by pharmaceutical companies because of the small patient population and lack of financial incentives . Drug companies tend to focus on the more lucrative `` blockbuster drugs '' for common diseases , at the expense of cures for rare conditions . The term `` orphan drug '' was originally coined because the pharmaceutical industry took little or no interest in discovery , development and marketing of drugs for rare diseases . According to the European Organization for Rare Diseases , rare diseases affect between three and five percent of the population in developed countries . In the European Union , a disease is considered rare if it affects fewer than five in 10,000 people , while in the United States the disease must affect fewer than 200,000 people . Many countries , such as the United States , Japan and the United Kingdom , have therefore attempted to encourage pharmaceutical companies to invest in orphan diseases by giving them tax incentives , fee reductions and extensions of patent rights . The United States was the first country to develop an orphan drug law in 1983 , followed by Japan and Australia . In 1997 , the Australian Orphan Drugs policy
LONDON , England -LRB- CNN -RRB- -- The 1992 Hollywood movie `` Lorenzo 's Oil , '' depicts the true story of Lorenzo , a five-year-old boy who suffered from adrenoleukodystrophy -LRB- ALD -RRB- , a rare and incurable disease that slowly destroys the entire nervous system . Michaela Damin and her son , Nick , who suffers from an extremely rare disease that affects the heart . The movie showed how Lorenzo 's grave physical and mental decline was finally stopped when his tireless parents found a treatment based on a mixture of oils , despite skepticism from doctors . The film illustrated perfectly the struggle faced by patients suffering from any of 6,000 known rare conditions worldwide , generally known as orphan diseases . Michaela Damin , founder of the Barth Syndrome Trust , whose son , Nick , suffers from this crippling syndrome that affects the heart and immune system and only has 130 known sufferers worldwide , told CNN : `` It can take years to get the correct diagnosis and even then there may be no expert who knows what to do next . '' The fact remains that rare diseases are still mostly overlooked