Orphan medicines and therapeutic innovation

Trabalho Final de Mestrado Integrado, Ciências Farmacêuticas, Universidade de Lisboa, Faculdade de Farmácia, 2014Rare diseases are by definition a disease that affects a small amount of people. However, adding up all the small number of rare diseases, the total number of patients will become significant, affecting millions of people around the world. The lack of interest in the development of orphan medicines and the patients' urgent medical need led the government and agencies to take action in order to promote and incentive the development of orphan medicines firstly in 1983 by the Food and Drug Administration and then followed by the European Union and several countries around the globe. These incentives not only enabled a growing knowledge pool on rare diseases but also the non rare diseases. During the last decade the concern about orphan medicines development has steadily increased to pharmaceutical and biopharmaceutical industry. This growth had obviously to do with the incentives, but also because industry saw the great opportunity that this underdeveloped market was and the vast income that could result from it. However, the access to these medicines is not equal to all the rare diseases' patients within different countries around the world and this is still a struggle that agencies are trying to find the best way to overcome. Therefore, most countries are trying to find a balance between the sustainability of the National Health Services and the equitable access of orphan medicines to patients with rare diseases