In 1980, Abbey Meyers was beginning to feel hopeful. She had enrolled her son, who had Tourette’s syndrome, into a clinical trial for the drug pimozide, and he was responding favorably. Then came the awful news… the trial was being halted and pimozide would no longer be available. “Why?†Meyers wondered. Because the drug was proving ineffective against schizophrenia, its primary target, she was told; and Tourette’s—the drug’s secondary target—is an “orphan disease,†meaning it wasn’t enough to merit further research on the drug. “I was devastated,†recalls Meyers.
After the sadness subsided, Abbey Meyers decided to take action. After all, the problem wasn’t as daunting as discovering a treatment… that had already been done. The problem was to find a way to get the treatment to market. She thought she might be able to help find a solution. She began by developing an understanding of orphan diseases.
A Rare Breed
An orphan disease is one that the pharmaceutical industry chooses to ignore because of its rarity. If a patient population isn’t large enough to justify the costs to develop, manufacture, and market a treatment, then it won’t happen. In the US the FDA has somewhat arbitrarily defined a rare disease as one that affects fewer than 200,000 people. By that definition, scientists have determined that there are about 7,000 rare diseases affecting 25-30 million Americans.
With this information, Meyers—previously a Tourette’s Syndrome Association volunteer—began to take a broader view of her advocacy. By addressing the larger issue of orphan diseases rather than just Tourette’s, she felt she could cast a wider net and marshal the necessary support and political clout needed for some kind of legislative action.
And that’s exactly what happened. Thanks to Meyers’ tireless work, a community of support groups representing families affected by rare diseases began to coalesce, culminating in the founding in 1982 of the National Organization of Rare Disorders (NORD). Then, in 1983, Congress passed the Orphan Drug Act (ODA), due largely to the efforts of NORD.
