An orphan drug is a medicine that is developed for the diagnosis, prevention or treatment of a rare disease. A rare disease, sometimes referred to as an orphan disease, is any disease that affects a small percentage of the population. In Europe a condition is considered rare when it affects less than one in 2,000 people.
A pharmaceutical company developing a drug for a rare disease can apply to the European Medicines Agency (EMA) for orphan designation. The EMA Committee for Orphan Medicinal Products (COMP) is responsible for evaluating applications for orphan designation. Receiving orphan designation entitles the company to avail of a number of incentives, including reduced fees for regulatory activities and longer market exclusivity than non-orphan medicines.
Assessment of orphan medicines in Ireland
Orphan medicines are assessed in the same way as all other medicines. However patient organisations have been advocating for changes to the processes assessing orphan medicines for many years. As a result of this, in November 2018, as recommended in the National Rare Disease Plan 2014 – 2018, the Rare Diseases Technology Review Committee (RDTRC) was established. The committee, which includes two patient representatives, is responsible for assessing rare disease medicines that have received a negative recommendation from the National Centre of Pharmacoeconomics (NCPE) after a Health Technology Assessment (HTA). The committee examine the impact of the specific conditions on patients and care givers, real world evidence along with scientific data. It does not have the authority to approve drugs but will make recommendations to the HSE Drugs Committee who makes the final decision about drug reimbursements.