Orphan Drugs for Rare Diseases: Grounds for Special Status

Strategy, Management and Health Policy Enabling Technology, Genomics, Proteomics Preclinical Research Preclinical Development Toxicology, Formulation Drug Delivery, Pharmacokinetics Clinical Development Phases I-III Regulatory, Quality, Manufacturing Postmarketing Phase IV In the European Union, rare diseases are defined as life-threatening or chronically debilitating diseases that occur at such a low prevalence that special initiatives are needed to address them. There is a continuing debate about the special registration, pricing, and reimbursement status that orphan drugs enjoy in many countries. Therefore, the aim of this commentary is to examine the arguments that argue in favor of granting special status to orphan drugs. We conclude that monetary arguments must be carefully balanced with social and ethical motives to avoid unfair treatment of patients with a rare disease. Also, we provide an overview of how the special status of orphan drugs can be put into practice with respect to registration, pricing and reimbursement. Adopting this special status in practice is possible, but not straightforward. Hence we propose a number of policy recommendations, such as risk sharing arrangements, to take account of the special status of orphan drugs.