What it Takes for New Drugs to be Tested and Reach Patients

What goes into researching and developing a cancer drug and what is required for approval by the Food and Drug Administration (FDA)? We sat down with Moshe Talpaz, M.D., Associate Director of translational research at the U-M Comprehensive Cancer Center, for a closer look at the FDA approval process.

Moshe Talpaz, M.D., has been involved in the FDA approval process of many cancer drugs. He leads translational research at the U-M Comprehensive Cancer Center.

Q: What is the general timeframe for a new drug to be approved by the FDA so it is available to patients?

From beginning to end, typical clinical drug development takes around 7-10 years. If a drug is showing remarkable activity and serves an unmet medical need, an accelerated process can go much quicker, maybe around 3 years after the onset of clinical trials.

Q: What is the first part of the process in developing a new drug?

The preclinical process is quite elaborate. Academic medical centers like U-M have the role of discovering what governs a disease, to discover the mutation that drives an illness like cancer. We first focus on the biology to show the full sequence of events in the body. We develop various laboratory approaches to demonstrate that shutting off the abnormal protein kills the cancer cell.

Drug companies then get involved. Medicinal chemists study many different molecules to identify one that has the characteristics of potentially becoming a drug.  This becomes the subject for extensive testing on animals to determine that it works safely in the body.

Q: When does research begin with actual patients?

Phase I testing in humans aims to find the maximum tolerated dose. We start with very low doses to check for toxicity. We end up with a safe dose to recommend for use in a Phase II study.

Phase II involves more extensive testing and is where we demonstrate whether the compound is effective and which tumors respond to it.

Phase III is a randomized study of large groups of patients that compares the potential drug to the current standard of care. If the compound reaches a significant goal, which is acceptable to the FDA, the FDA approves it.

Q: What about drugs that go through the accelerated process for FDA approval? Are there disadvantages when drugs go through the process quickly?

I believe the FDA approval process is streamlined and efficient. If a compound proves to be good and there is no question about it, it moves forward quickly in the approval process.

The FDA requires further testing on the drug once patients are using it, and the drug must meet additional milestones later.

Learn more about the clinical trials at the U-M Comprehensive Cancer Center.