Increasing pressures on health care budgets have led to a growing interest in the use of economic evaluation in reimbursement decisions for drugs and other health technologies. This has a great impact in the access of orphan drugs by rare disease patients.
In this scenario, insurance companies are starting to assess the value for money by comparing the incremental costs of the drug (compared to existing ones) with the incremental benefits, which is defined in terms of health gain.
How is this suitable for orphan drugs reimbursement evaluation if most of the times there’s no incremental costs for those?
Given the lack of cost-effectiveness for orphan drugs, funding it can only be justified if the patient population is willing to give up some of the overall health gain produced by the health care system. Considering that there is an urgent need to harmonize incentives for research with the potential for market access, it’s essential to be clearer on what, if anything, society is willing to pay for these treatments.
Reimbursement is a key issue for the continuous development of orphan drugs and their access by rare disease patients. Reason why, the program of next year’s World Orphan Drug Congress USA will be mainly focused on this matter.
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