Author
Dr. Nigel SB Rawson, Ph.D.
Introduction
In an analysis of Common Drug Review (CDR) recommendations for 33 drugs published between September 2003 and December 2005, all six drugs for rare disorders received a negative recommendation. A decade later, it is appropriate to review whether any change has occurred in the CDR’s evaluation of drugs for rare disorders.
Data and Methods
All submissions with final recommendations for first applications for new indications available on the CDR website at March 31, 2015 were accessed. Information about the date, indication, disease rarity, and recommendation was recorded. For rare disorder drugs, reasons for the final recommendation were examined.
Results
Of 250 final recommendations made between May 2004 and March 2015, 47.6% were negative. However, the negative recommendation rate for the 29 drugs for rare disorders was 65.5%, mainly due to a lack of clinically relevant outcome information and insufficient evidence of cost-effectiveness or significant clinical benefit.
Conclusion
Although the CDR’s overall negative recommendation rate has decreased slightly in recent years, it remains much higher for rare disorder drugs. The CDR review continues to be focused on requiring large randomized clinical trials with hard outcomes to provide evidence for clinical benefit. Most negative recommendations for rare disorder drugs seemed to place greater emphasis on their high cost, rather than their efficacy. Federal and provincial governments need to implement the Orphan Drug Regulatory Framework and coherent nationwide policies to ensure that drugs for rare disorders are funded quickly and equitably so that adequate healthcare is available to all Canadians that need these treatments.
