Jun 29, 2023

How Are New Medicines Discovered?

If you’re one of the many people in the United States that has participated in a clinical trial, then you’ve had a dramatic impact on the future of medicine.


The Pathway to Progress

Before a drug or device ever reaches a patient, it goes on a long and difficult journey. When an idea for a new treatment is conceptualized it must go through rigorous testing before coming to market. This begins in a research laboratory with beakers, cell cultures, Bunsen burners, and scientists with white coats. If the proposed treatment shows initial promise and doesn’t harm the cells, it moves on to be tested in animals. First, small animals like mice or rats; then, depending on the function of the drug or device, rabbits, dogs, or pigs. These experiments are carefully designed and approved by the International Animal Care and Use Committee (IACUC). Only about 1 in 5,000 of the drugs in this stage of development will go on to be approved (via New York Times). 

This goes to show just how difficult it is to develop new treatments for diseases. This pre-clinical phase can take years to gather enough research to prove that the drug can be effectively used in humans. Hundreds of thousands of dollars, sometimes even millions, are spent during this time on lab equipment, raw materials, labor, software programs, and other resources. All of these pre-clinical efforts are just laying the groundwork for clinical trial participation.




Once the drug is considered to be safe and effective for use in humans, it moves on to the clinical trial phase. This is the part where you come in! It is important and empowering for potential clinical trial participants to know exactly how he or she is impacting the study, and how the study may impact them. 

There are significant differences between the phases of a clinical trial. Phase I studies are specifically studying the safety of the drug in humans. Using the results of the laboratory and animal testing, they have a good understanding of what dosage to begin with. Typically, the drug is given to anywhere between 20 and 100 healthy volunteers and they are monitored for specific periods of time. This phase lasts several months. Phase II is specifically aimed to test the efficacy and side effects of the new drug or device. Several hundred people are enrolled in these studies, which can last up to two years. Phase III studies are where most of the data on safety and efficacy comes from. These studies enroll 300-3000 patients and can last up to four years. Phase IV studies occur after the drug is approved by the Food and Drug Administration (FDA). This is essentially a follow-up study that monitors thousands of patients who are actively taking the drug.


The Critical Component

Regardless of whether it was a phase I, II, III, or IV, the critical component of each of these is the participant–that’s right, you! These trials must reach a certain amount of participation before they can prove anything. All of the data gathered from laboratory research and animal testing is insignificant when compared to the results of human use in clinical trials.

Since 2000, there have been over 285,000 studies registered on ClinicalTrials.gov. This has resulted in the creation of an entire population of current and past clinical trial participants. This is a group of people that have tremendous insight and experience in the world of clinical research; a group who before now, did not have a voice. There is so much we can learn to help grow a culture of patient-focused clinical research. There is so much that can be passed on to future study participants.


The Power of Participation

At some point, you may have felt some concerns over participating in a clinical trial, such as, “Will this make my disease worse? How much time am I going to have to commit?” After some research and answers from your healthcare provider, you overcame the uncertainty and joined the study. With participation, there is a sense of pride in knowing that you are directly influencing the future of medicine. You are pioneering a future that may provide a glimpse of hope or a new beginning to someone suffering from a disease that is impacting their quality of life.

Not only can clinical trial participation give you access to a new treatment option, but the information gathered contributes to a pool of data that can give researchers tremendous insight into how to optimize the drug or device being tested. In some cases, this research is being done for diseases where the current treatments are ineffective or non-existent. Examples of these include multiple sclerosis (MS), Crohn’s disease, ulcerative colitis, Alzheimer’s disease, and cancer. 

Oftentimes, the delivery of the drug is inconvenient and frequent, depending on the disease. Patients may have to take significant time out of their lives to receive injections at a doctor’s office or clinic. The cost of co-pays and medication can make this experience even more challenging. Dealing with the pain and discomfort of the disease is one thing; however, watching your career come to a halt, or missing out on spending time with family or friends can be devastating.

The research for these cures can be done independently of human participants, but the only way these drugs can come to market is through people willing to get involved and be a part of the solution. As a participant in these clinical trials, you are on the front lines of developing life-saving treatments for you and future generations. It is not the researchers, physicians, and drug manufacturers that should receive all the credit for pushing the bar. They may be the facilitators, but you are the substance of the process. Michael J. Fox, who suffers from Parkinson’s disease and is a large contributor to research, once said, “Medical science has proven time and again that when the resources are provided, great progress in the treatment, cure, and prevention of disease can occur.” Money and equipment are the traditional resources that come to mind when you think of medical research. However, the real critical resource is the patient.