Clinical trials are critical to new medical treatments’ development and approval process. Researchers conduct these studies on volunteers to determine the effectiveness of medical, surgical, dietary, or any other new treatment method or device.
They use the results to evaluate if it’s better or less harmful than the standard treatment. Trials determine the safety, effectiveness, and side effects of medications or other medical products before they can be made available to the public.
Before a clinical trial begins, it must undergo an extensive review process by regulatory authorities such as the Food and Drug Administration (FDA). An independent review board (IRB) or ethics committee must also approve the trials. This article discusses how clinical trials work, the types of tests, and what to expect if you participate.
How Data Is Handled In Clinical Trials
Data is an essential part of clinical trials as it provides insight into the safety and effectiveness of treatment. All data collected from clinical trials must be carefully documented and securely stored. The Clinical Data Interchange Standards Consortium (CDISC) provides standard definitions of the information reported in a clinical trial. This information includes patient demographics, laboratory values, adverse events, and more.
The CDISC also developed the Analysis Data Model (ADaM). This standard template set allows data to be stored in a standardized format and easily analyzed. It will enable traceability between the source and the analysis data.
Examples of ADam datasets include vital signs (advs), laboratory chemistry data (adlbc), adverse events (adae), and concomitant medications (adcm). These examples of ADaM datasets are used in clinical trials to ensure data accuracy and consistency.
Stages Of Clinical Trials
Clinical trials are divided into four phases, with phase one being the most basic level of testing. Here is a detailed look at each phase:
Phase 1
The first phase is the most basic level of testing a new drug or therapy. It typically involves a small number of healthy volunteers. It focuses on determining if the treatment is safe, how the body metabolizes or reacts to the drug or therapy, and its optimal dosage. Phase 1 takes about 20–80 volunteers and 6–12 months before the next phase.
Phase 2
This phase focuses on determining if the drug or treatment works for its intended purpose. About 70% of the drugs or treatment methods from phase 1 will progress to phase 2. It involves a more significant number of volunteers (typically 100–300) and can take up to two years to complete.
In this phase, the testers consider the safety, short-term side effects, and effectiveness of drug or therapy. It’s aimed at evaluating if the drugs will work on people with certain conditions or diseases.
Phase 3
The third phase usually involves 1,000–3,000 volunteers from various locations. Only about 33% of the drugs will progress to this stage. This phase confirms the effectiveness and safety of the drug or treatment and its long-term side effects, depending on its use in a large population. The stage also evaluates how different populations respond to varying dosages in combination with other drugs. It typically takes up to three years to complete this phase.
Once a drug or treatment passes phase 3, the medication or treatment is submitted to the FDA for approval. The site or institution researching the drug will have to submit the New Drug Application (NDA), a detailed report of the research trial, and its results. The FDA will review the NDA and decide whether to approve the drug.
Phase 4
This final phase is also called post-marketing surveillance or Post-Approval Research and Monitoring. This phase evaluates any long-term side effects from using the drug or treatment. It also looks at how effective it is in a real-world setting compared to the clinical trial setting. This phase can last a minimum of 2 years and involves hundreds to thousands of volunteers.
Types Of Clinical Trials
Clinical trials can be divided into two types: interventional and observational studies. Here are ways these two types differ:
Interventional Studies
In interventional studies, the researchers provide treatment and observe its effects on a group of people. The study is typically randomized, meaning the volunteers are randomly assigned to receive the treatment or be part of the control group (placebo).
The treatment can be in the form of a medical device or a drug and is often compared to standard treatment. The researchers will measure the treatment’s safety, efficacy, or effectiveness by measuring specific outcomes, such as changes in symptoms, quality of life, or mortality rates.
Observational Studies
These studies involve observing the volunteers without necessarily giving them any intervention from the researcher. The researchers perform this by interviewing them and getting information about their health history, lifestyle and environment. The researchers will then analyse this data to look for patterns to help understand the treatment’s effects.
These studies are essential for providing evidence about specific treatments and drugs, which other researchers can use to develop new treatments or drugs or improve existing ones.
Conclusion
Clinical trials are an essential part of the medical research process. They help evaluate the safety, effectiveness, and efficacy of a drug or treatment before the general population can approve it.
In addition, the trials are conducted in several stages and involve thousands of volunteers. These trials provide vital information that helps researchers understand the effects of a specific drug or treatment.
