The origin of the Orphan Drug Act

Many people in the pharmaceutical industry today ask me questions about the origin of specific items in the Orphan Drug Act. The most common question is how and why we came up with the maximum population size of 200,000 Americans. I still have to chuckle to myself whenever I hear that question because no one could possibly imagine that the issue was decided in a ladies room on Capitol Hill. Other people would voluntarily give me their interpretation of specific items in the law, and I had to practice control of my temper because they were 180 degrees off base. Then there are those who tell me how they think the law should be changed, and I wonder why they would want to change something that has worked so well for 30 years?

This is why I decided to write this book. The reasons why the law was needed, how it came about, the political and ideological battles that were fought, and the continuous work of the patient community to make sure that all parties (the government, the drug industry, the medical professionals and the patient organizations) all live up to their responsibilities to ensure the law's success. Now, 30 years after the ODA was signed by President Ronald Reagan, I am continuously astounded by the law's accomplishments. The treatments and cures for rare diseases that are available today would not exist without the Orphan Drug Act, and orphan drug legislation in so many other nations of the world has accomplished similar relief from pain and death outside of the United States.