Is the current approach to reviewing new drugs condemning the victims of rare diseases to death?
Joe T.R. Clarke Joe Clarke is at the Department of Pediatrics, The Hospital for Sick Children and University of Toronto, Toronto, Ont. Correspondence to: Dr. Joe T.R. Clarke, Division of Clinical and Metabolic Genetics, Hospital for Sick Children, 555 University Ave., Toronto ON M5G 1X8; fax 416 813-5345; jtrc{at}sickkids.ca’ + u + ‘@’ + d + ”//–> Powerful financial incentives created by orphan drug legislation, particularly in the United States,1have resulted in the emergence of new, innovative therapies for a number of rare diseases. In Canada, approval for the commercial sale of new drugs is controlled by the Therapeutic Products Directorate of Health Canada, which adjudicates their safety and efficacy on the basis of information provided by the manufacturers. However, many of the new therapies are so expensive that, without financial support from provincial drug plans, access to them is a practical impossibility. For example, the annual cost of the treatment of Fabry disease with
Joe T.R. Clarke Joe Clarke is at the Department of Pediatrics, The Hospital for Sick Children and University of Toronto, Toronto, Ont. Correspondence to: Dr. Joe T.R. Clarke, Division of Clinical and Metabolic Genetics, Hospital for Sick Children, 555 University Ave., Toronto ON M5G 1X8; fax 416 813-5345; jtrc{at}sickkids.ca’ + u + ‘@’ + d + ”//–> Powerful financial incentives created by orphan drug legislation, particularly in the United States,1have resulted in the emergence of new, innovative therapies for a number of rare diseases. In Canada, approval for the commercial sale of new drugs is controlled by the Therapeutic Products Directorate of Health Canada, which adjudicates their safety and efficacy on the basis of information provided by the manufacturers. However, many of the new therapies are so expensive that, without financial support from provincial drug plans, access to them is a practical impossibility. For example, the annual cost of the treatment of Fabry disease with
