A Pill Is Born
Let a new drug show you how it (and other drugs) came to be.
Many Hurdles to Clear
I had been through a lot of experiments already, but I still had a long way to go. To move to the next step, the drug maker sponsoring me needed the FDA to approve tests in humans. The company showed the FDA how well I performed in the animal tests and explained how they would study me in people, in what's called a phase I clinical trial.
With a thumbs-up from the FDA, the researchers started looking for people to try me. They needed 20-100 healthy volunteers. The purpose of the study was not to see if I worked, but rather to test my safety and side effects in humans.
Some people had mild side effects, like headaches and upset stomachs. Hey -- no one's perfect! I'll bet you've given someone a headache before. The fact is, all drugs cause side effects sometimes. But I didn't cause any serious problems for the people in this study.
I Got a Name
I got my "nonproprietary" name around this time: noperalate. That's my generic chemical name, the one scientists use when they're talking about me. It's different from my brand name, which was given later by the companies that will sell me. A group called the United States Adopted Names Council assigns generic names to new pharmaceutical compounds. I never thought WBMD-523 was really me, so I was happy to be called noperalate.
So far, so good. But in the next step, a phase II trial, I had to prove that I worked. Up to this point, I only had to show that I was likely to work. Now I had to perform. The researchers wanted to see that I could inhibit that enzyme reliably, in a larger number of people -- around 100 to 500 -- without harming them. I would also be compared to a placebo, that is, to a dummy pill. The researchers and test subjects wouldn't know who took me and who took the placebo until after the study was done.
Seven years had passed since I was first noticed in the lab and chosen for development. A lot of time, brainpower, and money had been invested in me, but there was still a chance I might fail. Clinical trials are kind of like the Olympics. Often very promising athletes make it to the games but ultimately don't measure up. About four out of five drugs don't make it through clinical trials.
Not everyone was cheering me on, either. Many scientists in the field were skeptical. They thought the early study results weren't convincing. By the time the phase II studies wrapped up, however, a lot of people were getting excited. It was clear I would go on to phase III.
The final phase of clinical trials lasted four years. I had to be tested on thousands of people, and show beyond a doubt that I really worked and that my benefits far outweighed any potential problems.