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 Recerca
23 Setembre 2014
Presented by: Jordi Llinares
Head of product development scientific support

Tots tenim un passat…
Vall Hebron 2 Institut de Recerca September 29, 2014

contents
1. How the Agency supports research?
2. Orphan drugs Regulated
3. Where are the bridges between these and you?

contents

EMA Mission statement
5 Vall Hebron Institut de Recerca September 29, 2014

Human Medicines Research &
Development Support Division
6
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
Product
development
support
D-DS
Scientific
Advice
D-DS-SCA
Paediatric
Medicines
D-DS-PME
Orphan
Medicines
D-DS-OME
Regulatory & best
evidence
D-RE
Regulatory
Affairs
D-RE-REA
Best
Evidence
Development
D-RE-EVD
Vall Hebron Institut de Recerca September 29, 2014

7
Benefit
public
health
7
Development, evaluation and supervision
R&D support
Enable innovation
Anticipate and analyse scientific/regulatory issues
Remove regulatory and procedural hurdles

8
Benefit
public
health
Foster
Early dialogue
Workshop/Conferences
Guidelines
Communication
Scientific, Regulatory,
Operational Excellence
Core business
Knowledge centre
Competency and training
development centre

“Classical” development paradigm
Academic
research
Mechanism of
disease
Drug targets
Industry
sponsored
Meet patient
needs and
economic
demands
(modified from Patel and Miller)
Vall Hebron 9 Institut de Recerca
Preclinical
development
Animal safety
testing
Phase I, II, III
trials
Pharmacokinetics
Pharmacodynamics
Special toxicities
Surveillance
Adverse events
Product quality
Phase IV trials
September 29, 2014

A shift in the “regulatory” paradigm

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 models
Innovative
approaches
Enriched clinical
trial design
Genetic and
special
population
information
incorporated into
clinical studies
Genetic directed
surveillance to
decrease adverse
events
Rapid detection
of inferior
product quality
Compliance
across the entire
supply chain
Scientific
advice / protocol
assistance
Biomarker
qualification
Modelling and
simulation
Adaptive
licensing
PASE / PASS
Registers /
observational
studies
September 29, 2014

contents

How rarity affects patients and
products?

Why an orphan regulation

Objective of the Regulation
September 29, 2014

Designation criteria

Incentives
September 29, 2014

The designation process in the EU
DAY 1
DAY 60 (COMP
meeting)
List of questions
DAY 90 (COMP
meeting)
Decision (European
Commission)
Opinion
Intent to
file
letter
Application
submission
Evaluation
JOINT FDA/EMA?
Validatio
n
Oral discussion

Committee for Orphan Medicines (COMP)
September 29, 2014

COMP responsibilities
September 29, 2014

22
Status ooff OOrrpphhaann AApppplliiccaattiioonnss

23
Distribution ooff OOppiinniioonnss
ffrroomm yyeeaarr 22000000

24
Prevalence DDeessiiggnnaatteedd CCoonnddiittiioonnss
11221199 aapppplliiccaattiioonnss uupp ttoo 22001133

Protocol assistance
September 29, 2014

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

Authorisation of an orphan drug

28
Status of Orphan Marketing Authorisation
Applications: 93 granted to date

contents

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

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 oonn:: wwwwww..iirrddiirrcc..oorrgg

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

2012-2013 FP7 Health Calls
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
€ 200 million

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 )

• The EU’s 2014-20 programme
HORIZON 2020
for research & innovation
(around € 80 billion)
• A core part of Europe 2020, Innovation
Union & European Research Area
• Three priorities: Excellent science, Industrial
leadership, Societal challenges

Topics for Rare disease research in 2014-2015 Work
Programme
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)

Thank you for your attention
European Medicines Agency
30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom
Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555
Send a question via our website www.ema.europa.eu/contact
Do not use the slides without previous authorisation
Copyright EMA 2014
Follow us on @EMA_News

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