Clinical research is one of the main drivers behind advances in cancer treatment over the past decades. More precise therapies, innovative medications, increased survival rates, and improved quality of life for patients have only become possible because thousands of people voluntarily participated in clinical studies conducted with scientific rigor and ethical oversight.

But do you know how this process truly works? How does a medication go from its discovery all the way to the doctor's office or hospital? And what is the role of patients in this journey?

With the aim of expanding public access to qualified information about clinical research, strengthening patient autonomy, and promoting greater knowledge about studies conducted in Brazil, Instituto Projeto Cura is developing the "Caminhando com Confiança" (Walking with Confidence) project.

The initiative seeks to bring reliable and accessible information about clinical research closer to the public, contributing to a clearer understanding of how new treatments are developed and what rights and precautions are involved in participating in clinical studies. The project aligns with good clinical practices, Law No. 14.874/2024, the General Data Protection Law (LGPD), as well as national guidelines and sectoral benchmarks, including the Interfarma Guide.

The "Caminhando com Confiança" campaign is sponsored by GSK and, in this phase, we will simply explain how clinical research in cancer works and the steps involved until a medication can be made available to patients.

What is clinical research?

Before a medication is approved and made available for use, it must demonstrate that it is safe and effective. To do this, it undergoes rigorous studies that follow carefully planned and monitored stages.

In oncology, these studies typically involve people with cancer and are essential for the advancement of treatments available today and in the future.

Participation in clinical research is voluntary, occurs with the participant's consent, and follows strict safety and ethical protocols.

All studies must undergo ethical and regulatory approvals, and participants receive detailed information about the study through the Informed Consent Form (TCLE), a document that explains the objectives, potential benefits, risks, procedures, and rights of the participant.

The journey of a medication: the phases of clinical research

The development of a medication occurs in different stages. Each phase has specific objectives and generates important scientific evidence leading to potential regulatory approval.

Pre-clinical phase: everything starts in the lab

Before a treatment is tested in humans, it goes through an initial phase called pre-clinical.

At this stage, researchers study substances in the laboratory to understand their therapeutic potential and assess their initial safety. Chemical or biological compounds are analyzed using advanced technologies, artificial intelligence, and experimental models, including living organisms.

The main objective of this stage is to identify which substances have the potential to become promising treatments.

Only compounds that demonstrate consistent results and sufficient safety proceed to human studies.

Phase 1: the first human studies

After promising results in the laboratory stage, the drug can advance to Phase 1, considered the first contact with human participants.

These studies typically involve a smaller number of people, which can range from dozens of participants and, in some more modern research models, can grow as new results emerge.

The main focus of this phase is:

• Define a safe dose; 
• Understand how the body reacts to the treatment; 
• Evaluate tolerability and safety. 

In oncology, this stage usually involves patients for whom conventional treatments no longer yield the expected results.

Today, especially with more personalized therapies, many Phase 1 studies already observe promising responses early on, always with rigorous monitoring of participant safety.

Phase 2: evaluating if the treatment works

If initial safety is confirmed, the study advances to Phase 2, which involves a larger group of participants, typically between 100 and 300 people.

In this stage, researchers seek to better understand if the drug truly works against the disease, in addition to continuing to monitor its safety.

Among the main objectives are:

• Evaluate the treatment's efficacy; 
• Refine or confirm the ideal dose; 
• Monitor known side effects. 

In the context of cancer, this phase can also analyze indicators such as tumor reduction, disease control, or stabilization.

Phase 3: the definitive study

Phase 3 is one of the longest stages in drug development.

These studies typically involve hundreds or thousands of participants, often distributed across different hospitals, research centers, and even countries.

The main objective is to compare the new treatment with the existing therapeutic standard, to assess whether it offers additional benefits in terms of efficacy, safety, or quality of life.

In this phase, randomization may occur, a process in which participants are randomly assigned to treatment groups — similar to a lottery — to ensure neither the doctor nor the patient chooses the treatment, thereby guaranteeing greater scientific rigor and absence of bias in the results.

Another important aspect is the inclusion of diverse populations, which helps understand how the medication works in different patient profiles.

The data generated at this stage is often crucial for regulatory evaluation and eventual drug approval.

Phase 4: Post-Approval Monitoring

Even after a medication is approved and begins to be used, monitoring continues.

The so-called Phase 4, or post-marketing phase, monitors the treatment's behavior in the "real world."

At this stage, patients use the medication within their usual routine, considering daily factors such as diet, lifestyle habits, and other health conditions.

The objective is to:

• Identify rare or delayed side effects; 
• Observe long-term results; 
• Understand how the medication works outside the controlled environment of clinical studies. 

This phase helps reinforce safety and expand knowledge about the practical use of the medication.

The Patient's Role: Participation That Transforms Lives

Patient participation in clinical research is essential for the advancement of science.

Every person who chooses to participate contributes not only to the development of new treatments but also to benefit current and future patients.

In addition to strengthening scientific knowledge, clinical research can provide access to innovative treatments and highly specialized care, always within specific criteria and rigorous protocols.

It is important to remember that participating in clinical research is an individual, voluntary, and informed decision.

Walking with Confidence Promotes Accessible and Quality Information

Understanding how clinical research works is an important step to build trust, reduce misinformation, and empower more informed health decisions.

By promoting qualified and accessible information, the Caminhando com Confiança project, from Instituto Projeto Cura, aims to bring patients, families, and society closer to the world of clinical research, reinforcing its role in advancing oncology and building a future with more treatment possibilities.

Cure Project Institute

Cure Project Institute is a non-profit organization, established in 2016, dedicated to raising public awareness about the importance of clinical research for advancing cancer treatment in Brazil and Latin America.

With the mission to increase awareness and foster research in the fight against cancer, the Institute operates by promoting quality information, social mobilization, and encouraging investment in academic studies. 

Guided by the values of knowledge management, awareness, mobilization, social responsibility, and transparency, Cure Project Institute believes that combating cancer requires joint efforts among society, science, and various sectors, by expanding access to information, education, and innovation in healthcare.

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