Derek L G Hill1, Adam J Schwarz2, Maria Isaac3, Luca Pani3, Spiros Vamvakas3, Robert Hemmings3, Maria C Carrillo4, Peng Yu2, Jia Sun5, Laurel Beckett6, Marina Boccardi7, James Brewer8, Martha Brumfield9, Marc Cantillon10, Patricia E Cole2, Nick Fox11, Giovanni B Frisoni7, Clifford Jack12, Thomas Kelleher13, Feng Luo13, Gerald Novak14, Paul Maguire15, Richard Meibach16, Patricia Patterson17, Lisa Bain18, Cristina Sampaio19, David Raunig20, Holly Soares13, Joyce Suhy21, Huanli Wang6, Robin Wolz22, Diane Stephenson23. 1. IXICO Ltd., London, UK. 2. Eli Lilly and Company, Indianapolis, IN, USA. 3. European Medicines Agency, London, UK. 4. Alzheimer's Association, Chicago, IL, USA. 5. Eli Lilly and Company, Indianapolis, IN, USA; The University of Texas School of Public Health, Houston, TX, USA. 6. University of California, Davis, CA, USA. 7. IRCCS Fatebenefratelli, Brescia, Italy. 8. University of California, San Diego, CA, USA. 9. Coalition Against Major Diseases, Critical Path Institute, Tucson, AZ, USA. 10. Wellnessmanagments.com, Livingston, NJ, USA. 11. UCL Institute of Neurology, London, UK. 12. Mayo Clinic, Rochester, MN, USA. 13. Bristol Myers Squibb, Wallingford, CT, USA. 14. Janssen Pharmaceutical Research and Development, Titusville, NJ, USA. 15. Novartis, Basel, Switzerland. 16. Novartis, NJ, USA. 17. AstraZeneca, Wilmington, DE, USA. 18. Independent science writer, Elverson, PA, USA. 19. CHDI Foundation Inc., Princeton, NJ, USA. 20. ICON Medical Imaging, Warrington, PA, USA. 21. SYNARC Inc., Newark, CA, USA. 22. IXICO Ltd., London, UK; Department of Computing, Imperial College London, London, UK. 23. Coalition Against Major Diseases, Critical Path Institute, Tucson, AZ, USA. Electronic address: DStephenson@c-path.org.
Abstract
BACKGROUND: Regulatory qualification of a biomarker for a defined context of use provides scientifically robust assurances to sponsors and regulators that accelerate appropriate adoption of biomarkers into drug development. METHODS: The Coalition Against Major Diseases submitted a dossier to the Scientific Advice Working Party of the European Medicines Agency requesting a qualification opinion on the use of hippocampal volume as a biomarker for enriching clinical trials in subjects with mild cognitive impairment, incorporating a scientific rationale, a literature review and a de novo analysis of Alzheimer's Disease Neuroimaging Initiative data. RESULTS: The literature review and de novo analysis were consistent with the proposed context of use, and the Committee for Medicinal Products for Human Use released an opinion in November 2011. CONCLUSIONS: We summarize the scientific rationale and the data that supported the first qualification of an imaging biomarker by the European Medicines Agency.
BACKGROUND: Regulatory qualification of a biomarker for a defined context of use provides scientifically robust assurances to sponsors and regulators that accelerate appropriate adoption of biomarkers into drug development. METHODS: The Coalition Against Major Diseases submitted a dossier to the Scientific Advice Working Party of the European Medicines Agency requesting a qualification opinion on the use of hippocampal volume as a biomarker for enriching clinical trials in subjects with mild cognitive impairment, incorporating a scientific rationale, a literature review and a de novo analysis of Alzheimer's Disease Neuroimaging Initiative data. RESULTS: The literature review and de novo analysis were consistent with the proposed context of use, and the Committee for Medicinal Products for Human Use released an opinion in November 2011. CONCLUSIONS: We summarize the scientific rationale and the data that supported the first qualification of an imaging biomarker by the European Medicines Agency.
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