Next Generation Innovations in Regulatory Science

1 next Generation Innovations in Regulatory Science 28 October 2015 | Washington, DC. Better Science , Better Health Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be attributed to DIA, its directors, officers, employees, volunteers, members, chapters, councils, Communities or affiliates, or any organization with which the presenter is employed or affiliated. These PowerPoint slides are the intellectual property of the individual presenter and are protected under the copyright laws of the United States of America and other countries. Used by permission. All rights reserved. DIA and the DIA logo are registered trademarks or trademarks of Drug Information Association Inc.

2 All other trademarks are the property of their respective owners. 2015 DIA, Inc. All rights reserved. Key Question Do you believe that the Science of medicines development and the Science of regulation are progressing at an acceptable pace to ensure safe, effective, and accessible products are getting to patients? next Generation Innovations in Regulatory Science Panelists Hans-Georg Eichler, Senior Medical Officer, EMA. Carol Linden, Director of Office of Regulatory Science and innovation , US FDA. Sandra Milligan, Senior Vice President and Head of Global Regulatory Affairs and Clinical Safety, Merck Alastair Kent, Director, Genetic Alliance UK. Gigi Hirsch, Executive Director, MIT Center for Biomedical innovation ; Program Director, NEWDIGS.

3 Collaboration accelerates progress. Benefit- Risk Scientific Evidence Stakeholder input More than 7,000 medicines are in development globally. PhRMA Chart Pack 2015. Available at: Health authorities have made progress in accelerating approvals. CIRSCI Analysis. Available at: Additional information: next Generation Innovations in Regulatory Science Do you believe that the Science of medicines development and the Science of regulation are progressing at an acceptable pace to ensure safe, effective, and accessible products are getting to patients? Hans-Georg Eichler, Senior Medical Officer, EMA. Carol Linden, Director of Office of Regulatory Science and innovation , US FDA. Sandra Milligan, Senior Vice President and Head of Global Regulatory Affairs and Clinical Safety, Merck Alastair Kent, Director, Genetic Alliance UK.

4 Gigi Hirsch, Executive Director, MIT Center for Biomedical innovation ; Program Director, NEWDIGS. The Future of Regulatory Science at FDA Carol D. Linden, Director, O ce of Regulatory Science and Innova=on O ce of the Chief Scien=st for DIA: BeBer Science , BeBer Health next Genera=on Innova=ons in Regulatory Science October 28, 2015 9. Regulatory Science (FDA de ni=on) The Science of developing new tools, standards, and approaches to assess the safety, e cacy, quality, and performance of FDA- regulated products. 10. FDA Science and Mission at Risk Report of the Subcommittee on Science and Technology Prepared For FDA Science Board November 2007 11. Science and Mission at Risk 2007 Report Raised serious concerns regarding the health of the Science enterprise at FDA Causes iden= ed: Chronic underfunding Loss of scien0 c exper0se Increasing legisla0ve mandates/responsibili0es without corresponding resources to support them 12.

5 Addi=onal Legisla=ve Responsibili=es Since 2007 Report FDA Amendments Act (FDAAA 2007) Tobacco Control Act (2009) A ordable Care Act (2010) Title VII - generics Food Safety Modernization Act (FSMA 2011) FDA Safety and innovation Act (FDASIA 2012) Drug Quality and Security Act (DQSA 2013) 21st Century Cures and Senate innovation Act (2015) 13. Science and Mission at Risk 2007 Report Major recommenda=ons: Realign Science organiza0on to be?er manage/ coordinate emerging Science Strengthen the Science base Enhance the scien0 c workforce 14. Science Organiza=on at FDA Cross cutting coordination and Science promotion improved O ce of Chief Scientist established O ce of Regulatory Science and innovation O ce of Scienti c and Professional Development O ce of Countermeasures and Emerging Threats O ce of Health Informatics Senior Science Council 15.

6 Modernize toxicology to enhance product safety Stimulate innovation in clinical evaluations and personalized medicine Support new approaches to improve product manufacturing and quality Ensure FDA readiness to evaluate innovative technologies Harness diverse data through information sciences Implement a prevention- focused food safety system Develop medical countermeasures Strengthen social and behavioral sciences Strengthen global product safety 16. Regulatory Science at FDA Important to note all agency components also have program- speci c Regulatory Science ini=a=ves Includes intramural and extramural support Centers of Excellence Cri=cal Path Ini=a=ve 17. Science Board Updated Review Late 2013, Science Board requested to review progress since previous report Speci c ques=ons: Whether changes that FDA has made to its Regulatory Science programs have been a success Whether FDA should consider any other programma=c changes What opportuni=es, strategies, and frameworks for collabora=on, will best advance FDA's mission Whether FDA has taken su cient steps to strengthen the scien= c workforce & whether addi=onal steps may be necessary 18.

7 Outlines current status of Regulatory Science at FDA Details of current programs Examples of activities in each Regulatory Science priority thematic area 19. Progress Since 2007 The responsiveness of FDA to the Mission at Risk report and those responsible for overseeing its work has been extensive, transformative, and laudable. Many substantive changes have been made in FDA's organization, authorities, and programs that significantly address issues identified in 2007.. 20. Report Recommenda=ons Medical Product Innova=on Facilitate biomarker & surrogate endpoint quali ca=on Clinical trial e ciency Master protocols Clinical trial networks Data mining & analy=c tools Full deployment of Sen=nel; expand to devices 21. Report Recommenda=ons Product quality Genomics medical product quality and safety Foodborne and other outbreak inves=ga=on Analy=c methods for nanotechnology Novel technologies for lab & eld analy=cs 22.

8 Report Recommenda=ons Modernizing toxicology Pluripotent stem cells for toxicity evalua=on Bioimaging for neurotoxicity Mathema=cal modeling of systems In silico modeling for suscep=bility to toxicity Organ- on- a- chip technology 23. Report Recommenda=ons Expand extramural collabora=ons Speci c men=on of Reagan- Udall Founda=on Recruitment of 21st Century Workforce Resources to match responsibili0es 24. Overarching Regulatory Science Priority for Medical Products Study and support development of innova0ve ways to streamline/supplement/. speed medical product availability without nega0ve impact on the FDA gold standard of product e cacy and safety. 25. Addi=onal Priority Areas Methods development for use of real world data (esp.)

9 Pre- market) Adap=ve/innova=ve clinical trial design Pa=ent- focused drug development Social and behavioral Science 26. 5th GSRS in Parma Italy Focus area was bioinforma=cs 26 countries & 150 par=cipants Increasing opportuni=es for Regulatory Science interna=onally 27. next Generation Innovations in Regulatory Science Do you believe that the Science of medicines development and the Science of regulation are progressing at an acceptable pace to ensure safe, effective, and accessible products are getting to patients? Hans-Georg Eichler, Senior Medical Officer, EMA. Carol Linden, Director of Office of Regulatory Science and innovation , US FDA. Sandra Milligan, Senior Vice President and Head of Global Regulatory Affairs and Clinical Safety, Merck Alastair Kent, Director, Genetic Alliance UK.

10 Gigi Hirsch, Executive Director, MIT Center for Biomedical innovation ; Program Director, NEWDIGS. next Generation Innovations in Regulatory Science Gigi Hirsch, Executive Director MIT Center for Biomedical innovation & NEWDIGS. NEWDIGS: NNEWDIGS: New DParadIGmS. ew Drug Development rug Development ParadIGmS. Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be attributed to DIA, its directors, officers, employees, volunteers, members, chapters, councils, Communities or affiliates, or any organization with which the presenter is employed or affiliated. These PowerPoint slides are the intellectual property of the individual presenter and are protected under the copyright laws of the United States of America and other countries.