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Federal agency for medicines and health products
FAMHPSpeedy Assessment of Vaccines: EMA’s toolbox
Bart Van der Schueren Brussels , 9th September 2017
Disclaimer
The views expressed in this presentation are the personal views of the speaker and may not be understood or quoted as being made on behalf of or reflecting the position of EMA or one of its committees or working parties.
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Eligibility to PRIME scheme Based on Accelerated Assessment criteria
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Medicinal products of major publichealth interest and in particular fromthe viewpoint of therapeuticinnovation.
Potential to address to a significant extentan unmet medical need
Scientific justification, based on data andevidence available from nonclinical andclinical development
No satisfactory method or if method exists, bring a major therapeutic advantage
Introducing new methods or improving existing ones
Meaningful improvement of efficacy (impact on onset, duration, improving morbidity, mortality)
Justification for eligibility to PRIMEFor products under development yet to be placed on the EU market
• Unmet medical need Epidemiological data about the disease Description of available diagnostic, prevention and treatment
options/standard of care (SOC), their effect and how medical need is not fulfilled
• Potential to significantly address the unmet medical need Description of observed and predicted effects, clinical relevance,
added value and impact If applicable, expected improvement over existing treatments
• Data required at different stages of development
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Entry points PRIME eligibility and required evidence
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Proof of concept Sound pharmacological
rationale Clinical response efficacy and
safety data in patients (exploratory trials)
Substantial improvement Magnitude, duration, relevance
of outcomes to be judged on a case by case basis
Anysponsor
Proof of principle(For SMEs and academia only) Sound pharmacological
rationale, convincing scientific concept
Relevant nonclinical effects of sufficiently large magnitude and duration
Tolerability in first in man trials
SMEsAcademia
Confirmation
Nonclinical Phase I Exploratory Confirmatory
What do we expect to grant eligibility?
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Clinical exploratory data on relevant endpoint
Unmet medical needNo treatment or clear limitations of existing therapies
Nonclinical data supporting pharmacological rationale (e.g. gene therapy)
If uncontrolled, use comparable historical controli.e. need sufficient information on baseline characteristics
Magnitude of the effect size supporting major therapeutic advantage
PRIME was launched in March 2016
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Factsheet in lay
language
Q&A, templates, application
form for applicants
PRIME eligibility recommendations adopted by 20 July 2017
PRIME
> 120 eligibility requests28 granted*~ 50% SMEs
~ 50% Advanced therapies
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Features of the PRIME schemeEarly access tool, supporting patient access to innovative medicines.
Written confirmation of PRIME eligibility and potential for accelerated assessment;
Early CHMP Rapporteur appointment during development;
Kick off meeting with multidisciplinary expertise from EU network;
Enhanced scientific advice at key development milestones/decision points;
EMA dedicated contact point;
Fee incentives for SMEs and academics on Scientific Advice requests.
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Other development support activities
• The innovation task force– Informal early dialogue (SME)– Proactively identify scientific, technical and
regulatory issues related to emergingtherapies
• SME office• Scientific advice• Adaptive pathways
2: Focus on early access to innovation : Clinical trials
Vaccines for pandemics• Procedures to speed up the availability of vaccines to
protect against pandemic influenza– Mock up: strain of flu not circulating (quality, safety and
immunogenicity, Authorization under exceptionalcircumstances)
– Once pandemic declared: replace the flu strain in mock-up to strain causing the pademic rolling review variation
Vaccines for pandemics
• Procedures to speed up the availability of vaccines to protect against pandemic influenza– Emergency authorisation procedure: complete package
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Contact
Federal Agency for Medicines and Health Products –FAMHP
Place Victor Horta 40/40 1060 BRUXELLES
tel. + 32 2 528 40 00fax + 32 2 528 40 01
e-mail [email protected]
www.afmps.be
Your medicines and health products,our concern