1. Speedy Assessment of Vaccines: EMA’s toolbox
FAMHP
Speedy Assessment of Vaccines: EMA’s toolbox
Bart Van der Schueren
Brussels , 9th September 2017

3. 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.

6. 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

7. 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

8. 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

9. 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

10. 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

11. 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

12. 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.

13. 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

14. 2: Focus on early access to innovation : Clinical trials

17. 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

18. Vaccines for pandemics
Procedures to speed up the availability of vaccines to protect against pandemic influenza
Emergency authorisation procedure: complete package

19. 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
fax