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Open Knowledge for Real World Biomedical Health Benefits: NIH Strategies and Policies
Claire T. Driscoll
Director, Technology Transfer Office
National Human Genome Research Institute (NHGRI)
National Institutes of Health (NIH)
U.S. Department of Health & Human Services (DHHS)
Open Government Data Conference Sept 23, 2011Brisbane, Australia
Governments have Found a New, Cheap Way to Improve People’s Lives
Thaler, R. This Data Isn’t Dull. It Improves Lives, March 12, 2011, New York Times
Instructions:
•Take data various government agencies have already collected and post it on-line (tip: post it in an easy to use format )
•Wait (you won’t have to wait very long)
•Private sector gets busy reformatting the data to create new web sites, smart phone apps, etc.
•Public now has access to innovative, helpful new tools
U.S. Government’s Open Government Initiative http://www.whitehouse.gov/open
Memo on Transparency and Open Government issued by President Obama on Jan. 21, 2009 http://edocket.access.gpo.gov/2009/pdf/E9-1777.pdf
Government should be TRANSPARENT
Government should be PARTICIPATORY
Government should be COLLABORATIVE
Open Government Directive (Dec. 8, 2009)
Directs agencies to take specific actions (includes deadlines)
http://www.whitehouse.gov/open/documents/open-government-directive
Creation of Data.gov web site
Data.gov Users & UsesCore Users Use Avenues for
Interaction
General Public The general public can use the platform to download datasets.
The general public can also discover and access Federal data via third-party visualizations, applications, tools or data infrastructure.
Website, Tools (Agency-Provided and Third Party)
Application Developers
Application developers can develop and deliver applications by leveraging the raw data, APIs or other methods of data delivery.
APIs, Third-Party Data Infrastructure
Government Owners
owners can expand access to and leverage data from their public sector partners to enhance service delivery, drive performance outcomes and effectively manage government resources.
Website, Tools (Agency-Endorsed)
Data Infrastructure
Developers
Data infrastructure developers can increase the utility of Data.gov by enhancing its search capability, metadata catalog processes, data interoperability and ongoing evolution.
APIs
Research Community
The research community can help unlock the value of multiple datasets by providing insight on a plethora of research topics.
Website, APIs
Data Infrastructure
Innovators
Existing entities and new ventures developing innovative data and application offerings that combine public sector data with their own data.
Website, APIs, Bulk Downloads
U.S. Government Now Offers Prizes
Challenge.gov is a place where the public and government can solve problems together
National Institutes of Health
Largest financial backer of basic biomedical research in the world
Mission: Uncover new knowledge that will lead to improved public health by conducting and supporting research
Top biomedical research institution in U.S.
27 Institutes, Centers and Divisions (ICDs)
2010 budget: ~$32 Billion
Extramural (univ., hospitals, etc.) = 60,000 awards to 3,000 organizations worldwide per year
Intramural (on campus) = 18,000+ employees; 5,000 MDs and PhDs; 2000+ intramural R&D projects
Collaborate w/ public and private sector scientists
100s of technologies & inventions available for licensing
Getting to Know the National Institutes of Health
The 27 Institutes and Centers of
the National Institutes of Health (www.nih.gov)
NIH: A Public Sector Entity
FAIR
open, unbiased competition for grants, contracts & licenses
TRANSPARENT
Evaluation & eligibility criteria, standard forms, policies, budget info, etc. are in public domain
WE SERVE MANY STAKEHOLDERS
U.S. taxpayers, Congress, academia, scientists, clinicians, patients, small biotechs, the global biopharma industry, etc.
RePORTER
Research Portfolio Online Reporting Tool (RePORT) Expenditures and Results (REPORTER)—intramural & grantee data
Many query fields; easy access to NIH data
Easy sorting & downloading
Links to publications (PubMed & PubMed Central) & patents (USPTO)
http://projectreporter.nih.gov
Open Access in the Life Sciences: this isn’t new to NIH!
GenBank Contains human sequence data
collected by the Human Genome Project International Sequencing Consortium as well as sequence data from many model organisms
Numerous analysis tools & other features
Est. 1982
Free, full, open access to all
www.ncbi.nlm.nih.gov/Genbank
MedLine & PubMed Biomedical Literature databases
(1000s of journals)
Many full text & downloadable electronic files available for FREE (PubMedCentral)
1st federal agency to mandate “open access”; full text of all NIH-funded articles must be deposited in PubMedCentral within 6 months of publication (voluntary 2005 to 2009; mandatory 2009-present)
Free, open access to all
www.nlm.nih.gov
100’s of Other High Value NIH-Created and -Funded Databases &
Datasets…a few examples
Database of Genotypes and Phenotypes (dbGap) www.ncbi.nlm.nih.gov/gap
Phenotype-genotype associations (genome-wide associations studies, medical sequencing, molecular diagnostic tests, etc.)
PubChem pubchem.ncbi.nlm.nih.gov/
Small molecules (compound structures plus assay data from all Molecular Libraries-funded projects)
ClinicalTrials.gov www.clinicaltrials.gov
Info and data from all NIH-funded (or partially funded) human clinical trials
Database of All Known Approved Human Drugs….at last!!
Developed by NIH Chemical Genomics Center (NCGC), NHGRI
Open Access Database (>7,000)
Enables Repurposing and chemical genomics
Access to physical collection of these drugs is available to NCGC collaborators
Huang, R. et al. Sci Transl Med (2011) 3(80): 80ps16
Christopher P. Austin, M.D.Director, NIH Chemical Genomics Center
Senior Advisor to the NHGRI Director for Translational ResearchNational Institutes of Health
The Therapeutics for Rare and Neglected
Diseases (TRND) Program
“Desperately Seeking Cures”
Title of Newsweek cover story (May 24/31, 2010) Article subtitle “How the road from promising
scientific breakthrough to real-world remedy has become all but a dead end” “Basic research is healthy…but patients
aren’t benefitting.” “…look at the progress open-source software
has made in IT. Imagine the progress open-source research could make in biomedicine”
TRND Operational Model
In-house and/or contractor laboratories with expertise in preclinical drug development will collaborate with external laboratories with expertise in disease/target
Projects will be taken to phase needed for external organization to adopt for clinical development
Projects will enter at a variety of stages of development
Distinguishing features
Disease agnostic, will look explicitly for cross-cutting mechanisms
Processes will be established to incorporate learning from each project to operationalize continuous improvement
Science of preclinical drug development will be studied
Reasons for successes and failures will be investigated and published
Building on the Success of the HGP: Cumulative and Distributed
Innovation by the NIH (partial list) Human Genome Project (HGP) The Pharmacogenetics Research Network The Mammalian Gene Collection (MGC) Haplotype Map Project (HapMap) ENCODE Project (Encyclopedia of DNA Elements) Knockout Mouse Project (KOMP) Molecular Libraries Program Genetic Associations Information Network (GAIN) The Cancer Genome Atlas $1000 genome 1000 genomes ClinSeq TRND (Therapeutics for Rare & Neglected Diseases)
NHGRI Timeline for its “Open Access” Policies: Cost-free, Rapid, (Often) Pre-Publication & Unfettered Use of DNA Sequence Data
1990-start of Human Genome Project (HGP) 1991-NHGRI data release policy—requires release of data and materials
within 6 months of generation 1996-International Human Genome Sequencing Consortium adopts the
"Bermuda Principles”--automatic, rapid release of sequence assemblies to the public domain
1997- new data release policy requires grantees engaged in large-scale genomic DNA sequencing to release DNA sequence assemblies within 24 hours of their generation
2003-the Wellcome Trust convenes an international group of data producers, users, database personnel, journal editors and funding agency representatives Other large-scale research efforts, designated as "community resource projects,"
will increasingly be generating data and other resources; these should also be rapidly released in an unrestricted manner
Timeline Continued….the Going Gets Tough or Moving Beyond Sequence Data
1998-NIH Research Tools Policy goes into effect 1999-Mammalian Gene Collection (MGC) 1999-SNP Consortium
Single Nucleotide Polymorphism data/genetic variation data (no data on biological function or identification of “causative SNPs”, e.g. those linked to drug response or certain disease states are in the database)
2002-HapMap Genetic variation data; haplotype data (but again no disease associations in database)
2003-NIH Data Sharing Policy goes into effect 2003-Extension of NHGRI Rapid Data Release Policies to Large-scale
Sequencing and Other Community Resource Projects 2003-ENCODE
No longer dealing with just nucleic acid data but data still not of high commercial interest (still “pre-competitive” data)
2005-& beyond….KOMP, GAIN, Molecular Libraries, etc. Chemical structures, phenotype-genotype data, disease association data, etc. Moving closer to data & reagents that have possible commercial value…..
2009 (mandatory) NIH Public Access policy goes in effect 2010 TRND
Example NIH Policies
Many grants/cooperative agreements
must contain IP, Data sharing and/or Material Sharing plans
require grantees to adhere to Best Practices for the Licensing of Genomic Inventions guidance
http://ott.od.nih.gov/policy/genomic_invention.html
require adherence to “special” policies related to IP, publications & data access and use
Genome Wide Association Studies (GWAS) http://grants.nih.gov/grants/gwas/
Molecular Libraries projects
http://mli.nih.gov/mli/mlpcn/documents-definitions/
Relevant NIH Policies
Research Tools Guidelines (1999) http://ott.od.nih.gov/policy/research_tool.html
Data Sharing Policy (2003) http://grants.nih.gov/grants/policy/data_sharing/data_sharing_guidance.htm
NIH Policy on the Sharing of Model Organisms (2004) http://www.nih.gov/science/models/sharingpolicy.html
NIH Public Access (2008) http://publicaccess.nih.gov/
Key Assumptions for NIH Community Resource Projects
“Precompetitive” scientific data should be available in public databases without any restrictions on future use; this accelerates innovation without harming future commercial prospects
Many such data and reagent sets can be produced very effectively by Government entities, charities and their grantees & also via public-private partnerships
Univ. and companies have little incentive to “give away” potentially valuable data and licensable materials—governments need to lead the way (= if you want our money you have to play by our rules)
Companies come and go (or change business strategies) but NIH will always be there…
Lessons Learned
Do pilot studies—not just to see how things go for the scientific/technical aspects of the project but in terms of data access, use & IP policies as well Are “inappropriate” patent applications being filed?
Is there a problem with non-compliance? If yes, by whom?
Do the data generators and data users feel that the policies and any imposed restrictions are reasonable and necessary?
Were there any unintended negative consequences?
Directly involve legal, business development & policy folks from academic & commercial sectors from the beginning
For critical matters make sure that compliance/acceptance of the term/policy is an absolute requirement for the grantee; those holding the purse strings control the shots
Lessons Learned (continued)
Create a reasonable and logical governance structure and empower small working groups Get a wide range of input on draft polices
Chemists think differently than geneticists not just in terms of scientific issues but also in terms of what constitutes cultural or behavioral norms within their communities)
Tech transfer/legal professionals may not embrace a “limited IP” or “no IP” approach
Develop the reagent and data use, access, and related policies (ex. IP and publication) well in advance of announcing the availability of funds and long before grantees are selected
For more information:
Technology Transfer Office, NHGRI 5635 Fishers Ln Ste 3058, Rockville, MD 20852
tel: (301) 402-2537/ (301) 594-2235
fax: (301) 402-9722
e-mail: [email protected]
http://www.genome.gov (then click on Tech Transfer)
available NHGRI technologies
technology transfer information sources
E-mail links to scientific staff
http://ott.od.nih.gov Available NIH technologies (from all 27 ICs)
On-line technology transfer training module
