COVID brought clinical trial research into everyone’s home — it has made professionals like me, biostatisticians, and epidemiologists a household word.
This is a positive effect, however, there is a slight distortion as to what clinical trial research really involves. First, COVID trials were granted an expedited review process which IS NOT NORMAL and often not awarded. The clinical trial research process can be laborious, methodical, and worthwhile, particularly when you see gratitude from your patients. Let’s review what it takes to conduct a clinical trial.
Clinical research refers to scientific studies or investigations conducted on human subjects to evaluate the safety, efficacy, and effectiveness of medical interventions, treatments, drugs, devices, or procedures. It involves systematically gathering data and evidence to answer research questions, generate new knowledge, and improve healthcare outcomes.
Clinical research is essential for advancing medical understanding, developing new therapies, improving existing treatments, and enhancing patient care. It plays a vital role in evidence-based medicine by providing scientific evidence to support or refute the effectiveness and safety of medical interventions.
Clinical research can take various forms, including:
- Clinical trials: These are structured studies that involve human participants and aim to assess the safety and efficacy of new drugs, treatments, or interventions. Clinical trials follow specific protocols and guidelines, involve a control group or comparator, and often follow a phased approach to evaluate different aspects of the intervention.
- Observational studies: These studies observe and analyze individuals or populations to understand natural history, disease progression, risk factors, and outcomes without intervening or manipulating any variables. Observational studies can include cohort studies, case-control studies, cross-sectional studies, and longitudinal studies.
- Epidemiological studies: These studies investigate the patterns, causes, and effects of diseases within populations. Epidemiological research aims to identify risk factors, establish prevalence or incidence rates, and understand the distribution and impact of diseases in different populations.
- Outcomes research: This type of research focuses on evaluating healthcare interventions and treatments in real-world settings to assess their impact on patient outcomes, quality of life, cost-effectiveness, and healthcare utilization. Outcomes research often uses large databases, surveys, and statistical analyses to gather and interpret data.
- Translational research: Translational research aims to bridge the gap between basic science discoveries and their application in clinical practice. It involves translating laboratory findings into practical applications, such as developing new diagnostic tools, therapies, or medical devices.
Throughout the research process, ethical considerations and patient safety are paramount. Clinical research studies must follow ethical guidelines, obtain informed consent from participants, protect patient privacy and confidentiality, and undergo ethical review and approval by institutional review boards (IRBs) or ethics committees.
Collaboration between researchers, healthcare professionals, patients, and research organizations is crucial for conducting robust and reliable clinical research. The findings from clinical research studies contribute to medical knowledge, inform treatment guidelines, shape healthcare policies, and ultimately improve patient care and outcomes.
Can An OD Get Involved With Clinical Trials?
There are several methods an eye doctor can get involved with clinical trials. A provider can be asked by a pharmaceutical or medical device company to consider studying a new drug or device designed for a specific community of patients.
Clinical Trial Structure
There are three main phases of clinical trials – titled Phases 1 to 3. Some trials have a Phase 0 and may have a Phase 4. Phase 0 usually refers to lab or animal research and phase 4 trials are conducted after a drug or device has received approval from the FDA. These can be also referred to as post-marketing or Investigator Initiated Trials (IITs). Phase 4 studies can often be from a single site and have less requirements.
Phases 1-3 are rigorous phases of a trial, and each phase will have specific and targeted patients. The purpose of each phase is logical, providing information to support the next and expanded phase of a clinical trial. Some examples are:
- Phase I trials test if a new treatment is safe. The number of patients required during phase are very small and often are conducted on healthy volunteers. It is important to understand how a new drug or device behaves in a healthy subject before subjecting a person with the intended disease or condition to the study requirements. In other words, a trial sponsor needs to understand reactions from healthy persons so they can improve the predictions that may or may not occur in the desired study population. Phase 1 trails often require that an acceptable dose is determined. It can often be referred to as a dosing trial.
- Phase 2 trials take the information learned from Phase 1 and apply it to a slightly larger sized population (~100) that has the condition or disease the drug or device is targeting. This phase is generally designed to determine if the product is effective in the targeted population. It is important to isolate the study population regarding co-morbidities at this point until as much variability can be controlled and understood before expanding the size of the study.
- Phase 3 trials are generally expanded to include a more diverse population that may possess other co-morbidities. This is especially important so that the sponsor can understand the confounding other medications may have as well as if there are more side effects. Phase 3 studies can generally require hundreds of subjects.
Ultimately, new drugs and devices are compared to what is considered a “gold standard”. This gold standard is often the product or device considered to be the best solution currently available for commercial use. All phases of a clinical trial are required to have approvals from an IRB (Institutional Review Board) and the FDA (Food and Drug Administration) before commencing any phase of a clinical trial.
This process may sound daunting, but participating in a clinical trial can be very successful for your practice and your patients. Rewards range from providing novel and new research and improved Quality of Life (QOL) for your patients and increasing revenue. One of the best methods to get started in research is by publishing case reports or case series from data you currently have in your practice. Don’t discount the power of a case-report or case-series — these simple research projects are often the advent to the discovery pathway for new drugs and devices.
Written by Lynne Becker
References: Basics About Clinical Trials | FDA
Modernizing Clinical Trial Analytics for Greater Patient Centricity Playbook | SAS
Case Reports, Case Series – From Clinical Practice to Evidence-Based Medicine in Graduate Medical Education – PMC (nih.gov)