How To Prepare For An Fda Seminar

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1 MINISTRY OF FOREIGN AFFAIRS OF DENMARK THE EMBASSY OF DENMARK, WASHINGTON, DC FDA Seminar The Embassy of Denmark in Washington, DC is proud to present again its unique two-day FDA Seminar in collaboration with the U.S. Food and Drug Administration (FDA) officials. This seminar provides the latest updates, direction and forward-thinking from FDA speakers. Program topics Understanding Drug Regulation in the Overall Context of FDA FDA Meeting Process (including pre-ind meetings), Advisory Committees Rare Diseases, Expanded Access International collaboration from an FDA Perspective Ethics and Conduct of Clinical Trials Pediatric Activities under FDASIA The Content of a Pediatric Study Plans (PSP) The Process and Substance of FDA Review of PSPs and PPSRs Expectations for Revised Labeling for Drug Use in Pregnancy and Lactation Challenging Topics in Pediatric Product Development Future Developments in Pediatrics within FDA Hot Topic Forum Why Attend An opportunity for one-on-one meetings with FDA officials Understand the critical changes to create successful strategies in the US Benchmark your regulatory strategy against agency requirements Network with peers during and lunch and refreshment breaks Registration fee DKK per person which includes two-day seminar participation, materials, lunches/refreshments, and individual consultations with FDA officials. A certificate of participation will be issued after the seminar. Location Ministry of Foreign Affairs, Eigtveds Pakhus, Asiatisk Plads 2G, Copenhagen Denmark Information & Registration See enclosed program, speaker biographies, and registration form. Questions? Contact Lasse Eilenberg at laseil@um.dk Registration deadline February 27, 2015 Seating is limited, early registration is advised 1

2 Day 1 Program* Wednesday, March 18, :00 08:30 Registration & Light Refreshments 08:30 08:45 Welcome & Introduction 08:45 09:45 Understanding Drug Regulation in the Overall Context of FDA Overview of the FDA organization and basic differences in regulation of drugs, medical devices and dietary supplements. Details on CDER organization and how it facilitates sound scientific and regulatory review. 09:45 10:45 FDA Meeting Process (including pre-ind meetings), Advisory An overview on the types of meetings available to sponsors with the FDA during the product development process, general purpose and timing of those meetings prior to the submission of an NDA/BLA. Selection, screening and use of external experts (SGEs), the Advisory Committee process, and dispute resolution. 10:45 11:15 Break & Networking 11:15 12:15 Rare Diseases, Expanded Access An overview of some of the challenges in product development for rare diseases, including study designs, choice of control groups (natural history, use of placebo); the importance of patient engagement and the incentives available for rare disease product development (orphan designation, pediatric rare disease priority review voucher). In this context the FDA Expanded Access regulations (often called compassionate use ) and the challenges this presents for rare disease product development will be included. 2

3 Day 1 12:15 13:15 Lunch & Networking 13:15 13:45 Update on Pediatric Activities under FDASIA A review of the impact of FDASIA 2012 on pediatric product development, building on the legislative provisions of FDAAA Topics will include the establishment of the internal Pediatric Review Committee, changes in the timing of the submission and review of Pediatric Study Plans (PSP), the relationship of the required studies under a PSP and voluntary studies conducted under BPCA, the role of a Proposed Pediatric Study Request (PPSR), the establishment of Post-Marketing Requirements (PMR) and the ability of FDA to extend the deadline for submission of pediatric PMRs, and the ability of FDA to require post-marketing safety studies. 13:45 14:15 The Content of a Pediatric Study Plans (PSP) A review of the required PSP content, draft guidance on PSPs including anticipated changes and/or clarification. 14:15 14:45 Break & Networking 14:45 15:15 The Process and Substance of FDA Review of PSPs and PPSRs A more detailed review of the FDA internal review process of both PSPs and PPSRs by the FDA divisions and the Pediatric Review Committee. Selected scientific and ethical issues that arise in the development of a PSP and PPSR will be highlighted in anticipation of a more in-depth discussion of these issues in day 2 of the seminar. 15:15 15:45 International collaboration from an FDA Perspective Focus on the relationship between the FDA PSP and the EMA Pediatric Implementation Plan (PIP). The overlapping context of the PSP and PIP will be reviewed, along with a discussion of why a single application to both EMA and FDA would present challenges. The current collaboration between FDA and EMA will be reviewed, with examples of the results of that collaboration (e.g., Common Commentary, joint publications). 15:45 16:00 Adjourn 16:00 - One-on-one sessions with FDA officials (15 min. each) * Program can be subject to changes 3

4 Day 2 Program* Thursday, March 19, :00 08:30 Registration & Light Refreshments 08:30 08:45 Welcome & Introduction 08:45 10:15 Challenging Topics in Pediatric Product Development An in-depth discussion of selected scientific and ethical issues in pediatric product development such as the ethical framework for the additional protections for children in research, the selection and use of alternate trial designs, endpoint development including patient and parent-reported outcome measures, and the use of extrapolation. 10:15 10:45 Break & Networking 10:45 11:15 Expectations for Revised Labeling for Drug Use in Pregnancy and Lactation Presentation of the recently published Final Rule on the Content and Format of Labeling for Human Prescription Drug and Biological Products; Requirements for Pregnancy and Lactation Labeling (published December 4, 2014; effective June 30, 2015), with focus on the expectations and implementation of the rule (and the draft FDA guidance released in December 2014). The role of pregnancy registries and other post marketing surveillance tools for human data, and the challenges for obtaining data during lactation. 11:15 11:45 Future Developments in Pediatrics within FDA Conclusion on the pediatric portion of the seminar focusing on future developments in pediatrics within FDA (e.g., upcoming draft and final guidance, proposed rule-making on pediatric study plans). 4

5 Day 2 11:45 13:00 Lunch & Networking 13:00 14:00 Ethics and Conduct of Clinical Trials Reflections on the ethical challenges of conducting clinical trials in lowand middle-income countries set within the context of international ethical guidelines, the variability in local standards of care, and the requirement for equipoise. The controversy over the design and implementation of clinical trials for products to treat the Ebola Virus disease may be used to illustrate these issues. 14:00 14:30 Break & Networking 14:30 15:30 Hot Topic Forum ** An open forum for questions and discussion. 15:30 15:45 Adjourn Potential topics of interest could be: drug shortages and supply chain issues; FDA s approach to risk management and REMS; explorative clinical studies, limits of FDA oversight; biomarkers and combinations with (imaging/in vitro) diagnostics; adaptive designs and update on FDA experience and policies; and specific areas of FDA guidance for disease areas (e.g., inflammation, diabetes, cancer, cardiovascular). ** FDA speakers may be limited by knowledge/expertise in their ability to respond to all questions. 15:45 - One-on-one sessions with FDA officials (15 min. each) * Program can be subject to changes 5

6 Speakers Robert Skip Nelson, M.D., Ph.D., Deputy Director and Senior Pediatric Ethicist Office of Pediatric Therapeutics, Office of the Commissioner U.S. Food and Drug Administration Dr. Nelson joined the FDA full-time in 2009 having held positions in a number of advisory committees such as former Chair ( ) and member ( ) of the FDA Pediatric Advisory Committee and the Pediatric Ethics Subcommittee. He was a member of the Subcommittee on Research Involving Children of the Secretary s Advisory Committee on Human Research Protections ( ), and the Human Studies Review Board of the Environmental Protection Agency (2006). Dr. Nelson was a member of the Committee on Clinical Research Involving Children of the Institute of Medicine ( ), and a member and former Chair of the Committee on Bioethics of the American Academy of Pediatrics ( ). Dr. Nelson is the Editor-in-Chief of the AJOB Empirical Bioethics, which publishes empirical research in bioethics. Prior to joining FDA he was Professor of Anesthesiology, Critical Care and Pediatrics at The Children s Hospital of Philadelphia and University of Pennsylvania School of Medicine. Melissa S. Tassinari, PhD Senior Clinical Advisor, Pediatric and Maternal Health Staff, Office of New Drugs Center for Drug Evaluation and Research, U.S. Food and Drug Administration Melissa Tassinari joined the FDA after retiring from Pfizer, Inc. where she held several leadership positions in the toxicology laboratories and in regulatory affairs. Prior to joining Pfizer, she was Assistant Professor in Cell Biology at the University of Massachusetts Medical Center and taught at both Simmons and Wellesley College. She is a past-president of the Teratology Society and has broad experience in both developmental toxicology and pediatric drug development. 6

7 Registration deadline February 27, 2015 Return completed form to Lasse Eilenberg at Registration Form FDA Seminar Yes, I want to attend the FDA Seminar on at the Ministry of Foreign Affairs, Eigtveds Pakhus, Asiatisk Plads 2G, Copenhagen Denmark. DKK per person (mark/check required) The registration fee includes two day seminar participation, materials, lunches/refreshments, and individual consultation with FDA officials. An invoice and certificate of participation will be issued after the seminar. Registration is binding. Name: Title: Company: Address: Phone: My interest(s) in the seminar are: Consultations: I would like to reserve a one-on-one consultation of 15 minutes with the following speakers* Dr. Skip Nelson. FDA Dr. Melissa Tassinari, FDA Elizabeth Dempsey Becker, Danish Embassy My preference for the day of the consultation is: Wednesday, March 18 Thursday, March 19 * If overbooked, consultations will be assigned on a first-come, first-served basis. Signature Date Send completed registration to Lasse Eilenberg at laseil@um.dk before February 27 7