药物经济学：开放获取

Pharmacoeconomic Considerations in the Management of Rare Diseases: Challenges and Opportunities

Abstract

Anne Zemella

Pharmacoeconomic considerations play a pivotal role in the management of rare diseases, presenting a complex interplay of factors that challenge traditional healthcare economics. Rare diseases often referred to as orphan diseases, affect a small percentage of the population, typically fewer than 200,000 individuals in the United States or fewer than 1 in 2,000 people in Europe. Despite their low prevalence, these diseases collectively impact millions worldwide. Managing rare diseases involves unique challenges, including limited treatment options, high costs, and difficulties in conducting clinical trials due to small patient populations. One of the primary challenges in managing rare diseases is the high cost of treatment. Pharmaceutical companies invest significant resources in research and development to bring therapies to market for these conditions. As a result, drugs targeting rare diseases often come with a hefty price tag. The high cost of these treatments can pose substantial financial burdens on healthcare systems, payers, and patients alike. Additionally, the limited patient population means that the costs cannot be distributed across a large number of individuals, further exacerbating the economic challenge.