Transcript Slide 1

Programme for ERS Live on understanding the 2008 IMI calls
Introduction
The COPD call
The Severe Asthma Call
Summary and questions from participants
Leo Fabbri, ERS
Ingela Wiklund, GSK
Tim Higenbottam, Chiesi
Chris Compton and Leo Fabbri,
EFPIA and ERS
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The Innovative Medicines Initiative (IMI)
The 2008 Respiratory Calls:
COPD PRO
Ingela Wiklund
2008 COPD Patient Reported Outcomes (PRO)
call: Background
Explanation of the need:
• Efficacy evaluation of new therapies has
relied on demonstration of reduction of
airflow obstruction.
– European Regulatory guidelines
recommend the use of symptom
endpoints, in addition to lung
function measurements
•
A PRO is any report coming directly from
patients about how they function or feel
in relation to a health condition and its
therapy :
–
–
Without interpretation by physicians or
others
PRO Measurement tools should be
generated with adequate patient
input.
The areas that should be developed:
• Symptoms: A patient with COPD
experiences a variety of symptoms
including dyspnoea (shortness of
breath).
– Restricts patients’ ability to
exercise and perform daily
activities
– Results in psychological
consequences
– Significant impairments to overall
health status.
•
Exacerbations: Acute worsening of
symptoms that often require a
change in treatment or could result in
hospitalization.
There is a need for scientifically developed and validated PRO measures that capture
symptoms, exacerbations and their impact on patients
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COPD call: What is it about?
• Objectives
– Understanding of the patients’ experience of COPD to inform the strategy
to measure outcomes that are relevant to patients for assessing treatment
benefit
– Selection and/or Development of measures with good measurement
properties that capture the COPD patients’ experience of the disease and
effects of treatment. Eg PRO focussed on exacerbation:
• To inform the definition of exacerbation
• Understand day-to-day experience of symptoms characterizing an
exacerbation
• Evaluate the impact of these symptoms
• Scope
– Build consensus on the measurement strategy, especially PRO tools, by
working with the key consumers of the information on treatment benefit:
• Clinicians, academic clinical & health outcomes researchers, patients
and payers
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COPD call: Deliverables
A measurement strategy that includes specific PRO tools:
– Developed using robust psychometric methods
• With input from and review by the major customer
groups
– Validated for use in treatment evaluations across
Europe that can link to global efforts
• Using appropriate prospective studies
– Developed or adapted for capturing data using
electronic data capture (EDC) devices for daily
recording at home
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COPD call:
What are we expecting and going to do?
Project plan outline
• Planned in two phases :
– Phase A: Develop a framework to
understand the Patients experience
of COPD, especially in a broad
European context, to inform
strategies to measure outcomes
meaningful to patients in global
clinical trials
– Phase B: Develop/select and
validate PRO measurement tools,
to use in clinical trials evaluating
treatments for the disease
A Consortium or ‘Centre of Excellence’
• To develop a measurement strategy to
evaluate the benefits of COPD treatment
from a patients’ point of view
•
With contributions from the key
customers and ‘Subject Matter Experts’.
For example…
• University
• Clinical centres
• SME researchers
• Patient groups
• EMEA
• Payers
• Health Technology Assessment
agencies
• Agencies that make decisions
about reimbursement
It is important to link to and build on existing International ‘Initiatives for the
measurement of PROs in COPD’ to avoid redundancies
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The Innovative Medicines Initiative (IMI)
The 2008 Respiratory Calls:
Understanding Severe Asthma
Tim Higenbottam
Challenges to Drug Development in Severe
Asthma
Big gaps in care:
•
There is high unmet need for more
effective, convenient and safe
therapies, particularly for patients
with moderately severe and severe
disease.
•
10% of asthmatics with the most
severe disease account for up to 50%
of the total costs of asthma care.
1. Heterogeneous disease (the
“asthmas”)
2. Lack of standard definition of
severe
– Lung function
– Medication requirements
– Exacerbation frequency
– Symptom burden
3. What are the phenotypes of a
severe asthmatic?
4. Is there a genotype(s) of severe
asthma?
5. Effect and detection of noncompliance
6. Lack of regulatory path in severe
asthma
7. Lack of patient availability for
clinical trials
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Challenges to Drug Development in Severe
Asthma
Big gaps in care:
•
There is high unmet need for more
effective, convenient and safe
therapies, particularly for patients
with moderately severe and severe
disease.
•
10% of asthmatics with the most
severe disease account for up to 50%
of the total costs of asthma care.
1. What should we measure in
severe asthma and with what
instruments? Are they
validated?
2. What are appropriate study
designs and inclusion criteria in
severe asthma?
3. Are there biomarkers to identify
patient phenotypes and/or
follow therapy
4. Identification of key targets and
mediators of severe asthma
5. Understanding molecular
pathways of steroid resistance
6. Role of imaging and/or other
modalities for early decision
making in development
programs
7. Understanding mucosal
immune function in severe
asthma
Longitudinal study of a severe asthma Patient Cohort
Define disease
Innovative
Clinical
measurements
Recruit cohort
New model
development
Longitud
inal
study
Optimal clinical
measures of efficacy
Newly defined
cohort
New clinical study
design
Optimal dosing
ideal PK/PD and
high therapeutic
index
The deliverables for severe asthma over 5 years
First part of call deliverables:
1. EU and Globally agreed Diagnostic
criteria for severe asthma that are
aligned to EMEA requirements
2. A longitudinal cohort of severe
asthma patients with relevant
phenotype and genotype ~ recruited
with a standardised protocol, data
collection, and database with flexibility
for novel measures to be added in
during the project
3. Identification as well as validation of
novel targets for pharmacological
therapy and biomarkers to assess
response and predict effects on
clinical outcomes
Second part of the call:
Translating the cohort into a new therapeutic paradigm
1. Identification of targets relevant to specific
phenotypes or genotypes of severe asthma.
2. Understanding of aetiology and pathogenesis of
asthma exacerbations as mechanism to identify new
targets and therapeutic approaches especially with
regard to delaying time to exacerbation and reducing
severity of exacerbation, which ultimately will
deliver significant pharmaco-economic benefits.
3. Accurate targeting of an intervention to a particular,
well-defined patient sub-population.
4. Capability to develop translational models for
appropriate prediction of clinical relevance through
preclinical modelling.
5. Access to defined patients which will facilitate
enrolment and study of patients for clinical trials by
academic and pharmaceutical research.
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What is needed in expression of interest in
severe asthma
• A CRO or SME partner to provide management and
operational processes to the patient cohort
• A multi-disciplinary scientific management board that includes
clinical expertise, patients interest groups and laboratory
scientists
• An accepted definition ~ including the means for achieving
one and engagement that enables alignment with the EMEA
[FDA] requirements
• Measurements to phenotype patients and to have agreed
inclusion criteria for a database of patients
• A standardised patient recruitment process across Europe
• A standardised patient management using conventional
therapy and adequate instruments/tests to determine
compliance
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Strategic Research Agenda (SRA):
COPD, Asthma, Rhinitis
• SRA identified one priority respiratory disease based on
medical need: Asthma ~ severe or refractory was seen as the
most important be the EFPIA resp group
• As with each disease key bottlenecks fall into three main areas:
– Disease understanding: Epidemiology, diagnosis, assessment of severity,
phenotyping, biochemical and genomic markers
– Translational models: Identification and validation of new preclinical and
clinical models to facilitate translational research
– Measurable outcomes: QOL measures that are sensitive to
pharmacological interventions and can predict pharmacoeconomic
benefit
• Objective is to build a European Network or “Centre of
Excellence” for asthma through the life of IMI, starting with 2008
calls
– 2008 calls will be focussed to disease understanding for Asthma
and outcomes for COPD
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Joint working between the Industry, SMEs and Academia as Consortia:
IMI is different from other Framework Initiatives
Full Consortium
“
”
EFPIA + Public Consortium
1. Joint preparation of the final
proposal
2. Establish the process for
managing the proposal
3. Establish timelines for
deliverables
4. Select the partners and manner
of working
*
Shared research
agenda
Focus on
disease and
patient need
Tightly Managed
Investment
*Consortium includes EFPIA colleagues as active contributors not passive donors of money
The IMI is an independent company founded by EU
Commission and EFPIA Funding is 2 billion Euros over 8 years
In-Kind Contribution
A non-monetary contribution to the
consortium eg personnel, equipment,
consumables etc
2 Billion EURO
In kind + Cash
1Billion Euro
In kind
1Billion Euro
Potential in-kind contributions
Severe Asthma
• Project management
• Clinical FTE for protocol and CRF
technical input and development
and clinical monitoring of the
project in the field
• Technical input and resources
related to assays, imaging and
application of other technologies
• Database and informatics support
• Statistics expertise
• Facilities and management of
biobank for tissue samples
• Coordinate input from key
stakeholders where relevant e.g.
ERS/ ATS, EMEA, patient
organisations
COPD
• Project management
• Outcome Research Scientists and
clinical scientists to contribute to
both phase A and phase B,
including relevant framework and
protocol development and
monitoring of validation studies
• Statistics and database expertise
• Hardware for home monitoring
and electronic data capture
• Application and data
management/ reporting of the
questionnaire/instrument in phase II
and III studies
• Coordinate input from key
stakeholders where relevant e.g.
ERS/ ATS, EMEA, patient
organisations
The Call & Evaluation Process
Research Agenda
Annual Implementation Plan
Call definition
Call Topics
Call
Expression of Interest
≈ 5 months
1st Peer Review
Invitation to Submit Full
Proposal
≈ 3 months
Stage 1: Scientific excellence
•“Public” consortia
(academia, SME, patient
organisations) only. No EFPIA
Full Project Proposal
2nd Peer Review
1. Project Agreement
2. Grant Agreement
Stage 2: Feasibility and scientific
excellence. EFPIA join successful
consortium from stage 1
Contract Execution
Evaluation Process for Successful EoI at Stage 1:
EFPIA plus EC appointed independent experts
Note EoI only eligible if submitted through web-based system
1. Scientific and/ or technological excellence
•
•
•
•
Quality of the approach
Likelihood to meet key project objectives
Complementarities with EFPIA consortium
Innovation, progress beyond state-of-art, impact
2. Partnership case
•
•
Quality and relevant experience of individual participants
Appropriateness of role/ input of each applicant
3. Quality of the applicant consortium as a whole
•
Unique features, balance
4. Quality and soundness of the workplan
•
Timelines, budget
Call 2008: Planned timelines
• Publication of calls
Apr 30th
• Deadline for Expressions of Interest
Jul 15th
• Evaluation Stage 1 completes
Jul 16th - Sep 30th
• Submission period for full proposals
Oct 1st - Nov 30th
• Evaluation Stage 2
Dec 1st – Dec 3st
• Negotiation, signature of Grant
Agreements, first payments
Jan 1st – Feb 28th
The IMI IPR Policy Must Align with the
Objectives of IMI
• IMI aims to remove the bottlenecks in R&D by conducting
pre-competitive collaborative research utilising public and
pharmaceutical industry resources
IMI findings must be widely and readily available for research into
the discovery and development of medicines
IMI findings (Foreground IPR) must be widely and readily available
for research into the discovery and development of medicines
Information that Participants bring into a Project (Background IPR) that
is necessary for the research use of IMI findings (Foreground IPR)
must be widely and readily available for research into the discovery
and development of medicines
Note: The IMI IPR Policy Does Not Affect
Commercialisation of IPR
“Participants may use, exploit, sublicense or otherwise
commercialise their intellectual property rights as they see
fit beyond the Research Use rights described in this IPR
Policy.”
Total control for owner of Foreground and
Background IPR for non-research use
IMI Helpdesk can be accessed through IMI Website
• Includes call documentation, submission tool and FAQ
• Includes Helpdesk form for specific questions
• http://www.imi-europe.org/Pages/IMI_Call_2008_1.aspx
The Innovative Medicines Initiative (IMI)
IMI is public-private partnership between the European
Commission and the European Pharmaceutical industry to
promote biomedical innovation in Europe and to address the
bottlenecks in the R&D process.
Thank you very much for your attention!
http://www.imi-europe.org