This week is a MAJOR information dump from Canada’s leading expert on international approaches to Rare Disease Drug Access. What are the challenges to conducting additional clinical trials and/or collecting additional evidence? Description. 5 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 lieu of an orphaned drug strategy, Canada builds considerations for rare disease treatments into the existing negotiation scheme. Rare Diseases In recent years, great strides in research have been achieved in the field of rare diseases in Canada and internationally. It affects approximately 1 in 100,000 people in the general population, and 1 in 855 people in the Ashkenazi Jewish community. Panel Discussion: What are the indications for the extension of the proposed therapy for the specific (sub)population? CORD works with governments, researchers, clinicians and industry to promote research, diagnosis, treatment and services for all rare disorders in Canada. 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. 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. 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. The panel will consider recommendations to “unblock” barriers that should be built into the operations of "Canada’s Rare Drug Agency 2022.” In February 2019, the Canadian government committed $1 billion to a national Rare Disease Drug Strategy to be put in place in 2022. RPI Deficiency. Comprehensive national framework integrated with a Canadian Rare Disease Strategy Canada is also engaged in E-Rare, the European Union's main instrument for funding research in areas related to rare diseases. Although definitions vary by jurisdiction, diseases that affect approximately 1 in 2000 people are considered rare. Webinar 4 in CORD’s Rare Drug Strategy Consultation. 151 Bloor Street West, Suite 600 Realistic, feasible, sustainable and beneficial for all CanadiansSupported by allReady to go on January 1, 2022 “[The drug-pricing system] was never really designed for innovative therapies,” says Wong-Rieger. In addition, we are asking opinions about how specialty drugs should be available in a National Pharmacare program. 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. Sign the Petition: Help Kaysen access Zolgensma a life changing gene therapy for treatment of SMA before his 2nd birthday. New Rare Diseases jobs added daily. All rights reserved.GZCA.XLSD.18.12.0131 February 2019. In Canada, patients with rare diseases face the same issues as elsewhere, but with a few added complications. Toronto, Ontario M5S 1S4 Canada, Tel: (416) 969-7464 / 1-877-302-7273 “This is a major win for rare disease patients and their families that will help protect them from unexpected medical bills. In addition they held their annual conference and awards gala celebration. How Other Countries Provide Access to Rare Disease Drugs: What Canada can Learn … or Not Webinar 7: January 29 @ 11 am – 12 pm EST Please consult your healthcare professional if you have any questions about your health or treatment.​, Copyright © 2020. The provisions to end surprise medical billing were included in bipartisan legislation providing additional COVID-19 relief and to fund … Designing Pan-Canadian Rare Drug Framework Canadian Organization for Rare Disorders For Canada’s rare disease community, we have been offered the opportunity of a lifetime! Webinar 4 Slides: https://bit.ly/3pLQTEc Cronkhite-Canada syndrome (CCS) is an extremely rare disease characterized by various intestinal polyps, loss of taste, hair loss, and nail growth problems. 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. Welcome to 2nd Webinar in the 2021 CORD Consultations on the Pan-Canadian Rare Drug Framework. 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. This comprehensive strategy was released by CORD in May 2015 following a year of multi-stakeholder deliberations. Roundtable Panelists: This initiative enables scientists in different countries to collaborate on a common interdisciplinary research project, with a clear translational approach. Awareness: There is no known awareness day or organization for this condition. 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. Virtual Many thanks to all who contributed to our previous Crowdsourcing Webinar. Support & Resources Our top priority is meeting the needs of the rare disease community. Consultation Plan: Six Webinars addressing fundamental Issues: Progressive Multifocal Leukoencephalopathy (PML) Progressive multifocal leukoencephalopathy … Recordati Rare Diseases Canada Inc. 3080 Yonge Street, Suite 6060, Toronto, Ontario - Canada M4N 3N1. Fabry disease is a rare genetic disease that can be passed on from parents to their children. 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. Many thanks to our partners: Health information contained herein is provided for general educational purposes only. That amounts to 3 million people, and two-thirds are children. Why did Canada develop and never implement a Canadian Rare Disease Strategy and what has been the aftermath? RT @kc4ktaf: It’s Friday Follow! What was the rationale for Orphan Drug legislation in other countries? NEW Program! In February 2019, the Canadian government committed $1 billion to a national Rare Disease Drug Strategy to be put in place in 2022. What has been the experience with this therapy and what are the impacts on patients? An open letter to Canada's provincial minister of health for access to Zolgensma. 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. Webinar 3 Video Recording: https://bit.ly/3pNzTgUWebinar 3 Slides: https://bit.ly/3k4CMG5Presentation: 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. NEW Start date is October 2, 2020! Patient Partners Leadership Training. For Canada’s rare disease community, we have been offered the opportunity of a lifetime! Register Now! What are the challenges to accessing these preventive therapies through public and private drug plans? Our Goals MONITOR. 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. 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 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. A Canadian research-based pharmaceutical company. … Presentation:  The only national organization representing all rare disorder patient groups in Canada. It is difficult to treat because of malabsorption that accompanies the polyps. What has been the impact of those legislations? According to the Canadian Organization for Rare Diseases (CORD), rare diseases affect one in 12 Canadians (two Fax: (416) 969-7420 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). Together, people with rare diseases are not so rare. Webinar 1 Video Recording: https://bit.ly/2G1s45gWebinar 1 Slides: https://bit.ly/3mlVqdU Webinar 2 (Oct 23, 2020): How can we access preventive and risk-reduction therapies: examples from rare blood disorders. Webinar 1 (Oct 9, 2020): Does Canada need an Orphan Drug Policy to incentivize drug development and submissions? Apply Now. Browse the GARD list of rare diseases and related terms to find topics of interest to you. 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 … But time is short. Unlike other countries, Canada has not agreed on a common definition of rare diseases. The Canadian Organization for Rare Disorders (CORD) has released Canada’s Rare Disease Strategy, developed with experts from every sector. 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 ultimate goal is to achieve consensus on a national Rare Disease Drug Strategy that is: 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. Health information contained herein is provided for general educational purposes only. NEW TOPIC: Leave No One Behind: Rarest Among the Rare (Webinar 5 Registration: https://bit.ly/3j1Rf4W) Access to Specialty Drugs Under Pharmacare Survey. Gaucher disease is a rare, inherited metabolic condition, and the most common of a family of rare diseases known as lysosomal storage disorders (LSDs). Kevin Kuo (University Health Network)Dr. Katerina Pavenski (St. Michael’s Hospital)David Page (Canadian Hemophilia Society)aTTP, Thalassemia, Hemophilia, Patient Panel 10 Rare Autoimmune Diseases. Symptoms can mimic other diseases. Symptoms can mimic other diseases. If you suspect Gaucher disease, get tested. Symptoms can include severe pain in the hands and feet, red spots (angiokeratoma) on the midsection, and ringing in the ears. Pompe disease is a neuromuscular disorder that causes progressive muscle weakness and the loss of muscle tissue. Consultation Registration This survey is being conducted to learn from Canadians their experiences accessing specialty drugs. A rare disease is any disease that affects a small percentage of the population. Join us and become part of an active Canadian rare disorder community. Focus on: • Unituxin and Irinotecan, Temozolomide with GM-CSF for refractory and relapsed neuroblastoma • Kalydeco for pediatric cystic fibrosis patients carrying the mutation R117H PMPRB: Friend or Foe of Rare Disease Drug Strategy. Webinar 4: (Nov 20, 2020). For patient-based organizations, support groups or other charitable groups. 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. There are thousands of rare diseases—more than 6,800, according to the National Human Genome Research Institute. Patients with MPS I disease can experience a wide range of symptoms with variable degrees of severity. 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. Webinar 5: Dec 4 @ 12 pm ET. Your healthcare professional is the single best source of information regarding your health. What is the Definition of a Rare Disease? It’s our first priority to help protect the safety and lives of patients around the world. What are alternative treatments and how effective are these? A Canadian research-based pharmaceutical company. Panelists Please consult your healthcare professional if you have any questions about your health or treatment. In February 2019, the Canadian government committed $1 billion to a national Rare Disease Drug Strategy to be put in place in 2022. Inclusion on this list does not serve as official recognition by the NIH that a disease is rare. 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 note that CORD does not provide any clinical services and staff are not able to assist in making a diagnosis or referrals. Copyright © 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. What are possible ways forward to making this therapy available? 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. 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. CORD CADTH Consultation Feedback--Aligned Process. Do you know of any events not listed here? Cross Canada Consultation Dates Kimberly Robinson, Director, Pricing & Market Access, PDCIKim Steele, Director, Government and Community Relations, Cystic Fibrosis CanadaDr. Canada. In the United States, a rare disease is defined as one that affects fewer than 200,000 people. What are the benefits of therapies that can prevent or reduce the risk of serious symptomology in rare diseases? For the already vulnerable rare disease community, nearly 3 million people in Canada alone, the COVID-19 pandemic has presented a unique set of challenges. Why are these therapies especially relevant in the time of a pandemic? If you suspect Gaucher disease, get tested. The prevalence of a rare disease usually is an estimate and may change over time. For Durhane Wong-Rieger, the President & CEO of the Canadian Organization for Rare Disorders, this is a mistake. Recordati Rare Diseases is part of the rare diseases business within the Recordati Group, and is dedicated to developing innovative, high-impact therapies. They serve as our top priority and sit at the centre of everything we do Minister Hajdu, PMPRB: Your drug pricing approach is DEAD Wrong for Patients! 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.” A data lake for rare disease research.. Canada has a lot of lakes, more than every other country in the world combined, in fact. If you suspect MPS I disease, get tested. All rights reserved.GZCA.XLSD.18.12.0131 February 2019.​, 1 in 12 Canadians are living with a rare disease. 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. 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. GARD maintains a list of rare diseases and related terms to help people find reliable information. What are challenges in approval of these therapies through HTA process? ​ We all play a part in early diagnosis. General Information: info@raredisorders.ca. 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.