Orphan drug status

An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.

In the US and EU it is easier to gain marketing approval for an orphan drug, and there may be other financial incentives, such as extended exclusivity, all intended to encourage the development of drugs which might otherwise lack a sufficient profit motive. The assignment of orphan status to a disease and to any drugs developed to treat it is a matter of public policy in many countries, and has resulted in medical breakthroughs that may not have otherwise been achieved due to the economics of drug research and development. In the case of the rarest diseases that afflict fewer than 10,000 people, biotech companies who own the only approved drugs to treat those diseases "can charge pretty much whatever they want."

An orphan drug is a pharmaceutical agent which has been developed to treat a rare medical condition, the condition itself being referred to as an rare disease. A rare disease, also referred to as an "orphan disease", is any disease which affects a small percentage of the population. Most rare diseases are genetic, and thus are present throughout the person's entire life, even if symptoms do not immediately appear. Many rare diseases appear early in life, and about 30 percent of children with rare diseases will die before reaching their fifth birthday. With a single diagnosed patient only, ribose-5-phosphate isomerase deficiency is presently considered the rarest genetic disease. No single cutoff number has been agreed upon for which a disease is considered rare. A disease may be considered rare in one part of the world, or in a particular group of people, but still be common in another. As many as one-in-ten Americans suffers from rare disease. According to clinuvel.com, over 55 million people are estimated to suffer from a rare disease in Europe and in the US, global estimates are between 5000 and 7000 rare diseases and new rare diseases are discovered every week and many have no treatments available.

As of 2014^[update], there were 281 marketed orphan drugs and more than 400 orphan designated drugs in clinical trials. More than 60% of orphan drugs were biologicals. The US dominated the development of orphan drugs with more than 300 orphan drugs in clinical trial process, followed by Europe. Cancer treatment was the indication in more than 30% for orphan drug clinical trials.

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