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…

Vall Hebron Institut de Recerca2 12 April 2023

contents

1. How the Agency supports research?

2. Orphan drugs Regulated

3. Where are the bridges between these and you?


contents





EMA Mission statement

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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

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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7

Benefit public health

7

Development, evaluation and supervisionR&D support

Enable innovationAnticipate and analyse scientific/regulatory issues

Remove regulatory and procedural hurdles


8

Benefit public health

FosterEarly dialogue

Workshop/ConferencesGuidelines

Communication

Scientific, Regulatory, Operational Excellence

Core businessKnowledge centre

Competency and training development centre


“Classical” development paradigm

(modified from Patel and Miller)

Vall Hebron Institut de Recerca9

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



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





How rarity affects patients and products?


Why an orphan regulation



Objective of the Regulation

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Designation criteria



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



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


Authorisation of an orphan drug


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Status of Orphan Marketing Authorisation Applications: 93 granted to date

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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 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 )


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


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

30 Churchill Place • Canary Wharf • London E14 5EU • United KingdomTelephone +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 authorisationCopyright EMA 2014

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dr. jordi llinares: research, regulations and rare deseases. is there a meeting point?

Documents

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passatvall hebron

incentivesvall hebron

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orphan regulationvall

orphan drugvall hebron

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