A Review of recently Approved Orphan Designation Biological Drugs /

Abstract

Orphan Drugs are a critical resource for those suffering from rare diseases and there is an urgent need for many new drugs to address the thousands of serious rare conditions which affect millions of people across the world. The global industry of Orphan Biological Drugs has displayed some growth in recent years, however the number of drugs being approved each year by the FDA and the EMA regulatory authorities remains quite low, with an average of 2 to 3 biologic Orphan Drugs being approved each year. The regulatory authorities provide incentives to manufacturing companies to research and develop Orphan Drugs, which will often have a small target population. These incentives are crucial to ensure sustained and improved growth in this area. Many Orphan Biologics are developed and marketed by global multi-national pharmaceutical and biotech companies, after initially being researched by smaller specialist biotechnology companies. The cost of some Orphan Biologics can be very high, and at times prohibitive for both patients and health providers. This cost can be explained very often by the high cost of drug research and discovery and the higher risk associated with bringing Orphan Drugs to market. The aim of this thesis is to determine and review the numbers of monoclonal antibodies and other biological products obtaining Orphan Drug approval in both the EU and the US in recent years and the types of companies obtaining approval for these types of drugs. This thesis also looks at costs associated with Biological Orphan Drugs.