What is Clinical Research?

Clinical research is a type of research involving human volunteers, or participants (healthy or affected by a disease). Its aims are to improve knowledge of diseases, develop diagnostic methods and new interventions and treatments. Interventions and treatments can include:

Drugs: Testing new drugs or medications to develop more effective treatments.
Medical devices: Evaluating devices aimed at enhancing patient care.
Procedures: Investigating new procedures to improve current treatment methodologies.
Vaccines: Studying the efficacy and safety of new vaccines.
Behavioral programs (such as educational, diet or exercise programs): Studying how behavioral changes affect us, leading the way for new therapeutic approaches.

Clinical research must respect a set of precise rules and regulations shared worldwide and must:

Have the goal of increasing medical knowledge,
Take all necessary measures to protect participants – including obtaining their consent to participate,
Follow all the necessary legal and ethical steps.
Be carried out by trained personnel.

Video Source: “What is a Clinical Research Study” by EUPATI is licensed under CC BY-NC-SA 4.0. Available from: https://toolbox.eupati.eu/resources/what-is-a-clinical-research-study/ ‌

Let’s Talk About Clinical Trials for Drugs

Clinical trials for drugs provide the regulatory agencies and the scientific community the evidence needed to decide whether a medication should be widely available to those who could benefit from it. They also help Healthcare Professionals understand which patients are best suited for the medication and how to prescribe it.

These trials help researchers determine if:

The drug helps people feel better.
The drug can improve or prevent the progression of a disease.
The drug is safe to use.
There are any unwanted side effects and how to manage them.
How well people tolerate the drug's side effects.
The optimal dose of the drug to maximize benefits and minimize risks.

Before any new drug reaches your local hospital or pharmacy shelf, it must undergo thorough testing by highly trained researchers and medical professionals using precise methods.

Testing usually begins with animal studies in a lab, known as pre-clinical phase. If these animal studies show promising results, researchers can move on to human volunteers. These human studies are split into different "phases," each designed to carefully evaluate how well the drug works and how safe it is. With continued success, participant groups become increasingly larger in each phase.

Researchers are always seeking new participants to support studies. By joining a clinical trial, people are not only contributing to the advancement of medicine but also quality of life improvements that could benefit others in the future.

Female and male doctor talking to each other

Clinical research is conducted through studies. There are two main types of clinical studies people can take part in: interventional studies (also known as clinical trials) and observational studies.

1Clinical Trials
2Observational Studies

Clinical Trials

In clinical trials, participants receive new specific interventions or treatments designed for a disease or condition and undergo specific medical tests. This allows researchers to assess the impact of these interventions on health outcomes. Clinical trials can also test other aspects of care, such as ways to improve the quality of life for people with chronic illnesses. 

Clinical Trials Phases

Clinical drug trials are conducted in phases, with each phase answering different questions and building on existing insights. At each phase, potential participants receive information from the previous research to help them make an informed decision about joining a study. This step-by-step approach helps deepen and evolve our understanding of the drug's effects.

Phase 1
Phase 2
Phase 3
Market Authorization
Phase 4

Phase 1

Phase I is usually the first time a new drug is studied in human participants. It focuses on safety, determining how the drug interacts with the human body, and identifying any immediate side effects.

Phase I structure

Includes few participants (sometimes less than 10).
Participants are healthy volunteers.
In some cases, participants are patients who have the condition the drug is intended to treat.
Takes about two years to complete on average.

About 58% of drugs successfully advance to Phase II1.

Phase 2

Phase II assesses how the drug works in people with a specific condition and further examines its safety and side effects in a larger group. During this phase, researchers get a better idea of the ideal dosage, known as a dose finding study.

Phase II structure:

Includes a larger number of participants than Phase I.
Participants have the specific condition or disease the drug is designed to treat.
Participants given the drug may be compared with similar participants who receive a different drug, or no drug at all (this is called placebo).
Takes about three years to complete on average.

About 31% of Phase II trials successfully advance to Phase III1.

Phase 3

Phase III is the last stage of research before a drug can be approved for use. It expands evaluation to even larger groups to confirm safety and efficacy, monitor side effects, and compare it to commonly used drugs or a placebo.

Phase III structure:

Involves a larger number of participants.
Participants have the specific condition or disease the drug is designed to treat.
Takes about five years to complete on average.

About 60% of Phase III trials are successful enough to be submitted for regulatory approval2.

Request for marketing authorization

Once a drug successfully passes Phase I, II, and III trials, demonstrating its safety and efficacy for its intended use, the pharmaceutical company can apply to market the drug. Regulatory bodies like the FDA in the US, EMA in the EU, MHRA in the UK, PMDA in Japan, and others worldwide thoroughly examine all data submitted on the drug. Through this evaluation, they can determine whether to approve the drug for public use.

Phase 4

Phase IV trials occur after a drug has been approved for use. The goal is to observe how the drug works as part of people’s everyday lives, rather than in controlled settings like earlier phases. Additional information on safety, benefits, risks, and how the drug affects people's overall quality of life is collected during this phase. This data helps improve guidelines for using the drug, ensure its long-term safety, and demonstrate its effectiveness in real-world scenarios.

Phase IV structure:

Usually includes several thousand participants with a specific condition.
Helps determine the best way to deliver the drug.
Takes many years to complete.

References :

  1. FDA. Step 3: Clinical Research [Internet]. U.S. Food and Drug Administration. 2018. Available from:https://www.fda.gov/patients/drug-development-process/step-3-clinical-research
  2. Wong CH, Siah KW, Lo AW. Estimation of clinical trial success rates and related parameters. Biostatistics. 2019 Apr;20(2):273-286. doi: 10.1093/biostatistics/kxx069.Estimation of clinical trial success rates and related parameters | Biostatistics | Oxford Academic (oup.com)

Woman with a child playing in the fields

Why Join Clinical Trials?

Everyone participating in a clinical trial has their own individual reasons for joining. Some of the most common reasons people join a clinical trial are:

To explore a new option.
To gain increased access to specialized care and have more opportunities to consult with Healthcare Professionals, ask questions, and learn more about the condition or drug.
To keep track of any health changes and respond to them promptly.
To help improve outcomes for future patients with similar conditions.
To contribute to the advancement of medical science.

Anyone thinking about joining a clinical trial should carefully consider the pros and cons. Talk with your doctor to make sure you’re fully informed about the trial. You might discuss how the trial could affect your health, what’s involved in participating, and any other factors that could shape your decision.

Two doctors talking

Clinical Trial Safety

Feeling anxious about joining a clinical trial is completely understandable. Rest assured that all studies follow the highest ethical and safety standards. Each clinical study follows a careful study plan, called a protocol, which describes what the researchers will do. The principal investigator, or head researcher, is responsible for ensuring the protocol is followed.

As a participant, you’ll be valued and provided with excellent care. You’ll also be closely monitored throughout the trial to safeguard your health and the study’s success . Institutional Review Boards or Ethic Committees, informed consent, Data and Safety Monitoring Boards, and Observational Study Monitoring Boards are here to protect to protect volunteers participating to clinical trials.

If you decide to join a study, your participation is voluntary, and you can withdraw at any time if you change your mind; this will not affect your usual care or result in any loss of benefits to which you are otherwise entitled. We encourage you to speak with your healthcare provider first about your eligibility and what is involved

That said, you should consider some potential risks while participating:

You cannot choose the drug you receive, including the dosage level or if you get a placebo.
The new drug being tested may not be better than existing ones, or it could have a different side effect profile.
Taking part in a trial might involve more frequent doctor visits than usual.