Evaluating Canadians’ Values for Drug Coverage Decision Making

Abstract

Background: Decision makers are facing growing challenges in prioritizing drugs for reimbursement because of soaring drug costs and increasing pressures on financial resources. In addition to cost and effectiveness, payers are using other values to dictate which drugs are prioritized for funding, yet there are limited data on the Canadian public's priorities. Objectives: To measure the relative societal importance of values considered most relevant in informing drug reimbursement decisions in a representative sample of Canadians. Methods: An online survey of 2539 Canadians aged 19 years and older was performed in which 13 values used in drug funding prioritization were ranked and then weighted using an analytic hierarchy process. Results: Canadians value safe and efficacious drugs that have certainty of evidence. The values ranked in the top 5 by most of our subjects were potential effect on quality of life (65.4%), severity of the disease (62.6%), ability of drug to work (61.1%), safety (60.5%), and potential to extend life (49.4%). Values related to patient or disease characteristics such as rarity, socioeconomic status, and health and lifestyle choices held the lowest rankings and weights. Conclusions: Canadians value, above all, treatment-related factors (eg, efficacy and safety) and disease-related factors (eg, severity and equity). Decision makers are currently using additional justifications to prioritize drugs for reimbursement, such as rarity and unmet need, which were not found to be highly valued by Canadians. Decision makers should integrate the public's values into a Canadian reimbursement framework for prioritization of drugs competing for limited funds.

A systematic review of moral reasons on orphan drug reimbursement

“…Opposing views in this category concluded, for instance, that special status for reimbursement of OMPs was incompatible with the egalitarian principle to treat each individual equally [ 35 , 36 , 39 , 42 , 47 , 52 , 62 , 70 , 75 , 78 , 81 , 85 – 88 , 98 , 100 , 107 , 110 , 114 , 132 , 137 , 142 , 145 , 149 – 156 ]. On the other hand, an extensive body of literature argued that a special status was justified for reasons of equity [ 8 , 35 , 39 , 46 , 54 , 66 , 68 , 73 , 81 , 85 , 94 , 107 , 116 , 117 , 121 , 122 , 128 , 136 , 137 , 152 , 153 , 157 – 164 ]. Reasons related to this equity principle included: First, that rare disease patients should have the same access to treatments as all other patients should and that this was only possible by providing OMPs even if they were expensive [ 5 , 25 , 37 , 38 , 40 , 42 – 44 , 51 , …”

“…(2) Another feature justifying special status for the reimbursement of OMPs were unmet needs . Authors argued that in the case of unmet needs, it was unfair not to cover those treatments [ 24 , 36 , 57 , 69 , 75 , 77 , 79 , 87 , 89 , 99 , 102 , 114 , 116 , 122 , 129 , 130 , 136 , 178 , 184 , 189 , 202 , 203 , 205 , 208 , 220 – 224 ]. However, Sandman and Hofmann [ 184 ] found the concept of unmet needs unsustainable in this context and recommended instead considering disease severity.…”

“…However, Sandman and Hofmann [ 184 ] found the concept of unmet needs unsustainable in this context and recommended instead considering disease severity. (3) One extensively discussed reason (n = 68) related to unmet need was the lack of alternative treatment [ 8 , 24 , 37 , 44 – 46 , 51 , 54 , 56 , 60 , 62 , 64 , 70 , 75 , 77 – 79 , 83 , 84 , 87 , 96 , 111 , 112 , 114 – 116 , 122 , 123 , 128 – 130 , 135 , 139 , 141 , 142 , 147 , 150 – 152 , 161 – 163 , 167 , 174 , 177 , 178 , 184 , 193 , 199 – 203 , 206 , 208 , 210 – 215 , 217 , 219 , 223 , 225 – 228 ]: Since OMPs often represented the only hope for patients suffering from rare diseases, which justified their special status [ 70 ]. Relatedly, some studies also found a societal preference for covering drugs for diseases without treatment alternatives [ 45 , …”

“…These disadvantages include: the lack of drug development efforts before incentives were installed [ 97 , 117 , 173 , 175 , 229 ]; disadvantages in traditional cost-effectiveness procedures [ 43 ], as Quality-Adjusted Life Years (QALY) measures would disadvantage people with chronic disabilities [ 73 , 210 ]; the lack of public awareness; and difficulties in collecting effectiveness evidence for OMPs [ 165 ]. (5) A final justification for why rare disease patients were worse off is that OMPs were too expensive to be paid by patients themselves and therefore should be reimbursed [ 46 , 51 , 61 , 116 , 118 , 123 , 133 , 138 , 147 , 161 , 163 , 183 , 199 – 201 , 204 , 208 – 210 , 232 – 234 ]. It was also argued that rare disease patients already carried a substantial financial burden as compared to the general population [ 209 , 210 , 233 ], including indirect costs for transportation to specialized facilities or the inability of family caregivers to be in full-time employment [ 123 ].…”

“…In line with this argument, the reason given for special status of the reimbursement of OMPs was that rare disease patients were sick out of bad luck and could not be held responsible for their situation [ 47 , 137 , 177 , 231 ]. Rule of rescue A considerable body of literature (n = 48) discussed the controversial Rule of Rescue, which justifies the rescue of endangered lives of identifiable patients by moral intuition , no matter the cost [ 24 – 26 , 29 , 35 , 38 , 41 , 42 , 47 – 49 , 52 , 53 , 58 , 60 , 63 , 64 , 67 , 70 , 71 , 73 , 74 , 77 , 77 , 81 – 83 , 91 , 104 , 108 , 109 , 114 , 116 , 123 , 125 , 139 , 151 , 153 , 175 , 181 , 187 , 196 , 217 , 230 , 235 – 238 ]. Mass media coverage [ 151 , 236 – 238 ], the moral instincts of physicians [ 236 ] as well as society in general [ 175 , 217 , 237 ] were said to support the Rule of Rescue.…”