DIA Meeting on Companion Diagnostics October 29-30 Washington, DC PROGRAM COMMITTEE: Miu Chau, PhD Regulatory Program Director Genentech, A Member of the Roche Group Jennifer Dudinak, PharmD Vice President Regulatory Affairs, Oncology GlaxoSmithKline Jennifer Shen, PhD, RAC Scientific Reviewer Jeffrey N. Stuart, PhD, RAC Director Regulatory Affairs Novartis Pharmaceuticals Corporation OVERVIEW: Advances in scientific knowledge and technology are driving growth and innovation for companion diagnostics, thereby enabling personalized therapies that: Target those patients most likely to benefit from treatment Exclude those patients most likely to suffer harm Can be monitored during treatment for clinically significant changes Drug and device pairings allow treatment decisions to be tailored for each patient. However, development of companion diagnostics and targeted drug therapy still face challenges in clinical development as regulatory policy tries to keep up with and accommodate this growing field. One of the primary goals of the Safety and Innovation Act (SIA) involves the advancement and modernization of regulatory science. Such enhancements have had direct impact on both biopharmaceutical and in vitro diagnostic manufacturers. LEARNING OBJECTIVES: At the conclusion of this meeting, participants should be able to: Discuss the US regulatory framework for drug/diagnostic development and registration Identify key challenges facing companion diagnostic manufacturers as well as potential options to address these challenges Discuss the current status and emerging issues in the EU Register at DIAhome.org/PMCD14 DIA GLOBAL CENTER 21 Dupont Circle, NW, Suite 300 Washington, DC 20036 WORLDWIDE OFFICES Basel, Switzerland Beijing, China Horsham, PA, USA Mumbai, India Tokyo, Japan
2 CONTINUING EDUCATION CREDITS DIA has been accredited as an Authorized Provider by the International Association for Continuing Education and Training (IACET). As an IACET Authorized Provider, DIA offers CEUs for its programs that qualify under the ANSI/IACET Standard. DIA is authorized by IACET to offer.7 CEUs for the program. Participants must attend the entire program in order to be able to receive an IACET statement of credit. No partial credit will be awarded. If you would like to receive a statement of credit, you must attend the program, sign in at the DIA registration desk, and complete the online credit request process through My Transcript. To access My Transcript, please go to www.diahome.org, select Login to My DIA and you will be prompted for your user ID and password. Select My Transcript (left side bar) and Credit Request to process your credit request. Participants will be able to download a statement of credit upon successful submission of the credit request. My Transcript will be available for credit requests on Thursday, November 13, 2014. It is DIA s policy that anyone in a position to control the content of a continuing education activity must disclose to the program audience (1) any real or apparent conflict(s) of interest related to the content of their presentation and/or the educational activity, and (2) discussions of unlabeled or unapproved uses of drugs or medical devices. Faculty disclosures will be included in the course materials. To view DIA s Grievance Policy, please visit the CE page on DIA s website at www.diahome.org/ce DIA S CERTIFICATE PROGRAM This program is part of DIA s Certificate Program and is awarded the following: Clinical Research Certificate Program: 4 Elective Units Regulatory Affairs Certificate Program: 4 Elective Units For more information go to DIAHome.org/certificateprograms TO ACCESS PRESENTATIONS: Visit DIAHome.org Login to My DIA Enter your User ID and Password View My Presentation Downloads Please Note: DIA User ID and Password are needed to access presentations. If you have forgotten your DIA User ID and Password, or this is your first time logging into the DIA website, please use our Login Reminder
3 WEDNESDAY, OCTOBER 29 5:30-7:00pm COMPLIMENTARY TUTORIAL: COMPETING REGULATORY OVERSIGHT OF INVESTIGATIONAL TESTS, INCLUDING LDTS, FOR CO-DEVELOPMENT PROGRAMS Moderator: James A. Boiani, JD, MS Senior Counsel Epstein Becker Green Panelists: Andrew Fish, JD Executive Director AdvamedDX Christine Gathers, MS, RAC Global Regulatory Affairs, Diagnostics Eli Lilly Elizabeth A. Mansfield, PhD Director, Personalized Medicine Staff, Gary Paul Director Quality Assurance Covance Central Laboratory Services Inc. The recent draft guidance, Framework for Regulatory Oversight of Laboratory Developed Tests (LDTs), proposes a risk-based framework for regulatory oversight of a subset of in vitro diagnostics (IVDs) referred to as laboratory-developed tests (LDTs). s pre- and postmarket requirements for IVDs would apply under certain circumstances to LDTs developed in laboratories, which are also regulated under the Clinical Laboratory Improvement Amendments (CLIA). The importance of investigational tests, including LDTs, to diagnosis and treatment decisions is increasing, especially with the advancement of personalized medicine and companion diagnostics. Stakeholders agree that the tests, regardless of their source, must be analytically and clinically valid as well safe for patients. What impact will the oversight have, and where are the overlaps and potential conflicts with CLIA regulation? What is the most effective means of assuring clinical validity of LDTs? Will approvals under the proposed framework be able to keep pace with technology based advances in testing? How can patient safety be best balanced with innovation? These and other questions will be discussed in depth from the perspectives of regulators, diagnostics developers, laboratories, and pharma sponsors. Rosanne Welcher, PhD, MBA, RAC Quality Assurance Regulatory and Clinical Affairs Dako North America, an Agilent Technologies Company
4 THURSDAY, OCTOBER 30 7:30-8:00am REGISTRATION AND CONTINENTAL BREAKFAST 8:00-8:10am WELCOME AND OPENING REMARKS 8:10-8:30am KEYNOTE ADDRESS Elizabeth A. Mansfield, PhD Director, Personalized Medicine Staff 8:30-10:00am SESSION 1: UPDATE Jennifer Shen, PhD, RAC Scientific Reviewer OIR Developments Eunice Lee, PhD Division of Molecular Genetics and Pathology (DMGP) Panel Discussion Joining the speakers: Reena Philip, PhD Division Director Division of Molecular Genetics and Pathology (DMGP) Gideon Blumenthal, MD Lead Medical Officer CDER Case Study Judith Finlayson, PhD Senior Manager, Regulatory Affairs Qiagen Jonathan Perkins Manager Personalized Healthcare Qiagen Andreas Köehler, PhD Head Pharmacogenomics Boehringer Ingelheim Pharma GmbH & Co. KG Development Germany 10:00-10:30am REFRESHMENT BREAK Connect with us! @DrugInfoAssn
5 10:30-12:00pm SESSION 2: MULTIMARKER DIAGNOSTICS AND NEXT-GENERATION SEQUENCING Jennifer Dudinak, PharmD Vice President Regulatory Affairs, Oncology GlaxoSmithKline Multiple marker diagnostics (multiplex) or next-generation sequencing (NGS) platforms offer the ability to test for a wide array of biomarkers and mutations at once. These platforms can be instrumental in fostering efficient health care utilization, maximizing sampling for tissue/specimen/biopsy and advancing innovations in molecularly-targeted therapeutic development and registration across a variety of therapy areas. There are several regulatory and development strategic considerations when employing a multiple marker diagnostic approach in a therapeutic development program. This session will explore challenges and opportunities of utilizing a multiple marker approach including test, bridging, analytical validation and labeling. Update Jennifer Dickey, PhD Regulatory Reviewer NCI Match Gregory F. Heath, PhD Senior Vice President, IVD Development Illumina, Inc Presentation Title TBA William Pignato W.J. Pignato & Associates, LLC Former Global Head of Regulatory Affairs Companion Diagnostic Novartis 12:00-1:00pm LUNCHEON 1:00-3:00pm SESSION 3: LEVERAGING ADVANCED COMPANION DIAGNOSTICS IN INNOVATIVE CLINICAL TRIAL DESIGNS: NEW FRONTIERS AND LESSONS LEARNED Eric Slosberg, PhD Translational Medicine U.S. Clinical Development and Medical Affairs Novartis Oncology Over the last decade, there has been rapid expansion in the knowledge of molecular mechanisms that drive disease and the associated diagnostics and therapies that detect and target these disease drivers. Increasingly, diseases are being recognized as more heterogeneous entities, potentially dividing patients into ever-smaller subsets for individualized therapy. As our knowledge of disease mechanisms continues to evolve, personalized medicine will likely become even more complex, straining the traditional drug-diagnostic development paradigm associated with large, single-sponsor clinical studies. This session reflects on recent efforts to reshape clinical trials for precision therapies and their companion diagnostics, and envisions future activities that may propel the advancement of innovative breakthrough therapies to biomarker-defined patients with high unmet medical need. Clinical: Patricia LoRusso, DO Associate Center Director - Innovative Medicine Smilow Cancer Center Yale University Roman Yelensky, PhD Biomarker and Companion Diagnostic Development Foundation Medicine, Inc. Stats: Meijuan Li Lead Mathematical Statistician CDER: Christopher Leptak, MD, PhD OND Biomarker and Companion Diagnostic Lead CDER Europe: Marisa Papaluca, MD Head of Scientific Support Office Specialised Scientific Disciplines Department European Medicines Agency 3:00-3:30pm REFRESHMENT BREAK
6 3:30-5:00pm SESSION 4: GLOBAL PERSPECTIVE: EU, CONSULTANT, JAPAN, ROW Miu Chau, PhD Regulatory Program Director Genentech, A Member of the Roche Group Currently companion diagnostics do not require pre-marker authorization by a regulatory authority in the EU. The manufacturers only need to perform conformity assessment on the in vitro diagnostics (IVDs), i.e., self-certification, for CE marking. In other countries, such as China and Japan, companion diagnostics registrations are independent of drug approval. However, with the increasing number of drugs requiring companion diagnostics to identify patients for treatment the global regulatory framework is gradually changing to keep up with the innovations. For example, the European Commission put forward a proposal for a regulation on in vitro diagnostic medical devices in 2012. This session will explore the potential impact of the changing global regulatory framework on different aspects of companion diagnostic development, e.g., analytical and clinical performance requirements, risk-based classification, regulatory approval/ certification process, etc. How the CPDx will be reviewed Revised IVD directive Chinese perspective Speakers: Julie S. Engel, PhD, RAC Director Companion Diagnostics Regulatory Affairs Ventana Medical Systems Rosanne Welcher, PhD, MBA, RAC Quality Assurance Regulatory and Clinical Affairs Dako North America, an Agilent Technologies Company Drug Safety: Is Your Staff Prepared? DIA s Drug Safety elearning Program is Your Training Solution to Reduce Risk Drug safety is a primary concern throughout the medical product development life cycle. Developed with DIA expertise to meet the unique needs of its stakeholders and members, this online safety program provides the knowledge your staff needs, from regulations and requirements through premarket review and postmarket monitoring. Make Sure Your Staff is Ready. Enroll today at diahome.org/safetyelearning or contact Katie.Hill@diahome.org for information on group discounts and licensing. Save Now! Buy all 6 modules and get 20 % off individual prices. Group discounts and licensing available for 10 or more users. Drug Safety Training in Six Modules Use these self-paced modules to meet your individual or organization s training needs. Introduction to Drug Safety Drug Safety Regulatory Requirements Premarketing Clinical Trial Safety Postmarketing Safety Management Basics of Signal Detection and Pharmacoepidemiology Safety Audits and Inspections (Available in October)