Why does reimbursement of orphan drugs require a specific approach? Review article

Abstract

The greatest problem concerning the reimbursement of orphan products is their high price. In the case of orphan drugs, reimbursement and pricing decisions are highly interrelated. In decisions concerning reimbursement of newly marketed health technologies, their cost-effectiveness or cost-utility should be taken into account. Rigorousness and thoroughness of evaluating an orphan drug prior to a reimbursement or pricing decision should depend on the cost of treatment and it should be stressed that for most orphan interventions costs of treatment depends mainly on the price of the orphan product. Low cost orphan drugs should be managed as all other drugs which do not generate high treatment costs. In the case of high and very high cost orphan drugs much more restrictive procedures should be applied in its assessment. Apparently, assessment of a very high cost orphan drug should be performed and appropriate analyses should be prepared by the applicant. Alternatively, the technology may be assessed by the HTA Agency itself. Appraisal should be performed by a separate advisory body, e.g. The Rare Diseases Team to the Minister of Health. The final decision should be made by the Minister of Health, who will bear political responsibility. In Poland appropriate state authorities have to undertake specific professional pricing activities with respect to orphan products in egalitarian approach and introduce risk sharing agreements by Law.