Orphan drugs explained

"Orphan drugs" are drugs developed for the treatment of rare diseases. A disease is classified as "rare" in the United States if fewer than 200,000 people suffer from it.

Pharmaceutical companies have difficulty making money from the development of orphan drugs because the pool of potential customers is small. It is not financially viable to develop these drugs for the marketplace. The US federal government and other governments around the world have developed schemes to support the development of orphan drugs. Congress passed the Orphan Drug Act (ODA) in 1983 to assist the research and development of treatments for rare diseases. The act has lead to the acceleration of rare drug treatment with more than 300 treatments being developed in the past 25 years.

The ODA and the Office of Orphan Products Development

The term orphan drug had long been used by the pharmaceutical industry to classify drugs for rare diseases. The US Food and Drug Administration (FDA) also had a department to identify drugs that were for rare diseases for almost a year before the ODA was ratified.

The Office of Orphan Products Development (OOPD) was established in 1982 by the FDA to identify orphan drugs, and then later administer the ODA and provides information on drug development and support. Since its inception, the OOPD has overseen the development of more than 1700 drugs and biologics indentified as orphans. It is estimated that rare diseases affect more than 25 million Americans.

Benefits available to ensure the development of orphan drugs

The OOPD supports all levels of orphan drug development, from initial research to production. The help comes in the form of physical support of research to the wavering of charges to take the drugs to the marketplace. Some of the specific assistance provided is:

• The cost of human clinical testing of drugs classified as orphan drugs is subsidized by the government through the Orphan Products Development Grant Program.

  The annual budget for the Orphan Products Development Grant Program, which includes biologics, medical devices or medical foods, is $14 million in 2008. Grants are funded for up to three years. The first phase is granted from $200,000 and second and third from $350,000.

  Up to 15 news projects are awarded grants each year.

• Research into the treatment of rare diseases is supported with tax credits and assistance with research design.
• Companies that bring orphan products to the market are protected from competition by a seven-year period of exclusive marketing.

The OOPD offer support throughout an orphan drug development from research to delivery to the marketplace.

The grant program is only available to clinical studies for rare diseases as classified by the FDA. The product being studied must also be a new product, or an existing product being studied afresh as a treatment for a rare disease.

Since the program strived to support all levels of drug development, the grant program is open to universities, research institutes, large and small businesses.

Government assistance for orphan drugs is designed to reduce cost and balance the economy-of-scale for the product. Orphan drugs are still required to meet safety and efficacy standards set by the FDA to be approved for the market.

There is no fast-track process to get orphan drugs approved quicker than other drugs; however because most orphan drugs are for life-threatening illness they tendto go through the approval process faster.

Orphan Drug Programs in Other Parts of the World

Japan is the second-largest pharmaceutical manufacturer in the world. Japan has close to 1400 pharmaceutical companies employing more than 200,000 people. The country has an orphan drug support program similar to the US model. One difference is that the definition of rare disease is not a specific number but any disease or condition affecting 0.05 per cent of the population or less.

The European Medicines Agency has also formed the Committee of Orphan Medicinal Products (COMP) to identify orphan products. The COMP considers rare diseases those that affect no more than five in a population of 10,000. COMP identifies orphan drugs and rare diseases for the 27 European Union states.