PRIME SME workshop, 3 October 2016 Presented by Jordi Llinares and - PowerPoint PPT Presentation

PRIME SME workshop, 3 October 2016 Presented by Jordi Llinares and Zahra Hanaizi Scientific and Regulatory Management Department An agency of the European Union

PRIME scheme - Goal & Scope To foster the development of m edicines w ith m ajor public health interest. Reinforce scientific and regulatory advice Building on ?  Foster and facilitate early interaction ! existing  Raise awareness of requirements earlier in development framework; Eligibility according to Optimise development for robust data generation existing  Focus efficient development ‘Accelerated  Promote generation of robust and high quality data Assessment criteria’ Enable accelerated assessment  Facilitated by knowledge gained throughout development  Feedback of relevant SA aspects to CHMP 1

Features of the PRIME scheme Early access tool, supporting patient access to innovative medicines.  W ritten confirm ation of PRI ME eligibility and potential for accelerated assessment;  Early CHMP Rapporteur appointm ent during development;  Kick off m eeting 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. 2 PRIME, SME workshop 2016

Entry points PRIME eligibility and required evidence Nonclinical Phase I Exploratory Confirm atory Confirm ation Any SMEs sponsor Academ ia Proof of principle Proof of concept  Sound pharmacological rationale (For SMEs and academia only)  Sound pharmacological rationale,  Clinical response efficacy and convincing scientific concept safety data in patients (exploratory trials)  Relevant nonclinical effects of  Substantial improvement sufficiently large magnitude and duration  Magnitude, duration, relevance  Tolerability in first in man trials of outcomes to be judged on a case by case basis 3 PRIME, SME workshop 2016

Overview of PRIME scheme  Iterative Scientific advice National  Enhanced regulatory guidance Early identification of scientific MAA review under  therapeutic Incremental knowledge gain advice accelerated innovation in unmet  Proactive dialogue assessment. medical needs.  Promote use of existing tools Post- Nonclinical Phase I Exploratory Confirm atory Evaluation authorisation SA 1 ( SAW P) SA 2 Accelerated ( SAW P) SA n Assessm ent Eligibility ( SAW P) confirm ation ( CHMP) ( CHMP) Any Early CHMP Rapporteur appointm ent SMEs sponsor Academ ia Early CHMP Rapporteur appointm ent 4

PRIME eligibility criteria and request 5 PRIME, SME workshop 2016

Eligibility to PRIME scheme Based on Accelerated Assessment criteria No satisfactory method or if Medicinal products of major method exists, bring a major public health interest and in therapeutic advantage particular from the viewpoint of therapeutic innovation. Introducing new methods or improving existing ones  Potential to address to a significant extent an unm et m edical need  Scientific justification, based on data and Meaningful improvement of efficacy (impact on onset, evidence available from nonclinical and duration, improving morbidity, clinical development mortality) 6 PRIME, SME workshop 2016

Justification for eligibility to PRIME For products under development yet to be placed on the EU market Unm et m edical need  Epidemiological data about the disease  Description of available diagnostic, prevention and treatment options/ standard of care, their effect and how medical need is not fulfilled Potential to significantly address the unm et m edical 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 developm ent Justification assessed by EMA’s scientific com m ittees 7

Justification for eligibility to PRIME  Short background on disease & product  Unmet medical need  Epidemiology of disease  Available treatment  Supportive evidence  Nonclinical pharmacology  Clinical data (eg exploratory efficacy + safety)  Conclusion on claim of major public health interest  Is there an unmet medical need in the proposed indication?  Is the data sufficient to support product’s potential to significantly address unmet medical need? 8

Focus on nonclinical data & proof of principle • Sound pharmacological rationale, convincing scientific concept • But, new pharmacological target or mechanism of action is not sufficient • Relevant nonclinical effects • in vitro and in vivo data from relevant models, with comparison to results from other products if possible • Observed effects: sufficiently large and/ or of long duration • Compelling results to outweigh many uncertainties of very early stage • Early clinical data from first in man trials • Acceptable tolerability • PK to confirm sufficient exposure so that nonclinical effect may be observed in man 9 PRIME, SME workshop 2016

Assessment of Eligibility: 40-day procedure CHMP SAW P CAT* Application request Report Day 40 Day 30 adoption EMA SAWP scientific reviewer officer * For ATMPs Confirmation of eligibility Outcom e Outcom e to centralised procedure letter letter Accepted Rejected 10

Transparency  Monthly report in CHMP highlights  Broad characteristics  Active substance/ I NN for eligible products  High-level statistics regularly updated 11 PRIME, SME workshop 2016

PRIME eligibility requests received as of 29 June 2016 12

Support to eligible products 13 PRIME, SME workshop 2016

What happens next? Kick-off m eeting Rapporteur nom ination Scientific advice 14

 Facilitate initial interaction between applicant and EU regulatory network;  Introduction of product and development Kick-off meeting status by applicant;  Discuss the overall development plan and regulatory strategy;  Multi disciplinary meeting with relevant  Regulatory guidance and awareness on experts from SAWP and CHMP and other committees; requirements  To take place, at EMA, shortly after  Provide recommendation on milestones and eligibility confirmation; identify issues for scientific advice;  Plan interactions with regulators. 15

Role of SAWP coordinator Support to be channelled through Scientific Advice  One SAWP coordinator to follow all by SAWP/ CHMP iterative Scientific Advice  Discuss detailed development plan,  Recommend next milestones for SA design of pivotal studies requests Early CHMP Rapporteur  Discuss key issues for MAA, at major milestones appointment  Expectation of iterative advice,  Continuity with life-cycle approach higher frequency of interactions  Dialogue on regulatory pathway/ MAA  Continuity will facilitate sharing of requirements knowledge from development to  Promote use of tools/ initiatives life-cycle  Fee waivers for SMEs and academia  Knowledge gained during development to facilitate accelerated assessment 16

In summary • PRIME aims at strengthening support to medicines that target an unmet medical need. • For medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients with no treatment options. • EMA will offer early, proactive and enhanced scientific and regulatory support to optimise the generation of robust data and enable accelerated assessment. • This will allow patients to benefit from therapies that may significantly improve their quality of life as early as possible 17 PRIME, SME workshop 2016

PRIME webpage and supporting documents Factsheet in lay language Q&A, tem plates, application form for applicants 18 PRIME, SME workshop 2016 prime@ema.europa.eu

If PRIME is not the right tool EU I nnovation innovatio Task Force n netw ork EMA still can SME Scientific Advice office provide support through… Paediatric ATMP early certification interaction m eetings Pre- Accelerated subm issio Assessm en n m eetings t 19 PRI ME, SME workshop 2016

Thank you for your attention Further information Jordi.Llinares@ema.europa.eu Zahra.Hanaizi@ema.europa.eu European Medicines Agency 30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom Telephone + 44 (0)20 3660 6000 Facsim ile + 44 (0)20 3660 5555 Send a question via our w ebsite www.ema.europa.eu/ contact Follow us on @EMA_ New s