Regulatory Approach To Orphan Medicines In The United States And EU

Abstract

Introduction : Rare diseases represent a growing significant public health problem and a challenge for health systems. For most diseases no treatment has been developed yet. Due to the lack of economic profitability, the pharmaceutical industry often refrains from investing time and money in the development of orphan drugs. There is a special legislation with main purpose to stimulate the research, production and marketing of these medicines. The most frequently cited regulations are those of the Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The aim of this publication is to produce a comparative analysis of regulatory approaches to the authorization of orphan drugs between the European Union (EU) and the USA and to identify common trends and benefits for faster access to patients therapies. Materials and Methods : A literature review and a SWOT analysis were conducted of current regulatory practices, availability, accessibility and reimbursement in the USA and the EU. The databases Medline, PubMed and EMBASE were searched with the keywords “rare diseases, orphan drugs, orphan drug regulation, orphan drug designation, orphan drug legislation, orphan drug indication, Orphan Drug Act, EU orphan drug statistics”. Results : The detailed comparative analysis by the EMA and the FDA highlights a number of similarities and differences between the incentives provided to pharmaceutical companies. Both regulatory agencies provide: shortened authorization periods; exceptions to the payment of certain fees; assistance under the protocol and the development process and financial relief; facilitation in cases of concurrent approval of orphan drugs. Differences are found in cases of market exclusivity, tax incentives and subsidies granted. Conclusion : In recent years, the EU and the USA have worked together to regulate orphan drugs, as well as in areas such as information exchange and standardization of marketing authorizations for the benefit of millions of patients on both sides of the ocean suffering from rare diseases.