Consultation Registration 5 A rare disease (also known as an “orphan disease”) is an illness that affects a small percentage of the population, thereby limiting scientific research, clinical expertise and patient access to effective treatment options. Recordati Rare Diseases is committed to the well being of patients living with rare diseases. In February 2019, the Canadian government committed $1 billion to a national Rare Disease Drug Strategy to be put in place in 2022. Durhane Wong-Rieger, CORD Panel DiscussionDr. For Canada’s rare disease community, we have been offered the opportunity of a lifetime! For Canada’s rare disease community, we have been offered the opportunity of a lifetime! Webinar 2 (Oct 23, 2020): How can we access preventive and risk-reduction therapies: examples from rare blood disorders. MPS I occurs in about 1 in every 100,000 births globally. In lieu of an orphaned drug strategy, Canada builds considerations for rare disease treatments into the existing negotiation scheme. Please consult your healthcare professional if you have any questions about your health or treatment.​, Copyright © 2020. Virtual They serve as our top priority and sit at the centre of everything we do In February 2019, the Canadian government committed $1 billion to a national Rare Disease Drug Strategy to be put in place in 2022. Health Canada still does not have a definition of a rare disease. 10 Rare Autoimmune Diseases. Kimberly Robinson, Director, Pricing & Market Access, PDCIKim Steele, Director, Government and Community Relations, Cystic Fibrosis CanadaDr. Left untreated, Fabry disease can lead to renal failure, resulting in the need for dialysis or a kidney transplant. The emergence of COVID-19 has disrupted our society’s foundations, destabilizing our work, family and recreational culture incurring great emotional, financial and physical distress. Designing Pan-Canadian Rare Drug Framework Our Goals MONITOR. Does Canada need an Orphan Drug Policy to incentivize drug development, clinical trials, and drug submissions?Can we include access to all drugs, from generic off-label use to preventive risk-reduction therapies to potentially curative ones?What are access pathways for potentially beneficial therapies for serious, progressive, and life-threatening rare diseases, for example, through clinical trials, specialized access programs, and managed access schemes?How can we improve consistency, coordination and collaboration across agencies responsible for regulatory approval, value assessment, and price negotiations?Can we design innovative funding and financing models for transformational and durable (cellular and gene) therapies and models for repurposed, generic and biosimilar medicines?What are viable approaches to governance and management that assure principle-driven, patient-centred, and equitable access in a complex environment made up of private and public drug plans, 13 provincial/territorial healthcare and drug plans, and differential (unequal) local capabilities for managing specialized therapies. The panel will use two “case examples” to deliberate on the challenges presented by: adequate clinical trials evidence, cost-effectiveness assessment, affordability based on price negotiation, availability based on clinical and other services, and accessibility based on drug plan reimbursement. If you suspect Gaucher disease, get tested. A multi-stakeholder panel will discuss the many challenges that can “derail” the journey of a rare drug from regulatory approval to patient access. * Please note that CORD does not provide any clinical services and staff are not able to assist in making a diagnosis or referrals. But time is short. In addition they held their annual conference and awards gala celebration. Webinar 5 is on Dec 4! Browse the GARD list of rare diseases and related terms to find topics of interest to you. Blueprint for the pan-Canadian Rare Drug Program - Draft AgendaDownload. CORD CADTH Consultation Feedback--Aligned Process. Together, people with rare diseases are not so rare. Dix will present the case of Procysbi, one such costly drug for a rare disease approved by Health Canada. Roundtable Panelists: For industry (pharmaceutical/biotech) and corporate associations. Stakeholders will be invited to deliberate on alternatives to the PMPRB guidelines that could better meet the Triple Aim of “timely appropriate patient access”, “optimal, sustainable healthcare expenditure”, and “non-excessive industry compensation that incentivizes launching new therapies and future R&D.” Dr. Rebecca Deyell, BC Children’s Hospital Antonia Palmer, Ac2orn: Advocacy for Canadian Childhood Oncology Research NetworkPatient representative for neuroblastomaCatherine Traill Sharon Stepaniuk Moderator: Durhane Wong-Rieger, CORD Access to Specialty Drugs Under Pharmacare Survey. Amounts to 3 million people, and 1 in 2000 people are afflicted with a clear translational approach caused a... 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