What is an 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. 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. For example, In EU rare diseases, are diseases affecting less than 1 in 2,000 persons or a maximum of 250,000 citizens. 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 may not otherwise have been achieved, due to the economics of drug research and development. Below is the orphan drug legislation development: 1983 – First Orphan Drug Act in the United States 1990s – Orphan Drug Legislation adopted by Singapore (1991) Japan (1993) and Australia (1997) 1999 – Adoption by the European Parliament of the Regulation on Orphan Medicinal Products 2000 – Creation of the Committee for Orphan Medicinal Products (COMP) at the EMEA (European Medicines Agency) 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. In late 2007 the FDA and EMA agreed to use a common application process for both agencies to make it easier for manufacturers to apply for orphan drug status but, while continuing two separate approval processes. Below are some incentives provided by the EU regulation for orphan drugs manufacturers: • Market exclusivity in the EU. Similar competitive products cannot be placed on the market for 10 years after the granting of marketing authorization and 12 years if pediatric studies are performed. • Protocol assistance. Scientific advice is provided to pharmaceutical companies by the EMEA (European Medicines Agency, based in London) to optimize drug clinical development meeting European regulatory requirements. • Access to the centralised procedure. Orphan drugs have direct access to the EMEA centralized procedure for the application for marketing authorization. • Fee reductions. Fee waiver for orphan designation and reduced fees for marketing authorization, inspections, variations and protocol assistance. • EU-funded research. Pharmaceutical companies developing orphan drugs may be eligible for grants from EU and Member State programs and initiatives supporting research and development, including the Community framework programs. Global statistics 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. Examples for selected diseases Cystic fibrosis In the 1980s, people with cystic fibrosis rarely lived beyond their early teens. Drugs like Pulmozyme and tobramycin, both developed with aid from the ODA, revolutionized treatment for cystic fibrosis patients by significantly improving their quality of life and extending their life expectancies. Now, cystic fibrosis patients often survive into their thirties and some into their fifties. Familial hypercholesterolemia The 1985 Nobel Prize for medicine went to two researchers for their work related to familial hypercholesterolemia, which causes large and rapid increases in cholesterol levels. Their research led to the development of statin drugs which are now commonly used to treat high cholesterol. Wilson's disease Penicillamine was developed to treat Wilson's disease, a rare hereditary disease that can lead to a fatal accumulation of copper in the body. This drug was later found to be effective in treating arthritis. Bis-choline tetrathiomolybdate is currently under investigation as a therapy against Wilson's disease. Phospholipase 2G6-associated neurodegeneration In 2017 FDA granted RT001 orphan drug designation in the treatment of phospholipase 2G6-associated neurodegeneration (PLAN). By Aliyev F. References https://en.wikipedia.org/wiki/Orphan_drug. https://www.eurordis.org/content/what-orphan-drug http://www.evaluate.com/thought-leadership/pharma/evaluatepharma-orphan-drug-report-2018