Many patients may not be aware of the vital role they play in medical discoveries, or that without their participation there would be no medical discoveries. CheckOrphan recently presented a “how-to” article explaining what patients should know if they are thinking of participating in a clinical trial.
About Scientific Research
Personalized medicine is a two-way street. Not only does it entail doctors targeting an individual’s disease, but it also means that patients should get personally involved in their own diagnosis. As an example, it would suggest that the patient not be intimidated when reading their CBC (complete blood count) test results. Hospitals have simplified blood count results for easier comparison.
The Institute of Medicine recommends improving patient engagement and, as an example, making cancer care more patient-centered.
The National Institute of Health (NIH) emphasizes the rights of clinical trial participants governing biomedical research. The NIH encourages research participants to understand, ask questions, give opinions, and communicate their wishes.
What Are Clinical Trials?
Most new medical treatments go through a lengthy research process before they reach patients. Once the efficacy and safety are confirmed, the intervention is ready to be tested with real patients. These studies take place in a series of clinical trials.
Clinical trials can be very expensive. The average cost of a clinical trial required to get a new medication approved by the FDA is nineteen million dollars. In total, it costs around two to three billion dollars to develop and test a new drug.
The purpose of clinical trials is to test, improve or modify health conditions collectively termed “intervention”. That would include therapies, devices used for treatments, diagnostic tests, and medications.
New medical treatments travel a long and complex path toward approval. The process begins in the lab. The proposed interventions must be tested until they reach a point where scientists are able to prove their safety and efficacy. Until such time, the drug (or proposed treatment) may not be tested on humans.
On average, it costs $19 million for FDA approval of a new medication. The total amount for development and testing can run as high as two to three billion dollars. Funding can come from many sources such as the NIH or other government agencies, private foundations, drug companies or treatment manufacturers.
The Four Phases of Clinical Trials
A clinical trial uses certain criteria for recruiting subjects. This would include a specific condition as well as age, ethnicity or gender. Prior to the onset of the trial, the participant will be informed of the risks. The risks will be detailed in the consent document and also explained by a member of the study
Phase O trials are first in the series of studies that are conducted on humans. They are usually experimental. The goal of these trials (involving about ten to fifteen patients) is to determine the effect of the drug and how it is processed in the body.
Phase I trials generally test different doses of a drug to determine the optimum dose with the least side effects. The doctors will begin with a low dose in one arm of the study. Other patients will receive higher doses until either the side effects worsen or the desired effect is reached. If the drug is proven to be safe the study will proceed to a Phase II trial.
Phase II trials will give a further assessment of whether the drug is effective. The number of participants in this phase is generally larger than the number of participants in Phase I. The drug or combination of drugs are usually tested in patients who have a similar type of disease. At this stage, the drugs are still not compared to the most commonly used regimen called “standard-of-care”. If the results of the study show that the drug is effective, the study moves on to Phase III.
Phase III Trials generally enroll over one hundred patients. Usually, the trials are randomized. Patients are put into groups (trial arms) by chance rather than by choice to reduce bias. For example, doctors cannot assign patients to a particular group. A computer program is used for these random assignments.
Generally, there will be a control group that receives the standard-of-care. A second or possibly a third group would receive the new drugs. A comparison is made of the new drugs to the current standard-of-care. Side effects are studied and a determination is made about which drugs are the most effective. If it is determined that the new drugs are more effective then the researchers would apply for FDA approval.
Phase IV trials test the new drugs in hundreds or thousands of patients after they have been approved by the FDA. The size of the study allows a broader view of side effects, efficacy, and whether the drugs can be used with other treatments.
In the long run, millions of people who have had their quality of life improved can thank those who volunteered to participate in clinical trials. These trials have achieved new medical advances now and for future generations.
Have you or anyone you know participated in clinical trials?
