An Overview About Rare Diseases in Saudi Arabia and Reimbursement of Orphan Drugs

Abstract

Due to the high cost of specialty drugs and the increasing pressure on health care budget, the use of economic evaluation in reimbursement decisions has become a necessity. Although economic evaluation methods have been established internationally, doubts have been raised about their use in orphan drugs. In the absence of Health Technology Assessment (HTA) agencies in the Arab world, it is almost impossible to make conscious decisions in regard funding drugs used not only in rare conditions but in all disease areas. The use of economic evaluation is increasingly growing and comparable to regulatory bodies role especially in the context of the current national transformation program 2020 and the Saudi vision 2030. One of the vision objectives is to optimize the utilization of available resources and the efficiency of government spending, which is going to be impossible to achieve with the current practice. Challenges accompanied by reimbursement decisions are and always will be a limitation in the absence of a proper framework that addresses limitation with the use of traditional methods in assessing the value of orphan and non-orphan drugs. This paper mentions the prevalence of rare conditions in Saudi Arabia, discusses the current reimbursements methods and policies in other countries, explore challenges with the use of typical methods of economic evaluation used in different countries, and provide a potential solution to tackle some of the limitations in funding rare conditions.