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Andrea Baruchin, Ph.D.Senior Advisor to the President
India-US Technology SummitNovember 19, 2014
Purpose Created by Congress:→ To support the NIH in its mission; → To advance collaboration with biomedical researchers from universities,
industry and not-for-profit organizations.
Structure• 501(c)(3) not-for-profit organization;• Independent Board of Directors;• NIH Director and FDA Commissioner ex-officio Board Members
Highlights• Raised over $800 million since 1996;• Supported over 500 projects;
• research partnerships • scientific education/training• conferences/events • capital programs
Building partnerships for discovery and innovation to improve health.
Our role…
Identify:• Important scientific problem• Key players• Resources required and sources of
support• Neutral convener; trusted party to
provide safe harbor for discussions
Establish:• Highest level of ethical standards• Clear goals and milestones• Effective mechanism to generate
scientific consensus• Nimble infrastructure and project
expert project management
Facilitate: • Discussions with key opinion leaders
and regulatory decision makers• Integrated approach to cross-sector
partnerships• Communications; ensure all partners’
voices are heard;
Enable: • Sharing of data and expertise to
collaboratively address medical needs
• Resource mobilization• Manage grants, contracts, and
projects; oversee and conduct research
What we do, how we do it…
Lessons Learned
• A matrix that is greater than the sum of its parts;
• Well defined objectives, budgets, milestones and deliverables;
• Common governance, rules and legal framework;
• Realistic funding goals, expectations and timelines;
• Projects aligned with donor interest;
• Appreciation of the value of gifts;
• Collaboration adds complexity: must “play nice with others!”;
• Nimble, transparent and accountable.
Partnership “must-haves”
How we Fund Our Programs
Industry
Government
Associations Foundations
Individuals
FNIH has no endowment
Funding History
$808 million
Our history of support for hundreds of projects since 1996 offers many models for partnerships between the public and the private sectors, ranging from simple fund transfers to highly complex and interactive programs.
TrainingEventsCapitalResearchAgencyFamily FundsUnrestrictedNIH Contribution
$816 M
Support for over 500 projects
Funding & Partnership Models
FNIH
NIH
Private Sector Funders
Intramural or Extramural
Lab
$$ Flow
FNIH
NIH
Private Partners
$$ Flow
Scientific Interaction
Intramural or Extramural
Lab
FNIHSteering
Committee/Project Team
Model 1 Model 2
Scientific Programs &
Research
(and/or)
Select Programs“When you’ve seen one partnership…you’ve seen one partnership.”
• Public-private partnership with NIH, 10 companies, non-profits.
• Aims to distinguish targets of disease most likely to respond to new therapies in:
• Alzheimer’s Disease;• Type 2 Diabetes;• RA/SLE.
• Partners have developed research plans; are sharing costs, expertise, resources.
• Launched in 2014, $230M investment over five years on pilot projects.
• FNIH acts as a neutral convener, secures the funding and scientific expertise, establishes partnership governance and provides program and project management for AMP.
Accelerating Medicines Partnership (AMP)
Industry members
Government members
Non-profit members
AMP Participants
• Qualifies biomarkers for specific applications in diagnosing disease, predicting therapeutic response, and improving clinical practice.
• Generates information useful to inform regulatory decision-making.
• Employs rigorous, inclusive governance and project management with clearly defined goals and milestones.
• Addresses a broad range of disease / therapeutic areas – cancer, neuroscience, metabolic disorders, immunity & inflammation.
• Pre-competitive; makes consortium project results broadly available to the entire scientific community.
Our Founding Partners: FDA, NIH, FNIH, PhRMA, BIO, CMS
Fosters the exchange of knowledge and expertise among industry, academic, and government leaders
Biomarkers Consortium
Alliance
ECOG-Acrin
NRGNCI-C
SWOGNational Clinical Trials
Network
S1400Master Protocol
Thank You !
Developing effective new medicines takes too long, costs too much and fails too often.
AMP
Success
Efficacy Failures
Other Failures
Success
Other Failures
Current targets Animal models Cell lines
AMP targets Emerging Technologies
• DNA sequencing• Proteomics• Single-cell analysis• Bioengineered cells• Imaging
Extensive Human Data• Tissue/blood samples• Clinical information• Demographics
Big Data Tools
Efficacy Failures
Lack of efficacy currently accounts for more than half of all drug failures in Phase II clinical studies
AMP’s target validation efforts aimed at improving efficacy and increasing success rate
Efficacy
AMP Governance & Membership
Alzheimer's diseaseSteering Committee
FNIH program management
Extended Executive Committee Core Executive Committee
Members•Francis Cuss, BMS
•Richard Hodes, NIA•Kathy Hudson, NIH•Tom Insel, NIMH
Co-chairs• Mike Decker, AbbVie• Neil Buckholtz, NIA
EC Liaison• Richard Hodes, NIA
Members• Maria Carrillo, ALZ• Xiaoming Guan, GSK• Tim Harris, Biogen Idec• Walter Koroshetz, NINDS• Nick Kozauer, FDA• Mark Mintun, Lilly • Pat Walicke, NINDS
Type 2 DiabetesSteering Committee
Co-chairs• Andy Plump, Sanofi• Phil Smith, NIDDK
EC Liaison• Griffin Rodgers, NIDDK
Members• Keith Demarest, J&J• Joe Hedrick, J&J• Dermot Reilly, Merck• Hartmut Ruetten, Sanofi• Melissa Thomas, Lilly• Jeff Pfefferkorn, Pfizer• Liangsu Wang, Merck• Dan Rader, UPENN
RA, SLE & related diseasesSteering Committee
Co-chairs• Marty Hodge, Pfizer• Bob Carter, NIAMS
EC Liaison• Bob Carter, NIAMS
Members• Christopher Arendt, Sanofi• Beth Axtell, Arthritis Foundation• Carolyn Cuff, AbbVie• Ellen Goldmuntz, NIAID• Satwant Narula, BMS• Lisa Olson, AbbVie• Dan Rotrosen, NIAID• Susana Serrate-Sztein, NIAMS• Jonathan Zalevsky, Takeda• Dennis Zaller, Merck• Jeff Browning, ALR/RFI
• Bill Chin, PhRMA• Bill Hait, J&J• Jim Sullivan, AbbVie• Patrick Vallance, GSK• Rupert Vessey, Merck
• Doug Williams, Biogen Idec• Janet Woodcock, FDA• Tachi Yamada, Takeda• Elias Zerhouni, Sanofi
•Steve Katz, NIAMS•Rick Lifton, YALE•Jan Lundberg, Lilly•Griffin Rodgers,
NIDDK
Co-chairs•Francis Collins, NIH
•Mikael Dolsten,
Pfizer • Steve Hoffmann, FNIH• Maria Vassileva, FNIH• David Wholley, FNIH
AMP – Research Topics
Disease area Research plan topics Deliverables and approach
Alzheimer's disease
Exploratory biomarker validation in clinical trials and network analysis on human tissue
• Embed (ie, incentivize the use of) exploratory biomarkers in NIH-funded clinical trials to develop biomarkers of disease progression and surrogate endpoints
• Conduct network analysis in human brain samples to identify genetic nodes & networks linked to AD to support target identification & validation
Type 2 DiabetesSequencing & phenotyping of targets of interest and a tool to enable easy interrogation of all available data
• Create a knowledge portal containing comprehensive T2DM (& diabetic complications) genotype/phenotype data sets – apply informatics to identify predictors of risk and potential drug targets
• Conduct targeted sequencing/genotyping of high priority targets of interest (as defined by industry
RA, SLE & related autoimmune
diseases
Immune module deconstruction with blood/tissue and cross-disease comparisons
• Conduct extensive profiling of key immune modules in highly refined subsets of relevant cells in informative cohorts to establish pathway/network maps of RA & SLE; high priority targets identified from pathway analysis to be validated via RNAi. The data will be made available in a knowledge portal
- Informative cohorts include: Early RA, Established RA (responder/non-responder), Lupus Nephritis, Skin Lupus
Funding for AMP projects are split 50/50 between industry participants and NIH
AMP – Budget
($Millions) Total Project Total NIH Total Industry
Alzheimer’s Disease 129.5 67.6 61.9
Type 2 Diabetes 58.4 30.4 28
Rheumatoid Arthritis & Lupus 41.6 20.9 20.7
Total 229.5 118.9 110.6
AMP – IP & Data Sharing
• Research supported by AMP will be precompetitive
• All data will be shared broadly
• AMP supported research will not be patented
Biomarkers ConsortiumContributing Partners
For-Profit CompaniesActelionAmgenAstraZenecaCrescendo BioscienceDaiichi Sankyo, IncEisai, IncJohnson & JohnsonEli Lilly & CompanyLundbeckMerck Sharpe & Dohme Corp.MetabolonMitsubishi Tanabe Pharma America, IncMyriad RBMPfizer, IncRegeneron Pharmaceuticals, Inc.SanofiTakeda Pharmaceuticals USA, Inc
Non-Profit OrganizationsAlzheimer’s AssociationAmerican Diabetes AssociationAmerican Orthopaedic Society for Sports MedicineArthritis FoundationAutism SpeaksBiotechnology Industry OrganizationCalifornia Dairy Research Foundation (CDRF)Centre for Proteomic and Genomic ResearchCHDI FoundationDairy Research InstituteFoundation for Health Improvement and TechnologyJuvenile Diabetes Research FoundationPharmaceutical Research and Manufacturers of AmericaPROOF Centre of ExcellenceRadiological Society of North AmericaUS Pharmacopeia
Biomarkers ConsortiumGovernance Structure
Executive CommitteeNIH / FDA / CMS / industry / FNIH
Inflammation & Immunity
Steering Committee
NeuroscienceSteering Committee
Metabolic Disorders
Steering Committee
CancerSteering
Committee
Multiple Project TeamsRepresentatives from NIH, FDA, Industry, Subject experts from academia
Biomarkers ConsortiumLaunched Projects
Neuroscience PET Radioligand in Neuroinflammation Alzheimer’s Plasma Proteomics Alzheimer’s CSF Proteomics* Alzheimer’s / MCI Placebo Data Analysis*
Executive Committee■ Kidney Safety Biomarkers■ Skin Infection Pneumonia
(CABP/ABSSSI)■ Hospital-Acquired & Ventilator Acquired
Bacterial Pneumonia
Cancer• FDG-PET in Lung Cancer• FDG-PET in Lymphoma I-SPY 2 Trial** Minimal Residual Disease in ALL
Inflammation and Immunity■ Osteoarthritis Biomarkers
Metabolic Disorders Adiponectin Carotid MRI Reproducibility Sarcopenia Consensus Definition■ Atherosclerosis In-Silico Modeling■ Beta Cell Clinical Studies■ Bone Quality
■ Ongoing Projects Completed Projects * Final publication pending ** Transitioned to standing trial
Biomarkers ConsortiumAccomplishments
16 Total projects launched
8 Projects completed
3 New projects recently approved
3 FDA guidances completed with BC contributions
4 Drugs advanced toward approval with support from BC projects
20+ Journal articles published and mentioned in 50 others
• Multi-arm Master Protocol
• Homogeneous patient populations & consistent eligibility from arm to arm;
• Each arm independent of the others;
• Infrastructure facilitates opening new arms faster;
• Phase II-III design allows rapid drug/biomarker testing for detection of “large effects.”
• Screening large numbers of patients for multiple targets by a broad-based NGS platform reduces the screen failure rate.
• Provides a sufficient “hit rate” to engage patients & physicians.
• Bring safe & effective drugs to patients faster.
• Designed to facilitate FDA approval of new drugs.
Rationale for Lung-MAP Protocol
Lung-MAP Protocol
S1400DFGFR
FGFR ampl, mut, fusion
S1400CCDK4/6
Cell cycle gene alteration
S1400EHGF
C-MET Expr
S1400BP13K
PIK3CA mut
S1400ANon-match(Anti-PD-L1)
Arm1 Arm2
1:1
Arm1 Arm2
1:1
Arm1 Arm2
1:1
Arm1 Arm2
1:1
Arm1 Arm2
1:1
1 1 Medi47362 2 DocetaxelMedimmune
1 GDC-00322 DocetaxelGenentech
1 1 Palbociclib2 2 Docetaxel
Pfizer
1 AZD45472 DocetaxelAstrazeneca
1 1Rilotumumab+ erlotinib
2 2ErlotinibAmgen
Alzheimer’s Disease Neuroimaging Initiative (ADNI)
• Natural history study of normal cognitive aging, mild cognitive impairment (MCI) and early Alzheimer’s disease (AD).
• Launched in 2004 at over 50 clinical sites in the US and Canada.• Designed to develop methods, acquire data, and form a collaborative network of clinical and
imaging sites to facilitate the utilization and evaluation of neuroimaging and other biomarkers for use in clinical trials aimed at slowing the onset and progression of Alzheimer’s disease.
• Public-private partnership, funded through the NIH ($100M) and the private sector, ($40M) coordinated by the Foundation for the NIH
• World-wide network to improve AD studies and clinical trails for disease-modifying treatments
• Optimize and standardize biomarker methods for early detection and disease progression.• Validate biomarkers for use in clinical trials:
– As measures of change– As diagnostics or predictors (symptomatic and re-symptomatic)
• ADNI 3 planning now underway
About ADNI
• Largest public-private partnership on AD for industry leaders to work together in a proven, pre-competitive environment:– Cost sharing and cost savings;– High level interactions with top clinical/biomarker AD investigators;– Idea and data sharing not possible in competitive environment;– Interaction with FDA – ongoing guidance on endpoints and validation.
• Study design and objectives address industry needs:– Standardization of all methods for regulatory approval;– Identification of patients at risk in the pre-dementia stage;– Validation of biomarkers to measure change and treatment effects;– Validation of biomarkers to identify early AD pathology;– Longitudinal data with biomarkers for design and powering of trials;
• ADNI plays a major role in:– Providing new information concerning the pathophysiology of AD;– Developing early detection methods and improved treatment trials;– Determining diagnostic requirements;– Providing tools for evaluating milder patients;– Understanding disease progression/rate of change during different stages of
disease.
Current ADNI 2 PPSB Partners
Board of Directors
• Charles Sanders, MD – ret. GSK• Mrs. William McCormick Blair – Lasker
(Emeritus)• Kathy Bloomgarden, PhD – Ruder Finn• Mrs. William Cafritz – Kennedy Center• James Donovan – Goldman Sachs• Joseph Feczko, MD – retired Pfizer• Maria Freire, PhD – FNIH • Miles Gilburne – ZG Ventures• Paul Herrling, PhD – Novartis• Ronald Krall, MD – ret. GSK• Sherry Lansing – Sherry Lansing Foundation• Freda Lewis-Hall, MD – Pfizer• Edison Liu, MD, PhD – Jackson Labs• Ann Lurie – Lurie Investments, Inc.
• Joel Marcus – Alexandria Real Estate Equities• Steven Mayer – ret. Human Genome Sciences
& CoGenesys• Paul Montrone, PhD – Perspecta Trust• Martin Murphy, PhD – AlphaMed Consult• Garry Neil, MD – retired Johnson and Johnson • Steven Paul, MD – Weill Cornell Med College• Hon. John Porter – Hogan Lovells• Mrs. Jillian Sackler, DBE – AMS Found• Mrs. Lily Safra – Safra Foundation• Ellen Sigal, PhD – Friends of Cancer Research• Solomon Snyder, MD – Johns Hopkins• Nina Solarz - philanthropist• Samuel Thier, MD – Harvard Medical School• Anne Wojcicki – 23andMe
NIH Director/FDA Commissioner ex-officio Board members