Canada's Progress Toward an Orphan Drug Framework: 2014

摘要

The following papers have been brought together in a mini-symposium describing the challenges facing Canadian patients, academic institutions, clinicians, and regulators in the development of an improved process for clinical investigation of orphan drugs. Commitment to creation of a national orphan drug framework was announced by the federal Minister of Health on October 3, 2012, triggering an active discussion that continued throughout 2013. A process of early engagement with key stakeholders included two meetings bringing together researchers, clinicians, regulators, interested organizations, and patient/family representatives. These took place in Montreal in February 2013 with a focus on rare genetic disorders and in Ottawa in May 2013 with a focus on oncology.