Why Are Clinical Trials Important?

Clinical trials are critical tools to help us better understand and improve human health and health care. They allow us to evaluate new medical interventions – including new drugs, devices, vaccines, and lifestyle modifications – and identify those that are most likely to benefit patients. They show us what we cannot learn in a lab or in animal testing – how these interventions affect actual people.

How Clinical Trials Work

The Clinical Trial Protocol

Before a clinical trial begins, a research team develops a clinical trial protocol. This is the research plan that describes:
  • Who can join the trial
  • Which treatments the participants will receive
  • What questions the researchers will try to answer
  • How long the trial will last
  • How participant privacy and safety will be protected throughout

Clinical Trial Volunteer Participants

Conducting clinical research – studying what keeps people healthy – relies on clinical trial participants who volunteer their time and effort to help make research happen. Some clinical trials study the effects of a new treatment, such as a drug, vaccine or device, in healthy volunteers, but most clinical trial participants are individuals with chronic illnesses or other diseases who are seeking treatment. If a participant is interested in joining a clinical trial, they meet with clinical research professionals at one of the trial sites. This meeting might be a hospital or a doctor’s office, or it can even from the participant’s home through phone and video. Someone from the trial site explains the trial, the treatments, the eligibility requirements, and the expectations for the participant, including how often they will need to visit the trial site and the medical tests they will have at each visit, as well as what they may need to do at home.

5 Reasons Why Clinical Trials Are Important

1) The Results Can Affect Many More Patients

When a new treatment enters clinical trials, it is often studied in a small number of participants. As more is learned about the experimental treatment, later stage trials study the effects of the drug in larger and more diverse populations. The entire clinical trial process might involve hundreds or even thousands of participants. The data collected from these participants allows government regulatory agencies, , to determine whether the treatment should be approved for use by people outside clinical trials. As such, the results of clinical trials have the potential to affect the medical care and treatment options available to an entire patient community.

2) Clinical Trials Lead to Better and Improved Medications

Clinical trials (and clinical trial participants) are responsible for raising the standard of care for conditions and diseases. For example, in clinical trials of cancer treatments, late stage trials will often compare a new treatment with the current standard of care for the type of cancer being studied . For the new cancer treatment to be approved, it needs to demonstrate a meaningful benefit to patients. This might mean a treatment that improves outcomes, treats a wider range of symptoms, or has fewer side effects. It might also mean a treatment that is more convenient for patients, such as a new formulation that requires use once a week instead of daily. Clinical trials also lead to the development of treatments for people with diseases who historically have had few or no treatment options. For example, in the last decade, advances in gene therapy have given people with rare genetic diseases treatment options that address the cause of their disease on a genetic level. Before these advances, treatment options for many of these patients were limited to treating their symptoms.

3) They Provide Good Information

Clinical trials are a cornerstone of “evidence-based medicine.” They provide the evidence of a treatment’s effectiveness and safety that can be used to guide patient care. The clinical trial protocol ensures that a trial will be a carefully planned research study of the experimental therapy, with predefined outcome measures and objective methods of data collection. The design of a clinical trial is scrutinized by government regulatory agencies in countries where the trial will be held to ensure that research questions are relevant to patients, the results are measured and analyzed properly, and the trial data are handled appropriately. Once a new treatment is approved, clinical trial data give health care providers, patients, and the rest of the medical community a better understanding of what to expect from that treatment.

4) They Test Safety and Efficacy

Many people think the purpose of a clinical trial is to answer the questions, “Does the treatment work?” “Does it reduce the symptoms or improve the prognosis of the disease it is intended to treat?” and “Is this treatment associated with greater improvements than the current standard of care?” The main purpose of many clinical trials is to answer these questions. However, clinical trials are also designed to assess treatment safety. Is the treatment associated with fewer adverse events (unexpected medical issues), less severe adverse events, or fewer serious adverse events that require hospital care? Clinical trials are conducted in stages, or phases. There are 4 phases of clinical trials. During an early clinical trial phase (Phase 1), the main objective is often to assess the safety of a new treatment,. Experimental treatments will only advance to the next stages if they pass certain safety and efficacy standards defined in the trial protocol. Often, independent reviewers, who are part of a Data and Safety Monitoring Board, will review the data and agree that the treatment meets prespecified safety and/or efficacy standards before recommending that the research can advance to the next phase.

5) They Take out a Physician’s Bias

In a randomized controlled trial, the effects of treatment with a new drug are compared with that of a control group – either a placebo (a treatment that looks identical to the new drug but contains no active medicine) or the current standard of care. Treatment assignments are “randomized,” which means that neither the trial doctors nor the participants choose which treatment a participant will receive. Instead, treatment assignment is usually determined by a computer program. The computer program makes sure that the treatment groups are balanced – that the treatment groups are similar in terms of average age, disease severity, and other factors that could affect the trial results. But a given individual’s treatment assignment is decided randomly (by chance) rather than by a doctor’s decision or a patient’s preference. Randomized controlled trials help prevent bias in clinical research. Bias occurs when the trial results are influenced by choices that people involved in the trial might make, such as who should receive which treatment.