The EU Regulation on medicines for rare diseases (‘orphan medicines’) and the Paediatric Medicines Regulation especially for children are intertwined and partly complementary as they both intend to redirect investment in previously neglected therapeutic areas through a scheme of incentives, rewards and obligations. The scope of this evaluation will be focused on the output and results of the two regulations: in what respect have patients’ needs been fulfilled, what have been the societal consequences and what has been the synergy between the two. It will also focus on the cost-effectiveness when providing the incentives and rewards incorporated in the legislation and how they have been used in practice; it will make a cost-benefit analysis, both from a general point of view and per group (patients, industry, payers etc).
Deadline: 8 January 2018
Website: http://ec.europa.eu/