Blueprint for the pan-Canadian Rare Drug Program - Draft AgendaDownload. Copyright © 2020. Webinar 1 (Oct 9, 2020): Does Canada need an Orphan Drug Policy to incentivize drug development and submissions? Kimberly Robinson, Director, Pricing & Market Access, PDCIKim Steele, Director, Government and Community Relations, Cystic Fibrosis CanadaDr. Webinar 4: (Nov 20, 2020). Recordati Rare Diseases Canada Inc. 3080 Yonge Street, Suite 6060, Toronto, Ontario - Canada M4N 3N1. How Other Countries Provide Access to Rare Disease Drugs: What Canada can Learn … or Not The prevalence of a rare disease usually is an estimate and may change over time. Unlike other countries, Canada has not agreed on a common definition of rare diseases. Your healthcare professional is the single best source of information regarding your health. Our focus is mainly on those who we believe need it most—people affected by rare diseases. Join us and become part of an active Canadian rare disorder community. Webinar 2 (Oct 23, 2020): How can we access preventive and risk-reduction therapies: examples from rare blood disorders. Description. In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discovering treatments for it, except by the government granting economically advantageous conditions to creating and selling such treatments. They state that about 1 in 12 Canadians have a rare disease but later note that there is no common definition of an orphan drug to treat a rare disease. A data lake for rare disease research.. Canada has a lot of lakes, more than every other country in the world combined, in fact. Why did Canada develop and never implement a Canadian Rare Disease Strategy and what has been the aftermath? Today’s top 197 Rare Diseases jobs in Canada. A Canadian research-based pharmaceutical company. Gaucher disease is a rare, progressive, inherited condition that causes a fatty substance, called glucosylceramide (also called GL-1), to build up in certain areas of the body, including the spleen, liver, and bone. This week is a MAJOR information dump from Canada’s leading expert on international approaches to Rare Disease Drug Access. 151 Bloor Street West, Suite 600 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 Gaucher disease is a rare, inherited metabolic condition, and the most common of a family of rare diseases known as lysosomal storage disorders (LSDs). What are the challenges to accessing these preventive therapies through public and private drug plans? Canadian Organization for Rare Disorders Re-imaging Canada’s Rare Drug Strategy. If you suspect Gaucher disease, get tested. New Rare Diseases jobs added daily. A multi-stakeholder panel will discuss the many challenges that can “derail” the journey of a rare drug from regulatory approval to patient access. Comprehensive national framework integrated with a Canadian Rare Disease Strategy 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. Apply Now. This session presents two case examples of current significance to provide context for understanding the challenges and possible solutions in a pan-Canadian Rare Drug Strategy that is directed to meeting patient needs and “leaving no one behind.” This list includes the main name for each condition, as well as alternate names. If you suspect MPS I disease, get tested. Fax: (416) 969-7420 Patients with MPS I disease can experience a wide range of symptoms with variable degrees of severity. Recordati Rare Diseases is committed to the well being of patients living with rare diseases. What are alternative treatments and how effective are these? * Please note that CORD does not provide any clinical services and staff are not able to assist in making a diagnosis or referrals. Designing Pan-Canadian Rare Drug Framework 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.” But Genomics4RD is the first Canada-wide data lake for rare disease research, providing a centralized repository of structured and unstructured data from 5,000+ participants.You can’t canoe across this lake, but it will power rare disease research across the country. NEW Start date is October 2, 2020! Patient Partners Leadership Training. Webinar 3 in CORD’s Rare Drug Strategy Consultation, a multi-stakeholder panel will consider the potential impact of the PMPRB revised guidelines on entry and access of rare disease therapies, using “real” case examples. In Canada, patients with rare diseases face the same issues as elsewhere, but with a few added complications. Approximately 1 in 12 Canadians suffer from 1 of over 7,000 different rare diseases, and the number of rare diseases identified increases every week especially with the emergence of personalized medicine. The Canadian Organization for Rare Disorders (CORD) has released Canada’s Rare Disease Strategy, developed with experts from every sector. This survey is being conducted to learn from Canadians their experiences accessing specialty drugs. This initiative enables scientists in different countries to collaborate on a common interdisciplinary research project, with a clear translational approach. Webinar 2 Video Recording:https://bit.ly/3dVAdo9Webinar 2 Slides: https://bit.ly/37GUJrM But time is short. Left untreated, Fabry disease can lead to renal failure, resulting in the need for dialysis or a kidney transplant. Webinar 7: January 29 @ 11 am – 12 pm EST Although definitions vary by jurisdiction, diseases that affect approximately 1 in 2000 people are considered rare. For Canada’s rare disease community, we have been offered the opportunity of a lifetime! Inclusion on this list does not serve as official recognition by the NIH that a disease is rare. Durhane Wong-Rieger, CORD Panel DiscussionDr. Consultation Registration That amounts to 3 million people, and two-thirds are children. Consultation Plan: Six Webinars addressing fundamental Issues: Your healthcare professional is the single best source of information regarding your health. Here are 10 in the category of rare autoimmune diseases: 1 Canada is one of only a few developed countries without a national “orphan drug” program to protect patients with rare diseases from exorbitant drug costs. Do you know of any events not listed here? Minister Hajdu, PMPRB: Your drug pricing approach is DEAD Wrong for Patients! The panel will consider recommendations to “unblock” barriers that should be built into the operations of "Canada’s Rare Drug Agency 2022.” The Canadian Organization for Rare Disorder's (CORD) is a non-profit organization dedicated to the enhancement of lives of all persons affected by rare disorders through … Pompe disease is a neuromuscular disorder that causes progressive muscle weakness and the loss of muscle tissue. The Strategy details the extraordinary burden faced by Canadian families with rare illnesses. CORD submits House Health Committee HESA hearings on disastrous PMPRB drug pricing rules; supposed to lower prices but instead creates barrier to new medicines for rare diseases #Canada4Rare and other life-saving medicines. The only national organization representing all rare disorder patient groups in Canada. Focus on: • Unituxin and Irinotecan, Temozolomide with GM-CSF for refractory and relapsed neuroblastoma • Kalydeco for pediatric cystic fibrosis patients carrying the mutation R117H What are possible ways forward to making this therapy available? Support & Resources Our top priority is meeting the needs of the rare disease community. 10 Rare Autoimmune Diseases. Progressive Multifocal Leukoencephalopathy (PML) Progressive multifocal leukoencephalopathy … Welcome to 2nd Webinar in the 2021 CORD Consultations on the Pan-Canadian Rare Drug Framework. New Topic "Leave No One Behind: Rarest Among the Rare”, For Canada’s rare disease community, we have been offered the opportunity of a lifetime! In particular, a number of Canadian biopharmaceutical companies and institutional researchers have developed drugs for rare diseases (DRDs) that are saving and improving patients’ lives. Canada is also engaged in E-Rare, the European Union's main instrument for funding research in areas related to rare diseases. FOR IMMEDIATE RELEASE: October 23, 2020 New PMPRB guidelines can’t fix flaws in regulations that will stall new treatments for Canadian patients, Webinar Series: CORD Consultation Toward Canada’s Rare Disease Drug Strategy. Leverage your professional network, and get hired. For individuals, patients or family members. GARD maintains a list of rare diseases and related terms to help people find reliable information. Dix will present the case of Procysbi, one such costly drug for a rare disease approved by Health Canada. Register Now! CCS occurs primarily in the older population (average age 59) and predominantly occurs in males. Webinar 5: Dec 4 @ 12 pm ET. Our Goals MONITOR. Leanne Ward, Professor, Medical Director of the CHEO Bone Health Clinic, Scientific Director of the Ottawa Pediatric Bone Health Research Group  Fred Horne, Former Alberta Minister of Health and Senior Advisor to 3Sixty Public AffairsMarissa Poole, Country Lead, Sanofi Canada and General Manager, Sanofi Genzyme Canada Webinar 3 Video Recording: https://bit.ly/3pNzTgUWebinar 3 Slides: https://bit.ly/3k4CMG5Presentation: Canada. The provisions to end surprise medical billing were included in bipartisan legislation providing additional COVID-19 relief and to fund … Webinar 5 is on Dec 4! It affects approximately 1 in 100,000 people in the general population, and 1 in 855 people in the Ashkenazi Jewish community. Webinar 7: How Other Countries Provide Access to Rare Disease Drugs: What Canada can Learn … or Not, January 29, 2021 CORD’s Submission to PMPRB on the Revised Draft Guidelines & CORD Responses to Consultation Questions for the Proposed Alignment of CADTH Drug Reimbursement Review Processes. 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. What are challenges in approval of these therapies through HTA process? In addition, we are asking opinions about how specialty drugs should be available in a National Pharmacare program. Very Limited Enrolment. National consultation forum: December 2020Provincial consultation forums: January to April 2021“What We Heard” Consolidated Feedback: May 2021Public Consultations (Survey and focus groups): June to August 2021Collaborative Document: October 2021Ready to go on January 1, 2022 All rights reserved.GZCA.XLSD.18.12.0131 February 2019.​, 1 in 12 Canadians are living with a rare disease. The ultimate goal is to achieve consensus on a national Rare Disease Drug Strategy that is: In addition they held their annual conference and awards gala celebration. To affect positive policy change in the rare disease environment within the Canadian health care system to improve the health of Canadians with rare disorders and improve patient access to orphan medicines. Symptoms can mimic other diseases. This comprehensive strategy was released by CORD in May 2015 following a year of multi-stakeholder deliberations. Webinar 5 (Dec 4, 2020): Roundtable: How can we provide access to treatments for specific “subgroups” of populations who are not included in access or reimbursement protocols because they were not part of the original clinical trials, not part of the value-assessment submissions, or have limited evidence of effectiveness in real-world settings. What has been the experience with this therapy and what are the impacts on patients? NEW TOPIC: Leave No One Behind: Rarest Among the Rare (Webinar 5 Registration: https://bit.ly/3j1Rf4W) Kevin Kuo (University Health Network)Dr. Katerina Pavenski (St. Michael’s Hospital)David Page (Canadian Hemophilia Society)aTTP, Thalassemia, Hemophilia, Patient Panel Symptoms can include difficulty performing physical activities, trouble chewing and swallowing, and an inability to rise from a seated position. In Canada, 1 in 12 people are afflicted with a rare disease. According to the Canadian Organization for Rare Diseases (CORD), rare diseases affect one in 12 Canadians (two Recordati Rare Diseases is part of the rare diseases business within the Recordati Group, and is dedicated to developing innovative, high-impact therapies. You can also get involved! Awareness: There is no known awareness day or organization for this condition. Presentation:  General Information: info@raredisorders.ca. Health Canada still does not have a definition of a rare disease. What is the Definition of a Rare Disease? Topics:Similar but not Same: France, Germany, UK, Italy and SpainWho Does it Best for Patients with HPP, XLH, MPS IV, SMA, and CF?Value of A Rare Drug: What is Right “ICER” … or Does It Matter?Whither EDRD Supplemental Process … or Does It Matter?Principles for Rare Drug Framework/Program/StrategyKeynote Speaker: Dr. Tania Stafinski, PRISM (Promoting Rare Disease Innovations Through Sustainable Mechanisms). Roundtable Panelists: CORD provides a strong common voice to advocate for health policy and a healthcare system that works for those with rare disorders. Why are these therapies especially relevant in the time of a pandemic? For patient-based organizations, support groups or other charitable groups. Many thanks to our partners: Health information contained herein is provided for general educational purposes only. For Durhane Wong-Rieger, the President & CEO of the Canadian Organization for Rare Disorders, this is a mistake. With little more than a year to start up, the Canadian Organization for Rare Disorders is launching an ambitious consultation plan, starting October 2020 to end of 2021. Virtual It costs 60 times more than a similar, though older, treatment. A Canadian research-based pharmaceutical company. NEW Program! Today, one area of policy and decision making that lags behind is that for treatment of rare diseases. Access to Specialty Drugs Under Pharmacare Survey. PMPRB: Friend or Foe of Rare Disease Drug Strategy. For Canada’s rare disease community, we have been offered the opportunity of a lifetime! Canada is one of the few developed countries without a national rare diseases strategy to guide diagnosis, treatment and management of rare diseases. But time is short. In February 2019, the Canadian government committed $1 billion to a national Rare Disease Drug Strategy to be put in place in 2022. What was the rationale for Orphan Drug legislation in other countries? Many thanks to all who contributed to our previous Crowdsourcing Webinar. The Lymphoma Research Foundation Canada (LRFC) is a non-profit organization that was founded in 1998 to provide support for those affected by lymphoma and for individuals who conduct research in the diagnosis, treatment, and cure of these diseases. We will continue to endeavor maintaining a constant supply of our critical care products. MPS I disease is a rare inherited genetic disorder caused by a deficiency in an enzyme called alpha-L-iduronidase. CORD provides a strong common voice to advocate for health policy and a healthcare system that works for those with rare disorders. Please consult your healthcare professional if you have any questions about your health or treatment. It is difficult to treat because of malabsorption that accompanies the polyps. 5 Webinar 1 Video Recording: https://bit.ly/2G1s45gWebinar 1 Slides: https://bit.ly/3mlVqdU If you suspect Gaucher disease, get tested. Fabry disease is a rare genetic disease that can be passed on from parents to their children. Realistic, feasible, sustainable and beneficial for all CanadiansSupported by allReady to go on January 1, 2022 What is Canada’s Rare Disease Strategy and how has it influenced healthcare? This September, in the Speech from the Throne, the government further committed to an overall Canadian Rare Disease Strategy, first presented to Parliament in 2015. There are thousands of rare diseases—more than 6,800, according to the National Human Genome Research Institute. RPI Deficiency. “This is a major win for rare disease patients and their families that will help protect them from unexpected medical bills. In lieu of an orphaned drug strategy, Canada builds considerations for rare disease treatments into the existing negotiation scheme. Interestingly, the proposed Supplemental Process reinforces the recommendations for a “managed access” process to rare disease drugs outlined in Canada’s Rare Disease Strategy. Enhancements to NORD’s Rare Disease Database and website were made possible through a grant from the Anthem Foundation, the charitable arm of Anthem, Inc.. NORD’s Rare Disease Database provides brief introductions for patients and caregivers to specific rare diseases. But time is short. Toronto, Ontario M5S 1S4 Canada, Tel: (416) 969-7464 / 1-877-302-7273 What has been the impact of those legislations? Browse the GARD list of rare diseases and related terms to find topics of interest to you. Panelists Rare Diseases In recent years, great strides in research have been achieved in the field of rare diseases in Canada and internationally. Gaucher disease is a rare, progressive, inherited condition that causes a fatty substance, called glucosylceramide (also called GL-1), to build up in certain areas of the body, including the spleen, liver, and bone. All rights reserved.GZCA.XLSD.18.12.0131 February 2019. What are the benefits of therapies that can prevent or reduce the risk of serious symptomology in rare diseases? Cross Canada Consultation Dates Durhane Wong-Rieger, CORD Panel DiscussionRebecca Yu (Takeda Canada)Nicola Worsfold (Jesse’s Journey Canada)Sandra Anderson (Innomar Strategies)Nahya Awada (PhD Candidate Carleton University) Webinar 4 Slides: https://bit.ly/3pLQTEc ​ We all play a part in early diagnosis. Presentation: … Panel Discussion: What are the indications for the extension of the proposed therapy for the specific (sub)population? MPS I occurs in about 1 in every 100,000 births globally. Webinar 3: (Nov 6, 2020). Fred Little, Canada Lead, Rare Disease, Pfizer CanadaChristopher McMaster, Scientific Director, CIHR Institute of Genetics Ferg Mills, Director, Strategic Consulting, Innomar StrategiesBlaine Penny, CEO, MitoCanada CORD (Canadian Organization for Rare Diseases) hosted a breakfast reception for all members of the rare disease community to meet with Members of the Provincial Parliament and other supporters. 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. 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