Giovina Ruberti MIUR Delegate in the IMI - States Representatives Group

National Research Council

Institute of Cell Biology and Neurobiology, Monterotondo (Roma) gruberti@ibc.cnr.it

Salute in Horizon 2020:

Giornata Nazionale di Lancio dei bandi 2014-15

Roma, 3 dicembre 2013 Ospedale Pediatrico Bambino Gesù

Innovative Medicines Initiative: Achievements, Ongoing Projects, Call 10

and Call 11 and IMI-2 SRA

IMI– challenges and opportunities

Achievements and ongoing IMI projects

IMI2 and Horizon 2020

IMI Call 10 and Call 11

The Innovative Medicines Initiative: Focus, Outcomes and Experiences

The European Union and the pharmaceutical industry

have joined forces to make

drug R&D processes in Europe more innovative and efficient,

enhance Europe’s competitiveness

& address key societal challenges

by forming

the biggest PPP in Life Science

4

Innovative Medicines Initiative: Joining forces in the healthcare sector

Open collaboration in public-private consortia

(data sharing, dissemination of results)

“Non-competitive” collaborative research for EFPIA pharma companies

Competitive calls to select partners of

EFPIA companies (IMI beneficiaries)

Key Concepts

Private

Investment

in kind

(€ 1 billion)

EU Public

Funding

cash

(€ 1 billion)

EFPIA

ACADEMIA

HOSPITALS

PATIENTS’ ORGANISATIONS

SMALL AND MEDIUM-SIZED ENTERPRISES

REGULATORS

Pharma

1

Pharma

2

Pharma

3

Pharma

4

Pharma

5

Pharma

6

A Typical IMI Consortium

Applicant consortia can comprise academics, SMEs, patient groups, regulators, non-profit bodies and intergovernmental organisations. There must be at least two independent legal entities within the applicant consortia at stage one, although in order to cover all requirements of the call topics, applicant consortia are likely to be larger than this. The applicant consortia at stage one does not include EFPIA members. At stage two at least two EFPIA companies must join the consortia. The above-mentioned organisation types are eligible for funding from IMI only if they will carry out the research in an EU Member State or a country associated to the framework programme. Organisations that are not eligible for funding from IMI (for example, non-EFPIA companies that are not classified as SMEs) can participate in the applicant consortia, but must bear their own costs.

Who is eligible to form part of the applicant consortia?

SMEs and IMI funding eligibility To be eligible for IMI funding, SMEs must meet the EU’s official definition of a SME which can be summarised as follows: has fewer than 250 employees; has an annual turnover not exceeding €50 million, and/or; has an annual balance sheet total not exceeding €43 million Under IMI’s rules, SMEs that meet the EU definition when the project starts remain eligible for IMI funding throughout the project, even if they lose their SME status during the project.

Funding rates are the following (only for beneficiaries): - Research activities: a maximum of 75% of total eligible

costs - Other activities, including management and training

activities: a maximum of 100% of total eligible costs Indirect costs - actual indirect cost - or a flat rate of 20% of total eligible direct costs (excluding subcontracting costs and the costs of resources made available by third parties which are not used on the premises of the beneficiary) for organisations which do not aggregate their indirect costs at a detailed level, but can aggregate them at the level of the legal entity.

Help and advice Questions about Calls for proposals, application procedures, and funding rules: e-mail Hugh Laverty (hugh.laverty@imi.europa.eu) Questions about intellectual property: - Visit the intellectual property (IP) web page - E-mail the IMI IP Helpdesk (IMI-IP-Helpdesk@imi.europa.eu) Find project partners through our Partner Search Tool Get advice through your national IMI States Representatives Group member

14 regulators

22 patient

organisations

Key figures of 42 on-going projects

Collective intelligence networks Improved R&D productivity of pharma industries

Innovative approaches for unmet public health needs

113 SMEs

600 Academic

& research

teams

388 EFPIA teams

IMI portfolio – budget breakdown

0,0 1,0 2,0 3,0 4,0 5,0

Rheumatology

Pharmacology & Pharmacy

Endocrinology & Metabolism

Neurosciences

Clinical Neurology

Biochemistry & Molecular Biology

Psychiatry

Genetics & Heredity

Research & Experimental Medicine

Immunology

Overall

IMI papers EU papers

average 2.04 average 1.13

19% of IMI publications are highly cited

Citation impact 2010-1012

13

IMI stimulates collaboration across EU Co-authorship between IMI-supported researchers Calls 1-4

Pre IMI funding award Post IMI funding award

Data & analysis: Thomson Reuters (Custom Analytics & Engineered Solutions)

14

IMI accelerates the development of new therapies for major yet unmet public health needs and

diseases where treatments are lacking

The Anti-Microbial Resistance Conundrum

- Anti-microbial resistance kills 25 000 European citizens every year

- Annual costs are estimated at €1.5 billion

- Only 2 new antibiotic classes developed over the last 30 years

- Low level of industry investments in antibiotic innovation

How IMI addresses infectious diseases

Invested €229 million in projects aiming at:

Solving scientific challenges

Fostering new models of industrial collaborations for antibiotics and diagnostics development

Developing networks for clinical trials

Revisiting regulatory environment

Providing necessary incentives for industry to reinvest in the area

The conundrum of brain disorders

Brain disorders affect 1 in 3 Europeans And cost the economy € 798 billion / year

Developing new drugs to treat brain disorders takes longer, costs more than

for other diseases

There are few effective treatments in this area

IMI projects are tackling these issues

How IMI addresses brain disorders

Invested €148 million in projects aiming at:

Shedding new light on the underlying causes of autism (EU-AIMS)

Combining data to pave the way for new treatments for schizophrenia & depression (NEWMEDS)

Identifying new targets for drugs for chronic pain (Europain)

Developing models to predict the efficacy of drug candidates in Alzheimer’s patients (PHARMACOG)

Revisiting regulatory environment for drug development

Providing necessary incentives for industry to reinvest in brain disorders

Identified brain functional abnormalities that can be reversed in ASD patients by modulating brain 5HT - outcome predictor for clinical trials

Identified significant differences in cortico-cortical anatomical connectivity in ASD patients - outcome predictor for clinical trials

Identified significant differences in brain functional response to emotional sounds in infants at risk for autism - risk predictor

Published first study of EU wide prescribing in ASD

Advances in Autism

6 EFPIA companies; 15 Public organizations; 3 SME 1 patient org; Total Budget: 35.9 Mi €

2 Largest Clinical Trials for Early Detection and Monitoring of ASD Children

Identification of biomarkers that precede ASD onset

− Prospective study of 300 high-risk infants (3 and 7 months) with older siblings with ASD, and 100 low-risk

− Infant cognition, behavior, neuroimaging, neurophysiology

− Relation to symptoms/diagnosis of ASD at outcome

Accelerated longitudinal study for validation of ASD biomarkers in children and adults

− 450 ASD patients and 300 controls

− magnetic resonance imaging, event-related potential, eye-tracking

Platelets: APP metabolites, 55 kD and 25 kD fragments - AlzProtect

Lymphocytes: ~ 150 RNA transcripts related to Abeta pathway, inflammatory pathway and to immune mechanism - Exonhit (now Diaxonhit)

Red blood cells: binding of Abeta1-42 on cellular membrane and change in PKC conformation in red blood cells - Innovative Health Diagnostics (IHD)

Red blood cells: panel of inflammatory markers- Innovative Concept in Drug Development (ICDD)

COMPLETED RECRUITMENT OF 150 MILD COGNITIVE IMPAIRED PATIENTS FOR BIOMARKER MATRIX CLINICAL TRIAL

Novel Disease Markers for Alzheimer’s Disease

12 EFPIA companies; 13 Public organizations; 5 SME Total Budget: 27.7 Mi €

IMI DIABETES PLATFORM Memorandum of Understanding between IMIDIA, SUMMIT and DIRECT

for exchange of data and confidential information

€38.5 million invested in Education and Training programs

More than 1000 scientists have been trained so far

Many more scientists take advantage of industrial internships

Enhanced learning experience

IMI trains a new generation of industrial scientists and regulators, and reduces the cost of continuous

professional development for companies

Catalogued >5000 masters, PhD, CPD and short courses taught in 21 languages, from 39 countries, covering over 60 scientific, therapeutic and biomedical areas from about 1000 universities.

More than 320 students participated in various courses (50% from EFPIA companies) in 2012. Provided 20 new courses in drug safety sciences.

58 students are following the flexible and fully e-learning programme covering medicines risk identification and quantification, medicines and public health, medicine risk communication, assessing the benefits of medicines, and regulatory processes.

24 universities network + 16 universities affiliates. 685 students have been following various courses (28% from EFPIA companies).

Europe’s first Patients’ Academy on Therapeutic Innovation aiming to provide scientifically reliable information on medicines and R&D, as well as an online public library that will empower patients to engage more effectively in the development and approval of new treatments and become true partners in pharmaceutical R&D.

IMI Education and Training programs > 1000 trainees so far

On July 2013, the European Commission released its proposal for IMI2. The EC proposal is now being discussed by the European Parliament and the Eu Member States. 1. IMI2 will build on the successes and lessons learnt under

IMI. Goals: innovative vaccines, medicines and treatments to tackle Europe’growing health challenges and secure competitiveness of Europe’s pharmaceutical industry

increasing number of older people burden on public healthcare systems more reliable and faster clinical trial better regulation new service and products

IMI2

2. What is new? Common rules for all of Horizon 2020. Derogation for IMI2 limited to industry commitments (large industry will not receive EU funding) and the IP rules. Lighter financial rules. Option for other industrial partners to contribute in kind 3. Budget The budget proposed for IMI2 is €3.45 billion. EFPIA €1.5 billion in kind, other life sciences industries may contribute a further €225 million if they join IM2 4. Duration IMI will start in 2014 and will run for 10 years

IMI2

IMI2 SRA

Disease areas of focus

11 priority disease areas (Antimicrobial resistance, Osteoarthritis, Cardiovascular diseases, Diabetes, Neurodegenerative diseases, Psychiatric diseases, Respiratory diseases, Autoimmune diseases, Ageing-associated diseases/conditions, Cancer, Orphan Disease) have been highlighted in the SRA as being of high priority for both the European healthcare system and the pharmaceutical industry.

SRA IMI2 Draft – IMI2

Draft available at http://bit.ly/1axI076

Comments and suggestions are welcome: SRAconsultation@efpia.eu

Strategic Research Agenda for IMI2:

The right prevention and treatment for the right patient at the right time

Innovation Investment Package and Horizon 2020 IMI2 is part of a wider €22 billion Innovation Investment Package which includes proposal for: 4 other JTI • Fuels Cells and Hydrogen 2 (FCH2) – to expand the use of

clean and effcient technologies in transport, industry and energy

• Clean Sky 2 (CS2) – to develop cleanerm quiter aircraft with significantly less CO2 emission

• Bio-based Industries (BBI) - to use renewable resources and innovative technologies for greener everyday products

• Electronic Components and Systes (ECSEL) - to boost electronics manufacturing capabilities

Joint Programmes with Member States European and Developing Countries Clinical Trials Partnership 2 (EDCTP2) European Metrology Research Programme (EMPIR) Eurostars 2 (for SMEs) Active and Assisted Living Research and Development Programme SESAR Joint Undertaking European Air Traffic Management System (SESAR)

Rationale for IMI2

• Innovation in creating effective treatments for diseases is under significant stress/challenges: declining R&D productivity for new drug development; patent expiration; lack or loss of return on investment.

• Mismatch grows between where public health needs lie and where industry invests and succeeds in bringing new interventions to patients.

• Current model is unsustainable. The loss of this industry from Europe is a significant current risk.

• Industry cannot invest alone in disease areas that require complex and costly R&D with uncertain financial returns (market failure).

• Biopharmaceutical capabilities and data are dispersed across Europe, public intervention at individual Member State level is not enough for mobilising the necessary critical mass of knowledge and financial resources.

IMI 2 Building on succes of IMI (1)

IMI accelerates development of new therapies for major yet unmet public health needs and diseases where treatments are lacking

Improving tools for pharmaceutical research

– Developing better tools to predict safety of existing and future medicines

– Developing reproducible models of human disease, thus accelerating development of novel treatments

Public Health Impact of IMI2

IMI2 more focused on public health impact through explicit

reference to WHO Priority Medicines for Europe and the

World report, launched on 9 July

Wider scope and openness to industries beyond pharma

improves public health impact because approaches other

than pharmaceuticals can be addressed

Developing technologies and innovations that improve

people's lives, e.g. prevention and early diagnosis,

technologies that allow people to live safely in their homes

General Objectives of IMI 2

To provide European citizens with timely access to new and effective diagnostics and treatments that improve their health and wellbeing, and

Helping safeguard the future international competitiveness of the European biopharmaceutical industry and secure growth and jobs.

SME Involvement (1)

Very strong SME involvement in IMI: already now around 20% of IMI financial contribution goes to SMEs; this is much better than FP7 Health:

In 2007-2009, less than 10% of EU financial contribution from FP7 Health was awarded to SMEs

Corrective actions were taken by introducing special calls, imposing minimum amount of EU contribution going to SMEs etc.

No such ´forcing' was necessary under IMI

IMI2 will strongly contribute to the target of 20% of EU contribution from Societal Challenge 1 going to SMEs

SME Involvement (2)

Expanded scope and openess to other health-related industries further improves chances for SME involvement in IMI2

Also the possibility to exceptionally fund medium-sized companies offers the potential to even more funding from IMI2 going to SMEs

Widening Participation

Participation in IMI from EU13 Member States is currently weak

Large communication effort to promote IMI in these countries has been undertaken

Widening scope of IMI2 by addressing the entire life science research and innovation value chain and enlarging participation to health-related life science industries beyond pharma is expected to improve opportunities for participation from newer EU Member States

Call 10: just launched – deadline 28/01 Immunological assay standardisation & development for use in assessments of correlates of protection for influenza vaccines.

Call 11: to be launched mid-December

In the pipeline for Q4 2013

Call 11 currently has the following indicative topics: 1. Applied public-private research enabling

osteoarthritis clinical headway (APPROACH) 2. European platform for proof of concept for prevention in Alzheimer’s Disease (EPOC-AD) 3. Blood-based biomarker assays for personalized tumour therapy: value of latest circulating biomarkers 4. Zoonoses anticipation and preparedness initiative (ZAPI).

5. Generation of Research Tools to enable the translation of genomc discoveries into drug discovery projects 6. ND4BB: Epidemiology research and development of novel systemic antibacterial molecules against healthcare-associated infections due to gram-negative pathogens 7. ND4BB: Development of novel inhaled antibiotic regimens in patients with cystic fibrosis (CF) and patients with non-CF bronchiectasis (BE) 8. Ecorisk prediction (ERP)

How to partecipate

1. Visit the IMI website (understand the aims of IMI and the ongoing IMI Projects for sinergy);

1. Study the topic of your interest in details -expertise and/or tools required;ongoing collaborations with FP7 and/or IMI Projects listed in the call as relevant for interaction/collaboration etc.; 3. Partecipate to the IMI webinar; 4. Use the IMI Partner Search Tool; 5. Contact your national representative (gruberti@ibc.cnr.it; 06-90091263)