Using open science to shorten the time lag between discovery research & clinical use

Transcription

1 Enhancing Translational Research and Clinical Development in Alzheimer s Disease and other Dementia: The Way Forward November 2014, Lausanne Using open science to shorten the time lag between discovery research & clinical use Professor Martin Rossor NIHR National Director for Dementia Research Lorna Harper, Dementia Research Centre, University College London

2 Traditional Model of Publically Funded Research Funding Access to unique data is the primary commodity available to researchers to generate funding and subsequently publications Unique Data The ability to sustain this circular economy ultimately determines the success of most labs. The research community alone currently lacks the resources to drive this change and break from these traditional models of discovery. Publications Support from governments, charities, funders and other public bodies at an international level are required in order to bring about meaningful change in health research.

3 Guidance from the OECD Openness Flexibility Transparency Legal conformity Protection of intellectual property Formal responsibility Professionalism Interoperability Quality Security Efficiency Accountability Sustainability Opportunity to operationalise these guidelines for dementia research

4 Open-Science Success Stories - Particle Physics BIG Data 1 million petabytes of raw data per year per experiment Smart Storage Flexible to cover the variety in the data and adaptable to changing requirements Sharing Solutions Other areas of physics are adopting this successful sharing model Distributed Load Globe spanning network of distributed computing Image CMS Higgs-event: simulated data modelled for the CMS particle detector on the Large Hadron Collider

5 Open-Science Success Stories - Genomics Information freely released to the public domain Rapid release within 24hrs Prevented the exploitation and control of human sequence information. Genome-Wide Association Studies (GWAS) Policy 2014 Genomic Data Sharing Policy Genotypes and Phenotypes database

6 Greater Openness in Clinical Trials Adopted by the EFPIA and PhRMA in 2013

7 Clinical Trial Data Sharing Initiatives (including access to patient-level data) ClinicalTrial StudyTransparency.com

8 Criteria for Access to Trial Data Sponsor s Criteria for Listing Study Products approved by the US/EU regulatory agencies Primary manuscripts accepted for publication Impossible for individuals to be reidentified (rare diseases, small sample size, single-site data is unlikely to be released) No technical constraints to providing data access No legal, contractual or consent provisions that prohibit transfer to third parties Researcher Criteria for Access Independent review panel are convinced of: Scientific relevance of proposal to medical science or patient care Ability of the plan to meet it s objectives Publication plan for the research No conflicts of interest which may impact on any part of the study The experience of the team to conduct the proposed research Ability of the team to adhere to the Data Sharing Agreement

9 Public/Private Data Sharing Initiatives The Global Alzheimer Association Network Online research platform Access to both research data and tools Advance global sharing, investigation and discovery The European Medical Information Framework Framework for consistent reuse of available patient-level data Includes population-based registries, hospital databases, national registries and biobanks Plans in place to link the two systems

10 Public/Private Data Sharing Initiatives ALZHEIMER S DISEASE Coalition Against Major Diseases PARKINSON s DISEASE Creating new tools and methods to increase the efficiency of the development process of new treatments for Alzheimer s disease and Parkinson s disease Focus on: Qualification of biomarkers Development of common data standards Creation of integrated databases for clinical trials data Development of quantitative model-based tools for drug development

11 Case Studies The Alzheimer s Disease Neuroimaging Initiative - a worldwide, data sharing project for research on the pathology, prevention and treatment of AD The Swedish National study on Aging and Care (SNAC) - four longitudinal individual-based data collections describing the ageing process and encompassing the care system IMI AddNeuroMed - A cross European data repository, designed to improve biomarker discovery for Alzheimer s disease The UK Biobank - A large-scale national health resource, addressing bio-medical and lifestyle influences on health - a key component of the UK Dementias Platform 11

12 Principles for Good Practice and Reporting? Dementia Principles Toronto (2009) Nature 461, (Sept 2009) OECD Guidance (2007) Fort Lauderdale (2003) Bermuda Principles (1996)

13 What to Share? Establish Centres of Excellence to generate and share complex data Open Data Avoid a lower threshold on cohort size Encourage harmonisation of data collection tools Open Tools Open Science Open Access Make provisions for greater integration of data from population studies Provide infrastructure to share computing resources Publishing portal for negative results 13

14 When to Share? After data from every N patients has been acquired Encourage multicentre collaboration to speed up data collection Establish unique publication opportunities based on data generation Provide safeguards and incentives for data collection sites Pre-publication? Encourage data collection sites to lead on guidelines relating to the harmonisation of data collection tools

15 How to Share? Responsibly Internationally agreed: Patient consent forms Anonymisation standards for patient-level data Data sharing policies with endorsement from funding bodies Efficiently Effectively Seek support for change from both commercial and public funding bodies and publishers Engage governments, charities and other public bodies to push for change at an international level Web-based information framework Timely release of data Pooled computing resources and harness cloud computing technology Develop a business model for curation Diagram by Michael Nielsen