Clinical Trials 101 - Your Guide to Endometriosis & Research

Each year thousands of clinical trials occur. Learning about clinical trials can feel like you are learning a new language. The good news is that there are lots of resources to help support and guide decision-making before enrolling in a clinical trial. Check out 10 things to know about clinical trials and participating in trials. Clinical trials are the backbone of treatment breakthroughs Clinical trials allow researchers to understand the impact of a treatment. When it comes to treatments or drugs, clinical trials provide critical information about the drug’s safety and effectiveness. In addition, they can lead researchers down of path to develop other new treatments. Ultimately, clinical trials make a difference not just for today’s patients, but also potentially to those who’ll be diagnosed in the future. Regulatory and ethics groups oversee clinical trials All places that conduct clinical trials have strict ethical duties to participants and regulations they must adhere to. In order to ensure that the ethical and regulatory guidelines are followed there is a team of people who are independent of the research team who are asked to review t and oversee the clinical trial. This team of people is known as an Institutional Review Board (IRB), sometimes known as Independent Ethics Committee or Ethics Review Boards. The IRB must review the research protocol before any participants can enroll in a study. Risks and benefits are different for each person Each clinical trial has different risks and benefits that are explained in the study protocol. Since each participant is different it can mean that each person may experience the risks and benefits differently. For example, it could be possible that one participant may experience a side effect that another participant does not experience. The exact risks and benefits can be difficult to predict since a component of some clinical trials is exploring the impact of a treatment. During the Informed Consent Process, a member of the research team will review the risks and benefits of the clinical trial. There are several key players involved There can be lots of different people involved in clinical trials, but there are key players to know about. Key players include the Principal Investigator (PI), who is the lead researcher leading the clinical trial. There is the trial sponsor, who is paying for research to take place. The research staff, which can include doctors, nurses, social workers or clinical research staff who will work directly with patients. Lastly, patients and their caregivers, without these key players medical advances wouldn’t be possible! Participants are able to leave a trial at anytime If you enroll in a clinical trial and then find that you are no longer able to participate, you are allowed to drop out of the trial at any stage. Trial participants do not have to provide any reason for leaving if they do not want to. If a participant decides that they no longer wish to partake in the clinical trial it is the research team’s responsibility to remove the person from the trial in a way that is safe for the participant. The health and safety of participants is the top priority One of the primary responsibilities of the research team is the care and safety of participants who have volunteered for the trial. Researchers may go by unfamiliar titles, like Principal Investigator or Study Coordinator, but they are, also, doctors, nurses, and trained research professionals. As such, they monitor participant safety and well-being. They will adjust study-related care when they feel it’s in the participant’s best interest. This may mean withdrawing a participant from a clinical trial to prevent harm. There are rules about who can and cannot participate The factors that allow an individual to participate in a clinical trial are called the inclusion criteria. The factors that disqualify an individual from participating in a clinical trial are called exclusion criteria. Inclusion and exclusion criteria are typically based on factors such as age, gender, health status, previous treatment history, and other medical conditions. There is a lot of thought and time put into the development of inclusion/exclusion criteria as these factors may have an impact on the findings of the clinical trials. The inclusion and exclusion criteria are applied to all possible study participants equally before they enroll in the study. Clinical trials occur in various settings Clinical trials could take place at a hospital, a university, a community clinic, or a doctor’s office. In addition to a variety of sites, there are a variety of researchers. Some medical professionals devote their career to conducting clinical trial research, while other research professionals conduct research for some of the time and the remaining time they are providing patient care in a healthcare setting. Evaluating the safety and efficacy of new drugs is a multi-step process When you peek inside your medicine cabinet do you ever wonder the journey that a medication underwent in order to allow it to prescribed by a doctor and land itself in your cabinet? It’s a long journey and thorough one. Every potential treatment undergoes multiple phases of research, each with a different goal. In clinical trials that are studying an interventional drug, treatment, or therapy there is pre-clinical research that occurs in laboratories, usually with cells or animals. If the pre-clinical studies show promising results, the treatment team must get permission from the US Food and Drug Administration (FDA) to test in humans. The research team submits an Investigational New Drug (IND) application to the FDA and must be approved before any next steps. People have lots of different reasons why they participate There are numerous reasons to participate in a clinical trial. Some might decide to participate to gain access to new or alternative treatments. Others may join to have access to personalized and expert medical attention. Some may participate as it can provide access to medications and procedures at no cost to the participant. Some people are motivated to participate to contribute to advancing medicine. Choosing to participate in a clinical trial is an individual decision and each person may have their own personal reasons for deciding to take part in a clinical trial. [lead_gen showIfLoggedIn="0" campaign="shortcode" source="site" title="Sign up for emails from {{site-name}}:"] When many people think of clinical trials, they may think of testing a new experimental drug or taking a placebo pill. But, not all clinical trials involve treatment! Even among clinical trials that focus on treatment, many don’t involve medication. Researchers conduct clinical trials to study and improve many aspects of healthcare. Here are some of the most common types of clinical trials: Treatment trials Treatment trials may involve a variety of different types of treatment, including medication, a combination of medications, or a new surgery or procedure. While some treatment trials compare a group of people receiving a medication to a group who is receiving a placebo (sometimes known as a sugar pill or an inactive medication), that is not always the case! Many clinical trials are being conducted to find new treatments for people with serious illnesses. In some situations, it would be unethical to not provide any type of treatment to a trial participant. Often, trials will compare a group of people taking the current best available treatment option, sometimes called the ‘gold standard’, to a new experimental treatment. In other situations, researchers may be conducting a clinical trial to test new dosages for an already approved treatment. People in these trials would not receive a placebo medication and may even receive a medication that is available to the public (often for free!). Prevention trials These trials look for ways to prevent disease in healthy people or look for ways to prevent a disease from coming back. Some prevention trials focus on non-medical ways to prevent disease. For example, a trial where participants make a certain lifestyle change could be studying prevention efforts. Other prevention trials may use new drugs or therapies to try and prevent disease. For example, a vaccine trial to prevent an infectious disease. Screening trials Clinical trials focusing on screening aim to find the best ways to detect health conditions or disease. It’s important to note that screening is different than diagnosis. Screening looks to detect a disease in someone before they have developed any symptoms, while a diagnosis confirms a disease after symptoms have presented. Screening trials may include different imaging tests, such as MRI or CT scans, or laboratory tests, like blood or urine tests. These trials may also look at genetics to better understand what role inherited genes play in disease development. Diagnostic trials Diagnostic trials study or compare different tests or procedures that are used to diagnose a disease or health condition. For some conditions, there is no single test that is able to confirm a diagnosis. Many people will need to have several different tests to rule out other conditions before being diagnosed. Diagnostic clinical trials can hopefully make diagnosis an easier process for future patients. Quality of life trials Quality of life trials, sometimes known as supportive care trials, do not focus on curing a disease or treating a health condition. Instead, these trials focus on improving the comfort or quality of life of people with different health conditions. These trials recognize that while new treatment breakthroughs are important, overall quality of life is also extremely important to patients. Behavioral trials Trials that focus on behavior aim to compare or evaluate different ways behavior can impact health. While many behavior trials may also focus on prevention, like those looking at diet or exercise, others may aim to better understand how our brains work to shape behaviors. [lead_gen showIfLoggedIn="0" campaign="shortcode" source="site" title="Sign up for emails from {{site-name}}:"],Clinical trials can provide an opportunity for patients to receive the latest treatment, provide a new treatment option for those whose previous treatment didn’t work, and allow patients to be a part of research that can help guide treatment in the future. While these possibilities are exciting, it’s also important to prepare for any costs of treatment. Types of expenses Costs in clinical trials are divided into two categories: patient care costs and research costs. Patient care costs are expenses related to normal care of a patient, including doctor visits, hospital stays, standard treatment, laboratory tests, and imaging tests, like x-rays or magnetic resonance imaging (MRI). These expenses are usually covered by health insurance, although each individual plan has its own deductibles and limits. Research costs are expenses related to the clinical trial, including the study drug or procedure, lab tests solely for research purposes, and any additional imaging tests for research purposes. Insurance won’t cover these costs, but the trial’s sponsor may pay for them. Being a part of a clinical trial usually involves additional doctor’s visits to check on the treatment’s effectiveness and monitor for any side effects. While these visits may be covered by insurance, there may be additional expenses incurred by patients, like transportation costs or child care.1 Plan ahead Before you begin treatment in a clinical trial, talk to your doctor and your insurance to discover what costs will be covered. Some insurance companies require preauthorization before expenses are incurred, and you may need to provide documentation about the clinical trial for them to review. Your doctor or hospital may have someone on staff that can help you, like a financial counselor or research coordinator. These people deal with many insurance companies and are familiar with what materials insurance companies may need to approve expenses, like journal articles about the study treatment or a letter from your doctor. Keep track of any calls you make to your insurance company (including the date and who you spoke with), and keep copies of any documentation you send them. This information may be helpful later on.2 Additional resources For costs that aren’t covered by the research trial or your insurance, there are organizations that may help with financial needs, including: Medicare – This government-assisted program can help pay for costs related to clinical trials. Medicare covers adults in the U.S. over the age of 65. TRICARE – TRICARE is a health benefit program for all uniformed service members and their families. TRICARE has a program for costs related to clinical trials. Veterans Affairs – Eligible veterans can apply for their costs related to clinical trials to be covered by the Department of Veterans Affairs. Your doctor’s office or hospital’s financial counselor may be able to suggest additional organizations that can help in your state. [lead_gen showIfLoggedIn="0" campaign="shortcode" source="site" title="Sign up for emails from {{site-name}}:"] Making decisions can be tough, especially when they relate to your health! One decision you may face is whether or not participating in a clinical trial is right for you. There are several types of clinical trials, and each have different goals and requirements. So, while one clinical trial may not be a good fit for you, there may be others that are. If you’re thinking about exploring clinical trial options but still aren’t sure, ask yourself some questions to determine if a clinical trial might be right for you: Am I willing to work with a new healthcare team during the trial? While you can still see your doctor while enrolled in a clinical trial, you may also be seeing a new group of healthcare professionals who are working on the study. Clinical trials are conducted by a group of researchers. The lead researcher for a trial is called the principal investigator (PI), who is usually a medical doctor or other healthcare professional. The research team may also include other doctors, nurses, and researchers. This research team is often different from the healthcare team you see for your regular care. It’s important to inform your usual healthcare team of the clinical trial you are thinking of participating in and to discuss how the process will work. Am I willing to follow the trial protocol? The clinical trial protocol is a detailed plan of what the trial will involve. When you agree to participate in a clinical trial you will sign an informed consent document, which outlines the protocol and any tests, procedures, or appointments that are expected of you. These expectations may impact your day-to-day life. When reviewing the trial protocol be sure to note when you will need to travel to appointments and how often you may be required to have tests or procedures, like blood draws or imaging scans. You may also be asked to stop taking medications you are currently on while you are in the clinical trial. Keep an up-to-date list of all medications, including over the counter medications, and show this to the clinical trial team to be sure you can continue taking them during the trial. Am I okay with potential unknowns? Some clinical trials are blinded, or masked, meaning that participants will not know what medication they are receiving. Some studies are double-blind studies, meaning both the participant and the research staff will not know what medications a participant is on. This is done to prevent any bias or influence and to allow the participant and researchers to measure the true effect of a drug. In blinded trials, you will be informed of all possible treatments that will be part of the trial, including no treatment (placebo). Before agreeing to participate, it is important to ask yourself if you would be okay with receiving any of the possible treatments, and if you could handle not knowing which treatment you may be given. Am I okay with the possible risks? The research protocol and informed consent document will outline your rights as a clinical trial participant. This document will note the potential benefits for participating in a clinical trial and will also describe potential risks. Clinical research is approved and monitored by an Institutional Review Board (IRB) that ensures any risks are minimal. While the research team and IRB will do everything possible to minimize risks, it cannot always be guaranteed. It is important to take note of the potential risks you may incur and decide if these possible risks are worth the benefits. Will I be okay with the possibility of not being selected? Clinical trials are designed with specific criteria and researchers are often looking for a select group of participants with certain medical histories. These characteristics and medical histories are outlined in the trial’s inclusion and exclusion criteria. One of the main reasons people are excluded from a study is to maintain their safety. For some clinical trials, you may agree to participate and sign an informed consent document, but then be found to be ineligible for the trial after further testing. If you are not selected for a particular trial, this does not mean that you cannot try to enroll in a different clinical trial. There may be other questions you have about clinical trials. You may want to talk with friends, family, and your doctor, or ask the community for their experience. Ultimately, it’s important that you make a decision that you feel comfortable with. [lead_gen showIfLoggedIn="0" campaign="shortcode" source="site" title="Sign up for emails from {{site-name}}:"] Understanding Informed Consent and Your Rights Informed consent is the term that signifies a person has been given information regarding all possible consequences for a treatment, including both potential risks and benefits. While informed consent frequently is applicable in any medical procedure or treatment, it is especially important in clinical trials. The U.S. Department of Health and Human Services has established regulations that require any research trial to obtain informed consent from any human subject.1 Before taking part in a clinical trial, you will be provided with an informed consent form, which includes important information about the trial, including its purpose, how long it is expected to last, any tests or procedures that are expected, and potential benefits and risks (like side effects). The informed consent form must be signed before you can take part in a research trial, and your signature confirms that you have been given and understand the information contained in it. In addition, informed consent continues throughout the research trial, as new information about the treatment becomes available, and you have the right to leave the trial at any time.2 While you may receive benefit for your health condition by participating in a clinical trial, there is also a chance that you will not receive any benefit, and you may be exposed to unplanned risks. Informed consent aims to give you all the information you need to make a decision about participating in the research trial.3 What are your rights in a clinical trial? When you sign an informed consent form, it means you have been given the required information about the trial and that you understand it. Your signature does not bind you to participate in the research trial, and you have the right to discontinue or leave the trial at any time for any reason. In addition to your rights before beginning in a research trial, you have the right to be informed throughout the trial as new information becomes available, such as when new risks or findings emerge during the course of the study. Children who participate in clinical trials also have special protection. In most cases, both parents must provide legal consent for the child to participate, and many trials allow children aged 7 years or older to also provide their agreement to participate.4 Questions you may want to ask As a potential participant in a clinical trial, you will be given the opportunity to ask questions. Some questions you may want to consider include: What is the purpose of this study? What tests or procedures will be performed on me? What are the expected outcomes or benefits for this treatment? What are the possible risks, side effects, or complications? How long is the research trial expected to last? What costs are covered and what costs am I responsible for? What happens after the trial is completed? How might my participation in this trial impact my life? Are there other treatments that are options for me?4 [lead_gen showIfLoggedIn="0" campaign="shortcode" source="site" title="Sign up for emails from {{site-name}}:"]

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