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Transcript of Boosting Drug Development Through Public-Private Partnerships – The IMI Model WRIC Brussels, 05...
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• Outdated clinical designs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 of
EFPIA 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
of c
olla
bora
tions
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