Canadian Organization of Rare Diseases has proposed a strategy for Canada which encompasses a five-point action plan that will address unnecessary delays in testing, wrong diagnoses and missed opportunities to treat. This strategy is the result of cross-Canada consultations and contributions from a wide range of stakeholders, including governments, researchers, individual patient organizations, policy experts and others.
First, in terms of timing and priority, the strategy recommends the implementation of the federal Orphan Drug Regulatory Framework. Second, along with the regulatory framework the strategy emphasizes the need for a tailored evaluation and funding approach to ensure timely and equitable patient access to orphan drugs.
Third, a key-integrating factor is the Center of Excellence, to generate and support research and patient care, which could be linked through a new Canadian Partnership for Rare Diseases or reference network. Fourth, provide dedicated and increased research funding for rare diseases, potential through Public Private Partnerships. At the same time, allocated resources for patient organizations and ORPHANET Canada is one of the few developed countries that do not provide some support for patient groups, to September 14, 2015 4 contribute to the health system, to support the work with their patient community, and to do outreach and awareness
And finally, adopt national program for Newborn Screening with clear guidelines for adding new diseases based on evidence and international best practices (USA) and implement state of the art DNA and genomic screening with “core sites” and national outreach. CORD is working with stakeholders to take action immediately, and many are already doing so. They are also planning to take this on the road across the country in order to build awareness, gain feedback and earn the support of other health system stakeholders.