Orphan Drug Designation

Patients suffering from rare medical conditions, like narcolepsy, are every bit as much in need of innovative new treatments as those who suffer from more common diseases like heart disease or diabetes. Unfortunately, the rarity of a condition such as narcolepsy means that pharmaceutical companies are less motivated to carry out research in that area (simply because there are fewer patients who might benefit from an innovative new treatment, and so the potential for sales and a commercial return on the necessary investment is lower).

To address this issue, and to promote the development of treatments for rare diseases, regulatory authorities such as the European Medicines Agency will grant a potential treatment for a rare disease what is called “orphan drug status”. In the EU, this is done where the potential new medicine is for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that affects not more than five in 10,000 people in the European Union, or where the medicine is unlikely to generate sufficient profit to justify research and development costs.

The most important benefit to the pharmaceutical company of being granted an orphan drug designation is that, one authorised, the medicine is given ten years of market exclusivity. In other words, once they are approved authorised orphan medicines benefit from ten years of protection from market competition by similar medicines for the treatment of similar diseases.with similar indications once they are approved. This extended period of freedom from competition gives the pharmaceutical company more time in which to recoup its investment in developing the medicine and to make a commercial return, and is therefore intended to benefit sufferers from rare diseases by stimulating the development of innovative new treatments that might otherwise never be produced.