Why Clinical Trials Are Important

Learn why clinical trials are important, why we do clinical trials and why their role is necessary for effective treatments and medical advancements.

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Reviewed by Lola Ravid, BSN, RN

Published 20 December 2023

Clinical trials are essential to help medical professionals gain a thorough understanding of human health, helping to bring in new treatments and practices that can improve healthcare across the world. This could be an overall improvement in healthcare outcomes, better treatment of symptoms, or reducing side effects. However, clinical trials are also designed to assess the safety and efficacy of a medical treatment.

In this article, we will outline why clinical trials are important, their different types, benefits, and common misconceptions.

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Types of clinical trials

Clinical trials can be divided broadly into therapeutic and non-therapeutic trials, each of which has a specific function in medical research.

In therapeutic clinical trials the main goal is to assess the safety and efficacy of brand-new interventions or treatments. These studies aim to identify more effective approaches to treat or manage a range of illnesses and conditions by evaluating the efficacy, safety, and ideal dosage of new medications, medical devices, or techniques.

Therapeutic clinical trials may be designed to investigate:

  • New medications
  • Medication combinations
  • Surgical procedures
  • Medical devices

On the other hand, non-therapeutic trials do not offer patients any form of direct treatment, but rather examine important factors that could improve our understanding of treatments and their effects. This can range from screening and prevention to understanding survival rates.

As these trials are not intended to treat patients directly, they instead concentrate on investigating different aspects of health and illness. They may involve understanding the natural progression of diseases, genetic predispositions that contribute to specific conditions, developing new diagnostic tools, comprehending environmental risk factors, or behavioral factors that might have an impact on a person's health.

Clinical trial phases

According to their intended goals, clinical trials are typically divided into a number of phases.

  • Phase 0: The main focus of Phase 0 is to better understand the drug. That includes whether or not it seems to be working the way scientists expect it to. This helps scientists determine if the new drug shows enough promise to study further, saving time and money in later phases.
  • Phase I: In this phase researchers evaluate the safety profile of new drugs or treatments in studies, usually involving 20-100 participants, including those living with the disease, like cancer patients, as well as healthy volunteers. These trials aim to identify the most appropriate and safe drug dosage range, as well as uncover any possible adverse effects.
  • Phase II: These trials represent a significant step forward as they move beyond the initial safety evaluations. In this phase, researchers aim to not only understand the treatment's efficacy but also continue safety monitoring, often enlisting several hundred people to form a more diverse range of participants.
  • Phase III: Perhaps the most important phase of clinical trials, phase 3 trials involve a comprehensive evaluation where a new intervention is compared to the existing standard treatment. The studies conducted in this trial phase further widen the group of participants by opening additional study sites across the country and enrolling several thousand participants with the disease to assess both the treatment efficacy and potential adverse reactions across different demographics, forming the basis for drug approval.
  • Phase IV: Also known as post-marketing surveillance studies, phase 4 clinical trials monitor any adverse reactions, treatment consequences, and negative impacts across diverse populations over an extended period of time.

The various clinical trial phases vary from interventional to observational, with each contributing to our comprehension of a treatment's safety, effectiveness, and best usage.

Other types of clinical trials

Alongside the phased clinical trials, there are also other clinical trial types that contribute to the exploration of drugs and treatments. These are generally divided into two main categories - interventional and observational studies.

These are generally divided into 3 sub-types: preventive trials, screening trials, and quality-of-life trials.

  • Preventive trials assess the most effective techniques for identifying specific diseases or health conditions.
  • Screening trials evaluate the best ways to prevent their recurrence.
  • Quality-of-life trials, sometimes referred to as supportive care trials, explore methods to enhance the comfort and quality of life for individuals with chronic illnesses.

Why clinical trials are necessary

Clinical trials are key to the future of medicine, providing a number of advantages to volunteers who take part in them, particularly when traditional treatments have been unsuccessful.

These trials, which use a varied approach to explore new treatments, form the basis of advancing medical knowledge, guaranteeing that each new treatment meets the strictest safety and efficacy standards, improving quality of life, and catering to the diverse requirements of people around the world.

Safety Assurance

The safety of medical treatments and procedures relies on information obtained from clinical trials and before being released to the public, all treatments undergo rigorous testing to establish their safety profile. This involves identifying potential side effects and their severity, as well as estimating the probability of adverse reactions.

Without comprehensive clinical studies, it would be impossible to determine whether a new treatment is more beneficial than it is harmful.The FDA has high regulations for all clinical studies and requires continuous monitoring during trials to identify and deal with unforeseen side effects.

Assessing the effectiveness of drugs and treatments

It is crucial to guarantee both the safety and effectiveness of a treatment, which is why clinical trials are conducted to evaluate the efficacy of a new drug or intervention and determine if it meets its intended objectives.

By comparing new treatments with existing ones or a placebo, researchers can determine if they are more effective, equally effective, or potentially less effective. This comparison is vital as any new treatment should ideally provide better outcomes or some other significant advantage over the current available treatments.

Determining drug dosages

The level of medication administered can significantly affect the effectiveness of certain treatments. If the dosage is insufficient, the desired outcome may not be achieved, while excessive dosing may result in adverse effects.

Clinical trials help determine the optimal dosage and the frequency of treatment, ensuring that it has the greatest impact with minimal risk. Maintaining this precise balance is essential for both patient safety and the overall success of the treatment.

Improving quality of life

Another goal of clinical trials is to improve the quality of life of patients, particularly for those with chronic conditions, rather than just to cure or treat diseases. Some clinical trials concentrate specifically on this aspect, looking into therapies or interventions that might not be able to heal a condition but can make living with it a lot more bearable and comfortable.

Clinical studies often look into methods to reduce symptoms, ease pain, and improve general well-being, allowing people to live fuller, more comfortable lives despite their medical conditions. Such trials can be extremely beneficial for people whose main objective is symptom management, rather than a full recovery.

Improving decision-making

Clinical trials provide doctors and other medical experts with evidence-based information that enables them to confidently suggest treatments. By analyzing the advantages and drawbacks of a treatment through the extensive and intricate data collected during clinical trials, they can suggest the most suitable options to their patients.

This understanding is necessary in guaranteeing that clinical decisions are backed by scientific evidence and work towards optimizing outcomes for patients.

Understanding disparities

Due to the inherent diversity in the population, the effectiveness of a treatment may vary depending on factors such as ethnicity, gender, age, and other demographics. Clinical trials play a vital role in determining how treatments may differ among different populations. By considering a wide range of variables, these trials help to identify the most suitable treatment responses for various demographic groups.

Taking these various factors into consideration, enables researchers to develop more inclusive and effective treatment methods, while doctors can make better-informed decisions tailored to individual needs by identifying how specific groups may respond to treatment and recognizing any disparities. This will ensure that healthcare solutions are diverse and meet the needs of the entire population.

Facilitating drug approval and market access

As more innovative, secure, and efficient treatments are introduced, they require the support of clinical trials. Regulators like the FDA (Food and Drug Administration) have strict guidelines for approving drugs, ensuring that only secure and efficient medications are released onto the market. 

Accurate and tangible data is needed by these organizations to assess proposed medications or treatments. Without clinical trials and studies, life-saving treatments might be delayed as regulatory agencies lack the foundational knowledge to make decisions, highlighting the importance of gathering evidence collected in a real-world scenario.

Importance of clinical trials

Clinical trials provide insights into what works and what doesn't, allowing medical professionals to better prevent, diagnose, treat, and care for patients. These trials also contribute to the progress of medicine by identifying the causes of illnesses.

Modern clinical trials are often used for benchmarking future care solutions, enhancing the quality of life for patients, while also ensuring that future patients receive consistently effective care.

Development of new treatments

Clinical trials are crucial for developing new therapies as they act as a link between laboratory research in Phase 0 and FDA approval to real-world use. Any new treatment that is released to the public must go through clinical trials to guarantee that it has been extensively scrutinized for quality, safety, and effectiveness, minimizing risks and maximizing advantages.

Therapy areas that commonly require clinical trials include:

  • Cancer
  • Cardiovascular & Circulatory Diseases
  • Digestive Diseases
  • Musculoskeletal Diseases
  • Infectious Diseases
  • Endocrinology & Metabolic-related Disease
  • Nervous System-related Diseases
  • Mental Health & Behavioral Disorders
  • Respiratory Diseases
  • Blood Diseases

Advancement of medical knowledge

Clinical trials provide invaluable insights into the biology and progression of diseases, enabling the medical community to gain a better understanding of complex medical conditions. This deeper understanding can lead to improvements in existing treatments, including changes to dosage and administration.

Additionally, clinical trials can uncover new uses for established therapies, allowing drugs to be repurposed for treating a range of conditions. Principal investigators leading clinical trials help carry out these comprehensive studies to advance or understand fundamental medical principles. Doing so enables better healthcare practices and improved treatment options.

Improving ethical considerations

Clinical studies must always maintain the highest ethical standards, while also ensuring the rights, safety, and well-being of participants are protected. Informed consent is one crucial aspect ensuring ethical standards are adhered to, with participants needing to receive adequate information about the trial's objectives, procedures, potential risks and benefits to make an informed decision about taking part.

A trial must also adhere to strict ethical standards governed by laws, both nationally and internationally, that aim to safeguard the safety, dignity, and well-being of participants. This is done through data and safety monitoring boards, which continually monitor the safety and effectiveness in ongoing trials.

Increasing patient benefits

Individuals who have medical conditions and limited treatment options can get the option to access new and innovative therapies before they become widely available to the public. Participants may receive comprehensive care and often, their health is closely monitored to quickly address any adverse reactions.

Participating in a clinical trial also allows individuals to actively contribute to medical research and aid in the development of potential cures for future patients. 

Positive impact on public health

The impact of clinical trials extends beyond specific treatments and greatly affects the health of the general public. They are important for developing and enhancing vaccines and therapeutic methods, aiding in the management, reduction, and even elimination of diseases that pose significant risk to the global population.

These trials allow key policymakers to make informed decisions by establishing the effectiveness and safety of new medical interventions, helping them to create viable public health strategies and recommendations.

The impact of clinical trials on health care

Clinical trials have a significant impact in terms of life-threatening conditions such as cancer, HIV and Hepatitis C. Numerous cancer therapies that have undergone a trial have given patients a better quality of life thanks to a reduction in symptoms, while many treatments have had impressive success rates in curing a person's cancer altogether.

The outcomes of clinical trials can influence the medical advice given by doctors, change and form new and existing healthcare policies, and increase global immunity against a range of conditions.

Common misconceptions about clinical trials

Clinical trials, despite their importance in advancing medicine, can often be misunderstood, with some people perceiving them as potentially dangerous and possibly exposing participants to untested and risky treatments. Nevertheless, participants are given full transparency of trial details and have the right to disenroll from a trial at any given moment.

Some individuals, such as in the case of cancer patients, may view these trials as a last resort or risky option, while others may worry that they could be used as "guinea pigs." There is also often a belief that clinical trials are not as strictly regulated or monitored as much as standard treatments, but full disclosure of the trial progression and protocol alleviates these concerns.

However, it is important to note that clinical trials are subject to thorough ethical reviews and ongoing monitoring to ensure participant safety, making it crucial to demystify these misconceptions in order to cultivate trust and enhance transparency in the relationship between the public and the medical research community.


Clinical trials are a vital component of medical research and are fundamental to evidence-based medicine, providing healthcare professionals and the scientific community valuable information about the safety, effectiveness, and optimal use of new treatments and interventions.

Without clinical trials, we would have to be forced to rely on anecdotal evidence or uncontrolled observations, which can often be misleading and result in ineffective or even harmful treatments.

Clinical trials also play a critical role in advancing our understanding of diseases by allowing researchers to test theories about disease mechanisms and progression, leading to new insights that can inform future research and treatment strategies.

Additionally, clinical trials provide patients with an opportunity to access potentially beneficial treatments that are not yet widely available.

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