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Andrea Baruchin, Ph.D. Senior Advisor to the President India-US Technology Summit November 19, 2014.

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Presentation on theme: "Andrea Baruchin, Ph.D. Senior Advisor to the President India-US Technology Summit November 19, 2014."— Presentation transcript:

1 Andrea Baruchin, Ph.D. Senior Advisor to the President India-US Technology Summit November 19, 2014

2 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.

3 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…

4 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”

5 How we Fund Our Programs Industry Government Associations Foundations Individuals FNIH has no endowment

6 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. Support for over 500 projects

7 Funding & Partnership Models FNIH NIH Private Sector Funders Intramural or Extramural Lab $$ Flow FNIH NIH Private Partners $$ Flow Scientific Interaction Intramural or Extramural Lab FNIH Steering Committee/ Project Team Model 1 Model 2 Scientific Programs & Research (and/or)

8 Select Programs “ When you’ve seen one partnership…you’ve seen one partnership.”

9 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)

10 Industry members Government members Non-profit members AMP Participants

11 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

12 Alliance ECOG- Acrin NRGNCI-C SWOG National Clinical Trials Network S1400 Master Protocol S1400 Master Protocol

13 Thank You !

14 Developing effective new medicines takes too long, costs too much and fails too often. AMP

15 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

16 AMP Governance & Membership Alzheimer's disease Steering 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 Diabetes Steering 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 diseases Steering 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

17 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 Diabetes Sequencing & 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

18 AMP – Budget ($Millions)Total ProjectTotal NIHTotal Industry Alzheimer’s Disease Type 2 Diabetes Rheumatoid Arthritis & Lupus Total

19 AMP – IP & Data Sharing Research supported by AMP will be precompetitive All data will be shared broadly AMP supported research will not be patented

20 Biomarkers Consortium Contributing Partners For-Profit Companies Actelion Amgen AstraZeneca Crescendo Bioscience Daiichi Sankyo, Inc Eisai, Inc Johnson & Johnson Eli Lilly & Company Lundbeck Merck Sharpe & Dohme Corp. Metabolon Mitsubishi Tanabe Pharma America, Inc Myriad RBM Pfizer, Inc Regeneron Pharmaceuticals, Inc. Sanofi Takeda Pharmaceuticals USA, Inc Non-Profit Organizations Alzheimer’s Association American Diabetes Association American Orthopaedic Society for Sports Medicine Arthritis Foundation Autism Speaks Biotechnology Industry Organization California Dairy Research Foundation (CDRF) Centre for Proteomic and Genomic Research CHDI Foundation Dairy Research Institute Foundation for Health Improvement and Technology Juvenile Diabetes Research Foundation Pharmaceutical Research and Manufacturers of America PROOF Centre of Excellence Radiological Society of North America US Pharmacopeia

21 Biomarkers Consortium Governance Structure Executive Committee NIH / FDA / CMS / industry / FNIH Inflammation & Immunity Steering Committee Neuroscience Steering Committee Metabolic Disorders Steering Committee Cancer Steering Committee Multiple Project Teams Representatives from NIH, FDA, Industry, Subject experts from academia

22 Biomarkers Consortium Launched 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

23 Biomarkers Consortium Accomplishments 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

24 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

25 Lung-MAP Protocol S1400D FGFR FGFR ampl, mut, fusion S1400C CDK4/6 Cell cycle gene alteration S1400E HGF C-MET Expr S1400B P13K PIK3CA mut S1400A Non-match (Anti-PD-L1) Arm 1 Arm 2 1:1 Arm 1 Arm 2 1:1 Arm 1 Arm 2 1:1 Arm 1 Arm 2 1:1 Arm 1 Arm 2 1:1 1 1 Medi Docetaxel Medimmune 1 GDC Docetaxel Genentech 1 1 Palbociclib 2 2 Docetaxel Pfizer 1 AZD Docetaxel Astrazeneca 1 1 Rilotumumab + erlotinib 2 2 Erlotinib Amgen

26 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

27 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.

28 Current ADNI 2 PPSB Partners

29

30 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


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