Clinical trials are an important part of clinical research and are at the heart of all medical advances. They are the primary way that researchers and doctors find out if new treatments are safe and effective and can provide hope and new opportunities for patients who may have exhausted current treatments and medications.

Clinical trials help us understand if a new treatment is more effective or not compared to current standard treatment options and what the potential side effects may be. They may also look at how to improve the quality of life for patients receiving cancer treatment, helping ease the physical pain that comes with a cancer diagnosis and providing relief and comfort to patients and their families.

A clinical trial is a scientific study which involves the organised testing and research of medicines and new treatment options for patients.

The results of a clinical trial can help prove if a certain medicine is safe and effective before introducing as a new treatment for a particular disease or condition.

If initial laboratory research, known as preclinical research, is successful, researchers send the data for approval to continue the research and test its effectiveness in humans.

Once approved, testing of experimental drugs and devices in humans is typically conducted in four phases. Each phase is considered a separate trial and, after completion of a phase, investigators are required to submit their data for approval before continuing on to the next phase.

The drug development process will normally proceed through all four phases over many years. In most cases it takes around 10 years for an experimental drug/treatment to move through all four phases.

Clinical trials are essential to the development, effectiveness and safety of new medical treatments, devices and diagnostic tests.

Medical treatments that are currently available are typically introduced following a clinical trial. Treatments for diseases and certain conditions, such as asthma or heart disease, are researched and developed through clinical research. Clinical trials often lead to new treatments that help people to live longer or with a better quality of life.

Clinical trials can help:

• Detect or diagnose diseases or conditions
• Treat diseases or conditions by testing a new medicine or other medical procedure
• Determine if an existing drug can be used for other conditions or diseases

Phase I trial – Is the drug/treatment safe?

A Phase I trial is the first time a treatment has been tested outside of the research laboratory in a patient and lasts days to weeks.

• These treatments are still experimental in nature but have shown positive results in the early research stage.
• Phase I trials can offer hope and opportunity for our patients with advanced or rare cancers, or where traditional lines of treatment are no longer effective. They can also empower patients to contribute to the future of cancer treatment.

Phase II trial – Does the drug/treatment work?

This phase aims to gather data on if the drug or treatment is effective on those who have the certain disease or condition that is being treated.

• These trials can last for months to years.
• Phase II trials also continue to study the safety of the treatment, including short-term side effects

Phase III trial – Is the drug/treatment better than what is already available?

These trials gather more information about safety and effectiveness through studying different populations and different dosages, or using the experimental treatment in combination with other standard therapies.

• The experimental drug/treatment is administered to a large group of people to confirm its effectiveness compared with standard of care or equivalent treatments
• These trials can last months to years
• If the Department of Health agrees that the Phase III trial results are positive, it may approve the experimental drug or treatment for use in the general public

Phase IV trial – What else do we need to know?

After a drug/treatment has been approved and made available to the public, researchers track its safety to seek more information about their risks, benefits and optimal use through a Phase IV clinical trial.

• Researchers monitor the treatment’s safety through identifying any rare or long-term side effects and evaluating the real-world effectiveness in long-term situations