Orphan Medicinal Products (OMPs) are medicines which are developed to provide solutions for the diagnosis, prevention or treatment of rare diseases. A rare disease is defined in the EU legislation as a life-threatening or chronically debilitating condition affecting not more than five in 10,000 persons in the Community*.
Research and development of OMPs is more complex, costly and time-consuming than for traditional medicines, due to several factors, such as the small number of patients, chronicity or severity of the disease, the lack of scientific knowledge and the lack of alternative treatment**.
Apart from this, OMPs-specific regulation at EU level (N. 141/2000) also recognises that patients suffering from rare conditions deserve the same quality, safety and efficacy in medicinal products as other patients. This legislation therefore provides regulatory and economic incentives for pharmaceutical companies to recoup additional investments in R&D and to ensure that in the future patients suffering from rare conditions can benefit from advances in medical innovation.
Since most orphan medicines are implicitly biotech medicines (i.e. using recombinant DNA technology and with biological components), EuropaBio has represented the voice of the orphan medicines industry during the past fifteen years. Since 2014, EuropaBio and EFPIA jointly represent an alliance of over 45 member companies specifically committed to the development of OMPs and to improving the lives of patients with rare diseases.
* see this link for more info
** more background information about OMPs and about their differences compared to the development of traditional medicines is available in the EuropaBio-EFPIA publication Development process of Orphan Medicinal Products