Constantinos Ziogas SME Office Stakeholder amp Communication Division European Medicines Agency Open Info Day Horizon 2020 Health demographic change and wellbeing Brussels 22 November 2013 ID: 781700
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The SME Office at the European Medicines Agency
Constantinos ZiogasSME Office, Stakeholder & Communication Division, European Medicines Agency
Open Info Day
Horizon 2020 'Health, demographic change and wellbeing‘
Brussels 22 November 2013
Slide2How can regulators proactively support drug development?
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Slide3Development challenges - seeing through the eye of the customer
Issues:
Solutions:
Perceived barriers to engaging with regulators – small companies, start-ups, academic groups (main developers of ‘advanced therapies’)
Easy access through ‘SME Office’
Early support from ‘Innovation Task Force’
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Slide4A proactive regulatory approach:
“
Be part of it and shape it together.
”
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Slide5Incentives/Assistance to drug development
ITF
Orphan
Scientific
Advice
Advanced
Therapies
Paediatric
SME
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Slide6Orphan Designation
Time
Knowledge
Medical Plausibility
Pos
Benefit-Risk
Compliance with
Ped
Invest Plan
Marketing Authorisation (To)(Clinical) Development
Scientific Advice
Benefit-Risk
Mgt
Plan
Paediatric Invest. Plan
Industry - Regulator interactions
ITF
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Slide7Support to small & medium sized enterprises (SMEs)
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Slide8Tailoring assistance for SMEs
To promote innovation & development of new medicinal products by SMEs
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Slide9SME Office
• A single interface/’one-stop shop’• SME assignment, public SME register• Fee incentives, regulatory assistance, translations• Facilitate communication• News bulletins, SME User Guide • Workshops
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Slide10Assignment of SME Status
For applicants established in EEASME criteria defined in Recommendation 2003/361/EC:Headcount < 250 and annual turnover not more than € 50 mil or balance sheet not more than € 43 milSubmit information to show compliance with criteria
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Slide11Incentives for SMEs
* Medicines based on genes / cells / tissues
•
Regulatory, administrative and procedural assistance
•
Fee reductions and deferrals
•
Certification of
Quality/Non-clinical data for advanced therapy medicinal products*
•
Translation of product information
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Slide12Fee Incentives for SMEs
90% reduction on :scientific adviceGMP, GLP, GCP, PhVig inspectionsscientific services100% ‘waiver’ on administrative servicesFor MAA, SME fee deferralorphan medicine: 100% waiver to 1st year post-licensingpaediatric use marketing autorisation: 50% fee reduction Conditional Fee ExemptionSubject to EMA scientific advice – payment only for positive outcome
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Slide13Experience with SMEs 2006-2012
1098 companies assigned SME status From 27 countries across EEA41% micro, 38% small, 19% mediumMajority human, 44 vet, 66 human/vet & 154 service providersPublic register of companies launched in 2010
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Slide14SME product pipeline – categories
Therapeutics (84%), vaccines (7%), diagnostics/imaging (9%)
Orphan medicines (20%)
Nanotechnology (4%), pharmacogenomics/biomarkers (3%)
Product development stages:
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Slide15Regulatory Assistance for SMEs
• Direct assistance:Queries dealt with by SME office e-mail/teleconBriefing meetings/telecon on regulatory strategy• Published SME User Guide on regulatory procedures• SME News bulletin • Annual training/workshops tailored for SMEs
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Slide16Scientific advice
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Slide17http://www.emea.europa.eu/index/indexh1.htm
under Application Procedures
Scientific advice and ‘protocol assistance’
•
EU advice on development & agreement of future strategy
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Working party of CHMP
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Note: Protocol assistance is scientific advice for orphan designated products, and does not only relate to clinical aspects.
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Slide18Scope of scientific advice
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Scientific Advice
can be provided on ANY scientific question
- Quality, non-clinical and clinical
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At any time point of the development
Early
advice with subsequent follow-up is recommended• Not only product specific Broad advice, Conditional approval/Exceptional circumstances - On the eligibility or on proposed developmentQualification of biomarkers and other novel methodologies
Note: Clinical trial authorisation is handled at the national level17
Slide19FAQs in Scientific Advice
Quality/CMC• comparability, stability, etc.Non-clinical• in vivo pharmacology for innovative products • animal models for products with human specific targets, animal models mimicking the human disease, surrogate molecules • carcinogenicity and reprotoxicity waivers, etc.Clinical• PK/PD, dose-finding, interactions• exploratory & pivotal trials: study endpoints, population, comparator, blinding, statistics (interim A, adaptive/seamless design), safety DB
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Slide20Key features of scientific advice
• Optional (upon company request)• Strictly confidential •
Robust
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Short procedures 40 to 70 days
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‘Pre-submission’ assistance
• Face-to-face meetings with scientific experts for 50% of advice
• Fee-related activity (significant fee waiver/reduction for orphan products/paediatrics/SMEs)• Written responses adopted by the licensing Committee, sent to the company ie. the ‘scientific advice letter’19
Slide21Figures for scientific advice
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Slide22Compliance with scientific advice is associated with positive MA outcome
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Slide23Scientific advice and MA application outcome (2004-7; N=188)
Who requests SA?Big pharma: 33%
Who complies with SA?
Company size is significantly associated with positive outcome of MA application: OR = 2.96 (95%CI: 1.92, 4.56)
Obtaining and
complying
with SA appears to be a predictor of outcome [compliant with SA vs. no-SA: OR 14.71, 95% CI 1.95; 111.2; non-compliant with SA vs. no-SA: OR 0.17, 95% CI 0.06; 0.47, p<0.0001)].
Regnstrom et al; Eur J Clin Pharmacol. 2010 Jan;66(1):39-48
Big pharma: 84%
Medium pharma: 60%
Small pharma: 25%
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Slide24Innovation Task Force (ITF)
Multidisciplinary platform for preparatory dialogue and orientation oninnovative medicines, technologies and methods
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Slide25Purpose of Innovation Task Force (ITF):
Provide a forum (soft landing zone) for innovation Complementary and preparatory to existing formal proceduresTools: Briefing meetings Scientific recommendations on classification Workshops (e.g. nano, stem-cell)
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Slide26A platform for discussing a wide range of topics
Preclinical and in vitro models
Biomarker Qualification
Adaptive designs
“-omics”
Methodologies and Statistics
Epigenetics
Synthetic Biology
Nanopharmaceuticals
Borderline products
Advanced therapies
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Slide27ITF briefing meetings
• Objective: facilitating the informal exchange of information and guidance in the development process, complementing and reinforcing existing formal procedures• Scope: regulatory, technical and scientific issues arising from innovative medicines development, new technologies and borderline products• Applicants: Consortia, Networks, Public/private partnerships, Learned societies, Pharmaceutical industry, Academia• Free of charge• Scientific discussions led by experts from the Agency network, working parties and committees• For pharmacogenomics, with PG Working Party
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Slide28‘Mutually benefitting’ dialogue
• Starting the regulatory dialogue with the Agency early• Facilitating knowledge exchange on innovative strategies: update on progress, address new science and questions to regulators, understand concerns and prepare for solutions• Providing orientation on regulatory science topics in drug development• Identify issue of particular interest to regulators in preparing for formal procedures (e.g. biomarkers qualifications, scientific advice, orphan medicines designation)
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Slide29ITF
briefing meetings
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Slide30SMEs in ITF briefing meetings
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Slide31Thank you for your attention
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