Orphan Drugs

Understanding the drug development and marketing process is key to understanding orphan drugs.

The development of the drugs used to cure disease and treat chronic conditions is a long, expensive process. It takes several years from the point of discovery, through research and development, and clinical trials, to the point where a drug can be marketed. According to a 2003 study, the process of bringing a drug to market often costs more than $800 million. Because of the time and expense involved, drug companies want to make sure that they are investing their funds in drugs that will have a viable market.

New drugs are covered by patents, giving the company that developed them the exclusive right to market the drug for a set period of time. While a drug company has a patent on a drug, no other drug company can make or sell the drug without permission of the patent holder. After the patent expires, other drug companies can market generic versions of the drug but without the brand name that the drug was originally sold under.

There are huge markets for drugs that lower cholesterol, treat cancer, and help manage diabetes. In addition to drugs that are prescribed for depression, high blood pressure, and other common conditions, these drugs are big money-makers for drug companies. However, drugs are also needed to treat, cure, and manage diseases, illnesses, and conditions that are not as profitable. Conditions such as cystic fibrosis, glioma, and multiple myeloma are three such diseases. Drug companies are less likely to travel down the long, expensive road of developing and marketing drugs for these conditions because it simply isn't profitable. Yet, the individuals who suffer from these diseases are just as deserving of treatment and cure as those who are prescribed drugs for more common ailments.

In January 1983, the United States initiated legislation designed to bridge the gap between drug companies that needed to make a profit and individuals suffering from relatively rare conditions that needed treatment. This legislation was called the Orphan Drug Act and its purpose has been to promote the research and development of drugs designed to treat, prevent, or cure what are often referred to as orphan diseases - those diseases that affect fewer than 200,000 people. Orphan drugs are drugs that will most likely never earn a profit for the companies that develop them, left to the free market.

In the past several years, the U.S. Food and Drug Administration has granted orphan designation to drugs designed to treat rare cancers (such as mesothelioma), anthrax infections, chronic graft vs. host disease, neuroleptic-induced tardive dyskinesia, Fabry's disease, and Pompe disease. Many rare conditions are recognized by the general public only when a celebrity or other public figure champions research and development of treatments for them through fundraising efforts or news reports. Muscular Dystrophy is one example of an orphan disease. Before the Orphan Drug Act, the only hope for individuals suffering from such illnesses was raising funds for the development of drug treatments through public awareness campaigns such as telethons.

The governments of the United States and other countries are attempting to entice drug companies to develop drugs to treat rare conditions, illnesses, and diseases by offering incentives such as tax breaks and other subsidies to make investing in orphan drugs more appealing. Drug companies are also offered a longer patent period during which to market the drug exclusively. At the discretion of the FDA, orphan drugs may face less stringent requirements for clinical trials during development. Since orphan diseases can rarely offer enough suitable subjects for clinical trials (i.e., there may not be thousands of individuals suffering from an orphan disease, therefore there are not enough individuals to participate in normal trials), the Orphan Drug Act allows drug companies to reduce the number of individuals required to produce an effective trial.

Nearly 2,000 drugs that have received orphan designation since the Orphan Drug Act went into effect. These include Alimta, which is now part of the standard treatment for mesothelioma. Onconase is also an orphan drug. The ODA has been somewhat successful in drawing drug companies into the orphan drug market, and in comparison with the days when telethons were the only way to pay for the development of orphan drugs, some credit has to be given to the agencies and drug companies willing to invest in what were, at one point, unprofitable ventures.

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