Importance of clinical trials and contributions to contemporary medicine: commentary

Abstract

Clinical trials are crucial to contemporary medicine, offering valuable insights into the safety, efficacy, and appropriate use of new treatments. They contribute to public health improvements, reduce healthcare costs, uncover new understandings of disease biology, and facilitate the development of therapies for rare conditions. Investing in clinical trials is therefore essential for advancing medical science and enhancing patient outcomes globally.

Graphical abstract

Clinical trials are essential in the development and approval of new drugs for medical use [1]. These trials offer invaluable insight into the efficacy, safety, and appropriate dosing of potential pharmaceuticals, allowing healthcare professionals to provide patients with the most effective treatment options available. In this Editorial, we will discuss the importance of clinical trials and their contributions to modern medicine.

Firstly, clinical trials help ensure the safety and effectiveness of new interventions. For example, before any drug is approved for use, it must undergo rigorous testing to determine its benefits and possible side effects. This process is necessary to identify any potential risks and to determine the appropriate dosage for the medication. Clinical trials also allow researchers to compare the new medication with existing treatments to determine if it is more effective or has fewer side effects [2].

Secondly, clinical trials can help improve public health by identifying new treatments for diseases that may have previously been untreatable. For example, clinical trials played a significant role in the development of antiretroviral drugs to treat HIV/AIDS [3]. Thanks to clinical trials, people living with HIV can now manage the virus effectively, leading to longer and healthier lives.

Clinical trials are a fundamental component of Evidence-Based Medicine (EBM) [4], which is also supported by systematic reviews and meta-analyses. The quality of meta-analyses often depends on the number of clinical trials they encompass, ensuring that the effectiveness of medical interventions is thoroughly analyzed and verified [5], thereby reducing the risk of biased conclusions. The Cochrane Library plays a key role in this effort by maintaining an updated database of systematic reviews and meta-analyses of clinical trial results, helping healthcare providers stay informed [6].

However, there are some critical challenges in clinical trial research. One major issue is participant recruitment; studies suggest that up to 86% of trials fail to meet their recruitment targets within the designated time frame [6]. Strict eligibility criteria can be a contributing factor to this problem of participant recruitment [7]. Moreover, 1 of 10 cancer trials are prematurely terminated, due to safety and recruitment issues [8]. Additionally, trial discontinuation and the non-publication of results are prevalent issues across various medical conditions and participant types [9].

Thirdly, clinical trials can help decrease healthcare costs by identifying medications that are more effective or have fewer side effects than existing treatments [10]. By providing healthcare providers with better treatment options, clinical trials can help to reduce the need for expensive hospitalizations and procedures, ultimately leading to cost savings for patients and healthcare systems. There is one thing that also needs to be noticed, the benefits of drug repurposing, which involves repositioning and testing existing drugs for the treatment of both common and rare diseases [11]. Recent drug repurposing trials conducted during the COVID-19 pandemic, for instance, evaluated several antiviral therapies, demonstrating its potential for addressing new and emerging health challenges.

Fourthly, clinical trials can also lead to new discoveries about the underlying biology of diseases. By studying the mechanisms by which drugs work, researchers can gain a deeper understanding of diseases and potentially develop new treatments based on this knowledge. For example, clinical trials have led to a better understanding of the genetic mutations that cause certain cancers [12], leading to the development of targeted therapies that are more effective and have fewer side effects.

Finally, clinical trials are essential in developing treatments for rare diseases [13]. Rare diseases affect a small percentage of the population, making it difficult to conduct large-scale studies on new treatments. Clinical trials offer a way for researchers to study these diseases and develop effective treatments, allowing patients with rare diseases to access potentially life-saving medications. While clinical trials are essential for developing treatments for both common and rare conditions, trials for rare diseases pose distinct challenges. Due to the limited number of eligible patients, cohort recruitment is particularly difficult, often requiring broader geographic recruitment or international collaboration. The small sample sizes also raise statistical concerns, making it harder to draw definitive conclusions. Furthermore, the lack of established outcome measures and the variability in disease progression can complicate trial design and analysis.

Recent advances in artificial intelligence could significantly enhance clinical trial practices, particularly in cohort recruitment. Currently, this process is largely manual, requiring medical practitioners to sift through electronic health records (EHRs) to identify potential candidates based on eligibility criteria. However, cutting-edge deep learning and machine learning models have the potential to automate this process, making it more efficient. Additionally, generative artificial intelligence is advancing information extraction techniques, allowing for the aggregation and summarization of clinical evidence from multiple sources [14]. As these technologies continue to evolve, they promise to streamline clinical research processes, ultimately leading to faster delivery of new treatments and improved patient outcomes.

When comparing clinical trials with evidence-based approaches like observational studies, we see that observational studies involve no intervention; instead, they observe outcomes in natural settings by classifying subjects based on existing exposures (such as lifestyle or environmental factors). These studies, which include cohort and case-control designs, are valuable for uncovering associations in real-world contexts, particularly when randomization is impractical or unethical, as in the study of disease risk factors. Nonetheless, observational studies have limitations, including confounding and selection bias, which can affect causal interpretations. While clinical trials provide stronger causal evidence, observational studies offer practical insights into long-term outcomes across larger, more diverse populations. Together, these methods complement each other in advancing medical knowledge: clinical trials emphasize the effects of interventions, while observational studies reveal correlations and implications in real-world scenarios.

Emerging trial designs like platform, basket, and umbrella trials are reshaping clinical research, especially in personalized medicine and oncology. Platform Trials: These trials evaluate multiple treatments for a single disease simultaneously within one overarching trial framework. Treatments can be added or removed based on interim results, allowing for flexible adaptation to new findings without launching separate trials [15]. Basket Trials: Designed to test the effectiveness of a single drug or intervention across multiple diseases or conditions, basket trials group patients by shared molecular or genetic characteristics, regardless of the disease origin. This design is particularly useful in cancer research for targeting specific mutations across various tumor types [16]. Umbrella Trials: Focusing on a single disease, umbrella trials test multiple treatments tailored to various subgroups within that disease [17]. For example, in lung cancer, different therapies may be assigned to patients based on specific biomarkers, making this trial type highly adaptable to personalized treatment approaches. These innovative designs optimize resource use and accelerate data collection, potentially speeding up the development of targeted therapies.

In conclusion, clinical trials play a vital role in modern medicine by providing valuable information about the safety, efficacy, and appropriate dosing of new drugs. They can help improve public health, decrease healthcare costs, lead to new discoveries about the underlying biology of diseases, and develop treatments for rare conditions. As such, investment in clinical trials is essential for continuing to advance medical science and improving patient outcomes around the world.

Funding Statement

This study had received no funding.

Ethics approval and consent to participate

Not applicable.

Consent for publication

Not applicable.

Author contributions

Haofuzi Zhang: Writing—original draft; writing—review& editing. Xiaofan Jiang: Writing—review & editing. All the authors have read and approved the final manuscript.

Disclosure statement

No potential conflict of interest was reported by the author(s).

Data availability statement

Data sharing is not applicable to this article as no new data were created or analyzed in this study.