TCHS Clinical Trials Guide

Clinical trials offer an opportunity for people with medical conditions or people who are interested in new approaches to healthcare to test the latest medical treatments or personal health. If you or someone you know is interested in or considering joining a clinical trial, this free guide will help you better understand how to find a trial, the risks and benefits of joining, the types of trials, and the stages of research you might participate in, patient protections, and how insurance coverage interacts with healthcare during a clinical trial.

What is a Clinical Trial?

Clinical trials are research studies that test new methods of prevention, detection, treatment, and care. The purpose of a clinical trial is to examine the effectiveness of a new intervention and compare it to existing ones. The main questions being asked by researchers are: “Does this work?”, “Is it better than the current standard treatmentStandard treatment – The form of treatment that is normally used and widely accepted. ?Standard treatment - The form of treatment that is normally used and widely accepted. ”, and “What are the harmful side effects?”

Types of Clinical Trials

Clinical trials take on several different forms depending on the information researchers hope to gather. A treatment trial is one of the more popular types of clinical trials among patient volunteersPatient volunteers – clinical trial volunteers with a specific disease or condition that is being studied.. The goal of a treatment trial is to find a better way to treat a condition or disease or broaden effective treatment options. This could include new behavioral interventions, vaccines, drugs, devices, surgical procedures, or a combination of those previously listed. Sometimes new modalities are not tested, rather, an altered sequence or combination of existing therapies is explored. Trials offer some patient volunteers the opportunity to test the efficacy of new medications that might extend or improve their quality of life with disease. In treatment trials, patients may be able to receive access to the most advanced treatment strategies for their disease. Patient participation also increases the knowledge that exists about specific conditions and how best to treat them. Participating trial volunteers value the role they play in advancing medical knowledge. Types of studies include: 

  • Natural history: investigate diseases, and collect information in order to better describe the disease. Tracks the disease and its progression. Natural history studies are mainly used for rare diseases because there is still a lot to learn about them.
  • Quality of life/supportive care: new ways of reducing end of life pain and symptoms associated with disease (drug, device)
  • Treatment: finding new ways to treat diseases. What is the best form of intervention? (drug, vaccine, device, behavioral, surgical, or combination treatment)
  • Screening: finding new ways to screen for and detect diseases. Which tests work better? How early should/can patients be screened?
  • Diagnostic: finding new ways to diagnose diseases. Which test is the most accurate and safe?
  • Prevention: finding new ways to prevent diseases. What actions prevent disease? What supplements prevent disease?


Clinical Trial Phases
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Clinical Trial Phases 

Clinical trials consist of five phases; Early Phase I and Phases I-IV. Each phase is designed to test a different aspect of the new drug, device, or method. Once the new form of treatment has made it through all five phases, it is eligible for review and FDA approval.Each clinical trial investigator must submit an Investigational New Drug (IND) application for review. An FDA review team assess the application for a month to determine whether the trial is safe enough for human subjects to participate. Once the trial is approved, it can begin. At this point, the FDA decides if the new treatment is as safe and effective as existing therapies. If successfully approved, the new product is introduced into the medical marketplace for a specific, approved use (clinical indication). That means many thousands or millions of new users, which can generate new findings that did not appear in the controlled trial. For that reason the new treatment will undergo ongoing testing even after receiving initial FDA approval to ensure it is safe for the general public. 

  • Early Phase I: Early Phase I trials test the initial safety and potency of the new treatment. These trials do not serve a diagnostic or therapeutic purpose. Since the purpose of this phase is to test the limits of the drug and determine how much a person can handle, most of the testing is done on animals rather than human subjects for safety reasons.
  • Phase I: Phase I trials aim to determine how effective the new drug, treatment, or device is and how the human body processes it. Researchers test varying dosages and methods of administering the treatment. It tests the capacity of the new treatment in order to figure out the safest and most effective dosage. Placebos are not used and tests are usually conducted on healthy individuals. Participants are also being monitored for possible side effects during this phase.
  • Phase II: Phase II trials investigate if the new treatment leads to prolonged life and/or better health outcomes. The purpose of this phase is to discover if the treatment worked as intended and if it improved the lives of the participants. Placebos are not usually used for patient volunteers. If a placebo is used, it is combined with the existing standard treatment. Researchers continue to look for adverse side effects.
  • Phase III: In a Phase III trial, researchers compare their new treatment to the existing standard treatment. They look to see whether the new treatment performs better than the current treatments available. In this phase, participants are separated into groups of those who receive the new treatment and those who receive the standard treatment. The new treatment may also be combined with other treatments to test what is most effective. Investigators may begin to request FDA approval during a Phase III trial.
  • Phase IV: In a Phase IV trial, the treatment being studied is one that has recently received FDA approval. Although the treatment has received approval it continues to be studied so that its long term effects can be monitored. If the treatment is found to have adverse side effects it may be taken off the market. Market-based comparisons between the new treatment and standard treatment are also made in this phase. A few of the questions the research may consider in this phase are: which treatment improves the patient’s quality of life? Which is more affordable and cost-effective?

Safeguards for Patient Volunteers 

An area of concern for many patients who are considering treatment in a clinical trial is “being treated like a Guinea pig.” Historical events can certainly reinforce that perception, but they have also fueled the push for comprehensive safeguards in clinical trials. The central tenet of all clinical investigative trials is that the patient remains in control of all decision-making throughout trial participation. The Institutional Review Board (IRB), sometimes known as the Ethical Review Board, is the premier safeguard for research study participants. Any organization that is conducting a study must have an IRB. An IRB can be composed of researchers, health professionals, clergy members, bioethicists, community members, lawyers, and anyone with expertise in the topic of study. Its board members monitor a study from the beginning to the end. They enforce three ethical principles – beneficence, justice, and respect for persons – to ensure the safety and wellbeing of participants. The principle of beneficence requires that the treatment being tested does not harm the participant, and if possible, benefits them as well. Justice promotes equality in the processes of recruitment and treatment distribution. Respect for persons safeguards a participant’s autonomy Autonomy – a patient’s right to make their own health care decisions if they are capable. The patient’s doctor is not allowed to decide for them.. The application of this principle can be seen through the participant’s right to informed consent, to agree to participate, as well as the decision to leave a trial.

Patient Advocate/Representative

A patient advocate is someone who helps a patient navigate the healthcare system. An advocate may have personal experience with being a caregiver or a patient volunteer. Advocates are trained to support and campaign for the patient volunteer. Trust between a patient and their advocate is extremely important because the advocate acts as a liaison between the patient and the patient’s physician, insurer or employer. The advocate can be a family member, friend, or a professional advocateProfessional Advocate - advocates who have been trained and certified to help patients navigate the health care system. Advocates may sometimes have experience as a person with a diagnoses in the health care system.Professional Advocate – Advocates who have been trained and certified to help patients navigate the health care system. Advocates may sometimes have experience as a person with a diagnoses in the health care system.. Most government institutions provide patient advocates (sometimes called a patient representative) for study participants. This can be especially helpful in large institutions because it offers participants a personal contact who can answer their questions about the trial. However if you prefer a personal or Board Certified Patient Advocate (BCPA), one can be found here.

Informed Consent   

Simply because a volunteer qualifies to participate in a clinical trial does not automatically mean it is the best choice for that individual. Before participating in a clinical trial the participant must be given documents, verbal explanations and other information that allow them to clearly understand the purpose, protocols, potential risks, benefits, and processes of the study. Informed consent ensures that patients are knowledgeable about the trial they are participating in. Patients have the right to request information, ask questions, and withdraw from a study at any point. Patients should also be given enough time to discuss their possible participation with their family, friends, or patient advocate/representative.

Transamerica Center for Health Studies® (TCHS) – a division of Transamerica Institute ® (TI) – is focused on empowering consumers and employers to achieve the best value and protection from their health coverage, as well as the best outcomes in their personal health and wellness. TCHS engages with the American public through national surveys, its website, research findings and consumer guidance. TCHS also collaborates with healthcare experts and organizations that are equally focused on health coverage and personal health and wellness. TI is a nonprofit, private foundation funded by contributions from Transamerica Life Insurance Company and its affiliates, as well as unaffiliated third parties. None of the contributors are major medical insurers.

TCHS and TI and their representatives cannot give ERISA, tax, or legal advice, and TCHS is not an agent of any government agency including, but not limited to, state or federal health benefit Exchanges. This material is provided for informational purposes only. TCHS and its representatives are not registered brokers, navigators, applicant assistors, or promoters. Although care has been taken in preparing this material and presenting it accurately, TCHS disclaims any express or implied warranty as to the accuracy of any material contained herein ‘or cited as source material’, and any liability with respect to it.