Orphan drug

An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.the revenue-generating potential of orphan drugs as great as for non-orphan drugs, even though patient populations for rare diseases are significantly smaller. Moreover, we suggest that orphan drugs have greater profitability when considered in the full context of developmental drivers, including government financial incentives, smaller clinical trial sizes, shorter clinical trial times and higher rates of regulatory success.Public resources went into understanding the molecular basis of the disease, public resources went into the technology to make antibodies and finally, Alexion, to their credit, kind of picked up the pieces.model of pharmaceutical research and development, the expectations that companies and patient groups have about how risk and reward is shared between the industry and a publicly funded NHS, and in the arrangements for commissioning expensive new treatments. An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy in many countries and has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development. In the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to encourage development of drugs which would otherwise lack sufficient profit motive to attract corporate research budgets and personnel. An 'orphan disease' is any disease which affects a small percentage of the population. Most rare diseases are genetic, and thus are present throughout a person's life even if symptoms do not immediately appear. Many rare diseases appear early in life, and about 30% of children with rare diseases die before reaching their fifth birthday. There is no universally accepted number for defining rare disease. A disease may be rare in one part of the world or in a particular group of people but common in another. As many as one-in-ten Americans suffers from rare disease. As of 2014, there were 281 marketed orphan drugs and more than 400 orphan-designated drugs in clinical trials. More than 60% of orphan drugs were biologics. The U.S. dominated development of orphan drugs, with more than 300 in clinical trials, followed by Europe. Cancer treatment was the indication in more than 30% of orphan drug trials. According to Thomson Reuters in their 2012 publication 'The Economic Power of Orphan Drugs,' there has been increased investment in orphan drug research and development, partly due to the U. S. Orphan Drug Act of 1983 (ODA) and similar acts in other regions of the world driven by 'high-profile philanthropic funding.' According to Drug Discovery Today, the years 2001 to 2011 were the 'most productive period in the history of orphan drug development, in terms of average annual orphan drug designations and orphan drug approvals.':660 For the same decade the compound annual growth rate (CAGR) of the orphan drugs was an 'impressive 25.8%, compared to only 20.1% for a matched control group of non-orphan drugs.':6 By 2012, the market for orphan drugs was worth USD$637 million, compared with USD$638 million for a control group of non-orphan drugs.