Health Economic Data in Reimbursement of New Medical Technologies: Importance of the Socio-Economic Burden as a Decision-Making Criterion

Abstract

Background: Assessment and appraisal of new medical technologies require a balance between the interests of different stakeholders. Final decision should take into account the societal value of new therapies.Objective: This perspective paper discusses the socio-economic burden of disease as a specific reimbursement decision-making criterion and calls for the inclusion of it as a counterbalance to the cost-effectiveness and budget impact criteria.Results/Conclusions: Socio-economic burden is a decision-making criterion, accounting for diseases, for which the assessed medical technology is indicated. This indicator is usually researched through cost-of-illness studies that systematically quantify the socio-economic burden of diseases on the individual and on the society. This is a very important consideration as it illustrates direct budgetary consequences of diseases in the health system and indirect costs associated with patient or carer productivity losses. By measuring and comparing the socio-economic burden of different diseases to society, health authorities and payers could benefit in optimizing priority setting and resource allocation. New medical technologies, especially innovative therapies, present an excellent case study for the inclusion of socio-economic burden in reimbursement decision-making. Assessment and appraisal have been greatly concentrated so far on cost-effectiveness and budget impact, marginalizing all other considerations. In this context, data on disease burden and inclusion of explicit criterion of socio-economic burden in reimbursement decision-making may be highly beneficial. Realizing the magnitude of the lost socio-economic contribution resulting from diseases in question could be a reasonable way for policy makers to accept a higher valuation of innovative therapies.

A systematic review of moral reasons on orphan drug reimbursement

“…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 , 57 , 60 , 61 , 63 , 67 , 70 – 72 , 78 – 81 , 87 – 89 , 93 , 95 , 96 , 100 , 101 , 103 , 105 , 106 , 112 , 115 , 118 , 126 – 129 , 139 , 140 , 142 , 151 ...…”

“… Appropriateness of cost-effectiveness criteria Some authors argued against special status, stating that OMPs could and should meet the same cost-effectiveness criteria as any other drug [ 38 , 45 , 53 , 62 , 73 , 78 , 82 , 85 , 90 , 104 , 105 , 111 , 114 , 127 , 134 , 135 , 142 , 150 , 194 ]. However, other authors mentioned that OMPs were unlikely to meet traditional cost-effectiveness criteria [ 24 , 35 , 36 , 38 , 41 , 43 , 45 , 54 , 57 , 59 , 62 , 64 , 65 , 68 , 72 , 73 , 75 , 87 , 89 , 109 , 116 , 118 , 120 , 122 , 128 , 140 , 143 , 144 , 151 – 153 , 155 – 158 , 162 , 178 , 196 , 199 – 201 , 206 , 208 , 220 ...…”

Improving Interactions Between Health Technology Assessment Bodies and Regulatory Agencies: A Systematic Review and Cross-Sectional Survey on Processes, Progress, Outcomes, and Challenges

“…Unlike regulators who may accept short-term or surrogate outcomes, payers usually prefer long-term clinical outcomes (3,7). Moreover, their assessment may involve a broader perspective such as the consideration of the potential social, legal, ethical, and political impacts of adopting the new technology (8,9). Given the differences in decision mandates of HTA/reimbursement bodies and regulatory agencies, their activities have been distinct from each other ( Table 1).…”

“…Unlike regulators who may accept short-term or surrogate outcomes, payers usually prefer long-term clinical outcomes ( 3 , 7 ). Moreover, their assessment may involve a broader perspective such as the consideration of the potential social, legal, ethical, and political impacts of adopting the new technology ( 8 , 9 ).…”

Multi-Criteria Decision Analysis for Assessment and Appraisal of Orphan Drugs

“…Assessment and appraisal of these medicinal products tend to be one of the most complicated HTA tasks. This assumption is particularly viable in regard to rare diseases and orphan drugs ( 10 – 13 ). Rare diseases represent a unique challenge to health authorities and payers, as they are life-threatening or chronically debilitating conditions with a low prevalence and a high level of complexity.…”