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 1 Summary of CDR^a recommendations for orphan drugs

Category	Number	Positive recommendations (n (%))^b
Total number of submissions	68	43 (63.2%)
Recommendations based on clinical parameters only (n (%))^c	33 (48.5%)	15 (45.5%)
Recommendations based on clinical/price parameters (n (%))^c	30 (44.1%)	26 (86.7%)
Recommendations based on price parameters only (n (%))^c	5 (7.4%)	2 (40.0%)
Submissions over different time periods:
2004–2009	22 (32.4%)	11 (50.0%)
Number of positive recommendations with a conditional price reduction^e		0 (0.0%)^d
2010–2011	7 (10.3%)	3 (42.9%)
Number of positive recommendations with a conditional price reduction^e		2 (66.7%)^d
2012–2013	12 (17.6%)	8 (66.7%)
Number of positive recommendations with a conditional price reduction^e		2 (25.0%)^d
2014–2015	12 (17.6%)	8 (66.7%)
Number of positive recommendations with a conditional price reduction^e		6 (75.0%)^d
2016	15 (22.1%)	13 (86.7%)
Number of positive recommendations with a conditional price reduction^e		11 (84.6%)^d
Recommendations with no ICUR (n(%))^f	36 (52.9%)	21 (58.3%)
Recommendations with an ICUR (n(%))^g	32 (39.2%)	22 (68.8%)
Recommendations based on a reconsideration	25 (36.8%)	8 (32.0%)

^aAbbreviations are: CDR Common Drug Review, ICUR incremental cost-utility ratio, QALY quality adjusted life-year
^bRefers to a recommendation for listing regardless of the specific type of listing; percentage is based on the total number of reviews in each category
^cCategories based on the sequence of factors listed in the reasons for recommendation in CDR reviews (see Methods)
^dPercent based on the total number of positive recommendations over each time period
^eConditions of recommendation include a substantial reduction in price or that price should not exceed the cost of a comparator therapy on drug plan formularies
^fIncludes recommendations which provided a cost per life year gained (n = 2) but excludes recommendations where the manufacturer requested that the ratio remain confidential (n = 2)
^gIncludes submissions where manufacturers requested that ICUR remain confidential (n = 2) but excludes assessment of cost/life year gained (n = 2)

ISSN: 1750-1172