Welcome to 2nd Webinar in the 2021 CORD Consultations on the Pan-Canadian Rare Drug Framework. For individuals, patients or family members. Progressive Multifocal Leukoencephalopathy (PML) Progressive multifocal leukoencephalopathy … Virtual Your healthcare professional is the single best source of information regarding your health. Health Canada still does not have a definition of a rare disease. Webinar 3 Video Recording: https://bit.ly/3pNzTgUWebinar 3 Slides: https://bit.ly/3k4CMG5Presentation: Webinar 5 is on Dec 4! Although definitions vary by jurisdiction, diseases that affect approximately 1 in 2000 people are considered rare. 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 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 Canadian Organization for Rare Disorders Consultation Registration 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. Roundtable Panelists: Kimberly Robinson, Director, Pricing & Market Access, PDCIKim Steele, Director, Government and Community Relations, Cystic Fibrosis CanadaDr. 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 The only national organization representing all rare disorder patient groups in Canada. 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. CORD provides a strong common voice to advocate for health policy and a healthcare system that works for those with rare disorders. For industry (pharmaceutical/biotech) and corporate associations. This initiative enables scientists in different countries to collaborate on a common interdisciplinary research project, with a clear translational approach. 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. 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. Minister Hajdu, PMPRB: Your drug pricing approach is DEAD Wrong for Patients! NEW Program! Webinar 1 (Oct 9, 2020): Does Canada need an Orphan Drug Policy to incentivize drug development and submissions? RT @kc4ktaf: It’s Friday Follow! MPS I disease is a rare inherited genetic disorder caused by a deficiency in an enzyme called alpha-L-iduronidase. What are challenges in approval of these therapies through HTA process? What are the benefits of therapies that can prevent or reduce the risk of serious symptomology in rare diseases? What was the rationale for Orphan Drug legislation in other countries? Our Goals MONITOR. 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. The Strategy details the extraordinary burden faced by Canadian families with rare illnesses. Why are these therapies especially relevant in the time of a pandemic? According to the Canadian Organization for Rare Diseases (CORD), rare diseases affect one in 12 Canadians (two That amounts to 3 million people, and two-thirds are children. Awareness: There is no known awareness day or organization for this condition. All rights reserved.GZCA.XLSD.18.12.0131 February 2019.​, 1 in 12 Canadians are living with a rare disease. RPI Deficiency. Recordati Rare Diseases Canada Inc. 3080 Yonge Street, Suite 6060, Toronto, Ontario - Canada M4N 3N1. 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. But time is short. For patient-based organizations, support groups or other charitable groups. … CORD provides a strong common voice to advocate for health policy and a healthcare system that works for those with rare disorders. CORD CADTH Consultation Feedback--Aligned Process. “[The drug-pricing system] was never really designed for innovative therapies,” says Wong-Rieger. 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). Fax: (416) 969-7420 Left untreated, Fabry disease can lead to renal failure, resulting in the need for dialysis or a kidney transplant. Webinar 7: How Other Countries Provide Access to Rare Disease Drugs: What Canada can Learn … or Not, January 29, 2021 CORD works with governments, researchers, clinicians and industry to promote research, diagnosis, treatment and services for all rare disorders in Canada. There are thousands of rare diseases—more than 6,800, according to the National Human Genome Research Institute. Here are 10 in the category of rare autoimmune diseases: 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. Many thanks to all who contributed to our previous Crowdsourcing Webinar. What has been the impact of those legislations? Sign the Petition: Help Kaysen access Zolgensma a life changing gene therapy for treatment of SMA before his 2nd birthday. 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. Your healthcare professional is the single best source of information regarding your health. 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. 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. MPS I occurs in about 1 in every 100,000 births globally. NEW TOPIC: Leave No One Behind: Rarest Among the Rare (Webinar 5 Registration: https://bit.ly/3j1Rf4W) Please consult your healthcare professional if you have any questions about your health or treatment.​, Copyright © 2020. What are possible ways forward to making this therapy available? Webinar 2 Video Recording:https://bit.ly/3dVAdo9Webinar 2 Slides: https://bit.ly/37GUJrM Re-imaging Canada’s Rare Drug Strategy. Copyright © 2020. “This is a major win for rare disease patients and their families that will help protect them from unexpected medical bills. Webinar 4 Slides: https://bit.ly/3pLQTEc 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. Pompe disease is a neuromuscular disorder that causes progressive muscle weakness and the loss of muscle tissue. Inclusion on this list does not serve as official recognition by the NIH that a disease is rare. 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. But time is short. 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. Webinar 5: Dec 4 @ 12 pm ET. Fabry disease is a rare genetic disease that can be passed on from parents to their children. Canada is also engaged in E-Rare, the European Union's main instrument for funding research in areas related to rare diseases. 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.” CCS occurs primarily in the older population (average age 59) and predominantly occurs in males. In the United States, a rare disease is defined as one that affects fewer than 200,000 people. In February 2019, the Canadian government committed $1 billion to a national Rare Disease Drug Strategy to be put in place in 2022. Consultation Plan: Six Webinars addressing fundamental Issues: * Please note that CORD does not provide any clinical services and staff are not able to assist in making a diagnosis or referrals. Today’s top 197 Rare Diseases jobs in Canada. Symptoms can mimic other diseases. 10 Rare Autoimmune Diseases. The ultimate goal is to achieve consensus on a national Rare Disease Drug Strategy that is: How Other Countries Provide Access to Rare Disease Drugs: What Canada can Learn … or Not They serve as our top priority and sit at the centre of everything we do Very Limited Enrolment. Kevin Kuo (University Health Network)Dr. Katerina Pavenski (St. Michael’s Hospital)David Page (Canadian Hemophilia Society)aTTP, Thalassemia, Hemophilia, Patient Panel This survey is being conducted to learn from Canadians their experiences accessing specialty drugs. Health information contained herein is provided for general educational purposes only. Unlike other countries, Canada has not agreed on a common definition of rare diseases. Cross Canada Consultation Dates If you suspect Gaucher disease, get tested. 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. 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. This comprehensive strategy was released by CORD in May 2015 following a year of multi-stakeholder deliberations. Canada. Patients with MPS I disease can experience a wide range of symptoms with variable degrees of severity. Recordati Rare Diseases is part of the rare diseases business within the Recordati Group, and is dedicated to developing innovative, high-impact therapies. What are the challenges to conducting additional clinical trials and/or collecting additional evidence? In lieu of an orphaned drug strategy, Canada builds considerations for rare disease treatments into the existing negotiation scheme. If you suspect Gaucher disease, get tested. PMPRB: Friend or Foe of Rare Disease Drug Strategy. Support & Resources Our top priority is meeting the needs of the rare disease community. Join us and become part of an active Canadian rare disorder community. Patient Partners Leadership Training. NEW Start date is October 2, 2020! A multi-stakeholder panel will discuss the many challenges that can “derail” the journey of a rare drug from regulatory approval to patient access. New Rare Diseases jobs added daily. 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. 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. Webinar 2 (Oct 23, 2020): How can we access preventive and risk-reduction therapies: examples from rare blood disorders. It costs 60 times more than a similar, though older, treatment. 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) Realistic, feasible, sustainable and beneficial for all CanadiansSupported by allReady to go on January 1, 2022 Why did Canada develop and never implement a Canadian Rare Disease Strategy and what has been the aftermath? Focus on: • Unituxin and Irinotecan, Temozolomide with GM-CSF for refractory and relapsed neuroblastoma • Kalydeco for pediatric cystic fibrosis patients carrying the mutation R117H Webinar 3: (Nov 6, 2020). A Canadian research-based pharmaceutical company. Register Now! Do you know of any events not listed here? 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 is the Definition of a Rare Disease? Durhane Wong-Rieger, CORD Panel DiscussionDr. Blueprint for the pan-Canadian Rare Drug Program - Draft AgendaDownload. Comprehensive national framework integrated with a Canadian Rare Disease Strategy 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.” In addition, we are asking opinions about how specialty drugs should be available in a National Pharmacare program. Access to Specialty Drugs Under Pharmacare Survey. 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 rare disease is any disease that affects a small percentage of the population. An open letter to Canada's provincial minister of health for access to Zolgensma. This list includes the main name for each condition, as well as alternate names. The provisions to end surprise medical billing were included in bipartisan legislation providing additional COVID-19 relief and to fund … Many thanks to our partners: Health information contained herein is provided for general educational purposes only. Toronto, Ontario M5S 1S4 Canada, Tel: (416) 969-7464 / 1-877-302-7273 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 … It affects approximately 1 in 100,000 people in the general population, and 1 in 855 people in the Ashkenazi Jewish community. What are the challenges to accessing these preventive therapies through public and private drug plans? This week is a MAJOR information dump from Canada’s leading expert on international approaches to Rare Disease Drug Access. 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! Interestingly, the proposed Supplemental Process reinforces the recommendations for a “managed access” process to rare disease drugs outlined in Canada’s Rare Disease Strategy. In Canada, 1 in 12 people are afflicted with a rare disease. 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