Advances in medicine were made possible through clinical research studies. Scientist would have difficulty finding out how to improve medicine if there were no actual tests conducted on humans.
Clinical research studies are performed after successful laboratory tests or animal studies. In this study, the experimental drug must pass a number of stages or phases in order to proceed to the next stage. The purpose of these tests is to determine whether the experimental drug has an acceptable "safety profile" (this means, the potential benefits outweigh the negative impact of an experimental drug). The U.S. Food and Drug Administration (FDA) formulized this system of ensuring the quality of medicine.
To conduct a clinical research study, the following must be done:
- Pre-clinical studies. In this stage you must be able to establish in laboratories a scientific ground for considering a drug as reasonably safe and offering potential benefits and effectiveness.
- Phase 1. This is the initial stage of human testing, enrolling fewer than a hundred volunteers. Your goal in this stage is to find the risks posed and the side effects of an experimental drug on humans.
- Phase 2. In this portion of the clinical research study, look for up to a few hundred individuals having the condition the drug is aimed to treat. This phase will provide further information on the risks posed by the experimental drug. The main focus, however, is the determination of the right dosage of the drug to be applied to an individual patient. You are not hindered from discovering benefits of the drug, however, phase 2 trial is generally too small to allow the extraction of data regarding the benefits of the experimental drug.
- Phase 3. Enlist hundreds or even thousands of volunteers if possible in multiple study sites across the whole country. Your main objective in this trial is to obtain the primary proof for safety and effectiveness that the FDA will take into consideration in deciding whether to approve the experimental drug or not.
- Phase 4. This is also referred to as post-marketing studies, an activity required by the FDA after the drug is approved. In this stage, you should place under observation the health of the individuals who received the drug to obtain further information on long-term safety and the best way to use the experimental drug.
Clinical research studies are not as simple as following all the above-listed steps. You must also see to it that the results of the tests are accurate. It was found out that people at times may feel better and will have improved performance in medical tests, simply because they believe that a treatment is aiding them. Doctors may also feel that treatment is producing a positive result because they care for their patients and want their conditions to improve.
These negative tendencies are eliminated with the aid of the following techniques:
- Placebo-controlled tests. This refers to a method wherein participants are randomly chosen to receive the experimental drug while others will receive the placebo, an inactive replica of the actual experimental drug.
- Double-blinded tests. This means that both volunteers and staff are unaware of who is getting the actual experimental drug and who is receiving the placebo, except for an independent Data Safety and Monitoring Committee that has access to this data.