Science is only as good as the evidence it is based upon. Considering the implications of medical science on health, it is imperative that every decision is based on strong clinical evidence. It is this evidence that is provided by the clinical trials and research. Thus NIH describes clinical trials as “research studies that explore whether a medical strategy, treatment, or device is safe and effective for humans” (1).
Importance of Clinical Trials
Clinical trials provide us with proof and ample information about the effectiveness and safety of a particular clinical intervention. Many clinical trials help us identify the better treatment strategy, thus, some clinical trials compare the two treatment methods to find out which one is better and safer.
Clinical trials do not study just the newest medications or interventions; they may be used to study the long-term effects of therapeutic approaches that are already in common use. Take for example hormone therapy, which is currently widely used, but recent clinical trials have found that hormonal therapy increases the risk of heart disease, stroke, blood clots and breast cancer. As a result, long-term use of hormonal therapy is no longer recommended in postmenopausal women (2).
Phases of Clinical Trials
When clinical trials are used to evaluate the application of any device or medicine, they are often divided into phases. Each phase has different objectives and questions to answer. Scientists usually start a clinical trial on humans, after they have found a particular medication or intervention effective in a laboratory or through animal studies. As per the FDA, clinical trials are carried out in four phases, with each phase building upon the knowledge gained from the prior phase (3),. If the trial fails to fulfill its objective at any particular phase, the study may be terminated (4).
- Phase I clinical trial
Generally carried out in a very small group of people (usually 20-100 healthy subjects). The main objective of this trial is to understand safety, and safe dosages in humans.
- Phase II clinical trial
In this phase, the trial is carried out on a larger group of people (several hundred people), and the objective is to know more about the effectiveness of medication or intervention, along with a collection of additional safety data.
- Phase III clinical trial
Once the drug or intervention has been found safe and effective, it is tested on a much larger group of humans (usually few thousand) over the period of few years. If found effective and safe enough, it is given a marketing approval by FDA.
- Phase IV clinical trial
The final phase, involving several thousand individuals suffering from the condition for which intervention or medication is being tested. Tests focus on the efficacy and safety on a much larger scale and in real life conditions.
It is also important to understand that not all clinical trials involve the study of new medications or interventions. For example, the genetic study of a group suffering from a certain disease, or effect of socio-economical status on cardiac health.
Why do people participate in clinical trials?
Everyone has different reasons to participate in a clinical trial. Healthy subjects mostly participate because they think that it is their duty to help science in moving forward. Many people suffering from diseases may participate in a hope of getting better or cured. There are many medical conditions in which there is no effective treatment to date, take an example of HIV infection, different kind of cancers, or even asthma.
Who can participate in a clinical trial is decided by the eligibility criteria of the study. Some trials require the participation of older people, while others focus on younger adults. Some may need healthy volunteers while others with a particular disease condition. Trials may also vary in duration and location where they would be carried out. In some trials, a person may need to travel or stay at the medical facility.
What are the benefits of participation in a clinical trial?
Benefits would essentially depend upon the phase or type of clinical trial. Clinical trials provide a person early access to the latest developments in the field of medicine. A person also gets attention from the best specialists in their field, along with much better diagnostics. Apart from the possibility of direct health benefits, individuals get an opportunity to play a role in the development of science.
How safe are clinical trials?
There is no guarantee that a person would benefit from any particular clinical trial. Some interventions or medications may cause unforeseen and severe side effects.
Therefore, it is important to know more about what is covered by health insurance and what is not. A person is informed about the possible risks when signing the informed consent. It is also important to know that informed consent is not a contract and a person can withdraw from the study at any given time.
What should a person know about the trial?
To understand clinical trials is really difficult for a person without a background in science, thus the official website of the US government for clinical trials has come up with a list of questions that may help a person to know more about the clinical studies. Below is the list of questions one should be asking, before giving consent to participate (5);
- Ask about the purpose the of the clinical trial.
- Ask about the possible benefits and risks from the trial, and if the trial with that particular intervention has been done earlier or not.
- Ask about the interventions involved in the trial, and what a person has to do for the participation, procedures involved, a number of hospital visits.
- Ask about the possible benefits of the intervention being tested, as compared to the treatment that is already being used.
- Ask if the study would require hospitalization or not.
- Ask about the length of the proposed clinical trial.
- Ask about the financial benefits for participation and coverage of participation costs.
- Ask about the long term care provided, after the clinical trial has ended.
- Ask about your options or compensation methods in case of injury.
- What Are Clinical Trials? – NHLBI, NIH. [cited 2017 Sep 10]. Available from: https://www.nhlbi.nih.gov/studies/clinicaltrials
- Why Are Clinical Trials Important? – NHLBI, NIH. [cited 2017 Sep 10]. Available from: https://www.nhlbi.nih.gov/studies/clinicaltrials/important
- What Are the Different Types of Clinical Research? [cited 2017 Sep 10]. Available from: https://www.fda.gov/forpatients/clinicaltrials/types/default.htm
- US FDA. The Drug Development Process – Step 3: Clinical Research. 2017 [cited 2017 Sep 10]. Available from: https://www.fda.gov/forpatients/approvals/drugs/ucm405622.htm
- Learn About Clinical Studies – ClinicalTrials.gov. [cited 2017 Sep 10]. Available from: https://clinicaltrials.gov/ct2/about-studies/learn