Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand

Table 2 Positive orphan drug recommendation rates for different ICUR threshold values

Cost-utility threshold	Number of submissions with cost-utility values in this range (N)^a	Positive recommendation rate (%)
≤ $50,000/QALY^b	6^c	66.7%
$50,000 - $100,000/QALY	11^d	72.7%
> $100,000/QALY^e	16	62.5%
Prior to 2016	7	42.9%
2016	9	77.8%^f

^aFor submissions with a range of values provided in the CDR recommendation, the lower value of the range was used for this analysis
^bAbbreviations are: QALY quality adjusted life-year, NHL Non-Hodgkin’s Lymphoma, MM Multiple Myeloma, ICUR incremental cost-utility ratio
^cIncludes deferiprone (iron overload) which was dominant and stiripentol (Dravet Syndrome) where ccost-utility ratio was $50,122/QALY
^dRatios for plerixafor (NHL (< $50,000/QALY) and MM ($50,000–$100,000/QALY)) and sunitinib (metastatic renal cell carcinoma and gastrointestinal stromal tumor both in the $50,000–$100,000/QALY category) are included twice in the calculations
^eThis category also includes drugs with a “confidential” recommendation (n = 2) based on the assumption that their ICURs were > $100,000/QALY
^fAll of the positive recommendations in this category had a conditional price reduction

ISSN: 1750-1172