Introduction
Healthcare systems and medical facilities are continuously evolving and have exponentially progressed in recent years. Diverse research strategies have been employed to achieve this rampant growth. Clinical trials are the prime propelling force in the development and utilization of new drugs, devices, and treatment interventions. This article navigates through various concepts of clinical trials and helps in a better understanding of the process. Broad knowledge of clinical trial procedures would widen the active participation of volunteers and clear conjuring beliefs related to clinical trials.
What Are Clinical Trials?
Clinical trials refer to research studies carried out in clinical settings, where volunteers (human subjects) are involved in testing or finding potential remedies to proposed health concerns. Generally, these trials are conducted by international or national government regulatory authorities like the United States Food and Drug Administration (FDA). The FDA follows multi-level safety and legal protocols in executing clinical trials. Some of the salient features of clinical trials are as follows -
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Potential therapeutic interventions are tested on selected human participants (volunteers) to assess their effects before approving their use on a wider common population.
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These therapeutic interventions include new drugs, medical devices, biological products such as vaccines, blood substitutes, gene therapy, or immunomodulating agents.
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Surgical interventions, radiological procedures, preventive care, and behavioral therapies also undergo clinical trials.
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Laboratory (preclinical) testing of animal models is mandatory prior to clinical trials. Potential treatment outcomes are first observed in animals before being tried on people to rule out the risk of toxic adverse effects in humans.
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Only those therapeutic interventions with approved safety results and promising profiles are taken to the next level in clinical trials.
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Clinical trials play a crucial role in evidence-based medicine by contributing to testing the working of new medications, diagnostic procedures, and vaccines.
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A large number of individuals are involved in clinical trials to ensure the safety and efficacy of new treatment perspectives.
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Clinical trials primarily involve two groups of participants, namely, the study group (on whom the new intervention is employed) and the control group (not exposed to the new intervention).
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Placebo - In clinical trials, a placebo ( pill, solution, or powder with no therapeutic value) is administered to the control group. The experimental compositions given to the study group are compared with the control group on placebo to determine the effectiveness of the new drug.
What Are the Ongoing Clinical Trials With Promising Breakthroughs?
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Cure for Alzheimer’s Disease - Monoclonal antibody targeting amyloid-β (primary cause for neurodegeneration) to treat cognitive impairment in patients with Alzheimer’s disease.
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Therapy for Metabolic Syndrome and Obesity - Long-term weight-loss intervention effects by minimal-calorie Mediterranean diet along with physical activities and behavioral assistance.
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COVID-19 Vaccination - Efficacy of vaccination in HIV-infected people with COVID-19 illness.
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Gene Therapy for Muscular Dystrophy - Editing of base pairs in mutated genes to repair the defective progenitor cells and rebuild normal muscle.
What Is the Need for Conducting Clinical Trials?
The prime focus of clinical trials is to ensure the safety and efficacy of newly developed drugs and treatment methods. Clinical trials are conducted for the following reasons -
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In order to evaluate whether the new treatment intervention is safe for the people and also provides an effective cure.
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In order to standardize the usage methods such as optimal drug dosage or exposure (radiological therapies) rates for easy and effective use, and also reduce adverse reactions.
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In order to study the effect of already existing drugs on newer study groups like children, pregnant and breastfeeding women, or elderly individuals.
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New drug combinations or concomitant use of two or more therapeutic interventions (chemotherapy with surgical removal of tumors) are also evaluated by clinical trials.
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New surgical approaches (minimally invasive techniques) or enhancing existing surgical techniques with recent advances (robotic surgery) are introduced into clinical practice after clinical trials.
What Are the Stages of Clinical Trials?
Clinical trials are research methodologies that are commonly executed following a step-wise approach. Each stage is precisely designed, accurately documented, and carefully reviewed before initiation and approval. The stages of clinical trials are categorized into the following four phases -
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Phase 1 Clinical Trial - The new drug is tested (first-ever) preliminarily on a small selected group of individuals (also known as subjects) to assess the safety of the dose range and also to detect the occurrence of any side effects.
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Phase 2 Clinical Trial - After passing through phase 1, the drug is tested on a larger population (human subjects) to evaluate adverse toxicities.
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Phase 3 Clinical Trial - Here, the study population is further expanded, involving people from geographically diverse regions or countries. This is the pre-final step before the new intervention goes for final approval.
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Phase 4 Clinical Trial - Phase 4 trials are conducted after authentic approval from regulatory bodies like the FDA. The population involved in the trials is widened, and the duration of the time frame is increased.
Who Can Take Part in Clinical Trials?
Participants are the major determinants of any clinical trial. All individuals, irrespective of age, gender, or any other related factor, could participate in clinical trials. Scientific committees postulate inclusion and exclusion criteria for a particular clinical trial according to the type of study conducted. People usually take part in clinical trials by volunteering themselves after being explained about the studies. Some of the facts regarding clinical trial participation are listed below:
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Healthy individuals taking part in clinical trials are often referred to as volunteers.
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Individuals of all age groups, including young adults and children, are involved in clinical trials.
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Individuals suffering from diseases, especially when there is no existing cure (rare diseases), or failed treatment regimes (cancer therapies) are included in clinical trials.
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People under medication with intolerable side effects (renal toxicity or hepatic toxicity) are also included in clinical trials if the newer drug is found to be well-tolerated.
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Healthcare providers like doctors, nursing staff, and lab technologists participate in clinical trials involving vaccines and preventive therapy. This commonly occurs when there is the emergence of new infectious agents (COVID-19 pandemic), and these individuals are at risk.
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People from diverse geographical regions and racial backgrounds are also included in clinical trials to evaluate the equity of drug response.
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All approved clinical trials frame guidelines for the eligibility criteria of participants.
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Previous medical history, family history, screening tests, laboratory investigations, and diagnostic scans are documented before participating in clinical trials.
What Is the Role of the FDA in Clinical Trials?
FDA closely monitors the clinical trials and ensures the medical interventions are safe as well as beneficial for the common people. All the drug formulae or compositions that underwent preclinical testing are strictly reviewed by the FDA. If the results are not satisfactory, the FDA does not approve them for human testing. FDA is a regulatory organization that does not develop new drugs or products of its own. It overviews and is vigilant about overall drug development processes. The staff and researchers employed at the FDA perform periodic and regular surveillance of sites under clinical trials. FDA protects the rights and privacy of the study participants and also verifies the data quality and integrity of the clinical studies.
Conclusion
Clinical trials are research protocols to study new drugs and treatment procedures and analyze their clinical efficacy and outcomes. People generally volunteer for clinical trials based on the established guidelines. Clinical trials are essential for successful medical advances and therapies. Clinical trials are carefully executed to prevent, identify, and cure diseases.
