Expanded Access Programs 2016

Transcription

1 REGISTER BY DECEMBER 11, 2015 & SAVE $300! Expanded Access Programs 2016 U.S. and Global Models for Stakeholder Collaboration, Program Design and Supply Equity FEBRUARY 23-24, 2016 DOUBLETREE BY HILTON BETHESDA WASHINGTON, DC Policy Is Propelling Progress as More Biopharmaceutical Products Offer Programs for Compassionate Use and Early Access Hear Thought Leaders Unite on a Path Forward 21st Century Cures Act Right-to-Try Laws FDA Guidance EAP Task Force Program Planning and Design Supply Equity Product Forecasting Stakeholder Collaboration Global Request Management Country-Specific Regulations EAP Partnerships Outsourcing Strategies Reimbursement Models Benefit/Risk Assessment Real-World Data Patient Reported Outcomes EAP Close Out and Transition Richard Klein, Patient Liaison Program Director, Office of Health and Constituent Affairs, FDA Amy McKee, Clinical Team Leader, Division of Oncology Products, FDA Catherine Blansfield, Vice President, Patient Services, National Organization for Rare Disorders (NORD) Durhane Wong-Rieger, President & Chief Executive Officer, Canadian Organization for Rare Diseases (CORD) Anne B. Cropp, Pharm.D., Vice President, Head Clinical Excellence, Compassionate Access Leadership, Global, Innovative Pharma, R&D, Pfizer Inc Nicole D Auteuil, Senior Vice President, Regulatory, Dyax Corporation Jodie Gillon, Head, External Interface Office/Chief Medical Office, AstraZeneca James C. Greenwood, President & CEO, Biotechnology Industry Organization (BIO) Richard Plotkin, Vice Chairman/Co-Founder, Max Cure Foundation, Inc. Jess Rabourn, CFA, Chief Executive Officer, Ax-S Pharma; Managing Director, ALS Emergency Treatment Fund Sean Turbeville, Ph.D., Executive Director, Global Medical and Scientific Affairs, Halozyme Therapeutics, Inc. Nicole Visitacion, PharmD, Director, Global Scientific Communications, Amgen PLUS! BENEFIT FROM TWO FOCUSED EDUCATION STREAMS: Keeping Pace with Progress U.S. Considerations Breaking Geographical Boundaries and Borders Global Considerations Educational Sponsor: Media Partners: CBI, a division of UBM Life Sciences REGISTER AT

2 Current legislation is quickly paving the path for easier patient access to experimental unapproved drugs. CBI s Expanded Access Programs is the industry s leading event dedicated to the nuances and complexities of designing and implementing early access programs. This multi-stakeholder event is focused on U.S. and global models for a variety of access management programs, including, but not limited to: Expanded Access Programs, Early Access Programs, Compassionate Use Programs, Named Patient Programs and Managed Access Programs. The FDA and a broad array of key stakeholders are coming together for an in-depth and meaningful discussion on Expanded Access Programs where the implications, prevailing policies and current industry approaches are discussed in detail. Don t miss the opportunity to join your peers and share best practices around providing investigational, pre-launch or end-of-lifecycle drugs to patients for treatment purposes. Previous Attendee Acclaim for Our Access Series: Meet the wide-range of professionals benefiting from the industry s leading event dedicated to EAPs: Great insights from a variety of perspectives including the patients, regulators and manufacturers, who all addressed real issues. - Vice President, AmerisourceBergen Excellent conference! I learned so much Thank you. - Senior Director, Pfizer Inc Very informative conference. Great speakers and collaborative meeting! Associate Director, Amicus Therapeutics Definitely a must-attend. Director, Global Medical Services, Med Communications Who Should Attend Medical Affairs 29% Clinical 25% Regulatory 21% R&D 11% Patient Advocacy 7% Medical Services 7% 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). You may represent one of the following function areas: 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. Educational Sponsor: A Great Place to Meet Your Market! Take advantage of the best opportunity to meet potential clients face-to-face. Build relationships while demonstrating thought leadership and sharing expertise. For more information on how to position your company as a sponsor or exhibitor, contact Alexa Moore at (339) or alexa.moore@cbinet.com.

3 DAY ONE Tuesday, February 23, :30 Main Conference Registration and Continental Breakfast 8:30 Chairperson s Welcome and Opening Remarks Anne B. Cropp, Pharm.D., Vice President, Head Clinical Excellence, Compassionate Access Leadership, Global, Innovative Pharma, R&D, Pfizer Inc 8:45 OPENING KEYNOTE ADDRESS Breakthroughs and Innovation Fueling Progress A New Era for Rare Diseases, Orphan Products, Stakeholder Collaboration and Access Surge in investment, research and commitment for rare disease treatment options Translational medicine Where are we today and where will this take us? The critical importance of stakeholder collaboration Role of academic institutions in bringing products to market Sustainability of the model moving forward in today s healthcare system James C. Greenwood, President and CEO, Biotechnology Industry Organization (BIO) 9:30 RECENT LEGISLATION AND POLICY UPDATE 21st Century Cures Act and Beyond Policy and Regulatory Frameworks Shaping Early Access Assess the current state of access programs based on recent legislation Dissect the implications of the requirements proposed in the 21st Century Cures Act Gain a robust understanding of what enhancements and procedures are needed to remain compliant pending the new legislation Discuss the status of Right-to-Try laws and Andrea Sloan CURE Act for early access David J. Farber, Partner, King & Spalding LLP Preeya Noronha Pinto, Partner, King & Spalding LLP 10:15 Networking and Refreshment Break 10:45 OHCA/FDA ADDRESS New Models and Guidance for Simplified Access to Investigational Medicine Delve into the implications of key changes in the February 2015 Individual Patient Expanded Access Applications: Form FDA 3926 guidance document Discuss streamlined programs and protocols to simplify access to investigational medicine Assess new forms and requirements for pre-approved treatment Richard Klein, Patient Liaison Program Director, Office of Health and Constituent Affairs, FDA 11:30 Q&A WITH THE FDA Advanced Discussions on the Path Forward for Early Access and Compassionate Use Interact directly with the U.S. regulatory authority on the path forward for expanded access/ compassionate use and collaborate on strategies to best utilize the development of these programs for streamlined patient access. During this unique opportunity attendees engage in an extended Q&A discussion with the FDA to learn more about expectations and responsibilities associated with their role in executing and maintaining an expanded access program. Participants: Richard Klein, Patient Liaison Program Director, Office of Health and Constituent Affairs, FDA Amy McKee, Clinical Team Leader, Division of Oncology Products, FDA 12:15 Networking Luncheon 1:30 STAKEHOLDER COLLABORATION COLLOQUIUM Fostering a United Front for Streamlined Access Programs Enhance physician collaboration models for simplified and accelerated access Optimize strategies for partnering with patient organizations Utilize compliant strategies on multimedia platforms to build program awareness for patients and physicians Implement ethical strategies for managing EAP requests Manage media pushes for treatment from the community Collaborate with solution providers to overcome common pitfalls Moderator: Hema Turabee, Head of EU Programme Management, Idis Managed Access Panelists: Jess Rabourn, CFA, Chief Executive Officer, Ax-S Pharma; Managing Director, ALS Emergency Treatment Fund Nicole Visitacion, PharmD, Director, Global Scientific Communications, Amgen Richard Plotkin, Vice Chairman/Co-Founder, Max Cure Foundation, Inc. 2:15 Bridging the Gap for Unmet Medical Needs Assess the Mechanisms Available for Access Explore the opportunities available to provide controlled and ethical access Assess the avenues accessible depending on the stage of development/product lifecycle

4 Weigh the options based on internal resources and requirements for compliance Jess Rabourn, CFA, Chief Executive Officer, Ax-S Pharma; Managing Director, ALS Emergency Treatment Fund 3:00 Networking and Refreshment Break 3:30 Benchmarks and Best Practices in Managed Access Program Planning, Protocol and Management Formulate a comprehensive planning strategy to ensure successful program roll-out Establish an EAP infrastructure and core team responsible for access programs Discuss the mechanics of building, implementing and managing the day-to-day operations Establish a standard template/protocol for programs that can be modified as needed Maintain strict oversight of treatment/ product delivery Jodie Gillon, Head, External Interface Office/Chief Medical Office, AstraZeneca DAY TWO Wednesday, February 24, :15 SUCCESS STORY SHOWCASE Inspiring Stories, Obstacles Surpassed and Innovative Efforts in the Pipeline During this multi-faceted and interactive discussion, attendees are encouraged to break out into small groups and network with patients, advocates, policy leaders, regulators and industry executives to gain tangible content, critical updates and innovative ideas to advance the dial and expedite access to lifesaving treatment. Through tailored dialogue between all of the key stakeholders, garner leading strategies and key takeaways to pave the path forward for early access and compassionate use. Anne B. Cropp, Pharm.D., Vice President, Head Clinical Excellence, Compassionate Access Leadership, Global, Innovative Pharma, R&D, Pfizer Inc 5:00 Close of Day One Networking, Wine and Cheese Reception immediately following the final session on day one 7:30 Continental Breakfast CHOOSE BETWEEN TWO EDUCATION STREAMS U.S. Keeping Pace with Progress 8:00 Track Chair s Welcome and Recap Anne B. Cropp, Pharm.D., Vice President, Head Clinical Excellence, Compassionate Access Leadership, Global, Innovative Pharma, R&D, Pfizer Inc 8:15 NORD ADDRESS Paving the Path for Early Access and Compassionate Use Discuss strategies for partnering with advocacy groups to assist with EAPs Examine patient considerations when participating in an EAP such as benefit/ risk assessment and patient end points for quality of life Evaluate funding opportunities for early access and delve into the patient protocol for access and reimbursement Catherine Blansfield, Vice President of Patient Services, National Organization for Rare Disorders (NORD) 9:00 Establishing an Internal Process for Managing Global EAP Requests Discuss important considerations for Case Study managing requests on a global scale Discover best practice for developing and implementing operating standards for EAP requests Breaking Geographical Boundaries and Borders 8:00 Track Chair s Welcome and Recap Rob Fox, Vice President Global Business Development, Idis Managed Access 8:15 CORD ADDRESS Advancements Abroad in Patient Access Discuss how Canada s Rare Disease Strategy positions rare diseases as a critical public health issue affecting all Canadians Explore multi-stakeholder initiatives to address barriers and improve care, including enhancing early detection and prevention, providing timely, equitable and evidenceinformed care, encouraging sustainable access to promising therapies and promoting innovative research Durhane Wong-Rieger, President & Chief Executive Officer, Canadian Organization for Rare Diseases (CORD) 9:00 Navigate the Complexities of Competing International Regulations Examine the types of access programs available and what they entail region by region Analyze the regulatory requirements governing various geographical areas Implement strategies for collaborating internationally while ensuring compliance Sean Turbeville, Ph.D., Executive Director, Global Medical and Scientific Affairs, Halozyme Therapeutics, Inc.

5 Assess key considerations for communicating with internal stakeholders and requestors (MD, patient, etc) Nicole Visitacion, PharmD, Director, Global Scientific Communications, Amgen 9:45 Leverage Real-World Data and Patient Reported Outcomes to Support the Regulatory Approval Process Ensure accurate and timely data collection Collaborate with patients and physicians for Case Study efficacy and safety data Garner tips for promoting the importance of the reportable information Hank Mansbach, M.D., Vice President, Medical Affairs, Ultragenyx Pharmaceutical 10:30 Networking and Refreshment Break 11:00 EAP Models and Frameworks for Small to Mid-Sized Bio/Pharmaceutical Companies Garner key strategies for building an EAP from Case Study the ground up Maximize resources and lean personnel for a centralized program structure Institute an ethical process for managing patient requests Nicole D Auteuil, Senior Vice President, Regulatory, Dyax Corporation 11:45 Navigate Ethical Reimbursement Models for Unapproved Medicine Assess the current process and requirements associated with charging for treatment Discuss who should cover the cost Evaluate who should absorb the cost of treatment Explore new models for patient reimbursement 9:45 Partnerships with EAP Providers Assessing Outsourcing Options for Streamlined Access Analyze internal resources to determine whether outside assistance is needed Discover the benefits of partnering with CROs and EAP service providers for accelerated access Investigate the pro s of wider EAP programs for greater distribution of treatment 10:30 Networking and Refreshment Break 11:00 International Product Supply Forecasting and Program Close-Out Implement strategies to gauge patient demand to ensure product supply Balance the impact to drug supply risks, cost, time and quality Establish a patient cap per program Discuss a timeline and valuable tips to ensure proper program close-out and transition Michele Barry, M.D., Ph.D., MBA, Worldwide Medical Director, Immunooncology 11:45 ROUNDTABLE DISCUSSION Best Practices for Extending Treatment Globally Pitfalls and Pearls of Wisdom Enhance global protocol/procedures Assess budgetary restrictions and company resources for international delivery Overcome distribution obstacles for providing treatment Share key takeaways for program management and surveillance Discover innovative solutions to surmount common challenges Facilitator: Lydia Falk, Head of Regulatory Affairs, Prothena Biosciences, Inc. 1:00 CLOSE OF EDUCATION STREAMS AND CONFERENCE About Our Educational Sponsor Idis Managed Access, part of Clinigen Group plc, is the global leader in the specialist management of ethical worldwide access to the most promising, innovative early stage medicines on behalf of pharmaceutical and biotech companies, to address unmet patient need. Our Managed Access Programs provide ethical and regulatory compliant strategic solutions for pharmaceutical and biotechnology companies, enabling a fast and efficient response to patient demand outside of traditional access routes.

6 REGISTER BY DECEMBER 11, 2015 & SAVE $300! Expanded Access Programs 2016 U.S. and Global Models for Stakeholder Collaboration, Program Design and Supply Equity FEBRUARY 23-24, 2016 DOUBLETREE BY HILTON BETHESDA WASHINGTON, DC SCAN HERE WAYS TO REGISTER NOW! WEB expandedaccess CBI, a division of UBM Life Sciences CBI 70 Blanchard Road Burlington, MA cbireg@cbinet.com PHONE outside the U.S. LIVE CHAT expandedaccess ANY QUESTIONS OR TO REGISTER CONTACT: Christian Alongi phone fax to my attention christian.alongi@cbinet.com Expanded Access Programs PC16193 REGISTRATION FEE: ADVANTAGE PRICING Standard Onsite Conference $1899 $2199 $2299 Register by December 11, 2015 and SAVE $300. Fee includes continental breakfast, lunch, wine and cheese reception, refreshments and conference documentation. Credit Card (Visa, MC, AMEX) 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. TEAM DISCOUNT: For every three paying registrations from your company, you will receive a fourth complimentary* registration to the conference (must register four at same time to qualify). To receive the team discount you must register with our customer service department by calling * 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 for further information. Advanced preparation for CBI conferences is not required. VENUE: DoubleTree by Hilton Bethesda 8120 Wisconsin Ave Bethesda, MD Phone Reservations: Hotel Direct Line: ACCOMMODATIONS: To receive CBI s special discounted hotel rate online or by phone, please go to: Online: Phone reservations: and mention CBI s Expanded Access Book Now! The DoubleTree by Hilton Bethesda 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. 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. 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 without notice. The opinions of the conference faculty do not necessarily reflect those of the companies they represent or CBI. REGISTER AT