Joining forces to accelerate biomedical innovation The Innovative Medicines Initiative www.efpia.eu
What s in IMI? In IMI the industry and the Commission are partners IMI delivers solutions for patients IMI supports evolution of collaborative research and moves boundaries of precompetitive collaborations IMI has been a preferred (or unique) mechanism of collaboration with industry for regulatory and other health authorities IMI addresses scientific, R&D productivity and regulatory challenges with direct impact on company portfolios IMI addresses access, healthcare systems and business models & other industry policy objectives www.efpia.eu 2
Drivers for value generation in IMI Address complex challenges and bottlenecks that a single pharmaceutical company cannot solve alone Access innovative capabilities / expertise / technologies Access to data and samples (preclinical and clinical, registries, big data approaches) Alignment with health & regulatory authorities (e.g. biomarker, clinical trial designs, market access) Share burden and risk (e.g. antibiotics, preclinical safety) Build critical mass
IMI a safe harbour for pre-competitive collaboration
Low hanging fruits have all been harvested (or very few are left) People live longer, diseases are more complex, burden of disease is higher Health budgets are under pressure and focused on sick-care rather than health-care New technologies and big data offer new (business) opportunities and challenges One company or one sector alone will not progress or will progress slowly Research and societal agendas need to converge Safe harbours are needed to develop and test new models to reduce attrition, accelerate innovation, improve patient outcomes www.efpia.eu 5
The Innovative Medicines Initiative: the largest public-private partnership for health research worldwide 5,276 billion IMI1 2 billion from 2008 2014 IMI2 3,276 billion from 2014-2024 Part of the EU FP7 and Horizon 2020 R&D funding www.efpia.eu 6 Source: http://www.imi.europa.eu/content/mission
Industry led agenda Industry/Commission decisions public/private implementation Industry defines priorities and projects IMI executive office drives consultations and review processes IMI Board (5 industry/5 European Commission) makes decision IMI programme office drives processes and facilitates and monitors implementation EFPIA Innovative Medicines Strategy Group (InnoMedS = ex RDG) SGGs = Strategic Governing Groups Advisory Bodies: State Representative Group IMI Scientific Committee EFPIA delegation led by Marc de Garidel, CEO Ipsen Paul Stoffels, Member of EFPIA delegation to the Governing Board 7
IMI2: Big themes all opportunities for integration Neuro-degeneration SGG Successfully prevent and treat dementia and other neurodegenerative diseases Prevention and treatment of immune-mediated disease SGG Advance immunological understanding to deliver new medicines and new and better vaccines Metabolic disorders SGG Tackle all phases of disease and its complications, including prevention and early interception Infection control SGG Address big societal problem related to multidrug resistance and develop new and better vaccines Translational Safety SGG Development of new human biology platform to predict toxicity and safety during early drug development Differentiating enablers for all themes Target validation based on human biology Stratified medicine, precision medicine Innovation in clinical trials Prevention, disease interception, patient adherence Effect on medical practice and outcomes (disease and health management) Regulatory framework Patient access Data generation/interpretation and knowledge management SGG Under consideration: Advanced Therapies (incl. manufacturing Oncology SGG (since 2016) Improve the outcome of cancer patients and reduce cancer s burden on society by driving integrative, dynamic personalized cancer treatment. www.efpia.eu 8
Increase R&D productivity, accelerate delivery and uptake of innovative solutions, improve patient outcomes What s in it for healthcare systems? What s in it for my business? What s in it for the health sector? What s in it for my sector?
What is precompetitive? Research Preclinical development Use and outcomes measures Clinical development Evaluation
IMI accelerates innovation Multiple companies join force where they would fail alone: Identify missing or weak links in medicines pathways that hold progress Combine (often) proprietary knowledge, data and assets Open them up for challenge by and collaboration with public partners Validate proposed solutions during project lifetime in R&D practice Work at Scale Solutions for diseases with high burden and cost for patients and society Solutions that challenge current business models and focus on value for patients and sustainable healthcare Tracking and addressing science gaps and inefficiencies from discovery to disease management www.efpia.eu 11
Example: Accelerating Alzheimer s Research AETIONOMY BIOMARKERS ROADMAP DEFINE AND MEASURE OUTCOMES EMIF-AD DATA AND MODELLING EPAD COHORTS AND ADAPTIVE TRIALS Pharma-Cog TOOLS TO MEASURE PROGRESSION www.efpia.eu 12
End-to-End: R&D accelerator Target validation ELF Clinical trials Networks EU-AIMS COMBACTE Hit to Lead Enable Big Data EMIF EHR4CR Biomarker acceleration SAFE-T www.efpia.eu 13
Impact on innovation cycle DISCOVERY New lines of enquiry in drug development (thanks to large data sets) Identification of new hits and leads (screening center with proprietary compounds) First human Beta cell line for diabetes research LATE DEVELOPMENT Definition of regulatory endpoints, e.g.; for autism, sarcopenia, asthma, pain Development of antibiotics and Ebola vaccines and diagnostics Clinical trial networks and fast-fail cohorts (pediatrics, antimicrobial resistance, autism) EARLY DEVELOPMENT Regulatory qualified safety biomarkers In silico predictive models Novel epigenetic mechanisms and early biomarkers for non-genotoxic carcinogenesis Normalised and structured data of about 8,000 legacy GLP toxicology reports from 13 Pharma companies, and about 100 predictive algorithms PATIENT ACCESS & USE Continued dialogue on merits of outcomes based and adaptive models Integration of patient voice in benefit risk evaluations Definition of evidentiary standards for pragmatic trials Methodological standards in pharmacovigilance
Regulatory impact examples: EU-AIMS (Autism) received several letters of support in Dec 2015 for clinical outcomes and 4 patient stratification methods SAFE-T received 2 letters of support in September 2016 for DILI and DIKI biomarkers PROACTIVE qualification for PROs (asthma) expected in 2017 MARCAR evidence for ongoing ICH S1 guidance on carinogenicty discussion EUROPAIN evidence for Guideline on clinical development of medicinal (including patient stratification PROTECT - SmPC-ADR Database, Inventory of drug consumption databases, Integration of recommendations on Good Signal Detection for pharmacovilance and pharmacoepidemiology
In tune with the healthcare systems New business models: BD4BO outcomes based healthcare systems Drive AB balance rational use and continued investment into new antibiotics Adapt Smart adaptive pathways based on outcomes measures www.efpia.eu 16 16
The Big Data for Better Outcomes programme at a glance "Big data for better outcomes" Goal: Support the evolution towards outcomes-focused and sustainable healthcare systems, exploiting the opportunities offered by big and deep data sources Design sets of standard outcomes and demonstrate value COORDINATION AND SUPPORT ACTION (CSA) DISTRIBUTED DATA NETWORK 1 2 3 4 Increase access to high quality outcomes data Use data to improve value of HC delivery Increase patient engagement through digital solutions Coordinatio n and operational topics Themes / Enablers ROADS: ALZHEIMER'S DISEASE HEMATOLOGIC MALIGNANCIES CARDIOVASCULAR Diseasespecific topics PROSTATE CANCER MULTI-DISEASE / MULTI-MORBID PATIENTS www.efpia.eu 17
All healthcare ecosystem stakeholders team up to improve outcomes in Hematologic Malignancies One of the largest funded project within "IMI projects are best practice in the industry" FDA representative 52 partners, including 45 public partners from 10 different EU countries Industry and public stakeholders as equal partners to co-create, shape and improve patient care Tech and biotech Pharma Regulators / HTAs Patient associations Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in HematologY Universities Medical associations Research inst. and hospitals
EFPIA Partners in Research address the need for quality technology integration The evolving societal and healthcare challenges as well as opportunities offered by new sciences and technologies, call for a much more integrated health research environment than in the past. Responding to the need for integrated cross-sector collaboration in the R&D, EFPIA has created "Partners in Research" as a constituent entity open to companies operating in associated sectors (imaging, MedTech, diagnostics, animal health, IT including e- and m- technologies, contract research organisations ). This membership status makes it possible to jointly design and implement collaborative R&D initiatives in the framework of IMI (the largest public private partnership in health research worldwide), which will ultimately implement new R&D, operational and business models. www.efpia.eu 19
From Data & Knowledge Management to Digitisation
SGG Data and Knowlegde Management Mission: Diseases & Mechanisms (Therapeutic) Development Optimization, Enabling Platforms Sustainability & data governance Budget Total investment to date IMI1+IMI2: 321,3 MILL Total number of DKM projects (ended or ongoing): 14 Total number of upcoming projects: 2 Total number of 2017 topics in preparation: 4 Astellas, AstraZeneca, Bayer, Biogen Daiichi-Sankyo, Grünenthal, GSK, Janssen, Eli Lilly, MSD, Novo Nordisk, Orion, Pfizer, Roche, Sanofi, Servier, UCB + EFPIA partners in research: Illumina, Psychogenics, SAS
IMI 1: Strategic Governance Group Data Knowledge Management ADVANCE: Accelerated development of vaccine benefit-risk collaboration in Europe (2013-2018) DDMoRe: Drug Disease Model Resources (2011-2016) EHR4CR: Electronic Health Records Systems for Clinical Research (2011-2016) EMIF: European Medical Information Framework (2013-2017) etox: Integrating bioinformatics and chemoinformatics approaches for the development of Expert systems allowing the in silico prediction of toxicities (2010-2016) etriks: Delivering European Translational Information & Knowledge Management Services (2012-2017) GetReal: Incorporating real-life clinical data into drug development (2013-2017) Open PHACTS: The Open Pharmacological Concepts Triple Store (2011-2016) WEB-RADR: Recognising Adverse Drug Reactions (2014-2017)
IMI 2 Project Portfolio of DKM (ongoing and upcoming projects and topics in 2017) Overall budget: 128 Mill (2015-2017) Big Data for Better Outcomes ROADMAP - Real World outcomes across the AD spectrum for better care: Multi-modal data Access Platform (2016 2018) HARMONY - Development of an outcomes focused informatics platform for patients with hematologic malignancies (2017 2022) DO->IT - Big Data for Better Outcomes - Coordination and Support Action (2017 2019) Call 7 Topic: Increase access and use of high quality data to improve clinical outcomes in heart failure (HF), atrial fibrillation (AF), and acute coronary syndrome (ACS) patients Call 10 Topic: How Big Data could support better diagnosis and treatment outcomes for Prostate Cancer 2017 Topic: Multi-morbid patients Sustainability and data governance 2017 Topic: FAIRfication of IMI and EFPIA data 2017 Topic: European Health Data Network (DDN) Development optimisation PRISM: Psychiatric Ratings using Intermediate Stratified Markers: providing quantitative biological measures to facilitate the discovery and development of new treatments for social and cognitive deficits in AD, SZ, and MD (2016 2019) Enabling platforms RADAR-CNS - Remote assessment of disease and relapse - Central nervous system (2016-2021) Call 12 Topic: Development and validation of technology enabled, quantitative and sensitive measures of functional decline in people with Alzheimer s Disease (RADAR- AD) 2017 Topic: Driving compliance and adherence through optimized digital information
Approved 2017 Scientific Priorities for SGG DKM ESTABLISHING A SUSTAINABLE LEGACY OF IMI DATA ASSETS: Develop solutions to make a significant portion of the data from IMI projects hosted in a sustainable way, accessible and interoperable. ACCESS, STANDARDS AND INTEROPERABILITY: Biomedical metadata registry - Develop sustainable, industry-wide metadata standards and allow queries within individual and across different databases Coordination and Support Action for building the basis for a common European biomedical 'language' DEVELOPMENT OF ENABLING PLATFORMS TO SUPPORT NEW RESEARCH PARADIGMS: Life science networks - Develop advanced network-based in silico approaches to get a better mechanistic understanding and hypothesis formulation Open Phacts Reasoning Engine - Tools and methods to facilitate the application of machine learning to predict biochemical activities of chemical structures making use of historical biological assay data Big Data for Better Outcomes (BD4BO) Distributed Data Network - Establishing a core distributed data infrastructure to allow real world evidence data repositories to be combined RADAR-Alzheimer s Personal health data ecosystem - Build data management systems that can provide individuals with ownership of their own health care data Adaptive designs: Develop solutions to improve the adoption of adaptive methods in R&D process DIGITAL SOLUTIONS FOR BETTER COMPLIANCE AND ADHERENCE
Accomplishments to date of SGG Data Knowledge management Good data management practices accepted Delivery of platforms for data sustainability in public private partnership environment Enabling platforms Faster translation of insights from real world health data to biomedical research and development approaches. Develop coherent and transparent framework to address data privacy and personal integrity issues inherent in the use of health records and personal genomic data. Improved data sharing and interpretation to allow the progress of new scientific insights to support and accelerate medicines development NEXT STEP: move towards digitalization in the pharmaceutical sector
The data network is part of the IMI2 Big Data for Better Outcomes (BD4BO) programme BD4BO" Goal: Support the evolution towards outcomes-focused and sustainable healthcare systems, exploiting the opportunities offered by big and deep data sources Design sets of standard utcomes and demonstrate value COORDINATION AND SUPPORT ACTION (CSA) EUROPEAN HEALTH DATA NETWORK (EHDN) 1 2 3 4 Increase access to high quality outcomes data Use data to improve value of HC delivery ROADS: ALZHEIMER'S DISEASE Increase patient engagement through digital solutions Coordination and operational topics Themes / Enablers HEMATOLOGIC MALIGNANCIES CARDIOVASCULAR PROSTATE CANCER Diseasespecific topics MULTI-DISEASE / MULTI-MORBID PATIENTS www.efpia.eu 27
JOIN: European Health Data Network (EHDN) Indicative duration: 5 years Call 12 launch: 19 July 2017 Submission deadline: 24 Oct 2017 Short proposals: 16 May 2018 Indicative contributions: IMI funding: 12 700 000 EFPIA inkind: 12 700 000 The central theme for the BD4BO Programme is the prospect of an outcomes-driven, sustainable healthcare. At the same time, it is recognized that reuse and analysis of healthcare data holds the key to the transition to an outcomes-based healthcare system, under the maxim that, you cannot change, what you do not measure. The EHDN initiative seeks to address this particular challenge by converting relevant datasets across Europe to a common format and standard so that they can be more efficiently used to their full potential within a federated network to achieve the objectives as mentioned above, while respecting patient privacy, local data provenance, governance and applicable regulations. T I T L E O F T H E P O W E R P O I N T 28
How does this relate to earlier efforts? EMIF & EHR4CR have proven that effective collaboration between public and private entities is required to overcome some of the barriers preventing access to retrospective data from different sources. There is significant potential to build on these efforts, while expanding the scope by involving the necessary public stakeholders (payers, HTAs, regulators, patient advocacy groups) to build trust and provide confidence to data providers. HTAs payers EMIF regulators EHR4CR data providers patient advocacy groups www.efpia.eu 29
Quo Vadis?
Digital agenda EFPIA Paying for Outcomes Vision (BD4BO) Value of health data Creating value through digital technologies and technology integration across the value chain European Medical Verification Systems IMI Think Big European Union Review of Digital Single Market Strategy (including open science data cloud, free flow of data, data reuse, e-prescriptions, etc.)
Conclusions from the Hever discussion Theme Examples of areas to consider Examples of impact sought R&D head sponsor Immunology/mic robiome Human biology across therapeutic areas incl. Neuroinflammation, immunometabolism, immunooncology Understanding and influencing immune system in all types of diseases (learning from ongoing translational research) E. Zerhouni, SANOFI J. Reed, ROCHE Antimicrobial resistance Antibiotics; vaccines; diagnostics Product development and incentives Integrated care high societal need P. Vallance, GSK Big data & digital health Digital devices/data generation and standardisation/ Data sharing models, digital markers, AI, regulatory pathways for RWE Integration of digital technologies and real world evidence in drug development and medical practice V. Narasimhan, NOVARTIS Regulatory pathways Optimised clinical trials, combinations, adaptive trials and associated regulatory practice Faster, cheaper and more effective trials that take advantage of variety of data and digital technologies P. Stoffels, JANSSEN M. Dolsten, PFIZER (US liaison) 32
Think Big timelines Preliminary ideas end May 2017 Workshops to define projects and programmes with critical mass of company support by mid July 2017 Finalisation of topics/programmes to be launched in 2018-2020 from October 2017 onwards 33
SGG Digital Health Reconciliation of different objectives in Q3 and review of mission Launch of Think Big projects (50 to 100 mln industry in kind contribution and matching EU funding)