Understanding clinical trials

By “clinical trials,” we mean the series of scientific studies carried out in humans that validate the research and development stages in the medicine discovery process.

This crucial step is subject to rigorous regulatory controls, for the purpose of obtaining a marketing authorization and determining the optimal use of a medicinal product to ensure therapeutic benefit for patients.

Clinical trials can last several years and must follow specific organization that complies with stringent research protocols to ensure participant safety.

Clinical research requires scientific rigor

The term clinical research comprises all scientific studies carried out on volunteers in order to develop medicines and medical devices.¹ Clinical trials are usually conducted in a hospital setting or a doctor’s office, under the supervision and responsibility of physicians.

Clinical trials are used to assess the safety, efficacy, and use of treatment in humans so that a new medicine can be made available on the market. They require the application of a rigorous scientific method in order to guarantee both the therapeutic benefit of the proven solution and participant safety. In addition to its therapeutic interest, clinical research also contributes to further developing medical and biological knowledge.²

The four phases of clinical trials

Pre-clinical trial validation is a prerequisite for starting a clinical trial. This phase involves testing the molecular entity on animals before administering the drug candidate to humans.

The development of a new medicinal product typically consists of four successive phases of clinical trials, which aim to observe the effects of the drug in the human body and measure its effectiveness.

• Phase I: Corresponds to the first study in humans and can take several months. The molecule is usually tested in a group of 20 to 100 people (healthy volunteers) to assess tolerance and pharmacokinetics (study of what happens to the drug in the body). This phase also determines the highest and safest dose to administer to patients.
• Phase II: Evaluates the effectiveness of the medicine in a group ranging from a few dozen to a few hundred patients. This phase checks whether the molecule is actively treating the target disease, determines the optimal dose, and measures adverse events.
• Phase III: Often referred to as a “pivotal study.” The molecule is tested more widely on a larger group of patients (up to several thousand participants). In this phase, the benefit-risk ratio of the molecule can be calculated and precautions for use are specified. At the end of this phase, a marketing authorization (MA) application can be submitted to the health authorities.
• Phase IV: Takes place after MA approval. In this phase, the tolerance and efficacy of the medicine are measured over the long-term in real life, because even after numerous tests, certain effects of the molecule may not be fully known.⁴

Currently, only one in ten medicines passes all four phases.³ However, for oncology or a rare disease, the clinical development process of a medicine can be accelerated: there can be fewer patients and certain development phases may be combined.

Pharmaceutical companies, researchers, doctors, and patients all play a part!

Clinical trials generally take several years – sometimes up to ten years – and entail the daily commitment and efforts of numerous stakeholders in order to find new therapeutic solutions that are vital for treating patients.

Most often, pharmaceutical companies initiate clinical trials and are responsible for their funding. All clinical trials are carried out by qualified health care professionals, called investigators. They are in charge ensuring they are carried out properly, and are responsible for the inclusion of patients in the study and for their safety. As for participants, they are always volunteers.

Ensuring transparent results within a stringent legal framework

New challenges ahead

• Digitization of clinical trials: Accelerating the digital shift is a challenge for all health care stakeholders. Digitizing clinical trials can be useful because it facilitates the participation of patients living far from the investigator centers, improving their compliance (e.g., through notifications) and providing easier access to real-time data.
• Patients at the heart of research: As the main players in clinical trials, patients are increasingly involved in the research and development process of pharmaceutical companies. They are in the best position to talk about their disease or condition, its course, and the treatment effects they experience. Involving patients, as early as possible, makes it possible to speed up research and deliver treatments that are best suited to their needs.