For a long time, people spoke about the problems involved with delivering drugs to people with rare diseases. Development costs were high and regulatory hurdles even higher. Costs were not economically justified by the limited returns expected from a medicine to treat a disease that impacting only a small number of people. Without an economic incentive, drug development for so-called orphan diseases languished.
That is where things stood in 1980. At that time, Congress held hearings on the issue and Maurice Klugman, a Hollywood writer and producer and brother of actor Jack Klugman, read about it. As a result, Maurice Klugman wrote an episode for his brother’s television series, Quincy, M.E., in which the gruff medical examiner dealt with the problem of orphan drug development. This episode, Seldom Silent, Never Heard, broadcast in 1981 and led to public awareness of the problem. Additional Congressional hearings followed at which Jack Klugman, himself, testified.
As a result of those hearings a bill was introduced in Congress to provide the necessary incentives for Orphan drug development. Specifically, it offered a significant tax credit for the cost of clinical trials for orphan drugs, seven-year exclusivity, and a clearly defined regulatory pathway. The bill ran into problems in the Senate where Orrin Hatch deleted the tax credit, dramatically decreasing the economic incentive to develop orphan drugs.
Guess what? Jack Klugman had the show’s writers do another episode in which he faced off with a fictitious Senator who was blocking the Orphan Drug Act. This episode, Give Me Your Weak, was broadcast in 1982He asks the senator to look out his window where he sees hundreds of people with signs saying “We want the Orphan Drug Act”. The producers, in fact, had cast over 500 extras who actually had the kind of rare diseases that the Act was intended to address. The senator in the episode gave in.
And, before too long, so did the real senator Hatch. The Orphan Drug Act was signed into law in 1983. Since then, hundreds of drugs have been approved to treat rare diseases, with many hundreds more in development.
Do you know if your drug qualifies for consideration under the Orphan Drug Act? To qualify, a drug must be intended to treat a condition or disease affecting less than 200,000 Americans. Pearl Pathways can work with you to prepare and submit your Orphan drug designation request. Pearl can also help you navigate FDA expectations, such as Annual Reports, once your drug has received Orphan status. We can also help you prepare and file your IND to begin the clinical trials to see if your drug will help patients. And, if it does, we can help you prepare and file your marketing application. That’s what Pearl Pathways is about: we help speed your product to patients.