Clinical research has revolutionized the world of science and medicine. Investigating the safety and efficacy of treatments, diagnosis techniques, medical devices, and prevention techniques is imperative to these interventions’ implementation in humans.
In a field as broad as clinical studies, one might wonder what the best uses for clinical research are. Research is typically very time-consuming and expensive, so knowing where to invest resources is invaluable for successful outcomes.
Broadly speaking, treatment, diagnosis, and prevention are the mainstays of clinical studies.
Clinical Studies and Treatment
Treatment utilizing interventions in healthcare is what most people think of when they think of clinical research. It is the end game for any concept or molecule introduced in preclinical, laboratory-phase research.
Clinical, or in-human, studies are tasked with establishing the safety and efficacy of a treatment in a given disease state. We will discuss exactly why clinical studies are so important in demonstrating an effective treatment below.
Bringing New Treatments to Market
Bringing a new treatment to market is the sole purpose of a large percentage of clinical studies. However, numerous regulatory, ethical, and scientific hurdles exist before an interventional treatment is made to the market.
The United States Food and Drug Administration, or FDA, requires rigorous testing of any new medicine or device before being available for human use. Treatments must be safe and efficacious in their intended use before obtaining the necessary approval.
This process can take many years and sometimes billions of dollars to achieve.
Clinical studies, therefore, are crucial to bringing a treatment to market. Well-thought-out protocol development, patient recruitment, and statistical analysis are required to establish a treatment worthy of market approval.
Generalizability to Many Patients
Treatment generalizability is an essential task for clinical studies to prove. Once a study exhibits safety and efficacy in a disease state, that evidence becomes more valuable by magnitudes when it also applies to other patients who aren’t in the clinical trial.
This metric can be determined when the patient population in a study closely mirrors the majority of the real-life patient population affected by the disease state.
Proper clinical trial recruitment, understandably, becomes imperative to promote trial generalizability. Focusing on the patient and creating patient-centric, well-thought-out protocols while implementing available resources can go a long way towards achieving recruitment success.
Value of Information Gained
Outside of facilitating treatments to market, clinical studies supplement the world of healthcare with invaluable information. When comparing and contrasting different interventions, clinical trials, when done correctly, allow healthcare providers to understand disease states and treatments more comprehensively.
New information can sprout new hypotheses and precipitate further studies. Commonly, incidental findings in studies through sub-group or post hoc analyses can be just as important as the initial, targeted findings.
Safety and Efficacy
The FDA does not approve anything before safety and efficacy are proven in clinical studies. Through Phase 1, 2, and 3 trials, sponsors can prove their drugs and devices are firstly safe and subsequently effective in treating the disease in question.
Safety studies are integral and take priority. Safety studies do not stop after approval, either. Post-marketing studies continue for many years to ensure an intervention is as safe as it was presumed to be at the time of approval.
Similar to the value of information gained from clinical studies, studies produce a significant degree of objective medical evidence that influences the way healthcare providers choose to practice. In turn, studies can reduce the amount of inter-physician bias and result in a society that practices in the realm of proven safe and efficacious medicine.
Healthcare providers may practice outside established medical guidelines and evidence if they feel it is in the patient’s best interests. Still, even these situations prompt new research questions and the opportunity to set up new clinical studies.
Clinical studies are important in understanding possible treatments for special populations, such as children, the elderly, and minority groups. Studies can be explicitly targeted to investigate potential treatments in special populations to gain information about how these populations differ from the general population.
Additionally, studies can use subgroup analysis to determine treatment differences between different populations, such as male versus female response rates.
Some special populations, such as children, need to have their own studies performed for FDA approval of a drug with specific indications for children.
Clinical Studies and Diagnosis
Without an adequate diagnosis, healthcare providers would never be able to sufficiently treat patients safely and effectively. Diagnostic techniques have vastly improved over the previous century, allowing us to detect even the smallest cancer cell and genetic abnormality.
According to the Department of Health and Human Services, or HHS, the FDA will not require laboratory test developers to submit a premarket approval application, premarket notification, or emergency use authorization for laboratory-developed tests as of August 2020.
However, certain tests need FDA approval as these are considered in vitro diagnostic products, or IVD, which are more in line with medical device regulations.
Let’s discuss why clinical studies are so important in approving IVDs, as well as what role diagnostic testing has in clinical research.
In Vitro Diagnostic Products
In vitro diagnostic products, or IVDs, are subject to premarket and postmarket regulation by the FDA. Some examples of IVDs are general-purpose reagents, analyte-specific reagents, and general controls. Anything used for use in the diagnosis of disease to cure, mitigate, treat, or prevent disease is subject to FDA regulatory authority.
IVDs can be Class I, II, or III, which dictates the level of authority the FDA deems necessary to ensure safety and efficacy. Class I includes low-risk general controls, Class II moderate to high risk, and Class III pertains to high-risk controls.
Clinical studies are useful in determining the safety and efficacy of IVDs, similar to drugs and devices used to treat disease. IVDs are also subject to categorization under the Clinical Laboratory Improvement Amendments, or CLIA ‘88, of 1988, which are used to establish quality standards for laboratory testing and an accreditation program for clinical laboratories.
Biospecimens can be used for a variety of reasons. In clinical trials, biospecimens help diagnose and classify diseases, such as identifying what type of cancer a person may have and what cellular characteristics it may express.
Additionally, and just as important, tissue can help monitor therapeutic response, such as if cancer is responding to therapy.
Human tissue specimens have experienced a rapidly increasing importance in recent years. This significance is paralleled with the rise in whole genome and informatics technologies advancements, so it is no coincidence that the demand for human tissue specimens has soared in addition.
As a result, human tissue specimens play a key role in the development of our understanding of the disease, personalized medicine, and targeted therapies.
Clinical Studies and Prevention
Prevention trials and observational studies that inform prevention strategies must be interpreted carefully in public health. Understanding the underlying disease and aspects of the intervention are essential in applying the preventative measure.
Some examples of preventative measures in clinical trials are counseling, vaccines, and lifestyle modifications. According to numerous preventative studies, most coronary heart disease and diabetes, two considerable contributors to morbidity and mortality in western countries, could be prevented by lifestyle modifications alone.
While preventative medicine can be just as or more important than clinical treatment studies, these trials have important considerations.
For example, the long duration of the study required to see a statistically or clinically significant effect can falsely bias study results to the null hypothesis or the hypothesis that there is no difference between the intervention groups.
Additionally, lack of adherence to interventions can have the same effect, such as when study participants fail to maintain a specific diet for the duration of the study.
What Is a Clinical Research Organization?
Clinical research organizations (CROs) are companies that specialize in clinical research and can be immensely helpful in orchestrating a clinical trial. CROs can considerably improve a clinical trial’s efficiency, scalability, and overall success.
While utilizing a CRO is an investment, some CROs can provide global clinical trial support and expertise in the fields of pharmaceuticals, biotechnology, and medical devices that can prove to be invaluable.
CROs can help navigate some of the problems and considerations in clinical studies, such as regulatory concerns and protocol optimization. At iProcess Global Research, you can expect exceptional teams and operations to provide cutting-edge research to help organizations with clinical trials.
The Bottom Line
Clinical studies have identified countless medical, surgical, and behavioral interventions that have transformed healthcare. Prevention, diagnostics, and treatments have all benefited from the rapid expansion and utilization of these studies.
With a constant demand for clinical trials, iProcess continues to meet the demand for specialists who can provide the high level of quality and competence required to facilitate clinical trial recruitment for success.
Contact iProcess to learn more.