The author of this article is Dr Niju Pegu, Sr Consultant and Incharge Oncosurgery at Health City Hospitals.
What is a clinical trial?
Clinical trials are research studies in which the safety and effectiveness of new tests or treatments are assessed.
Why are clinical trials needed?
Clinical trials are used for one of the following objectives:
- To learn if the new treatment is more effective, or can cause less side-effects than the standard treatment
- To test new ways to diagnose a disease earlier, at times even before symptoms appear
- To test ways to better prevent an ailment
- To learn how to improve life for individuals living with chronic or life-threatening conditions
- To check the role of support groups and caregivers in mitigating the health consequences
Clinical trials are initiated only when tests on the safety and effectiveness of the therapy on animal models show favorable results. Such trials are known as preclinical studies, and only about one in a thousand potential drugs being tested may enter the clinical trials stage.
What are the advantages of clinical trials?
Advancement in medical knowledge and improvement in patient care is made possible through clinical trials. They are the final step of an extensive process of drug or test approval.
The results of clinical trials provide important and reliable information about safety and efficacy of the new intervention in humans, which cannot be learnt through laboratory or animal studies.
Sometimes they also help doctors to know if the potential benefits of the new treatment can outweigh the side effects of standard treatment. For most of the diseases including cancer, many of the medical interventions have been developed through clinical research.
Often, they lead to new interventions that help people to live longer and better manage their condition.
As a patient enrolled for clinical trials, you may:
- Gain access to a new and possibly effective drug or treatment before it is available to the general population
- Be actively involved in your own health care
- Gain focused care with more frequent health reviews during the treatment
- Understand more about your disease
- Obtain more information about support groups
- Have a chance to help society access better treatment in the future
What are the phases of clinical trials?
Any new medical technique needs to cross four phases of critical evaluation before it is approved for use or made available for the general population.
The rules of these phases are outlined in a protocol and the investigators as well as volunteers participating in the study should abide by the preset guidelines.
This allows researchers to obtain reliable information about the drug or treatment in order to protect patients in each phase of the study.
Listed below are the phases of the clinical trials:
Phase 0: Phase 0 studies are small trials that help researchers know if a new agent works as intended before proceeding to phase I trials. They use only a few small doses of the new drug for a shorter duration in few people, usually less than 15 people.
Phase I: This phase involves testing of new drugs for the first time in a small group of people to determine a safe dosage range, route of administration and to identify side effects. The number of people involved in phase I trials could be around 15-30.
Phase II: Treatments that have been found to be safe in phase I are tested on a larger group of human subjects (less than 100) to know how well it works and monitor for any adverse effects.
Often, the drugs are tested either alone or in combination upon patients with specific types of conditions. The patients are closely monitored and the drug is passed to phase III trials, if it is found to work.
Phase III: Phase III trials involve larger populations (100 to several thousands) from varied religions and countries. They evaluate the effectiveness of the treatment, monitor side effects, and compare it with the standard drug care.
The pooled information about the drug or treatment is used to assess the safety of the drug for use. Hence, phase III trials are often the step preceding treatment approval.
Phase IV: When the treatment is found safe and effective after the first three phases, it is approved by the FDA for clinical use, although they continue to monitor its effects. Hence, phase IV trials are also known as post-marketing surveillance (PMS) studies.
They are extensive studies with longer timeframes, involving several thousands of patients. They provide more information related to side effects, long-term benefits or risks and the efficacy of the treatment when it is used for a wider population.
What is the selection process for clinical trials?
People of all ages can participate in clinical trials, including children. However, clinical trials have precise guidelines about who can participate in any particular study.
Several factors, such as age, gender, medical history, and general health of the person are considered before enrolling a volunteer. This helps to reduce the variation within the study, thereby enabling researchers to obtain reliable data.
Additionally, the potential treatment needs to be tested on individuals the drug or treatment is intended to help.
Also, it is important for the trial to include a diverse population as the response could vary within individuals.
Hence, not everyone who applies, may qualify to participate in the trials. Often the researchers decide whether you may qualify for the study.
From a patient’s perspective, the choice of participation is very personal and solely depends on his/her unique condition. You and your doctor can analyze the benefits and risks, and decide what is best for you.
Sometimes, people may participate in the clinical trials as the standard options haven’t worked, unable to sustain its side effects, or if there is no treatment for their condition. While, others may choose to participate in the trials so as to contribute to society and help the future generations to receive better care.
How can I find a clinical trial?
Earlier clinical trials were available only at major medical facilities. But, today clinical trials are being sponsored by many organizations, such as pharmaceutical companies, federal agencies, or individuals such as doctors or health care providers.
The location of the clinical trial is usually determined by the sponsors which could be universities, medical centers, hospitals or other research sites.
Your doctor is the primary source of information about clinical trials, and it is always worth putting forth your concerns before considering other options. Information about the current clinical trials can also be obtained from groups that provide ways to search for clinical trials.
They can provide reliable information about the study, eligibility criteria for participation and also help to find the appropriate information related to the specific type of disease and the type of the treatment you need.
https://clinicaltrials.gov/ is one such searchable registry that provides information about a trial’s purpose, eligibility, locations, and phone numbers for further details.
What should I find out about the clinical trial before joining it?
Before choosing to join a clinical trial, talk to your doctor about whether the clinical trial is related to your current health condition. You should also speak to the researchers about the potential risks and benefits of participating in the clinical trial.
Informed consent is the process that helps you to know what to expect as a volunteer and all the possible things that might happen during the study.
The patients have all the rights to make a voluntary decision to undergo the treatment or not. As a part of the informed consent process, you will have a chance to ask any questions related to the trial.
Following are some of the queries you may want to ask before enrolling for a clinical trial:
- Why is this clinical trial being done?
- What information are you trying to get from this study?
- What kind of tests and treatments does the study involve?
- Will I know what kind of treatment I am receiving?
- What are the possible risks, side effects and benefits of being a part of the trial?
- Where will the study take place?
- How long will I be in the study?
- What happens if my health condition gets worse while I am in the study?
- How might the study change my daily life?
- What type of long-term follow-up care can I expect?
These questions should help you and your oncologist make an informed decision about whether this clinical study is suitable for you or not.