Do you ever wonder how the medications that we use to treat or prevent illnesses are developed and eventually approved for use?  Do you wonder why the “miracle drug” that we read about in the news media takes years to get to market or never reaches our local pharmacies?

The process of developing a drug takes many years and is a combined effort involving the pharmaceutical company (sponsor), the Food and Drug Administration (FDA), an ethics and safety organization called an Institutional Review Board (IRB), local research sites, and most importantly, private citizens who are willing to donate their time to participate in clinical trials.

Before any human testing can be conducted, the pharmaceutical company gathers all the pre-clinical (animal) testing and presents it to the FDA.  The FDA is responsible for ensuring that drugs marketed in the United States are effective and safe.  All drugs marketed in the United States must be approved by the FDA.  The FDA rigorously analyzes the data as well as the pharmaceutical company’s plan for human testing.  They provide guidance for the types of clinical trials that the pharmaceutical company needs to conduct in order for the drug to be considered for approval.

Clinical trials are experiments that use human subjects to see whether a drug is effective and what side effects the drug might cause.  Before a sponsor may proceed with a clinical trial, the trial protocol must be approved by an independent IRB who is responsible for assuring that the study design is acceptable, that subject volunteers are fully informed of any known risks, that subjects have given their informed consent to participate, and that researchers are taking measures to protect patients from harm.

There are several phases of clinical trials that sponsors must conduct prior to presenting their drug to the FDA for consideration.  Initially, small trials are conducted using healthy volunteers to assess the activity of the drug in humans.  Next, larger trials are conducted to obtain preliminary data on whether the drug works in people who have a certain disease or condition. In these trials, the effect of the drug is often compared to similar patients who are taking an inactive substance (a placebo).  Finally, much larger trials are conducted to further evaluate the effectiveness and safety of the drug in a broader patient population.  The FDA meets regularly with the sponsor throughout this process to provide guidance and to assess the safety of the drug.

To conduct a large clinical trial in a population of patients who represent the diverse population of the United States, the sponsor hires local research sites across the country to work with patient volunteers to conduct the trial.  These sites are independent of the pharmaceutical sponsor and contract with the FDA to conduct the trial exactly as written in the protocol and to ensure patient safety.  The research sites, under the supervision of a local principal investigator, collect unbiased data to be presented to the sponsor.  All patient study information is submitted to the sponsor using anonymous code numbers to protect patient confidentiality.

The process of bringing a drug from the chemistry lab to the FDA for approval often takes about 15 years.  Proving that a drug is effective and safe is ultimately dependent on patient volunteers who are willing to sacrifice their time to participate in this process.  So, the next time your physician mentions a clinical trial, or you see an ad for a trial, consider that it may be your opportunity to be a part of the process to develop a breakthrough lifesaving new medication.