Limitations of the methods of HTA for decision to incorporate technologies for rare diseases

Abstract

 The increase in the budgets of health care has led various governments to use the methods of the Health Technology Assessment (HTA) to assist decisions regarding the reimbursement of medicines and other health technologies. The standard methods for Health Technology Assessment (HTA) are relevant in assessing allocative efficiency and budget impact within the context of prevalent and common diseases, but there is concern that these methods adequately reflect social preferences for the treatment of rare diseases. The objectives of this article are to analyze whether the standard methods of Health Technology Assessment (HTA) are suitable to aid decisions on orphan drugs; and discuss the social value of orphan drugs and issues surrounding its financing and use.