Abstract
BackgroundSince the introduction of the orphan drugs legislation in Europe, it has been suggested that the general method of assessing drugs for reimbursement is not necessarily suitable for orphan drugs. The National Institute for Health and Clinical Excellence indicated that several criteria other than cost and efficacy could be considered in reimbursement decisions for orphan drugs. This study sought to explore the multi-criteria decision analysis (MCDA) framework proposed by (Orphanet J Rare Dis 7:74, 2012) to a range of orphan drugs, with a view to comparing the aggregate scores to the average annual cost per patient for each product, and thus establishing the merit of MCDA as a tool for assessing the value of orphan drugs in relation to their pricings.MethodsAn MCDA framework was developed using the nine criteria proposed by (Orphanet J Rare Dis 7:74, 2012) for the evaluation of orphan drugs, using the suggested numerical scoring system on a scale of 1 to 3 for each criterion. Correlations between the average annual cost of the drugs and aggregate MCDA scores were tested and plotted graphically. Different weightings for each of the attributes were also tested. A further analysis was conducted to test the impact of including the drug cost as an attribute in the aggregate index scores.ResultsIn the drugs studied, the R2, that statistically measures how close the data are to the fitted regression line was 0.79 suggesting a strong correlation between the drug scores and the average annual cost per patient.ConclusionDespite several limitations of the proposed model, this quantitative study provided insight into using MCDA and its relationship to the average annual costs of the products.
Highlights
Since the introduction of the orphan drugs legislation in Europe, it has been suggested that the general method of assessing drugs for reimbursement is not necessarily suitable for orphan drugs
This might be because the proposed framework may have been developed from a pharmaceutical company perspective, since it is unlikely that a Health technology assessment (HTA) body would consider Manufacturing complexity and the Unique indication or not for which a drug is licensed as relevant criteria when assessing orphan drugs for reimbursement decisions
We suggest that future multi-criteria decision analysis (MCDA) frameworks take Incremental cost-effectiveness ratio (ICER) into account as an external “validation”
Summary
Since the introduction of the orphan drugs legislation in Europe, it has been suggested that the general method of assessing drugs for reimbursement is not necessarily suitable for orphan drugs. The National Institute for Health and Clinical Excellence indicated that several criteria other than cost and efficacy could be considered in reimbursement decisions for orphan drugs. Concern has been raised that because of their costs, in addition to the frequent lack of suitable comparators, and the difficulties of demonstrating robust efficacy in small patient populations, orphan drugs are not deemed (robustly) cost-effective under the standard methods of HTA [1, 3]. This potentially results in patients not having access to potentially valuable treatments [7]
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