Pharma industry

21 January 2025

Clinical trials: phases, methodology and regulations

Clinical trials are a crucial step in the development of new medications. They are conducted to assess the safety, efficacy, and tolerability of treatments before they are introduced into the market.

In addition to adhering to the strictest standards, these rigorous studies aim to ensure that the healthcare products brought to market are both safe for patients and effective.

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Table of contents

1. What is a clinical trial?
2. The different phases in clinical trials
3. Clinical trial methodology: how are they conducted?
4. International regulations and ethical framework for clinical trials
5. The importance of clinical trials for drugs and patients

In addition to adhering to the strictest standards, these rigorous studies aim to ensure that the healthcare products brought to market are both safe for patients and effective.

This article outlines the essentials of clinical trials: the definition of clinical trials, the different phases, as well as the associated methodology and regulations.

1. What is a clinical trial?

A clinical trial is a scientific study conducted on human volunteers to test a medication or treatment.

The primary objective of a clinical trial is to evaluate the efficacy, safety, and potential side effects of a healthcare product, primarily before its commercialization.

Clinical trials differ from preclinical studies, which are conducted in laboratories, often on animals or cell cultures, with the aim of ensuring the safety of progressing to human testing.

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2. The different phases in clinical trials

Clinical trials are structured into several phases, each with distinct objectives.

a. Phase 1 clinical trials: preliminary studies conducted on a small group of healthy volunteers.

Phase 1 is the first step in human evaluation. These trials typically involve 20 to 100 volunteers, often healthy individuals.

The primary objective is to determine the safety of the drug and establish an optimal dosage for subsequent phases. Researchers closely monitor participants’ responses, including side effects, physiological changes, and overall treatment tolerance.

Phase 1 clinical trials are a critical step in ensuring that the drug can be safely administered to patients. These trials take place before the drug receives marketing authorization (MA).

To learn more about marketing authorization (MA), explore our comprehensive guide.

clinical trials for market authorization

b. Phase 2 clinical trials: preliminary evaluation of efficacy

During phase 2 trials, the drug is tested on several hundred volunteers who have the targeted disease. Researchers assess whether the treatment produces the expected effects while monitoring for side effects.

The objective of phase 2 clinical trials is to adjust the dosage to maximize efficacy while minimizing the risks of adverse and side effects.

These drug tests provide an initial indication of therapeutic efficacy and lay the groundwork for subsequent phases.

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c. Phase 3 clinical trials: confirmation of efficacy in a large population

This phase involves several thousand participants recruited from different regions to represent a diverse population.

The objective of phase 3 clinical trials is to confirm the drug’s efficacy and safety. During this phase, trials typically compare the new treatment to a placebo or an existing standard treatment.

The data collected is used to prepare the marketing authorization (MA) application and validate the use of the treatment on a large scale.

d. Phase 4 clinical trials: post-marketing surveillance

Phase 4 trials commence once the drug has been approved and is available on the market.

This phase aims to achieve three objectives:

Monitor long-term effects.
Detect any rare or severe side effects.
Assess the treatment’s impact under real-world conditions.

This ongoing monitoring ensures that the medication remains safe and effective for patients over time.

3. Clinical trial methodology: how are they conducted?

Clinical trials follow a rigorous methodology to ensure the reliability of results. This methodology is based on:

Randomization: Participants are randomly assigned to different groups (placebo group and treatment group).
Double blind design: To avoid any bias, neither the patients nor the researchers know which treatment is being administered.
Inclusion and exclusion criteria: Only participants meeting specific criteria can take part in the different phases of clinical trials.
Statistical analyses: These validate the results and precisely assess the treatment’s effectiveness.

Each step of clinical studies is carefully planned to adhere to scientific standards and ensure participant safety.

ISO 17025 focuses on the competencies of laboratories conducting clinical trials. To learn more about the various ISO standards in the pharmaceutical industry, check out our dedicated article.

ISO standards ensuring medication safety

4. International regulations and ethical framework for clinical trials

Clinical trials are strictly governed by international and national regulations to ensure the safety of volunteers and the reliability of results.

The competent authorities:
- In France: The National Agency for the Safety of Medicines and Health Products (ANSM).
- At the European level: The European Medicines Agency (EMA).
- In the United States: The Food and Drug Administration (FDA) through its Center for Drug Evaluation and Research (CDER).
- In Canada: Health Canada via the Marketed Health Products Directorate (DPSC).
- In China: The National Medical Products Administration (NMPA, formerly CFDA).
- In Japan: The Pharmaceuticals and Medical Devices Agency (PMDA) and the Ministry of Health, Labour and Welfare (MHLW).
- In North Africa : Ministry of Health and Social Protection for Morocco, the Directorate of Pharmacy and Medicines (DPM) for Tunisia, and the National Agency for Pharmaceutical Products (ANPP) for Algeria.
Good Clinical Practices (GCP): These practices dedicated to clinical trials ensure compliance with ethical and scientific standards.
Informed consent: Each participant must receive clear and comprehensive information before agreeing to take part in medication testing.

5. The importance of clinical trials for drugs and patients

Clinical trials play an essential role in improving medical treatments and healthcare products. They ensure that new medications are both effective and safe while also helping to prevent risks associated with unexpected side effects.

For patients, clinical trials offer the hope of accessing innovative treatments, sometimes even before they are available on the market. This is how clinical studies contribute to the advancement of science and the strengthening of trust in pharmaceutical products.

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