Why Are Clinical Trials Important?

How Clinical Trials Work

The Clinical Trial Protocol

Before a clinical trial begins, the research team, including doctors and staff develops a clinical trial protocol. This is the research plan that describes:

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• Who can join the clinical trial
• Which treatments the participants will get
• Study procedures and tests
• What questions the sponsor wants to answer
• How long the clinical trial will last
• Rules that must be followed
• How participant privacy and safety will be protected

Clinical Trial Volunteer Participants

Clinical research relies on volunteers who help make research happen. Some clinical trials study investigational treatments, such as a drugs, vaccines, or devices in healthy volunteers, but most participants are people with diseases or other conditions who are looking for treatment.

If someone is interested in joining a clinical trial, they meet with clinical research professionals at a clinical trial site. This meeting might be at a hospital or a doctor’s office, or from home through phone and video. Someone from the clinical trial site explains the clinical trial, the treatments, who can join, and what is expected from the participant. This includes how often they will need to visit the clinical trial site and the medical tests they will have. They will also learn what they may need to do at home.

5 Reasons Why Clinical Trials Are Important

1) They Make Potential New Treatments Available to Patients in Need

Clinical trials help develop treatments for people with diseases or conditions who have had few or no options before. For example, in the last decade, gene therapy has given people with rare genetic diseases potential treatment options that target the genetic cause of their disease. Before these advances, treatments often only managed symptoms.

When an investigational treatment enters clinical trials, it is often studied in a small group of participants. As researchers learn more, later-stage trials study the effects of the drug in larger and more diverse groups. The whole process might involve hundreds or even thousands of participants. The data collected allows government agencies to decide if the treatment should be approved for use outside of clinical trials. This means that clinical trial results can affect the medical care and treatment options for many people.

2) Clinical Trials Lead to Better and Improved Treatments

Clinical trials and their participants help raise the standard of care for conditions and diseases. For example, in cancer treatment trials, late-stage trials will often compare an investigational treatment with the current standard of care. For the new cancer treatment to be approved, it must show a meaningful benefit to patients. This might mean better outcomes, treating more symptoms, or having fewer side effects. It might also mean a treatment that is more convenient, such as a new formulation that needs to be taken only once a week instead of every day.

3) They Provide Reliable Information About a Treatment

Clinical trials are a cornerstone of “evidence-based medicine.” They provide the evidence of how well a treatment works and how safe it is, which can help guide patient care. The clinical trial protocol is a detailed plan for the study. It ensures that the trial will be a carefully planned research study of the investigational treatment. It includes predefined outcome measures and objective methods for collecting data. Government regulatory agencies review the trial design to make sure that the:

• Research questions are relevant to patients,
• Results are measured and analyzed properly
• Data are handled properly

Once a new treatment is approved, the data from clinical trials helps health care providers, patients, and the medical community better understand what to expect from that treatment.

4) They Study Both Safety and Efficacy

Many people think the purpose of a clinical trial is to answer these questions:

• “Does the treatment work?”
• “Does it reduce the symptoms or improve the prognosis?”
• “Is this treatment better than the current standard of care?”

The main purpose of many clinical trials is indeed to answer these questions. However, they also check if a treatment is safe and ask:

• Is the treatment associated with fewer adverse events (unexpected medical issues)?
• Is the treatment associated with less severe adverse events?
• Is the treatment associated with fewer serious adverse events that need hospital care?

Clinical trials happen in stages, or phases. During an early phase clinical trial (phase 1), the main goal is to see if the investigational treatment is safe. Investigational treatments will only move to the next stages if they pass certain safety and efficacy standards set in the clinical trial protocol.

5) They Help Prevent Bias

Bias happens when the trial results are influenced by choices that people involved in the clinical trial might make, such as who gets which treatment. In a randomized controlled trial, the effects of an investigational treatment are compared with a control group. The control group might get either a placebo (a treatment that looks like the investigational treatment but has no active medicine) or the current standard of care. If participants are randomly assigned to each group, and the groups are well-balanced, then it is more likely that any differences between the groups are due to the treatments themselves.

Find an Astellas Clinical Trial Near You

Search Astellas Clinical Trials to see if any might be a match for you or for someone you care about.