Written by: Zeynep Demirag | Edited by: Zainab Bhatti and Nicole Xu
Understanding Clinical Trials
The clinical trial process is how a medical intervention such as a drug, device, or procedure moves from an idea to patient care (Mayo Clinic). Clinical trials are research studies that evaluate new ways to improve treatments and quality of life for people with diseases such as pancreatic cancer. Clinical trials are an option for all patients to consider and play an important role in improving health care. Results from clinical trials help develop new drugs, diagnostic tools, and clinical procedures. They can also evoke a new question for research which can lead to new discoveries over time and help us better understand diseases. All the drugs and medical devices used today were approved for use because patients volunteered for clinical trials.
Every clinical trial has “eligibility criteria” where a patient may need to have a certain medical condition, gender, or other specific quality to meet participation criteria (NIH). According to the beneficence principle of The Belmont Report, patients who want to volunteer need to understand potential benefits and risks (OHRP). Patients may personally benefit from receiving new treatments, therapies, or procedures. They also receive close supervision from clinical staff. On the other hand, patients might risk not benefitting from the new treatment and devoting a large amount of time to participate in a clinical trial. Informed consent is a process that helps patients to make a decision to consent whether to join a clinical trial or not. An informed consent document is provided and explains the purpose, procedures, potential benefits, and risks of the trial.
There are four different phases of clinical trials in which safety is emphasized. If a trial does not have a healthy and safe outcome at any phase, then it will not be allowed to proceed to the next trial phase. Before phase I begins, there is phase 0 where the new treatment has already been found to be safe in animals or healthy human volunteers (NIH). Phase I trials are designed to find the safe dose of treatment, determine how the treatment should be given, and learn how it affects the body. Phase I trials can last several months and approximately 70% of the new drugs move to the next phase (FDA). If a safe dose is found, the new treatment goes to phase II testing. Phase II is conducted with a larger patient group than the previous phase — usually having up to several hundred people. This phase studies how well a treatment or combination of treatments works for a certain type of disease. The length of Phase II can be anywhere from several months to two years, and 33% of drugs usually move to the next phase (FDA). At phase II, the eligibility criteria becomes more restrictive, and phase II studies can be either randomized or nonrandomized. If a new treatment is found to be safe and have benefits, it proceeds to phase III. Phase III trials are conducted in an even larger group of participants, and the length for Phase III can be anywhere from 1 year to 4 years. Approximately 25%-30% of drugs move to the next phase (FDA). Phase III involves a randomized design comparing the new treatment to the standard treatment. If a phase III clinical trial is positive, the new treatment will be approved and licensed for use in the US by the FDA. After the treatment is approved, phase IV clinical trials involve monitoring to continue assessing safety effectiveness, and optimal use.
Clinical trials play an important role in moving science forward and can be a hopeful option for many patients with certain diseases (NIH).
Bibliography:
Office for Human Research Protections (OHRP), OHRP. “Read the Belmont Report.” HHS.gov, US Department of Health and Human Services, 15 Jan. 2018, www.hhs.gov/ohrp/regulations-and-policy/belmont-report/read-the-belmont-report/index.html#:~:text=Beneficence.,to%20secure%20their%20well%2Dbeing.
Commissioner, Office of the. “Step 3: Clinical Research.” U.S. Food and Drug Administration, FDA, www.fda.gov/patients/drug-development-process/step-3-clinical-research.
NIH National Institute on Aging (NIA). “What Are Clinical Trials and Studies?” National Institute on Aging, U.S. Department of Health and Human Services, 2020, www.nia.nih.gov/health/what-are-clinical-trials-and-studies.
Mayo Clinic, “Clinical Trials – About Clinical Studies.” Mayo Clinic, 25 Feb. 2020, www.mayo.edu/research/clinical-trials/about-clinical-studies.