Many cancer patients inquire if they’re eligible to participate in clinical trials for their specific cancer. Such was the case with 47 y/o Melanie, mother of two daughters, who was diagnosed with an invasive form of breast cancer. Besides wanting the most up-to-date treatment for her cancer, Melanie was interested in the latest cancer preventive clinical trials which might apply to her daughters. Besides cancer, current clinical trials are investigating, for example, Alzheimer’s disease treatments, diabetes medications, and cardiovascular diseases.
Clinical Trials
Clinical trials are research studies designed primarily to validate the safety, and effectiveness of new drugs, vaccines, medical devices, medical procedures, and treatments. The trials are conducted on humans to determine the usefulness of the therapy under consideration.
The “gold standard” of clinical trials is the double-blind study. The participants are divided into two groups in which neither the researchers, nor the participants know which group is receiving the actual treatment, and which group is receiving a placebo. In this way, the experimental design helps to minimize bias and promotes objective results. There are four phases of the data driven, clinical trials.
Phase 1-Clinical Trials: Safety
The primary goal is to determine if the treatment is safe. A small group of healthy volunteers, or patients (ideally 20-100 people) participate in the double-blind study. If there is any concern about the safety of the individual, the researchers halt the trial.
Researchers want the answers to a few questions: Is the medication/treatment safe? What is the dosage range? What is the best dose? Are there side effects? Are there any interactions with food, or other medications?
Phase 2-Clinical Trials Does the Treatment Work?
Researchers continue to compile data concerning treatment safety in Phase 2. The primary goal in this phase of the trial is to determine whether or not the treatment works for a specific condition. Ideally between 100-300 volunteers are enrolled in Phase-2. Researchers ask, for instance, in cancer patients, is tumor size decreasing as a result of the therapy?
Phase 3-Clinical Trials: Treatment Comparisons
The primary objective of Phase 3 is to compare the experimental treatment to the standard ones. Thousands of patients (300-3,000+) participate in this phase of the clinical trial. Researchers want to know if the new treatment works as well or better than the standard of care for the same problem? Are there fewer side effects?
Phase 4-Clinical Trials: Long Term Monitoring
Researchers study the treatment effects after the drug’s/vaccine’s approval. Is it still effective? What are the side effects? Each phase builds on the last phase to validate, to ensure treatments are safe and effective.
One is reminded of the recent decision by the drug company Moderna to halt research concerning their experimental RSV vaccines in babies. The data from Clinical Trial Phase-1 indicated that the vaccine was unsafe and too dangerous to proceed any further with the experiment. The company stopped the trial; to proceed with the research was unwise, and irresponsible.
In contrast, the data gathered from Moderna’s clinical trials carried out in adults, received FDA approval as being safe and effective. Unfounded opinions, anecdotes, or political winds should not be a substitute for basic medical research. The outcomes of well-designed clinical trials have proved to advance the health and well-being of countless number of lives.
References
- FDA; Basics About Clinical Trials; May 8, 2023
- MD Anderson Center; Phases of Clinical Trials
- Definitive Healthcare; Most Common Clinical Trial Therapy Areas; Jan 2, 2024
This article is intended solely as a learning experience. Please consult your physician for diagnostic and treatment options.