REGISTER BY JANUARY 26, 2018 AND SAVE $300! Expanded Access Programs 2018 Design Sustainable Early Access Programs to Manage Global Complexities and Advance Real-World Evidence MARCH 28-29, 2018 EMBASSY SUITES BY HILTON ALEXANDRIA OLD TOWN WASHINGTON, DC Join Expert Thought Leaders in a Candid Discussion on Paving the Future of Expanded Access Programs in an Evolving Global Landscape CHAIRMAN: Beverly Harrison, Head of Patient Support, Office of the Chief Medical Officer, Johnson & Johnson Ramana Sonty, Director, Global Medical Organization, Johnson & Johnson Cartier Esham, Executive Vice President, Emerging Companies & Vice President, Science and Regulatory Affairs, Biotechnology Innovation Organization (BIO) Elena Gerasimov, MA, MPH, Director of Programs, Kids v Cancer Richard Klein, Former Director, Patient Liaison, FDA Paul Aliu, PharmD, MB Global Head Medical Governance, Chief Medical Office, Novartis Pharma AG Anne B. Cropp, Pharm.D., Chief Scientific Officer, Early Access Care Kevin Weatherwax, Director IND/IDE Assistance Program, University of Michigan Health System Alison Bateman-House, Research Assistant Professor, Division of Medical Ethics, NYU Erika Segear Johnson, Ph.D., RAC, Associate Director of Regulatory Affairs, Office of Regulatory Affairs and Quality, Duke University School of Medicine Hank Mansbach, VP Clinical Development, Ultragenyx Pharmaceutical Inc. Jess Rabourn, Co-founder and Managing Director, WideTrial June Wasser, Executive Director/CEO, Reagan-Udall Foundation Gretchen M. Randlett, Consultant Clinical Trial Commercial Product Strategy and Process, Eli Lilly and Company Brenda Gleason, President, M2 Health Care Consulting; Professorial Lecturer, GWU School of Public Health Christine MacCracken, Director, Patient Support, Office of the Chief Medical Officer, Johnson & Johnson Jennifer McNary, Board Member, Beauhawks Foundation and Manager, JMcNary Consulting Mariangela Demarcus, Ph.D., Associate Director, Managed Access Programs, Ph IV & Medical Strategy. Vertex Pharmaceuticals (Europe) Limited Emma James, Ph.D., Senior Director, Medical Affairs & Health Outcomes, Audentes Therapeutics, Inc. Key Topics to Be Addressed: Share best practices and review comparable processes and regulations between U.S. and global expanded access programs Streamline policies and procedures to enable the successful launch and sustainability of global EAPs Review industry trends in the collection of real-world data in expanded access programs Navigate pricing, payment and reimbursement policy for expanded access programs Discuss strategies to integrate EAPs into your drug development process at the busiest point of the drug development life cycle Understand the clinician s perspective on single-patient access and group EAPs Consider barriers to access Premier Media Partner: Supporting Media Partners: A division of UBM Americas REGISTER AT WWW.CBINET.COM/EAP 800-817-8601
CBI s Expanded Access Programs conference focuses on the design, development and practical implementation of early access programs. The summit convenes top industry stakeholders and regulators to examine current approaches in providing investigational, pre-launch or end-oflifecycle drugs to patients for treatment purposes. A multi-stakeholder faculty addresses key issues affecting EAPs and reviews the potential impact of the evolving legislative landscape. PREVIOUS ATTENDEE ACCLAIM FOR OUR ACCESS SERIES: fter ten years in the expanded Aaccess space, I learned something new and heard a lot from the patient s perspective. Associate Director, Expanded Access, TESARO reat insights from a variety Gof perspectives including the patients, regulators and manufacturers, who all addressed real issues. Vice President, AmericansourceBergen D efinitely, a must-attend. Director, Global Medical Services, Med Communications WHO SHOULD ATTEND This event will benefit all stakeholders involved in the considerations and development of special programs designed to expand access to investigational treatments (pharmaceutical and biotechnology companies; patient organizations and advocates; regulatory organizations). Medical Affairs Regulatory Affairs Patient Advocacy Clinical Affairs Rare Disease Products Managed Markets Market Access Pharmacovigilance Safety Legal This program will also benefit CROs, sourcing and logistics vendors and those who provide consulting services on the design or management of access management programs. CONFERENCE SPONSORS: A GREAT PLACE TO MEET YOUR MARKET! Maximize your access to decision-makers and align your brand with the life sciences industry s premier thoughtleaders and industry innovators. CBI s custom sponsorship programs are designed to support your organization s overall business development and marketing initiatives through meaningful prospect and customer interactions, brand assertion campaigns and content-rich thought-leadership opportunities. Capitalize on the life sciences community s premier platform for peer-to-peer exchange, solution driven content and first-in-class networking opportunities. For more information on how to position your company as a sponsor or exhibitor, contact Karen Hanover at 617-290-6113 or email karen.hanover@cbinet.com.
DAY ONE Wednesday, March 28, 2018 7:30 Main Conference Registration and Continental Breakfast 8:15 Chairperson s Welcome and Opening Remarks Beverly Harrison, Head of Patient Support, Office of the Chief Medical Officer, Johnson & Johnson 8:30 WASHINGTON UPDATE PANEL SESSION Multi-Stakeholder Perspectives on Legislation Impacting the Future of Expanded Access Hear key collaborators share their insights on: The current state of 21st Century Cures Act and where we are to date on its guidelines and the progress made on implementation The status of the Right-to-Try legislation How the streamlining of the IRB process is progressing and its potential impact on expanded access The FDA update on guidelines of how they use adverse effects data (i.e., practical applications not to negatively impact the drug development process) MODERATOR: Ramana Sonty, Director, Global Medical Organization, Johnson & Johnson PANELISTS: Cartier Esham, Executive Vice President, Emerging Companies & Vice President, Science and Regulatory Affairs, Biotechnology Innovation Organization (BIO) Elena Gerasimov, MA, MPH, Director of Programs, Kids v Cancer Richard Klein, Former Director, Patient Liaison, FDA Emma James, Ph.D., Senior Director, Medical Affairs & Health Outcomes, Audentes Therapeutics, Inc. 9:30 Navigate the Landscape for U.S. and Global Pre-Approval Access Review the factors of clinical drug development and EAPs, the global regulatory environment and the evolving landscape for expanded access programs Develop and implement a strategic approach to managing the operational challenges associated with global early access programs Understand the nomenclatures of EAPs globally and how to deal with specific scenarios Streamline policies and procedures to enable the successful launch and sustainability of global EAPs Examine the traditional barriers of expanded access faced by drug manufacturers and the emerging practices to overcome those barriers Paul Aliu, PharmD, MB Global Head Medical Governance, Chief Medical Office, Novartis Pharma AG 10:15 Networking and Refreshment Break 10:45 Evolving the Scope of the Expanded Access Navigator Hear an update on the role and intended impact of the Expanded Access Navigator, a comprehensive online information resource for expanded access launched in July 2017. Program evaluation to date Update on next steps in defining metrics to measure success Widening the scope Learn about the plans for the Navigator to apply to drugs that treat orphan disease Potential future initiatives June Wasser, Executive Director/CEO, Reagan-Udall Foundation 11:30 Clarifying the Regulatory Framework for Expanded Access Addressing Barriers to Access Discuss myths vs facts about the FDA expanded access process Review key components of the expanded access pathway Update on emergency expanded access process Addressing barriers to access * patient and physician knowledge of the program * potential impact of adverse event data on drug approval process Managing/balancing patient expectations of expanded access What is realistic to expect from expanded access Richard Klein, Former Director, Patient Liaison, FDA 12:30 Networking Luncheon 1:45 Making an Impact with EAPs Understand clinician and patient drivers for expanded access Build processes to efficiently manage patient inquiries for single-patient access Communicate your company s strategy for addressing unmet needs Examine current initiatives to advance awareness of all forms of Expanded Access Anne B. Cropp, Pharm.D., Chief Scientific Officer, Early Access Care 2:30 Determining Early Access at the Early Development Phase of Drug Development Key steps to consider: Anticipate the possibility that there will be requests for investigational agents prior to approval Developing pre-approval plan if necessary Consider operational elements and establish mechanisms for ongoing monitoring and management Jeffery Faris, Head of Global Medical Affairs Operations - Oncology, Janssen Global Services, Johnson & Johnson
3:15 Networking and Refreshment Break 3:45 Expanded Access Helps Patients and It Will Help Drug Makers Too Massive proliferation of clinical trials for new drug development Most patients can t get into trials and therefore the opportunity to learn from them is lost Operationalizing expanded access and capturing RWD will serve those patients and solve clinical trial proliferation Dennis Akkaya, Corporate Development, mytomorrows 4:30 CO-PRESENTATION Removing Barriers to Access Understand the Clinician s Perspective on Single-Patient Access and Group EAPs Assess the clinician burden of filling multiple single-patient requests for individual access, in comparison to enrolling patient in a traditional multi-site expanded access trial Build program awareness for physicians and patients Enhance collaboration models with physicians Discuss proven tools and templates to streamline clinician engagement Providing critical education for clinicians to ensure easier access Compare the IRB process for single-patient, investigator sponsored and drug company sponsored access programs Understand the unique circumstances and needs of physicians not connected with large universities or large healthcare systems Kevin Weatherwax, Director IND/IDE Assistance Program, University of Michigan Health System Erika Segear Johnson, Ph.D., RAC, Associate Director of Regulatory Affairs, Office of Regulatory Affairs and Quality, Duke University School of Medicine 5:30 Close of Day One Networking, Wine and Cheese Reception immediately following the final session on day one DAY TWO Thursday, March 29, 2018 7:30 Continental Breakfast 8:15 Chairperson s Review of Day One Beverly Harrison, Head of Patient Support, Office of the Chief Medical Officer, Johnson & Johnson 8:30 Managing Ethical Considerations for Expanded Access Programs Discuss decision-making protocols to determine what drugs to offer through expanded access programs and how many patients to include Define and assess patient risk levels when patients are willing to participate in any new technologies for a chance to treat their illness Align global access with regulatory filing strategy. If you do not intend to market a drug in a country, is it right to offer expanded access? And for how long? Managing the patient transition when the drug becomes commercially available but yet is not reimbursable or the patient cannot afford it Determine strategies to maintain fairness across all patients MODERATOR: Alison Bateman-House, Research Assistant Professor, Division of Medical Ethics, NYU PANELISTS: Christine MacCracken, Director, Patient Support, Office of the Chief Medical Officer, Johnson & Johnson Paul Aliu, PharmD, MB Global Head Medical Governance, Chief Medical Office, Novartis Pharma AG Paul Melmeyer, Director, Federal Policy, National Organization of Rare Disorders 9:30 Launching and Sustaining Expanded Access Programs Pitfalls and Lessons Learned Discuss the costs and infrastructure required to open and sustain EAPs Determine the key steps necessary to open and operationalize an EAP Formulate a comprehensive planning strategy to ensure success Identify calculated risks and challenges Both domestic and global Implement ethical procedures for managing requests for treatment Hank Mansbach, Vice President Clinical Development, Ultragenyx Pharmaceutical Inc. 10:15 Networking and Refreshment Break 10:45 Demystify Global Complexities in Expanded Access Review current global access programs and what they entail region by region Analyze the nuances of regulatory requirements that govern various geographical areas Balance the impact to drug supply risks, cost, time and quality Mariangela Demarcus, Ph.D., Associate Director, Managed Access Programs, Ph IV & Medical Strategy Vertex Pharmaceuticals (Europe) Limited
11:30 CONCURRENT WORKING SESSIONS Select one session for an interactive, deep-dive, facilitated discussion A Operationalizing Global Expanded Access Programs Examine global challenges/scenarios and potential solutions Managing the blurred lines between continued access and commercial off-label access Practical tips and applications to manage last minute global regulation changes Gretchen M. Randlett, Consultant Clinical Trial Commercial Product Strategy and Process, Eli Lilly and Company B Multi-Stakeholder Perspectives on Pricing and Reimbursement of Unapproved Medicines Examine current approaches and requirements associated with charging for treatment Explore potential new models for patient reimbursement under existing conditions Understand global differences in pricing of newly approved therapies Consider legislation and advocacy approaches to improved U.S. reimbursement policy Brenda Gleason, President, M2 Health Care Consulting; Professorial Lecturer, GWU School of Public Health 12:30 Networking Luncheon 1:30 Economic Sustainability and Expanded Access Programs Develop functional business models to align charitable, scientific and medical interests to achieve wider patient engagement Collaborate to manage risk, operational challenges and costs Understand the reimbursement landscape and uncover the potential for cost recovery Next steps for generation of real-world evidence and data utility Jess Rabourn, Co-founder and Managing Director, WideTrial 2:15 Define the Parameters of Data Collection for Expanded Access Advancing the Generation of Real-World Evidence to Address Multi-Stakeholder Needs Examine the potential impact and scope of generating real-world evidence within expanded access programs Clarify impact on drug approval process Discuss industry best practices for data collection Review current trends in the amount of data collected, how it is collected and potential global challenges in data collection Consider how expanded access data is being used and impact of collecting data on drug pre-approval process Explore cohort based EAPs versus single-patient access on data collection and costs Ensure accurate and timely data Educate patients and clinicians on the importance of data collection and their role in reporting MODERATOR: Beth E. Roxland, J.D., M.Bioethics, Senior Consultant on Law, Health Policy and Ethics PANELISTS: Hank Mansbach, Vice President Clinical Development, Ultragenyx Pharmaceutical Inc. Jess Rabourn, Co-founder and Managing Director, WideTrial Jennifer McNary, Board Member, Beauhawks Foundation and Manager, JMcNary Consulting 3:00 Close of Conference
REGISTER BY JANUARY 26, 2018 AND SAVE $300! Expanded Access Programs 2018 Design Sustainable Early Access Programs to Manage Global Complexities and Advance Real-World Evidence SCAN HERE MARCH 28-29, 2018 EMBASSY SUITES BY HILTON ALEXANDRIA OLD TOWN WASHINGTON, DC WAYS TO REGISTER NOW! WEB www.cbinet.com/eap A division of UBM Americas CBI 70 Blanchard Road Burlington, MA 01803 EMAIL cbireg@cbinet.com ANY QUESTIONS OR TO REGISTER CONTACT: PHONE 800-817-8601 339-298-2100 outside the U.S. LIVE CHAT www.cbinet.com/eap Christian Alongi phone 339-298-2131 email christian.alongi@cbinet.com Expanded Access Programs 2018 PC18193 REGISTRATION FEE: ADVANTAGE PRICING Standard Onsite Conference $1899 $2199 $2299 Register by January 26, 2018 and SAVE $300. Fee includes continental breakfast, lunch, wine and cheese reception, refreshments and conference documentation. Credit Card (Visa, MC, AMEX, Discover) or checks accepted. Please make checks (in U.S. funds drawn on a U.S. bank) payable to: CBI. (No personal checks accepted.) PLEASE NOTE: All advertised discounts are taken from the full, Standard Rate. GROUP RATE: Looking to bring your team? Contact Information Services to learn about potential group savings. Call 800-817-8601 or email cbireg@cbinet.com. * Advantage pricing rates do apply when applicable. Offer may not be combined with any other special pricing promotions. Offer may be used at CBI co-located events. SATISFACTION GUARANTEED: CBI stands behind the quality of its conferences. If you are not satisfied with the quality of the conference, a credit will be awarded towards a comparable CBI conference of your choice. Please contact 800-817-8601 for further information. Advanced preparation for CBI conferences is not required. VENUE: Embassy Suites by Hilton Alexandria Old Town 1900 Diagonal Rd Alexandria, VA 22314 Reservations: (800) 445-8667 Hotel direct line: (703) 684-5900 ACCOMMODATIONS: To receive CBI s special discounted hotel rate online or by phone, please go to: Online: www.cbinet.com/eap Phone reservations: (800) 445-8667 and mention CBI s Expanded Access Book Now! The Embassy Suites by Hilton Alexandria Old Town is accepting reservations on a space and rate availability basis. Rooms are limited, so please book early. All travel arrangements subject to availability. PLEASE NOTE: All hotel reservations for this conference should be booked directly with the hotel. CBI does not use Housing Bureaus and none are authorized to call on our behalf. SUBSTITUTION AND CANCELLATION: Your registration may be transferred to a member of your organization up to 24 hours in advance of the conference. All cancellations received in writing on or before 14 days prior to the start date of the event will be refunded, less a $399 administrative charge. No refunds will be made after this date; however, the registration fee less the $399 administrative charge can be credited to another CBI conference if you register within 30 days from the date of this conference to an alternative CBI conference scheduled within the next six months. In case of conference cancellation, CBI s liability is limited to refund of the conference registration fee only. Cancellation of a conference due to events beyond our control* are subject to a $399 administrative charge should you or a colleague be unable to attend the rescheduled date. CBI reserves the right to alter this program without prior notice. Please Note: Speakers and agenda are subject to change. In the event of a speaker cancellation, every effort to find a suitable replacement will be made. The opinions of the conference faculty do not necessarily reflect those of the companies they represent or CBI. *Events beyond our control include: severe weather conditions, natural and man-made disasters and any other similar events. REGISTER AT WWW.CBINET.COM/EAP 800-817-8601