter is a full-time employee and shareholder of Pfizer.
Jacky Vonderscher is a full-time employee and shareholder of F. Hoffmann-La Roche and also a shareholder of Novartis.
Agnes Westelinck has none beyond previous employment by F. Hoffmann-La Roche and current employment by GlaxoSmithKline.
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Goodsaid, F., Amur, S., Aubrecht, J. et al. Voluntary exploratory data submissions to the US FDA and the EMA: experience and impact. Nat Rev Drug Discov 9, 435–445 (2010). https://doi.org/10.1038/nrd3116
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DOI: https://doi.org/10.1038/nrd3116
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