Is our therapeutic innovation system in sync with public health needs? Many factors affect the health of individuals and communities. Global, socioeconomic determinants such as income and social status, education, families and communities, food and nutrition, environment, life style, and health care services have led to an epidemiological transition, leading to reduced fertility, lower mortality and ageing of populations OECD, 2016a). Despite significant advances in basic science and technology the vast majority of diseases can neither be prevented nor treated; rare diseases constitute a large part of these (Institute of Medicine, 2010). It has been argued that the current system of therapeutic innovation is not adequate to follow global health, societal and economic trends (Barker, 2016; OECD, 2015b). With a focus on rare diseases, this policy profile discusses policy measures in order to stimulate therapeutic innovation in areas of unmet medical needs.