Drug approval process

How research begins

Universities are not allowed to run private companies on their campuses. Instead, they "spin off" drug companies by licensing them exclusively to develop drugs or new therapies based on scientific breakthroughs discovered at the schools. The drug companies then develop clinical research trials to establish the effectiveness and safety of the drugs. A majority of today's largest biotechnology companies began that way.

Clinical trials

Basic science research provides the clues to help identify new drug candidates. Clinical research, in particular well-controlled clinical trials, assess the safety and efficacy of new drugs.

The most accurate study is called a double-blind, placebo-controlled trial. In this trial volunteers are split into two groups. One group gets the experimental drug and the other group gets an inactive drug (placebo).

Each patient receives a randomly assigned number. Neither the patients nor doctors know who is receiving the new drug and who is receiving the placebo. During the trial, all of the patients receive the same observation and tests. At the end, based on the patients' numbers, doctors can determine which patients were in each of the groups.

Drug approval

After clinical trials are completed, companies submit all scientific and clinical research information to the U.S. Food and Drug Administration (FDA) to request that a drug be approved for use. They also develop a "recommended label" to send to the FDA for FDA approval. The label contains suggested indications (what it will be used to treat) and dosing. Dosing of new drugs is based on the dosing used in the clinical trials.

If patients are prescribed the drug in a way that differs from what is suggested on the label, it is considered "off-label." Some insurance companies will not cover off-label uses.

Once the drug is submitted to them for approval, the FDA has 45 to 60 days to notify the company whether the drug has been accepted for review. Drugs receive 6-month priority review or the standard 10- or 12-month review. In order to receive priority review, the drug needs to treat a serious or life-threatening disease, or fulfill an unmet need.

The FDA reviews how the label was developed and whether it was written correctly. If the FDA disagrees with the label, the FDA and the company negotiate the change in the label. On average, the FDA takes a year or more to review drugs submitted for approval.