Birth of a New Medication

Enbrel, Remicade, and Celebrex are just a few of the newest weapons in the arsenal against arthritis. Ever wonder how these new drugs come to be?

It takes time, a long time, to develop a new medication. On average it takes 15.3 years from the beginning of an idea to FDA approval, if it gets that far. Of about 5,000 compounds screened for potential, only 250 of those get to laboratory testing. Of those 250, only 5 will make it into clinical testing in humans. Just one of those 5 will receive FDA approval. 1 out of 5,000 potential compounds make it to the drugstore shelves.

Not only is it time consuming to develop a new drug, but also it costs a pretty penny. According to the Boston Consulting Group, Inc., it costs an average of $500 million to develop just one new drug. 

A drug begins as the result of research. Scientists use computer modeling and combinatorial chemistry to search for compounds capable of affecting disease. 

Once the compound discovery has taken place, the pre clinical studies begin. Extensive toxicological tests and animal studies are done to determine whether the drug is likely to be safe and effective in humans. If it appears to be safe the FDA and review boards of the institution or institutions must approve applications where the trials are to be conducted.

Then the phase 1 clinical tests begin. Safety studies are conducted on 20 to 80 healthy volunteers. Potential side effects are identified, and a dosage range is determined.

Phase 2 clinical tests are done on 100 to 300 volunteers who have the targeted disease. This phase determines the drugs effectiveness. 

If the drug proves to be effective in phase 2 it moves on to phase 3. This is the big study. 1,000 to 3,000 (or more) patients are tested. The number and severity of side effects and the effectiveness of the drug is determined.

If data from all the tests prove the drug’s safety and effectiveness, the company submits all the results and files a New Drug Application with the FDA. If FDA agrees that the data proves significant safety and effectiveness, it will approve the drug. The FDA must also approve the official labeling, or guidelines for the drugs administration. 

Soon the drug is found on your pharmacy shelves, a long way from that idea over 15 years ago. The company and the FDA will continue to monitor the drug through post-marketing surveillance. This allows them to track its safety and efficacy when used by a larger number of patients than participated in the clinical trials.

PhRMA The Challenge of Drug Development