ILAE / IBE collaboration European perspective EUCARE session, Porto, August 27th, 2010 Michel Baulac, Paris ILAE Vice-President - PowerPoint PPT Presentation

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ILAE / IBE collaboration European perspective EUCARE session, Porto, August 27th, 2010 Michel Baulac, Paris ILAE Vice-President

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Cooperation: Ten Themes. 1 . Health. 2 . Food, agriculture and fisheries, biotechnology. 3 . Information and communication technologies. 4 . Nanosciences ... – PowerPoint PPT presentation

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Title: ILAE / IBE collaboration European perspective EUCARE session, Porto, August 27th, 2010 Michel Baulac, Paris ILAE Vice-President


1
ILAE / IBE collaborationEuropean
perspectiveEUCARE session, Porto, August 27th,
2010Michel Baulac, ParisILAE Vice-President
2
The European Scope
  • EUCARE Joint Task Force from ILAE and IBE
  • European branches
  • Commission on European Affairs (ILAE)
  • European Regional Executive Committee (IBE)
  • Europe EU and non EU countries

3
Potential targets for political action at EU level
  • Research funding FrameWork Programmes
  • Drug development, approval and indications (EMA)
  • Paediatric directive
  • Rare diseases programs

4
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5
Cooperation Ten Themes
1 Health
2 Food, agriculture and fisheries,
biotechnology
3 Information and communication technologies
4 Nanosciences, nanotechnologies,
materials and new production technologies
5 Energy
6 Environment (including climate change)
7 Transport (including aeronautics
8 Socio-economic sciences and humanities
9 Space
10 Security
6
FP 7 2011 Call Programme committee
7
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8
Research priorities in epilepsy for the next
decadeA representative view of the European
scientific community Summary of the ILAE
Epilepsy Research Workshop, Brussels, 1718
January 2008Volume 50, Issue 3, Date March
2009, Pages 571-578Michel Baulac, Asla Pitkänen
9
FP7 Calls word citations
  • 2011
  • Alzheimer 4
  • Parkinson 2
  • Multiple sclerosis 1
  • Stroke 2
  • Epilepsy 1
  • 2010
  • Alzheimer 12
  • Parkinson 0
  • Multiple sclerosis 0
  • Stroke 1
  • Epilepsy 0

Cooperation/ Collaborative / collaboration gt 150
BUT The specific section of the 2011 FP7 call
concerns only off-patent medicines. It aims at
improving medical evidence for drugs that are
extensively used in children the absence of real
safety and efficacy data New or recent AEDs are
not in the scope
10
GUIDELINE ON CLINICAL INVESTIGATION OF
MEDICINAL PRODUCTS IN THE TREATMENT OF EPILEPTIC
DISORDERS
E M A
  • First guideline (1995 2001)
  • First revision preparation, discussion,
    comments, adoption,
  • implementation (April 1998 - May 2001)
  • Second revision initiated in 2008 discussions
    with ILAE-CEA

11
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12
ILAE contact group Drug agencies from MS
Application Standardized dossier (CTD)
EMA
EU Commission (EPAR)
Positive opinion
CHMP
Global EU marketing authorisation
Comments from MS
Rapporteurs
External Experts
National Regulatory Agencies
Scientific Expertise
CTD Common technical Document
13
Different perspectives
14
EU Paediatric Regulation (entered into force on
26th January 2007)
  • Facilitating development and availability of
    medicinesfor children from birth to less than 18
    years
  • High quality dugs, ethically researched, and
    authorised appropriately
  • Transparent information
  • Without
  • subjecting children to
  • unnecessary trials,
  • or delaying the autorisation for use
  • in adults.

15
Paediatric investigation plans (PIPs) and
Paediatric Committee (PDCO)
  • New product applications have to include results
    of studies in the paediatric population, (unless
    deferral or waiver)
  • New indication for an already authorized product
    or new pharmaceutical form, or new route of
    administration
  • A PIP must be agreed in advance by the PDCO and
    is binding on companies with an age-appropriate
    formulation, in all paediatric subsets affected
    by the condition.
  • The Paediatric Committee PDCO,
    multidisciplinary, primarily responsible for the
    assessment and agreement of PIPs and waivers.
    Interacts with CHMP and other WP

16
Childhood epileptic encephalopathies
Revised AED guideline (2010)
  • Because of severity and potential age-dependant
    responsiveness
  • West syndrome, Dravet syndrome, myoclonic-astatic
    epilepsy, Lennox Gastaut Syndrome and Continuous
    Spike-Wave in Slow Sleep syndromes
  • Development in these conditions may start at the
    same time in children (exploratory) and adults
  • Study duration discussed according to the
    specific characteristics of the syndromes /
    seizure type as well as the outcome criteria.

17
Very young children
  • In young children (i.e. 1 month less than 2
    years), once efficacy has been shown in the older
    paediatric population,
  • short term assessment of response by using video
    EEG monitoring may be sufficient,
  • but PK and safety are often difficult to obtain
    in neonates and infants

18
Patient Organisations
EU Commission
Orphan drug program
Research Framework Programmes FP 6, FP7
National Rare Diseases Programs
19
The Concept of Rare Diseases
  • Severity serious, life-threatening or
    chronically debilitating
  • Nosologically and aetiologically homogeneous
  • Prevalence less than 5 in every 10 000 people in
    the European Union

20
Which Rare Epileptic diseases?
Rare Epileptic Conditions Age at onset frequency
Epileptic encephalopathies
Ohtahara - Early myoclonic encephalopathy First weeks 100 case-reports each
Migrating partial sz infancy infancy Few case-reports
Infantile Spasms / West Syndrome infancy Incidence 0.3 per 1000 live births
Dravet syndrome (SMEI) infancy Prevalence 1 per 40 000
Lennox-Gastaut syndrome childhood Prevalence 0.9 per 10 000
Landau-Kleffner syndrome CSWS childhood 1 per 10 000
21
Which Rare Epileptic diseases?
Rare Epileptic Conditions Age at onset frequency
Progressive myoclonic epilepsies variable lt 1/20 000
Lafora, Unverricht-Lundborg, MERRF Ceroid Lipofuscinosis, others. lt 1/ 20 000
Other specific diseases
Rasmussen 2 to 15 years Few cases per year
Ring 20 chromosome 6 months to adolescence Hundreds of cases reported
22
Rare Epileptic Diseases / Epileptic
encephalopathiesClinical Needs
  • Early diagnosis
  • Emergency situations (status, aggravation,
    adverse effect)
  • Technical facilities (EEG-video, brain imaging,
    genetic testing, molecular diagnosis)
  • Major and permanent concern for cognitive
    functions
  • Psychiatric or other comorbidities
  • Optimisation of AED therapies
  • Information to patients and families
  • Genetic counselling
  • Multidisciplinary research programmes including
    drug trials
  • Optimisation of the relay between
    neuropaediatricians and adult neurologists

23
Orphan drugs EU Incentives
Market Exclusivity For 10 yrs after grant of EU
marketing autorisation ( 2yrs if paediatric)
Centralised Procedure at the EMEA
Protocol assistance Free scientific advice to
optimise development
Fee Reductions
24
Committee for Orphan Medicinal Products (COMP)
Rare Epileptic Diseases
Orphan Drugs Candidates
  • Rare epileptic syndromes, designated as rare
    diseases most of them being epileptic
    encephalopathies
  • Infantile Spasms
  • Progressive myoclonic ep.
  • SMEI (Dravet)
  • Lennox-Gastaut
  • Drugs or candidate drugs
  • specifically targeting these rare diseases with
    incentive measures
  • Vigabatrin (FDA)
  • Brivaracetam (EMA)
  • Stiripentol (EMA)
  • Rufinamide (EMA)

X
25
Lobbying in Europe for Epilepsy
  • Perception of our advocacy activities in favour
    of Epilepsy
  • Professional medical association
  • Patients and families associations
  • Points of weakness
  • Points of strength

26
Organisation of laypersons and professionals
interested in the medical and non-medical
aspects of epilepsy. The IBE addresses such
social problems as education, employment,
insurance, driving licence restrictions and
public awareness.
27
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28
Responsibility / Credibility of our organizations
  • Among the missions of our organisations
  • educational programs,
  • standard guidelines for diagnosis and treatment
    of epilepsy
  • congresses and conferences
  • scientific and information materials
  • public advocacy campaigns
  • We represent expertise and authority to those
    inside and outside the field, physicians, patient
    associations, governmental, supranational
    administrations, and the public in general

29
ILAE / IBE Policy for limiting potential
Conflicts of Interests
  • We must ensure that our recommendations and
    advocacy activities are not influenced by any
    financial interests, whether they concern
  • individual members
  • the organization as a whole.
  • Our budgets are largely depending on the
    Pharmaceutical Industry
  • Crucial to reassure the public and interested
    parties that all steps have been taken to prevent
    inappropriate personal or organizational
    influence on ILAE/IBE recommendations or
    guidelines
  • Real and perceived COI need to be addressed
    transparently
  • Must ensure the integrity of ILAE / IBE
    decision-making processes, our recommendations
    and our reputation, as well as that of our
    individual members /associations / Chapters.

30
The Cinderella Syndrome
  • Is epilepsy really less well treated than other
    medical fields in terms
  • of care availability, surgery, research funding,
    social assistance, etc
  • Authorities may have difficulties in
    considering separately every
  • neurological domain, every single disease or
    ensemble of diseases

31
The newer AEDs positive or negative perception
? Need to better define pharmacoresistance (KWAN,
2009, ILAE task force)
Lacosamide
20
Oxcarbazepine
Fosphenytoine
15
10
32
Lobbying is competitive
  • Epilepsy versus other brain disorders
    (Alzheimers, Parkinsons..)
  • Brain versus other therapeutic areas (cancer,
    cardiovascular, diabetes)
  • Health versus other thematic priorities (food,
    agriculture, space, environment.)
  • Cooperation versus other specific programmes
    (ideas, people, capacities)
  • Framework versus other EU member state
    priorities

33
Lobbying in Brussels the EU Jungle
  • ..an army of 15,000 lobbyists represents some
    1,400 companies and interest groups, ranging from
    the European Bottled Watercooler Association to
    those concerned about endangered koala bears.
    They must navigate a matrix of increasingly
    powerful institutions, including the European
    Commission and the European Parliament, which has
    732 members and veto power over regulations,
    including health warnings on cigarette packs and
    airline passenger compensation.

34
Our strong points for advocacy
  • Diversity of epileptic conditions and of ages
  • Childhood, brain development
  • Needs for continuity of care between childhood
    and adulthood
  • Mortality
  • Cognitive and psychiatric comorbidities
  • Unique opportunities offered by epilepsy research
    (electrodes in the brain, living post-operative
    tissues, genes.)
  • News drugs, new mechanisms in the pipeline ?
  • Social limitations and stigma
  • Commonality of symptoms (seizures) with stroke,
    neurodegenerative diseases, Autism
  • Amonst others.

35
IBE / ILAE influencing EU authorities
European Commission
Cardiovascular lobby
Brain lobby
Cancer lobby
Member State interests
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