Speedy Assessment of Vaccines: EMA’s toolbox FAMHP Speedy 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.

Eligibility to PRIME scheme Based on Accelerated Assessment criteria 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) Medicinal products of major public health interest and in particular from the viewpoint of therapeutic innovation. Potential to address to a significant extent an unmet medical need Scientific justification, based on data and evidence available from nonclinical and clinical development

For products under development yet to be placed on the EU market 16 May 2018 Justification for eligibility to PRIME For 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

Entry points PRIME eligibility and required evidence 16 May 2018 Entry points PRIME eligibility and required evidence Nonclinical Phase I Exploratory Confirmatory Confirmation Any sponsor SMEs Academia 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 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

What do we expect to grant eligibility? Unmet medical need No treatment or clear limitations of existing therapies Nonclinical data supporting pharmacological rationale (e.g. gene therapy) Clinical exploratory data on relevant endpoint If uncontrolled, use comparable historical control i.e. need sufficient information on baseline characteristics Magnitude of the effect size supporting major therapeutic advantage

PRIME was launched in March 2016 16 May 2018 PRIME was launched in March 2016 Factsheet in lay language Q&A, templates, application form for applicants prime@ema.europa.eu

PRIME eligibility recommendations adopted by 20 July 2017 16 May 2018 PRIME eligibility recommendations adopted by 20 July 2017 > 120 eligibility requests 28 granted* ~ 50% SMEs ~ 50% Advanced therapies * NOTES 1 eligible product has subsequently been withdrawn from the scheme Out of scope: eligibility requests received but not started by EMA as they were deemed outside the scope of the scheme or with a format and content inadequate to support their review. These are not included in the breakdown by type of applicant or by therapeutic area. PRIME

Early access tool, supporting patient access to innovative medicines. 16 May 2018 Features of the PRIME scheme Early 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.

Other development support activities The innovation task force Informal early dialogue (SME) Proactively identify scientific, technical and regulatory issues related to emerging therapies 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 exceptional circumstances) 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

Federal Agency for Medicines and Health Products – FAMHP Contact Federal Agency for Medicines and Health Products – FAMHP Place Victor Horta 40/40 1060 BRUXELLES tel. + 32 2 528 40 00 fax + 32 2 528 40 01 e-mail welcome@fagg-afmps.be www.afmps.be