Dr. Jordi Llinares: Research, regulations and rare deseases. Is there a meeting point?
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Transcript of Dr. Jordi Llinares: Research, regulations and rare deseases. Is there a meeting point?
An agency of the European Union
Research , regulations and rare diseases, is there a meeting point?Vall d’Hebron Institut de Recerca23 Setembre 2014
Presented by: Jordi LlinaresHead of product development scientific support
Tots tenim un passat…
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contents
1. How the Agency supports research?
2. Orphan drugs Regulated
3. Where are the bridges between these and you?
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contents
1. How the Agency supports research?
2. Orphan drugs Regulated
3. Where are the bridges between these and you?
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EMA Mission statement
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Human Medicines Research &
Development Support Division
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The Division is in charge of:
providing scientific advice
fostering development of new drugs in orphan diseases and paediatric
populations
provision of regulatory intelligence
coordinating the development of best evidence to support regulatory decision
making
Human Medicines Research & Development Support
D
Scientific Advice
D-DS-SCA
Product development
supportD-DS
Paediatric Medicines D-DS-PME
Orphan Medicines D-DS-OME
Regulatory Affairs
D-RE-REA
Regulatory & best evidence
D-RE
Best Evidence
Development
D-RE-EVD
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Benefit public health
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Development, evaluation and supervisionR&D support
Enable innovationAnticipate and analyse scientific/regulatory issues
Remove regulatory and procedural hurdles
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Benefit public health
FosterEarly dialogue
Workshop/ConferencesGuidelines
Communication
Scientific, Regulatory, Operational Excellence
Core businessKnowledge centre
Competency and training development centre
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“Classical” development paradigm
(modified from Patel and Miller)
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Academic research
Mechanism of disease
Drug targets
Industry sponsored
Meet patient needs and economic demands
Preclinical developmentAnimal safety
testing
Phase I, II, III trialsPharmacokinetics
PharmacodynamicsSpecial toxicities
SurveillanceAdverse eventsProduct qualityPhase IV trials
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A shift in the “regulatory” paradigm
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A changing paradigm and (Some) Agency initiatives advancing regulatory science
Understanding genetics and
determinants of disease
Novel drug targets based on
mechanism of disease
Consortium approach for unmet needs
Integration of information from basic research and product development
More predictive in vitro and in vivo modelsInnovative approaches
Enriched clinical trial designGenetic and
special population information
incorporated into clinical studies
Genetic directed surveillance to
decrease adverse eventsRapid detection
of inferior product quality
Compliance across the entire
supply chain
Scientific advice / protocol
assistance
Biomarker qualification
Modelling and simulation
Adaptive licensing
PASE / PASSRegisters /
observational studies
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contents
1. How the Agency supports research?
2. Orphan drugs Regulated
3. Where are the bridges between these and you?
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How rarity affects patients and products?
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Why an orphan regulation
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Objective of the Regulation
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Designation criteria
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Incentives
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The designation process in the EU
List of questions
DAY 60 (COMP meeting)
DAY 1
DAY 90 (COMP meeting)
Decision (European
Commission)
Opinion
Intent to file letter
Application submission
Evaluation
JOINT FDA/EMA?
Validatio
n
Oral discussion
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Committee for Orphan Medicines (COMP)
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COMP responsibilities
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Status of Orphan Applications Status of Orphan Applications
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Distribution of Opinions Distribution of Opinions from year 2000from year 2000
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Prevalence Designated ConditionsPrevalence Designated Conditions1219 applications up to 20131219 applications up to 2013
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Protocol assistance
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Fee reductions
•Annually EU allocated special fund to cover fee reductions (approx. 6 million Euro)
•EMA has consistently kept maximum coverage for SMEs
•Academia and SME in 79% development of advanced therapies
•Policy reviewed annually, needed revision in 2013 according to current budget
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Authorisation of an orphan drug
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Status of Orphan Marketing Authorisation Applications: 93 granted to date
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contents
1. How the Agency supports research?
2. Orphan drugs Regulated
3. Where are the bridges between these and you?
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A non comprehensive list of opportunities
• Research in rare diseases and treatment Horizon 2020
• Applications
• Review
• Expert collaboration with IRDIRC
• Expert collaboration EMA procedures
• Sponsor applications orphan designation
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IRDiRC Policies & Guidelines
Common framework with separate sections for researchers and funders
Policy: principle which consortium members agree to follow. IRDiRC will periodically review its policies
Guideline: recommendations made by IRDiRC scientific committees/working groups that offer advice as to “best practices” at a given time
Document available on: www.irdirc.orgDocument available on: www.irdirc.org
The EU: A major player in funding health research in rare diseases
Over two decades of investment in the area
Over € 620 million invested in close to 120 projects launched in
FP7
Continued strong investment in Horizon 2020
47 projects
€ 64 million
59 projects
€ 230 million
~120 projects
>€ 620 million
EU collaborative research in rare diseases: main focus
areas in FP7
Europe wide studies of natural history and pathophysiology: in vitro/in vivo models, registries & bio-banks, identification of biomarkers
Development of preventive, diagnostic and therapeutic interventions including pharmacological and innovative approaches
8 research topics for rare diseases:
•-Omics for better understanding of diseases in view of development of new diagnostics and treatments *
•Databases, biobanks and clinical ‘bio-informatics’ hub
•Preclinical and clinical development of orphan drugs *
•Observational trials in rare diseases
•Best practice and knowledge sharing in clinical management
•Organisational support for IRDiRC
•Development of imaging technologies for therapeutic interventions in rare diseases *
•New methodologies for clinical trials for small population group
*industry/SME topic
2012-2013 FP7 Health Calls€ 200 million
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NATURE REVIEWS | DRUG DISCOVERY VOLUME 12 | APRIL 2013 | 253
Of the 12 projects in the article nine for
orphan designated products
Cordis website: three additional
projects granted that involve
development of orphan drugs:
•Drugsford (inherited photoreceptor
degeneration)
•Eurofancolen (fanconi anaemia)
•Aipgene (intermittent acute porfiria )
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HORIZON 2020
• The EU’s 2014-20 programmefor research & innovation (around € 80 billion)
• A core part of Europe 2020, Innovation Union & European Research Area
• Three priorities: Excellent science, Industrial leadership, Societal challenges
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ERA-NET: Rare disease research implementing IRDiRC objectives (WP 2014)
New therapies for rare diseases (WP 2015)
Clinical validation of Biomarkers (SME instrument: open call)
Topics for Rare disease research in 2014-2015 Work Programme
Thank you for your attentionEuropean Medicines Agency
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