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What are clinical trials and how do they work?

Clinical trials help deliver potentially life-saving treatments. Image:  Pexels.

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• Clinical trials are designed to determine if a medical, surgical, or behavioural intervention is safe for people.
• There are 420,000 clinical trials, across 221 countries, testing everything from exercise routines to gene therapies.
• Innovative uses of data technologies are improving the speed, costs, and reach of trials.

Scientists are discovering new cures for cancer, Alzheimer’s disease, and other diseases on a weekly basis; or at least that is what news headlines would suggest. Many of these press releases are based on preliminary research in cells or mice, while a majority of novel drugs end up showing no benefit when tested in people. Moving from cells to humans is the start of a highly-regulated and coordinated process called a clinical trial.

What are clinical trials?

Clinical trials are designed to determine whether a medical, surgical, or behavioural intervention is safe and effective for people. Regulators and researchers are continually looking for ways to accelerate the speed and reduce overall costs of clinical trials with the goal of bringing life-saving treatments to patients sooner.

As of September 2022, there were over 420,000 studies registered on ClinicalTrials.gov , a database of privately and publicly funded clinical studies conducted around the world. These studies spanned 221 countries recruiting millions of volunteers worldwide, testing everything from simple behaviours like exercise routines to complex procedures like gene therapies, all following a similar multi-phase process to evaluate their effect on human health.

Discovery to treatment: the clinical trial process

Basic research can often lead to the discovery of new drugs either intentionally or serendipitously. In between basic research and the start of a clinical trial is “ preclinical research ,” which involves lab experiments in cells or animal models to determine whether the drug is useful and if it is toxic before testing in people. This process takes multiple years and ends when researchers submit their data and detailed clinical trial plans to a regulatory agency like the US Food and Drug Administration (FDA).

Clinical trials are conducted in four phases , designed to evaluate a treatment, find the right dosage, and observe any side effects: Phase I usually involves a handful of people and judges a treatment’s safety and side effects; during Phase II and Phase III the number of participants increases and emphasis is given to the treatment’s effectiveness, after which the FDA may grant approval; Phase IV is designed to monitor long-term safety, identifying side effects that might not appear until years later.

While timelines greatly vary, the first three phases of clinical trials can take between 10-15 years to complete, costing companies anywhere from $1-2 million to more than $340 million . This process may appear overly complex for determining something as simple as a drug’s health impact but each step is carefully calculated, including sterile manufacturing practices, ethical patient consent, and clearly defined endpoints and measurements. Yet, even within this strict structure is room to experiment and try out new trial designs, with the goals of improving speed and reducing costs.

Have you read?

8 things you should know about gene therapy, new trial paradigms: increasing speed and decreasing costs.

Approximately 86% of drugs that enter clinical trials do not receive FDA approval. This has significant implications for how pharmaceutical companies approach clinical trials considering that the average total cost of developing a new drug is estimated to be between $2-3 billion . Although clinical trials are highly-regulated processes, innovation in trial design and active engagement with regulators can create flexibility to try new ways of evaluating drugs.

Unlike conventional clinical trials that are fixed and unchanged throughout the study, adaptive trials are flexible and agile, sometimes testing multiple drugs at once . As new data comes in, adaptive trials can drop or add doses, change the trial’s duration, or change the patient populations, which proved critical in quickly responding to the COVID-19 pandemic .

Advances in wearable health devices and AI technologies to gather and store large amounts of real-world, health-related data is impacting the design and execution of clinical trials. Companies are working with regulators to turn this data into real-world evidence , providing new insights into a drug’s safety and long-term effects, which traditional trials may not observe with such detail.

The COVID-19 pandemic has accelerated the adoption of decentralized clinical trials , bringing a proportion of trial activities to the patients rather than bringing patients to a trial site. Virtual technologies and services like telehealth and remote patient monitoring has enabled decentralization, potentially broadening trial access and improving participant diversity , a long-standing issue in drug development.

While momentum is building to improve participant diversity, particularly in the US , for individual clinical trials, reaching communities with some of the highest-disease burdens requires moving clinical trials to other countries.

Making better drugs for more patients

In 2021, the number of trials registered in high-income countries (HICs) was almost 100 times higher than in low-income countries, as reported by the World Health Organization.

Drugs developed in HICs should not be expected to work identically in low- and middle-income countries (LMICs) due to the vast biological diversity across regions. To identify and account for these differences, international collaborations and adaptive clinical trials are being used to create networks of sites across LMICs , helping to pool resources, share expertise, and accelerate data acquisition.

Long-term efforts to build clinical trial capacity (e.g. regulatory systems, manufacturing capabilities) in LMICs will be critical in developing drugs that are safe and effective for specific populations and deliverable within LMIC health systems.

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About Clinical Studies

Research: it's all about patients.

Mayo's mission is about the patient, the patient comes first. So the mission and research here, is to advance how we can best help the patient, how to make sure the patient comes first in care. So in many ways, it's a cycle. It can start with as simple as an idea, worked on in a laboratory, brought to the patient bedside, and if everything goes right, and let's say it's helpful or beneficial, then brought on as a standard approach. And I think that is one of the unique characteristics of Mayo's approach to research, that patient-centeredness. That really helps to put it in its own spotlight.

At Mayo Clinic, the needs of the patient come first. Part of this commitment involves conducting medical research with the goal of helping patients live longer, healthier lives.

Through clinical studies, which involve people who volunteer to participate in them, researchers can better understand how to diagnose, treat and prevent diseases or conditions.

Types of clinical studies

• Observational study. A type of study in which people are observed or certain outcomes are measured. No attempt is made by the researcher to affect the outcome — for example, no treatment is given by the researcher.
• Clinical trial (interventional study). During clinical trials, researchers learn if a new test or treatment works and is safe. Treatments studied in clinical trials might be new drugs or new combinations of drugs, new surgical procedures or devices, or new ways to use existing treatments. Find out more about the five phases of non-cancer clinical trials on ClinicalTrials.gov or the National Cancer Institute phases of cancer trials .
• Medical records research. Medical records research involves the use of information collected from medical records. By studying the medical records of large groups of people over long periods of time, researchers can see how diseases progress and which treatments and surgeries work best. Find out more about Minnesota research authorization .

Clinical studies may differ from standard medical care

A health care provider diagnoses and treats existing illnesses or conditions based on current clinical practice guidelines and available, approved treatments.

But researchers are constantly looking for new and better ways to prevent and treat disease. In their laboratories, they explore ideas and test hypotheses through discovery science. Some of these ideas move into formal clinical trials.

During clinical studies, researchers formally and scientifically gather new knowledge and possibly translate these findings into improved patient care.

Before clinical trials begin

This video demonstrates how discovery science works, what happens in the research lab before clinical studies begin, and how a discovery is transformed into a potential therapy ready to be tested in trials with human participants:

How clinical trials work

Trace the clinical trial journey from a discovery research idea to a viable translatable treatment for patients:

See a glossary of terms related to clinical studies, clinical trials and medical research on ClinicalTrials.gov.

Watch a video about clinical studies to help you prepare to participate.

Let's Talk About Clinical Research

Narrator: This presentation is a brief introduction to the terms, purposes, benefits and risks of clinical research.

If you have questions about the content of this program, talk with your health care provider.

What is clinical research?

Clinical research is a process to find new and better ways to understand, detect, control and treat health conditions. The scientific method is used to find answers to difficult health-related questions.

Ways to participate

There are many ways to participate in clinical research at Mayo Clinic. Three common ways are by volunteering to be in a study, by giving permission to have your medical record reviewed for research purposes, and by allowing your blood or tissue samples to be studied.

Types of clinical research

There are many types of clinical research:

• Prevention studies look at ways to stop diseases from occurring or from recurring after successful treatment.
• Screening studies compare detection methods for common conditions.
• Diagnostic studies test methods for early identification of disease in those with symptoms.
• Treatment studies test new combinations of drugs and new approaches to surgery, radiation therapy and complementary medicine.
• The role of inheritance or genetic studies may be independent or part of other research.
• Quality of life studies explore ways to manage symptoms of chronic illness or side effects of treatment.
• Medical records studies review information from large groups of people.

Clinical research volunteers

Participants in clinical research volunteer to take part. Participants may be healthy, at high risk for developing a disease, or already diagnosed with a disease or illness. When a study is offered, individuals may choose whether or not to participate. If they choose to participate, they may leave the study at any time.

Research terms

You will hear many terms describing clinical research. These include research study, experiment, medical research and clinical trial.

Clinical trial

A clinical trial is research to answer specific questions about new therapies or new ways of using known treatments. Clinical trials take place in phases. For a treatment to become standard, it usually goes through two or three clinical trial phases. The early phases look at treatment safety. Later phases continue to look at safety and also determine the effectiveness of the treatment.

Phase I clinical trial

A small number of people participate in a phase I clinical trial. The goals are to determine safe dosages and methods of treatment delivery. This may be the first time the drug or intervention is used with people.

Phase II clinical trial

Phase II clinical trials have more participants. The goals are to evaluate the effectiveness of the treatment and to monitor side effects. Side effects are monitored in all the phases, but this is a special focus of phase II.

Phase III clinical trial

Phase III clinical trials have the largest number of participants and may take place in multiple health care centers. The goal of a phase III clinical trial is to compare the new treatment to the standard treatment. Sometimes the standard treatment is no treatment.

Phase IV clinical trial

A phase IV clinical trial may be conducted after U.S. Food and Drug Administration approval. The goal is to further assess the long-term safety and effectiveness of a therapy. Smaller numbers of participants may be enrolled if the disease is rare. Larger numbers will be enrolled for common diseases, such as diabetes or heart disease.

Clinical research sponsors

Mayo Clinic funds clinical research at facilities in Rochester, Minnesota; Jacksonville, Florida; and Arizona, and in the Mayo Clinic Health System. Clinical research is conducted in partnership with other medical centers throughout the world. Other sponsors of research at Mayo Clinic include the National Institutes of Health, device or pharmaceutical companies, foundations and organizations.

Clinical research at Mayo Clinic

Dr. Hugh Smith, former chair of Mayo Clinic Board of Governors, stated, "Our commitment to research is based on our knowledge that medicine must be constantly moving forward, that we need to continue our efforts to better understand disease and bring the latest medical knowledge to our practice and to our patients."

This fits with the term "translational research," meaning what is learned in the laboratory goes quickly to the patient's bedside and what is learned at the bedside is taken back to the laboratory.

Ethics and safety of clinical research

All clinical research conducted at Mayo Clinic is reviewed and approved by Mayo's Institutional Review Board. Multiple specialized committees and colleagues may also provide review of the research. Federal rules help ensure that clinical research is conducted in a safe and ethical manner.

Institutional review board

An institutional review board (IRB) reviews all clinical research proposals. The goal is to protect the welfare and safety of human subjects. The IRB continues its review as research is conducted.

Consent process

Participants sign a consent form to ensure that they understand key facts about a study. Such facts include that participation is voluntary and they may withdraw at any time. The consent form is an informational document, not a contract.

Study activities

Staff from the study team describe the research activities during the consent process. The research may include X-rays, blood tests, counseling or medications.

Study design

During the consent process, you may hear different phrases related to study design. Randomized means you will be assigned to a group by chance, much like a flip of a coin. In a single-blinded study, participants do not know which treatment they are receiving. In a double-blinded study, neither the participant nor the research team knows which treatment is being administered.

Some studies use an inactive substance called a placebo.

Multisite studies allow individuals from many different locations or health care centers to participate.

Remuneration

If the consent form states remuneration is provided, you will be paid for your time and participation in the study.

Some studies may involve additional cost. To address costs in a study, carefully review the consent form and discuss questions with the research team and your insurance company. Medicare may cover routine care costs that are part of clinical trials. Medicaid programs in some states may also provide routine care cost coverage, as well.

When considering participation in a research study, carefully look at the benefits and risks. Benefits may include earlier access to new clinical approaches and regular attention from a research team. Research participation often helps others in the future.

Risks/inconveniences

Risks may include side effects. The research treatment may be no better than the standard treatment. More visits, if required in the study, may be inconvenient.

Weigh your risks and benefits

Consider your situation as you weigh the risks and benefits of participation prior to enrolling and during the study. You may stop participation in the study at any time.

Ask questions

Stay informed while participating in research:

• Write down questions you want answered.
• If you do not understand, say so.
• If you have concerns, speak up.

Website resources are available. The first website lists clinical research at Mayo Clinic. The second website, provided by the National Institutes of Health, lists studies occurring in the United States and throughout the world.

Additional information about clinical research may be found at the Mayo Clinic Barbara Woodward Lips Patient Education Center and the Stephen and Barbara Slaggie Family Cancer Education Center.

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• Phone: 800-664-4542 (toll-free)
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• Phone: 855-776-0015 (toll-free)

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• Phone: 507-284-8884
• Email: [email protected]

PART OF CANCER CARE

Clinical trials provide access to potential treatments under investigation, giving options to people who otherwise may face limited choices.

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Learning all you can about clinical studies helps you prepare to participate.

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