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Eric Godeck was recently fighting for his life against a rare muscle disorder. The infant had a severe type of “Pompe disease,” a genetic condition that caused his heart and respiratory muscles to build up abnormally high levels of glycogen. The total number of such cases in the United States is only 3,000 to 6,000.

With the fatal disease attacking his breathing and heart muscles, Eric’s only hope was to be accepted into a treatment study, thereby becoming eligible to receive a promising experimental drug. In the experimental treatment for Pompe disease, missing enzymes are administered to restore the muscles’ glycogen levels to normal.

There was not, however, enough medicine available to allow Eric to enter the study. The tragedy of this boy’s death reminds us that the development of new medical treatments should be encouraged and expanded.

An estimated 20 million Americans have any of more than 6,000 rare disorders, such as Pompe disease, and more familiar illnesses, such as sickle cell anemia, hemophilia and cystic fibrosis. Because each of these diseases affects fewer than 200,000 people in the United States, drug companies often ignored them. These illnesses, therefore, received the name “orphan diseases.”

To stimulate research in the area of orphan diseases, the government passed the Orphan Drug Act of 1983, which gave incentives to companies working on treatments for rare diseases. Since bringing a new prescription drug to market can take years and many millions of dollars, financial incentives are essential to the many smaller drug companies that develop these treatments.

Granting exclusive marketing rights for seven years to the company that first develops the orphan drug is another key enticement. This is very important because many orphan drugs are either natural substances that cannot be patented or older products that no longer have patent protection.

Orphan drug incentives have helped the development of approximately 200 new drug treatments in the past 16 years, compared to less than 10 orphan drug treatments developed in the decade before the Orphan Drug Act.

Even after new orphan cheap drugs are approved for use, they may not be immediately available to all patients. The drugs are often developed by smaller companies with limited manufacturing capabilities. As noted by Abby Meyers, president of the National Organization for Rare Disorders, “Patients had to initially enter a lottery to obtain new drugs for treating Lou Gehrig’s disease, as well as for one type of multiple sclerosis.”

The tragedy of Eric’s death reminds us that good programs like the Orphan Drug Act need to be supported and expanded to bring medicines more quickly to a greater number of patients. People should not have to play the “health treatment lottery” to receive life-saving remedies. Major pharmaceutical companies should increase their participation in the upbringing of our orphan drugs.