CORD has partnered with the Economic Club of Canada (ECC) to present Canada’s Rare Disease Strategy in Toronto on September 24, 2015 and other cities are planned across Canada.
Location and Date:
Rare Diseases: A Public Health Issue
In just a few years, Canada has emerged from a state of “not needing an Orphan Drug Policy” to becoming an acknowledged global leader in rare diseases, especially in research and patient advocacy. Which makes now a very good time to focus our efforts toward implementing Canada’s Rare Disease Strategy.
Admittedly, Canada has been late to embrace rare diseases. It was just five years ago that CORD pointed out that Canadians had access to only half of the orphan drugs approved in the USA and/or Europe. Approved drugs were routinely rejected for coverage by the public drug plans. Even today, there remains a lag of two to seven years for patients to get access. Similarly, over the past 30 years, while the USA, Europe, and Japan have invested hundreds of millions of dollars in rare disease research, Canada did not target research funding for rare diseases until 2012. But things are changing rapidly, with Canada’s Orphan Drug Regulatory Framework due to be implemented imminently and Canadian research sites now among the leaders in international collaborative projects.
Similarly, in 2009 when the European Union called for the development of national rare disease plans, almost all member states responded, as have countries outside the EU, such as Mexico and Peru. Integral to the national plans are Centres of Reference, for which the EU will issue a call for proposals in fall 2015.
Meanwhile in Canada, we have pockets of rare disease expertise, but most are neither linked to general practitioners and paediatricians nor formally to one another. Hence the need for the coalescing force of Canada’s Rare Disease Strategy and the call to collaborate on a five-point action plan so that collectively we can:
A key to success in Europe has been endorsement of “solidarity” as a fundamental value and “equal access” as a guiding principle for all patients regardless of what they suffer from or where they live. Rare diseases are not limited to a small population but are regarded as a public health issue affecting everyone.
Intended Audience: Senior Leaders from business, government industry, academic and media organizations.
Media Coverage: The dialogues are intended to be a high profile event with extensive media coverage. CPAC will be invited, as will leading health reporters and advocacy groups from across the country.
Moderated by: The Honourable Fred Horne, Former Minister of Health, Alberta
Register Now: Canada's Most Effective Patient Training Event. Canadian Expert Patients in Health Technology Conference in Toronto
Date: November 7 - 8 Location: Hyatt Regency Toronto, 370 King St W, Toronto, ON REGISTER HERE Statement of Need We are living in a rapidly evolving healthcare environment that offers significant opportunities and challenges to stakeholders. In particular, the development and availability of innovative health technologies, including therapies, devices, and medical procedures, are transforming once fatal diseases to chronic conditions, reducing the impact of physical and mental challenges, and significantly improving quality of life for patients and families. Patient advocates and patient organizations are playing key roles in the design and development of new therapies, and nowhere is this more evident than in the area of rare diseases where one third to one half of the innovative or breakthrough therapies are designated as orphan drugs. Public health programmes and private insurers are raising concerns about rising healthcare costs and budget impacts. Indeed, we are reaching a critical juncture (perfect storm) with innovations plus incremental improvements in current products meeting up with increasing demand from a population that is aging with more complex illnesses, aware of options, and desirous of having a say in healthcare decision making. In all sectors, patient-centred care and patient engagement are increasingly recognized as not only desirable but also essential to health policy and decision-making. However, not all patients are adequately represented or adequately prepared to participate in all phases of drug development and access, and indeed the opportunities for engagement are perhaps exceeding the capacity of the patient community to take fulsome advantage. Patient groups range from large, well-resourced multi-stakeholder not-for-profit or charitable organizations to small, family-based, single-cause, volunteer-run groups that may not be officially registered. The latter are especially predominant in the rare disease space. *Draft Agenda* Objectives Topics 1. Canada’s Strategy for Rare Diseases • Update on 5 Pillars: Diagnosis and Prevention, Expert Care, Community Support, Access to Therapies, Research • Progress on Ontario’s Strategy for Rare Diseases • Centres of Expertise: Platform for diagnosis, patient registries and databases, best practice care and management of drug therapies, coordination of community-based support, research initiatives 2. New Health Technologies • Emerging technologies in diagnostics (newborn screening, genome sequencing, “human phenotype ontology” • Orphan drug pipeline • Advances in transplantation therapy: bone marrow and stem cell transplant, gene modifications • Developments in precision/personalized therapies • Biosimilars • Repurposing drugs 3. Current Issues in Drug Access • Special Access Programme and Advanced Access Programmes • Managed access (aka Coverage with Evidence Development), Risk Sharing, Post-Market Monitoring • Proposed FPT Access to (Expensive) Drug for Rare Disorders • Engaging with pCPA • New Guideline from PMPRB • Private Drug Insurance 4. National Pharmacare • Convergence or Disparity: Comparison of Federal, Provincial, and Territorial Drug Coverage Programmes • What was Heard: Feedback from House of Commons Standing Committee on Health • Building Blocks of National Pharmacare Roadmap to Excellence in Drug Access • Roadmap to Excellence in Drug Access Educate: Inform patients and patient advocates about innovations in health technologies and emerging issues that affect access to therapies, diagnostics, and devices for rare diseases and beyond Enquire: Consult with patients, patient advocates, and patient organizations to ensure that the patient perspective is central in health policy and health-related decision making at all levels from individual to systems Empower: Provide training, support, and resources to increase capacity of patient organizations and patient advocates to advocate effectively on behalf of themselves and other patients at all levels of healthcare decision making Engage: Actively facilitate and provide channels and tools for patients and patient advocates to meaningfully engage with all other stakeholders in all processes of the development, review, use, and monitoring of health technologies and related services CORD is offering a small travel bursary to patient group representatives to help offset the cost of travel. Apply at here: https://www.surveymonkey.com/r/2016expertpatientapplication REGISTER HERE