Boosting Drug Development

Through Public-Private Partnerships –

The IMI Model

WRIC Brussels, 05 June 2013

Hugh Laverty, Senior Scientific Project Manager

2 Billion €

Partnership

Innovative Medicines Initiative:Joining Forces in the Healthcare Sector

I Billion €

Public

I Billion €

Private

IMI focus - Hurdles to better healthcare

• Disease heterogeneity

• Lack of predictive biomarkers for drug efficacy and safety

• Insufficient pharmacovigilance tools

• Outdated clinical designs

• Socio-economic approaches not adapted to tailored therapies

• Insufficient incentives to develop drugs for rare or complex diseases

• Poor EU market conditions to create biotech companies

• Lack of training programmes focusing on collaborative approaches

Open collaboration in public-private consortia (data sharing, wide dissemination of results)

“Non-competitive” collaborative research for EFPIA companies

Competitive calls to select partners ofEFPIA companies (IMI beneficiaries)

Key Concepts

EFPIA

IMI

Topic Definition& Launch

Applicant Consortia

EoI Submission

1st ranked EoI + EFPA

Full Project Proposal

SubmissionIMI

Governing Board

approval

Signatures & project kick-off

Academic research teams Hospitals

SMEs

Patients’ organisations

Regulatory authorities

Step 1 Step 2 Step 3 Step 4 Step 5

How it works

TheConsortium

18 weeks 9 weeks 6 weeks

1st ranked EoI

Selection

GB approval of 1st ranked FPP negotiations

start

Signatures & project kick-off

Call Launch

- Optimized timelines

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

How it works – Project Architecture

One IMI IP policy, multiple interests

Flexible Intellectual Property Rights policy allows to accommodate the interests of all stakeholders

Promotes knowledge creation, exploitation and disclosure → open innovation, open access

Ensures fair allocation of rights

Rewards innovation

Make Drug R&D processes in Europe more efficient and effective and enhance Europe’s competitiveness in the Pharma sector

Idea generation

Primary focus ofearly IMI calls

2007 SRA

Basic researchand non-clinicaltesting

Shift to addressing challenges in in society and healthcare

2011 SRA

Human testing Regulatory Approval

HTA and Pharmacovigi-lance

The Evolution of IMI From bottlenecks in industry to bottlenecks in society

SRA – Strategic Research Agenda

Key Figures from 40 on-going Projects

9 regulators

18 patient

org109 SMEs

594 Academic

& research teams

363 EFPIA teams

€ 580.7 mln IMI JU cash contribution

€587.5 mln EFPIA ‘n kindcontribution

Improved R&D productivity of pharma industriesInnovative approaches for unmet public health needs

~ 4500 researchers

Achievements from IMI Projects

IMI projects are delivering:

Robust validated models for drug development

Biomarkers and tools predictive of clinical outcomes - efficacy and safety

Potential new drug targets

Improving the design and process of clinical trials

“Big Data” solutions to leverage knowledge

Education and Training for new generation R&D scientists

Advancements in Autism research

Incidence• 1 in 88 births

Males vs females• 4x frequent in males

Strong Genetic link• 10-40% defined genetic alterations,

High penetrance

Treatment• No treatment for core symptoms• Risperdal and Abilify for irritability

Synapse disorder• Various targets

Autism spectrum disorders (ASD):Current situation

Courtesy: Autism Speaks – Rob Ring

Anti-depressants

Anti-convulsants

Sleep-medication

Stimulants

• No major strategy defined within Europe• No major or concerted efforts in drug discovery• No pre-clinical network• No clinical trial network • No translational network• No regulatory strategy• Late diagnosis and poor awareness (adults) • Poor knowledge of patients needs across life-course (teens into adulthood)• Wide range in treatment strategy with no evidence of efficacy

Autism spectrum disorders (ASD):European Situation 2012

a concerted effort of key stakeholders is neededPrivate Public Partnership

EU-AIMS

Launched April 2012

Identified 4933 de novo mutation based on sequencing 78 Icelandic parent–offspring trios (219 distinct individuals).

As a man ages, de novo mutations increase in his sperm offspring at increased risk of autism or schizophrenia.

Developed a new animal model replicating nonsyndromic autism. Demonstrated a reversal of the condition with specific therapy in mice opportunity clinical development of new treatments for autism.

Advancements in Autism research

Uncovered a key mechanism for controlling synaptic properties NL1 isoform-specific cis-interactions with ionotropic glutamate.

Concept paper on the development of Medicinal products for the treatment of Autism Spectrum Disorder

21 March 2013

Clinical Investigator Networks

COMBACTECombatting Bacterial Resistance in Europe

Establishment and training of European clinical investigators with the expertise and resources

required to participate in global trials

Innovative rodent touchscreen technology

The rodent touchscreen technology is applied, validated and extended in NEWMEDS (schizophrenia) and PharmaCOG (Alzheimer’s)

Jointly developed as industry standard by Orion, UCAM, Janssen, Lundbeck, Roche, Abbott, Lilly, Pfizer

Pharmacologically validated for testing of pro-cognitive agents

Optimizing schizophrenia trials

Proposed ways to reduce required numbers of patients needed for antipsychotic trials while preserving 90% power (p<.05)

Based on resampling of data from 34 such trials (n=11,670 patients) data from Astra Zeneca, Janssen, Lilly, Lundbeck, Pfizer

Sample size can be reduced from 79 to 46 patients per arm by targeting trials

In addition the trial duration can be reduced from 6 to 4 weeks

Current mix =70% female; 20% early episode; 40% enriched

Enriched=prominent positive and negative symptoms

Early episode=under 3 with 4 or more years of illness

Note: Per patient cost 6wk study $70,000-$100,000

Systems Biology Approach in Severe Asthma

Systems Biology Approach in Severe Asthma

Developed various “omics” platforms based on genetic, proteomic, metabolomic, breathomic biomarkers

Generated a preliminary phenotype ‘handprint’ by combining molecular, histological, clinical and patient-reported data

Cohort clinical trial recruitment target reached 1025 subjects, 726 adults, 299 children - 14 European centres

175 000 samples, 1 500 variables measured, and ~ 3 000 data points

An integrative system biology approach to understanding pulmonary diseasesAuffray C. et al. 137: 1410, 2010

Diagnosis and definition of severe asthma: an international consensusBel EH et al. 66: 910, 2011

http://www.ubiopred.european-lung-foundation.org/

Patient Reported Outcome tools in COPD

Selected activity monitors for precisely capturing the Physical Activity (PA)

Developed a conceptual framework around PA

Selected the most appropriate parameters based on patient input

Conducted pre –validation study on 230 subjects

Official interactions initiated with EMA for qualification advice

European Lead Factory

All partners will have access to unique high-quality Joint European Compound Library

≥ 300.000 compounds from industry partners – €60m200.000 compounds from public partners

Industry-like lead discovery platform available for public projects - focus on value generation Addressing ‘intractable targets’ 48 high throughput screening projects per anno Sustainable model to establish independent business entity

European Lead Factory Coordinated by Bayer, Managing Entity: TI Pharma

Exploiting Electronic Health Records

Academic perspective– Provide tools and services to better plan and conduct academic trials – Facilitate comparative effectiveness research

Pharmaceutical perspective– Improve patient recruitment process and study design – Better understanding of real patient populations– Support observational and outcomes research studies in real-world settings– Enable more cost effective research and clinical trials

General Healthcare perspective– Facilitate the re-use of EHR data to more efficiently manage public health issues – Enabling safer and more evidence-based diagnosis and treatment

28

‘Think Big’Research on human diseases at an

unprecedented scale

Access to information on 40 million patients through EHR Alzheimer’ disease: research on 10-times more subjects than ADNI (Alzheimer's

Disease Neuroimaging Initiative)

Metabolics research on > 20,000 obese & Type 2 diabetes subjects Linkage of clinical and “omics” data Development of a secure (privacy, legal) modular platform Continue to build a network of data sources and relevant research

58 partners (3 consortia + Efpia)>200 scientists involved

14 European countries representedTotal budget €56.4m “3 projects in one”

Advancements in benefit-risk assessment methodologies

Case study Natalizumab

• Indication - Relapsing remitting

• approved 2004• license withdrawn 2005 • reintroduced due to patient

demand 2006• severe side effects:

Progressive Multifocal Leukoencephalopathy

• Data source - EPAR• Comparators: placebo, Avonex, Copaxone

30

Within the next 5 years, the Patients‘ Academy will…

develop and disseminate accessible, well-structured and user-friendly

information and education on medicines R&D

build expert capacity by training patient advocates,and competencies among patients and the public

create the leading public library on medicines R&D: 7 languages, “creative commons” license

facilitate patient involvement in R&D to partner up with academia, authorities, industry, ethics committees

Mapping Collaborative Networks – Call 1-3Collaborative publicationsamong IMI researchers

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

(until 08)

Collaborative activity fostered by IMI

2007 2008 2009 2010 2011 20120

400

800

1,200

1,600

2,000

Within-Sector Cross-Sector

Num

ber o

f col

labo

ratio

ns

Collaborations between researchers engaged in IMI projects – Call 1-3

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

Addressing key scientific challenges

Developing tools to translate scientific advances into regulatory guidelines

Considering new pathways to accelerate patient access to innovative therapies

Providing a neutral platform that fosters collaboration between stakeholders

How public-private partnerships move personalized medicine forward

IMI - The Neutral Trusted Party

Fosters large scale industry collaboration and engagement with scientific community

Promotes active involvement of patients, regulators and payers

Enables innovation via join effort where singular approach has failed so far

Facilitates Intellectual Property agreements

SUCCESS

The measures of success

New model developed &

published

Setting new standards

In house implementation

by industry

Impact on regulatory guidelines

THANK YOU !www.imi.europa.eu