RARE DISEASE DAY CONFERENCE 2018: Building on Research Excellence to Improve Patient Care Two-Day Interactive Conference
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1 March 21-22, 2018 DELTA OTTAWA CITY CENTRE, 101 Lyon Street Ottawa, Ontario How this Conference Will Make a Difference (for Rare Diseases in Canada) Day 1 (AM) In May 2015, the Canadian Organization for Rare Disorders, with multistakeholder collaboration, launched Canada s Rare Disease Strategy. It has become both the cornerstone and the touchstone for building Canada s Rare Disease Alliance, encompassing diagnosis and prevention, comprehensive expert treatment and care, community-based support, access to therapies, and research. This session will provide examples of excellence in rare disease spanning these areas, with a focus on application of genomics in diagnosis, models of multi-disciplinary/multistakeholder rare disease networks, Canadian discovery and development of drugs and devices, and support communities. Day 1 (PM) This multi-stakeholder session is a sobering reflection on real-world access to rare disease therapies (and other supports) in Canada. How well does Canada live up to good? Do patients get timely access to new (and promising) therapies through clinical trials, expedited approvals, early access programmes, patient-centred assessments, and individualized managed access programmes? And how good is the process? Are patients meaningfully engaged throughout the lifecycle? Are timelines respected? And are there procedures to assure transparency and accountability? AGENDA Day 1(March 21) 8:00 a.m. 8:45 a.m. Breakfast and Registration 8:45 a.m. 9:00 a.m. 9:00 a.m. 9:45 a.m. Welcome and Morning Objectives Canada s Rare Disease Strategy: Building on Today s Capabilities to Create Tomorrow s Opportunities Accelerating rare disease diagnosis: Canada s excellence from research to clinical practice How did FORGE improve rare diagnosis through genome (exome) sequencing?
2 How has the Care4Rare Consortium contributed to translation of gene knowledge to treatment improvement? What can we hope from the next big collaboration: C4R-SOLVE Kym Boycott, Children s Hospital of Eastern Ontario 9:45 a.m. 10:30 a.m. Rare Disease Centres of Expertise: what we are learning and what wll we need to sustain and expand? Neuromuscular Disease Network/SMA - (TBD) Bone Disease Network - Cheryl Greenberg, Winnipeg Regional Health Authority Rhett Syndrome Community: Clinic, Registry, Research, Support - Victoria Siu, London Health Sciences Centre 10:30 a.m. 10:45 a.m. 10:45 a.m. 11:45 a.m. Enhancing Canada s footprint in the development of therapies for rare diseases Discovery, adaptation, and targeting drugs for rare conditions - Daniel Drucker, Lunenfeld Tanenbaum Research Institute Novelion Therapeutics - Andrés Treviño, Novelion Therapeutics Medical devices to improve capabilities and support quality of life - Pamela Borges, B-TEMIA Inc How can we create caring communities that support and empower? (TBD) Scleroderma Society of Canada 11:45 a.m. 12:00 p.m. 12:00 p.m. 1:00 p.m. Wrap Up All Lunch
3 1:00 p.m. 2:15 p.m. Welcome and Afternoon Objectives: Bringing Rare Disease Drugs to Canada: Opportunities, Challenges, and More Challenges 1. Access to Rare Disease Drugs through Canada s Approval Process (Regulatory, Pricing, HTA, Funding) 2. How Patients Experience Access: Clinical Trials and Early Access of Orphan Drugs in Canada; SAP Regulatory Review: Expedited and Early-Stage Aprovals HTA Process for Rare Disease Drugs Funding through Private and Publc Plans Accessing Medical Devices for Rare Diseases 2:15 p.m. 2:30 p.m. 2:30 p.m. - 3:30 p.m. Current and Future Reality of Canadian Environment for Orphan Drugs Is Canada attracting clinical trials for rare diseases? How much support do researchers and innovative startups receive in Canada? Do companies feel Canada (still) needs Orphan Drug Regulatory Framework despite pathways to approval with existing legislation and regulations? Has review of rare disease drugs through the regular process for common drugs resulted in appropriate assessments and recommendations? Is a RDD pathway still needed? Does review through pcpa and public drug programs providing timely and appropriate access for patients with urgent, progressive, and life-threatening conditions to therapies that may have been approved with high uncertainty and high per patient costs? Is Canada bringing in alternative access solutions for drugs that do not fit the common paradigm? How will proposed amendments to PMPRB affect rare disease drugs? Where are private plans now in coverage for rare diseases and what are the future directions?
4 3:30 p.m. 4:00 p.m. Rapprochement: Where are we now, where are we heading, and what do we need to be doing to assure timely patient access to rare disease drugs: What are we learning from the patient experience? What has improved over the past decade since the initial Fabry programme? What are we hoping for and what are we afraid of with reform taking place among all players in the drug lifecycle? What are the solutions for Canada? Comimg in Day 2. Health Canada Session at Canadian Organization for Rare Disorders (CORD) Meeting AGENDA Day 2 (AM) March 22 8:00 a.m. 8:30 a.m. Breakfast and Registration 8:30 a.m. 8:45 a.m. Welcome and Opening Remarks Durhane Wong-Rieger, President & CEO, CORD Cathy Parker, Director General, Biologics and Genetic Therapies Directorate, Health Canada 8:45 a.m. 9:15 a.m. 9:15 a.m. 10:15 a.m. 10:15 a.m. 10:30 a.m. Canadian Regulatory Framework for drugs for rare disease Fiona Frappier, PhD, Senior Policy Analyst, Health Canada Regulatory Review of Drugs and Devices (R2D2) Megan Bettle, Director, Regulatory Innovation and Business Operations, BGTD, Health Canada
5 10:30 a.m. 11:50 a.m. 11:50 a.m. 12:00 p.m. Panel Discussion on R2D2: Collaboration amongst health portfolio partners Brian O Rourke, CADTH, Doug Clarke, PMPRB Luc Boileau, INESSS Imran Ali, pcpa Megan Bettle, Health Canada Suzanne McGurn, Ontario Drug Programme, MOHLTC Pamela Fralick, Innovative Medicines Canada Closing Remarks Agnes Klein, MD, Senior Medical Advisor, BGTD AGENDA Day 2 (PM ) University of Alberta PRISM and Integrative Health Institute Workshop: Systems Thinking to Design Managed Access that Works Detailed AGENDA in development 1. What have we learned from successful Canadian experiences with various forms of managed access to rare and specialty drugs? 2. What are best international examples of access to orphan drugs, including early and expedited access? 3. How can we better engage patients and caregivers to develop relevant outcome measures and to participate as partners in managed access? 4. What are system process requirements and contribution of all stakeholders to assure success of managed access to reduce risk and uncertainty while expediting access to patients with urgent and chronic needs?
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Wednesday October 14, 2015 Startup Acceleration Training (8:00-15:00) 08:00-15:00 Invitation only. For more information, please visit http://goo.gl/forms/fpm0vdyciz. Biocluster Workshop - Internationalization
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