title: Precompetitive Data Sharing as a Catalyst to Address Unmet Needs in Parkinson's Disease.
abstract: Parkinson's disease is a complex heterogeneous disorder with urgent need for disease-modifying therapies. Progress in successful therapeutic approaches for PD will require an unprecedented level of collaboration. At a workshop hosted by Parkinson's UK and co-organized by Critical Path Institute's (C-Path) Coalition Against Major Diseases (CAMD) Consortiums, investigators from industry, academia, government and regulatory agencies agreed on the need for sharing of data to enable future success. Government agencies included EMA, FDA, NINDS/NIH and IMI (Innovative Medicines Initiative). Emerging discoveries in new biomarkers and genetic endophenotypes are contributing to our understanding of the underlying pathophysiology of PD. In parallel there is growing recognition that early intervention will be key for successful treatments aimed at disease modification. At present, there is a lack of a comprehensive understanding of disease progression and the many factors that contribute to disease progression heterogeneity. Novel therapeutic targets and trial designs that incorporate existing and new biomarkers to evaluate drug effects independently and in combination are required. The integration of robust clinical data sets is viewed as a powerful approach to hasten medical discovery and therapies, as is being realized across diverse disease conditions employing big data analytics for healthcare. The application of lessons learned from parallel efforts is critical to identify barriers and enable a viable path forward. A roadmap is presented for a regulatory, academic, industry and advocacy driven integrated initiative that aims to facilitate and streamline new drug trials and registrations in Parkinson's disease.
authors.affiliation: Critical Path Institute, CAMD, Tucson, AZ, USA.
authors.forename: Diane
authors.initials: D
authors.lastname: Stephenson
authors.affiliation: Nuffield Department of Clinical Neurosciences, University of Oxford, Neurology Department, Level 3, West Wing, John Radcliffe Hospital, Headley Way, Headington, Oxford, UK.
authors.forename: Michele T
authors.initials: MT
authors.lastname: Hu
authors.affiliation: Critical Path Institute, CAMD, Tucson, AZ, USA.
authors.forename: Klaus
authors.initials: K
authors.lastname: Romero
authors.affiliation: Scientific consultant, London, UK.
authors.forename: Kieran
authors.initials: K
authors.lastname: Breen
authors.affiliation: Henry Wellcome Building, Faculty of Medical Sciences, Newcastle University, Newcastle upon Tyne, UK.
authors.forename: David
authors.initials: D
authors.lastname: Burn
authors.affiliation: University of Bristol, Canynge Hall, Bristol, UK.
authors.forename: Yoav
authors.initials: Y
authors.lastname: Ben-Shlomo
authors.affiliation: US Food and Drug Administration (FDA), New Hampshire Avenue, Silver Spring, MD, USA.
authors.forename: Atul
authors.initials: A
authors.lastname: Bhattaram
authors.affiliation: EMA, 30 Churchill Place, Canary Wharf, London, UK.
authors.forename: Maria
authors.initials: M
authors.lastname: Isaac
authors.affiliation: U of Rochester Medical Center, Crittenden Blvd, Rochester, NY, USA.
authors.forename: Charles
authors.initials: C
authors.lastname: Venuto
authors.affiliation: Michael J Fox Foundation for Parkinson's Research, Seventh Avenue, New York, NY, USA.
authors.forename: Ken
authors.initials: K
authors.lastname: Kubota
authors.affiliation: Aston University and MIT, Aston University, Aston Triangle, Birmingham, Media Lab, Massachusetts Institute of Technology, Cambridge, MA, USA.
authors.forename: Max A
authors.initials: MA
authors.lastname: Little
authors.affiliation: Sage Bionetworks, Seattle, WA, USA.
authors.forename: Stephen
authors.initials: S
authors.lastname: Friend
authors.affiliation: Department of Psychiatry, University of Oxford, Warneford Hospital, Oxford, UK.
authors.forename: Simon
authors.initials: S
authors.lastname: Lovestone
authors.affiliation: Department of Clinical Neuroscience, UCL Institute of Neurology, London, UK Department of Neurology, Royal Free Hospital, London, UK Neurology, National Hospital for Neurology, London, UK.
authors.forename: Huw R
authors.initials: HR
authors.lastname: Morris
authors.affiliation: Institute of Neuroscience and Psychology, University of Glasgow, Scotland, UK.
authors.forename: Donald
authors.initials: D
authors.lastname: Grosset
authors.affiliation: National Institute of Neurological Disorders and Stroke (NINDS), Neuroscience Center, Bethesda, MD, USA.
authors.forename: Margaret
authors.initials: M
authors.lastname: Sutherland
authors.affiliation: Department of Psychiatry, University of Oxford Hospital, Warneford, Oxford, UK.
authors.forename: John
authors.initials: J
authors.lastname: Gallacher
authors.affiliation: Department of Clinical Neurosciences, John Van Geest Centre for Brain Repair, University of Cambridge, E.D. Adrian Building, Forvie Site, Robinson Way, Cambridge, UK.
authors.forename: Caroline
authors.initials: C
authors.lastname: Williams-Gray
authors.affiliation: Independent Scientific & Medical Writer, Savits Drive, Elverson, PA, USA.
authors.forename: Lisa J
authors.initials: LJ
authors.lastname: Bain
authors.affiliation: Critical Path Institute, CAMD, Tucson, AZ, USA.
authors.forename: Enrique
authors.initials: E
authors.lastname: Avilés
authors.affiliation: Institute of Neurodegenerative Diseases, Parkinson's Progression Markers Initiative, Suite 8B, New Haven, CT, USA.
authors.forename: Ken
authors.initials: K
authors.lastname: Marek
authors.affiliation: Laboratory of Neuro Imaging, Keck School of Medicine of USC, University of Southern California, CA, USA.
authors.forename: Arthur W
authors.initials: AW
authors.lastname: Toga
authors.affiliation: Teva Pharmaceutical Industries Ltd., Petach Tikva, Israel.
authors.forename: Yafit
authors.initials: Y
authors.lastname: Stark
authors.affiliation: Boehringer Ingelheim Pharmaceuticals, Inc., Ridgefield, CT, USA.
authors.forename: Mark
authors.initials: M
authors.lastname: Forrest Gordon
authors.affiliation: Parkinson's UK, London, UK.
authors.forename: Steve
authors.initials: S
authors.lastname: Ford
mesh_terms: D015415:Biomarkers; D002986:Clinical Trials as Topic / Q000592:standards*; D055808:Drug Discovery / Q000592:standards*; D006801:Humans; D033181:Information Dissemination; D010300:Parkinson Disease / Q000188:drug therapy*; D012107:Research Design
chemical_list: D015415:Biomarkers
keywords: Data standards; collaboration; data integration; privacy; quantitative disease progression; regulatory science