Recent update of the guidance for Parallel EMA/ FDA scientific - PowerPoint PPT Presentation

Recent update of the guidance for Parallel EMA/ FDA scientific advice Industry stakeholder platform on research and development support, 15.11.2017 Presented by Thorsten Vetter An agency of the European Union

Experience with parallel FDA interactions 16 14 • Limited uptake of parallel interactions by 12 applicants 6 10 • Reasons include lack of awareness of PSA tools, perceived process challenges, pSA 8 5 7 observer preference to discuss with Agencies 6 4 3 3 ad hoc 6 separately, and the level of convergence 1 4 4 2 These considerations triggered a review 3 2 3 5 5 4 5 of the General Principles for PSA 2 3 2 1 2 3 which have been published in April 2017 1 1 1 1 0 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 1 Recent update of the guidance for Parallel EMA/ FDA scientific advice 15 November 2017

Parallel Scientific Advice (FDA-EMA) 2 Recent update of the guidance for Parallel EMA/ FDA scientific advice 15 November 2017

PSA updated General Principles (1/ 3) • The goal of the PSA program is to provide a mechanism for EMA assessors and FDA reviewers to concurrently exchange w ith sponsors their view s on scientific issues during the development phase of new medicinal products • I ncrease dialogue between the two agencies and sponsors from the beginning of the lifecycle of a new product, provide a deeper understanding of the bases of regulatory decisions , optim ize product developm ent , and avoid unnecessary testing replication or unnecessary diverse testing m ethodologies . • Voluntary and usually occur at the request of the sponsor 3 Recent update of the guidance for Parallel EMA/ FDA scientific advice 15 November 2017

PSA updated General Principles (2/ 3) • Focus on sharing inform ation and perspectives . Achieving harm onization and increased convergence is a potential beneficial outcom e of the PSA process • Candidates : im portant m edicinal products , [ ..] for indications lacking developm ent guidelines , or if guidelines do exist, those for which EMA’s and FDA’s guidelines differ significantly. In addition biosim ilars , products with significant clinical safety, anim al toxicology, or unique m anufacturing concerns . Previous PSAs have involved medicinal products for oncology, anti-infectives, rare diseases, the paediatric population, and cardiovascular disease, as w ell as post-licensure com m itm ent clinical trials . 4 Recent update of the guidance for Parallel EMA/ FDA scientific advice 15 November 2017

PSA updated General Principles (3/ 3) • A PSA request does not guarantee the PSA procedure will be granted • After a PSA procedure, each agency w ill retain its individual regulatory decision-m aking authority regarding drug developm ent issues and m arketing applications . • The advice of each agency may still differ after the joint discussion • However, both agencies w ill strive to provide PSA responses that are convergent . 5 Recent update of the guidance for Parallel EMA/ FDA scientific advice 15 November 2017

New element: Consultative Advice • Alternatively both agencies can engage in a “ consultative advice ”. In this case, a limited number of experts from either side will be invited to participate in the discussions of the other agency. • Allows sponsors to request scientific advice from one regulatory agency and concurrently notify the other regulatory agency of the request. At the invitation of the first agency, the second w ill participate in the sponsor m eetings or teleconferences as able . Unlike the parallel scientific advice process, the second agency will be expected to only engage on top level issues . The review and sponsor meeting will follow the timelines of the regulatory agency from whom the sponsor initially seeks scientific advice. Only the initially contacted regulatory agency w ill provide w ritten scientific advice 6 Recent update of the guidance for Parallel EMA/ FDA scientific advice 15 November 2017

Examples for parallel EMA/ FDA scientific advice Type of Product Topic Outcom e engagem ent DILI Proposal for a panel of FDA observed F2F meeting and pursued Participation Qualification markers to improve independent Qualification procedure, FDA and as observers Advice SAFE-T / DILI monitoring in EMA issued Letters of Support IMI clinical trials Agreement on design Licensed Tripartite meeting and discussions between Formal of PAES medicinal agencies allowed to harmonise the PAES parallel SA/ QA (post-authorisation product design efficacy study) Very helpful discussion on programme design, Formal Investigational Genetic GI disease / use of natural history data to identify suitable product parallel SA/ QA paediatric development efficacy endpoints, historic controls, FU planned on final Ph2/ 3 design 7 Recent update of the guidance for Parallel EMA/ FDA scientific advice 15 November 2017

Take home messages • Parallel interactions are very helpful tools and uptake should be encouraged • Important to chose right tool for purpose • Particular benefit foreseen in the context of PRIME/ Breakthrough Designation • Update of General Principles provides clear framework and indicates feasibility without significant increase in procedural burden Revised general principles: http: / / www.ema.europa.eu/ ema/ pages/ includes/ document/ open_document.jsp? webContentId= WC500014868 8 Recent update of the guidance for Parallel EMA/ FDA scientific advice 15 November 2017

Any questions? Further information Scientificadvice@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

PSA Timeline Chart 10 Recent update of the guidance for Parallel EMA/ FDA scientific advice 15 November 2017