IMI2: Strategic research agenda and overall approach Hugh Laverty Senior Scientific Project Manager Introducing IMI2: Vienna 16 July 2014
The way in which pharmaceutical companies develop new medicines is changing Regulators Rising R&D cost EU Pricing Patent cliff Pharma HC Reform Generics Declining R&D productivity
New approaches needed Deciphering the complexity of human diseases and finding safe, cost-effective solutions that help people live healthier lives requires collaboration across scientific and medical communities throughout the health care ecosystem. Indeed, we must acknowledge that no single institution, company, university, country, or government has a monopoly on innovation.
Innovative Medicines Initiative: Joining Forces in the Healthcare Sector The biggest public/private partnership in Life Science aiming to: Make drug R&D processes in Europe more innovative and efficient Enhance Europe s competitiveness Address key societal challenges Features: 1:1 funding, joint decision making All EU funds go to SMEs, academia, patient organisations and regulatory agencies Large pharmaceutical industry, represented by EFPIA, contributes in-kind
The IMI Portfolio
The IMI Community Calls 1-8 46 projects > 6000 researchers 714 academic & research teams 410 EFPIA teams 135 SMEs 23 patient org. 14 regulators 61% of projects reported some form of PATIENT INVOLVEMENT REGULATORS ON BOARD OF 12 PROJECTS 50% of projects have REGULATORY AUTHORITIES representatives in Scientific Advisory Boards
The measures of success SUCCESS New models developed & published Setting new standards In house implementation by industry Impact on regulatory guidelines Better Science = Better Decisions
IMI2
New approaches needed Deciphering the complexity of human diseases and finding safe, cost-effective solutions that help people live healthier lives requires collaboration across scientific and medical communities throughout the health care ecosystem. Indeed, we must acknowledge that no single institution, company, university, country, or government has a monopoly on innovation.
The Vision For IMI2 The Right Prevention And Treatment For The Right Patient At The Right Time Biologically heterogeneous patient population effective Adverse events not effective Dx Test e.g. biomarker effective not effective adverse events A B C Trial and Error vs Information based treatment decisions Graphic adapted from C. Carini, C. Fratazzi, Eur. Pharm. Rev. 2008, 2, 39-45
Science is driving advances in diagnosis: breast cancer is actually 10 different diseases Thursday April 19 2012 A landmark study has reclassified the country s most common cancer in breakthrough research that could revolutionise the way we treat breast tumours scientists found breast cancer could be classified into 10 different broad types according to their common genetic features. http://www.nhs.uk/news/2012/04april/pages/breast-cancer-genetic-diversity-mapped.aspx 11
The Evolution of IMI: From bottlenecks in industry to bottlenecks in Industry and Society Make Drug R&D processes in Europe more efficient and effective and enhance Europe s competitiveness in the Pharma sector Idea generation Basic research and non-clinical testing Human testing Regulatory Approval HTA and Pharmacovigilance Daily Medical practice Primary focus of early IMI calls 2007 SRA Shift to also addressing challenges in in society and healthcare 2011 SRA IMI 2 includes real life medical practice 2013 SRA
Objectives of IMI2 what the regulation says Increase the success rate in clinical trials Where possible, reduce the time to reach clinical proof of concept in medicine development Develop new therapies for diseases for which there is a high unmet need and limited market incentives Develop diagnostic and treatment biomarkers for diseases clearly linked to clinical relevance and approved by regulators; Reduce the failure rate of vaccine candidates in phase III clinical trials through new biomarkers for initial efficacy and safety checks; Provide support for the development of tools, standards and approaches to assess efficacy, safety and quality of regulated health products.
For integrated healthcare solutions Alignment with Horizon 2020 objectives of the Health challenge Addressing healthcare priorities identified by the WHO 2013 report on priority medicines for Europe and the world Strategic Research Agenda aimed at progressing the vision of stratified medicines: prevention, treatment and health management Entire cycle from discovery, through development to healthcare delivery and access models Collaboration across sectors to harness all knowledge and technologies which can contribute to IMI2 vision - diagnostics, imaging, IT, medical devices,
Strategic Research Agenda Comprehensive framework for a 10-year programme Prepared with input from 80+ organisations Project ideas from industry and third parties will be screened against it http://goo.gl/jqmp9g
Ischaemic heart disease Cerebrovascular Unipolar depression Diabetes mellitus Hearing loss COPD HIV/AIDS Alcohol use disorders Lower respiratory infections Alzheimer and other dementias Trachea, bronchus, lung cancer Osteoarthritis Cirrhosis of the liver Colon and rectum cancer Breast cancer Prematurity and low birth weight Birth asphyxia and birth trauma Neonatal infections and other Malaria Tuberculosis 2013: WHO report on priority medicines for Europe and the World: societal challenge reflected in the IMI2 SRA 12 10 8 6 4 2 0 WHO report: Percentage of DALYs for top 20 high burden diseases and conditions 1.3. Building on the strengths of Europe... 2.Establishing the Research Priorities for IMI2... 3.Research Objectives of IMI2... 4.Enabling Technologies... 5.Implementation strategies... 1.4. Learning from the Innovative Medicines 2.1. Challenges facing the healthcare ecosys 2.2. The role of Research & Development in 2.3. Regulatory, health technology assessme 3.1. Four major axes of research... 3.1.1. Axis 1: Target validation and biomarker 3.1.2. Axis 2: Adoption of innovative clinical tr 3.1.3. Axis 3: Innovative Medicines... 3.1.4. Axis 4 : Patient tailored adherence prog 4.1. Excellence in Data Management... 5.1. Education and Training required to imp 6.European Health Priorities to be addressed by IM 7.Translating research to tangible benefits for Euro (no 5.2. priority order) Excellence in clinical trial implementatio 6.1. Antimicrobial resistance... 6.2. Osteoarthritis... 6.3. Cardiovascular diseases... 6.4. Diabetes... 6.5. Neurodegenerative diseases... 6.6. Psychiatric diseases... 6.7. Respiratory diseases... 6.8. Autoimmune diseases... 6.9. Ageing-associated diseases... 6.10. Oncology... 6.11. Rare/Orphan Diseases... 6.12. Vaccines... Europe World Therapeutic Areas in IMI2 SRA
IMI2: Major Axes of Research Reclassification of disease by molecular means Biomarker identification/validation (precision medicine) Innovative methodologies to evaluate treatment effect Target Identification and validation(human biology) Determinants of drug /vaccine Safety and efficacy Target & Biomarker Identification (safety & efficacy) European Health Priorities Innovative clinical trial paradigms Adoption of innovative clinical trial designs Benefit/Risk Assessment Innovative drug delivery methodologies Innovative Medicines Patient tailored adherence programmes Healthcare delivery: focus on the treatment programmes not just the medicine Manufacturing for personalised medicines Discovery and Development of novel preventative and therapeutic agents Innovative adherence programmes Drive change in delivery of medical practice
First five big themes (1) Therapeutic Areas and Cross-cutting Themes Differentiating Enablers for all themes 1. Neuro-degeneration Successfully prevent and treat dementia and other neurodegenerative diseases 2. Prevention and treatment of immunemediated disease Advance immunological understanding to deliver new medicines and new and better vaccines 3. Metabolic disorders Tackle all phases of disease and its complications, including prevention and early interception (type 2 diabetes, obesity, dislipidemia, hypertension) 4. Infection control Address big societal problem related to multidrug resistance and create incentives for reinvestment (including antimicrobials, antivirals, vaccines) and develop new and better vaccines 5. Translational Safety identification of predictors of safety and development of point of care for safety biomarkers & Development of new human biology platform to predict toxicity and safety during early drug development Towards early and effective patient access to innovative prevention and treatment solutions (Medicines Adaptive Pathways to Patients, MAPPs): Target validation based on human biology Stratified medicine, precision medicine Innovation in clinical trials Data generation and interpretation (knowledge management) Prevention, disease interception, patient adherence (incl. societal acceptance of vaccines) Effect on medical practice and outcomes (health/disease management) Regulatory framework (including pharmacovigilance) Patient access
First five big themes (2) Prioritisation/selection criteria Field of unmet need Patient-centric approach The science appears ready to make a big change over the next decade Added value of PPP to make a difference (including collaboration with other industry sectors/technologies) Synergies/complementarity with similar initiatives While keeping focus on prioritised questions, there is sufficient room for other projects within the SRA e.g. Oncology; Rare/Orphan Diseases; Psychiatric Diseases; Respiratory Diseases
IMI2 - Broad Participation to be able to set ambitious goals: Bigger budget: >3 Billion Euro, equally shared by EU and industry Not limited to EFPIA members: open for other industries / companies, which can contribute to the PPP goals (Healthcare IT, medical devices, ) SME Funding: Better conditions for medium sized SMEs than in IMI1 (companies according to former EU rules to big for funding but too small to be able to afford non-funded participation) Standardized financial framework: 100% Funding plus 25% overhead, as in general in HORIZON2020
The Role Of The Programme Office A neutral broker: To implement programmes and activities in the common interest of all stakeholders To monitor the use of public funds and industry investment To guarantee fair and reasonable conditions for optimal knowledge exploitation and dissemination To facilitate the interaction between stakeholders, including Intellectual Property agreements To actively communicate and promote IMI and its activities
IMI2: The First Call Two topics: Translational approaches to disease modifying therapy of type 1 diabetes mellitus (T1DM) Discovery and validation of novel endpoints in dry agerelated macular degeneration and diabetic retinopathy Submission date: 12 November 2014
Questions? Hugh.Laverty@imi.europa.eu www.imi.europa.eu