This section of the website was kindly written by Ancora.ai and medically reviewed by Corinne Jordan, MD
Introduction to trials
Clinical trials aim to establish better ways to prevent, diagnose, or treat cancer or manage patient symptoms. Every advance in cancer treatment in recent years has come out of a clinical study. Without clinical trials, new therapies could not be approved, so clinical trials are essential for advancing medical progress.
Yet, only 15% of cancer patients are aware of clinical trials as a treatment option and less than 5% take part in clinical trials. This low participation rate can be explained by many reasons, one of them being the lack of information about clinical studies and the difficulties that patients face when navigating the clinical trials jungle in order to find a study suitable for their needs and preferences.
This lack of information about clinical trials has led me to team up with Ancora.ai. They are a Swiss healthcare technology company with the vision of using technology and a patient-first approach to democratize clinical trial access, so that every patient has the best chances of recovery and leading a healthy life. Ancora.ai has kindly provided a stack of information about clinical trials in order to help you understand your options when discussing your treatment plan with your medical team. Participating in a clinical trial may not be for everyone (whether down to personal choice or on the advice your medical team), but having the information in order to ask more questions and make an informed decision is really important. And remember, like everything that relates to your treatment, it’s always important to discuss it with your oncologist/medical team.
This page contains a lot of information because there is a lot to say on the topic. This page will cover:
- The difference phases of clinical trials
- Facts and figures on trials
- How do I find a trial I am eligible for?
- How often and how far will I have to travel?
- What are the goals of the trial and do they match what I’m looking for?
- What does the informed consent document say?
- What about cost?
- Will I receive any follow-up care after the trial is over?
- What is the difference between an interventional and an observational trial?
- What are the different types of treatments that can be investigated in a trial?
- Why do some drugs have bizarre or multiple names?
In addition to the wealth of information on this page, you can find more information on this site in the following locations:
- Introduction to clinical trials
- A list of questions to ask if you are considering taking part in a trial
- A list of further resources where you can find out more about clinical trials
And please also visit www.ancora.ai for more information about clinical trials and their interactive database where you can find details of clinical trials that might suit you, and which you can discuss with your medical team.
Clinical trials have different phases
Before we go on to talk about the process for enrolling in a trial, there are a few introductory remarks we need to make about clinical trials. Clinical trials usually consist of 3 phases, each with a different purpose. A trial only moves to the next phase if the previous phase is successful based on pre-approved criteria. Each type of clinical trial has pros and cons and it is important you make an informed decision before enrolling in one.
The main goal of phase 1 clinical trials is to determine what dose of the drug can be given safely without provoking serious side effects. Additionally, researchers look for signs that the disease responds to the treatment. These studies usually include a low number (20 to 100) of healthy volunteers or people with the disease/condition and usually last several months to a year. The drug dosage is slowly increased for the clinical trial participants and side effects are closely evaluated. Approximately 70% of drugs in phase 1 move to phase 2.
Once a dose or range of doses of a new medication has been determined to be reasonably safe in phase 1, a phase 2 clinical trial can then be initiated in order to test the efficacy of the treatment. Depending on the type of treatment, doctors might look at a different type of benefit or response. For example, they could look at whether cancer shrinks or disappears, or if there is a longer period of time before cancer comes back. Sometimes, quality of life is also evaluated. Often it is determined if the medication allows patients to live longer than they would have without the test medication. Usually, phase 2 studies recruit between 25 and 300 patients who are suffering from the same type of illness and the studies last for about 2 years. Approximately 33% of drugs in phase 2 move to phase 3.
If a treatment has been shown to work without harmful side effects in a phase 2 study, it will be further tested in a phase 3 clinical trial before it can be approved for general use and sold on the market. The purpose of phase 3 studies is to evaluate how well a new treatment works. Participants are usually randomized and either get the new treatment or the standard of care. Both patients and doctors are usually blinded, meaning none of them will know which treatment each participant is receiving. This reduces bias in the study. Phase 3 studies are larger and involve up to 3,000 people. These phase 3 clinical trials can last for several years. Approximately 25 to 30% of drugs in phase 3 are eventually approved.
Facts and figures on trials
- Currently (as at the day of writing this text in July 2020) there are 2,352 breast cancer clinical trials underway and these studies need to recruit 2 million patients in order to be completed.
- Only 3% of breast cancer patients join a clinical trial, while half or more could be eligible for one.
- Minorities are currently underrepresented in clinical trials, and this is a problem because it means access to innovative medicines is not equal and also that research to make sure medicines are safe and effective in different populations is not getting done.
- A main barrier to participation in clinical trials is lack of awareness among physicians and patients of clinical trials as an option.
- Physician recommendations are a main reason patients pursue trials, but only around 20% of patients hear about trials from their doctors.
- Breast cancer trials almost never use placebos. The treatment being studied will always be compared to a standard of care treatment.
- If you choose to participate in a clinical trial you can ALWAYS change your mind and are free to stop at any time – a critical part of research ethics!
If you are interested in exploring clinical trials, here is a list of top 10 things patients and their loved ones should ask when they are looking for a trial:
Who can participate in a clinical trial?
- How do I find a trial I’m eligible for?
In order to find a clinical trial, you have the possibility to use governmental registries such as Be Part of Research (NHS), ClinicalTrials.gov or the EU Clinical Trials Register, where you can find studies conducted in a chosen location and for your specific disease. However, most patients find it difficult to navigate these websites as they are primarily made for researchers, scientists, and doctors. You might find it overwhelming to have to go through a very long list of clinical trials as there are many in breast cancer.
To know if you may be eligible to participate in a trial, you have to check the inclusion and exclusion criteria that make up “eligibility criteria” in each trial listing. This is where the profile of patients the trial is looking for is described.
- The inclusion criteria are a list of characteristics that allow a person to enlist in a trial. For example, some studies will require that you are not older or younger than a certain age, or that you have a specific genetic mutation.
- The exclusion criteria, on the other hand, are all the factors that would disqualify you from participating in a study. For example, you might not be eligible to enter a specific study if you are taking other medications or if you are pregnant.
Going through the numerous eligibility criteria written in medical jargon can be time consuming and confusing. Some patients find it easier to identify clinical trials matching their needs and characteristics by using a clinical trial search tool, such as Ancora.ai. This website will help you narrow down a list containing only the trials you are potentially eligible for. The search tool will ask you a list of specific questions around your health status, age, gender, and previous treatments and will automatically filter down to the trials that could be suitable for you. In order to obtain the most appropriate results, we recommend that you write down any questions you are unsure about and ask your doctor for input. If you are still unsure about whether you qualify for a study, you can ask to be put in contact with the study coordinator, who can then help you assess your eligibility.
Ancora.ai also offers additional features, such as the possibility to save all the trials of interest to you in a dashboard, so that you can easily come back to them anytime in the future and get notified of updates, such as if the trial starts or stops recruiting. Moreover, you can also directly share a trial listing with someone else, such as a family member or your doctor, by entering their email address. Ancora.ai will also automatically send you a notification to your inbox whenever a new trial you are eligible for is opened, that way you can be sure you don’t miss any new developments.
2. How often and how far will I have to travel?
Traveling to a clinical trial site can be an unexpected challenge for many patients, especially if you are already feeling unwell because of your disease or side effects caused by treatment. In order to limit the distance you will be required to travel for treatment, you can check if the trial of your choice is available locally. To help you with that, most clinical trial search tools offer an option to narrow down to studies that are located near your home.
It is also important that you ask the trial coordinator how often you will be required to travel to the trial center. Also, keep in mind that phase 3 studies are generally conducted in multiple trial sites, while phase 1 studies are usually more restricted in the number of trial locations. Sometimes, travel costs will be reimbursed by the trial sponsor, however, it is best to verify this is the case by asking the trial staff.
In most cases, when you participate in a trial you will need to go to the trial site for some aspects of your care associated with the trial, but you will still also see your own oncologist and some aspects of your care will be handled as usual. You should ask the trial coordinator how they plan to cooperate with your normal physicians so this is clear to you and your doctor before deciding to participate.
3.What are the goals of the trial, and do they match what I’m looking for?
You will find this information in the study protocol, which is a written plan describing how a clinical trial will be conducted. It is important that you have a clear picture of the differences between the new treatment being tested and the other established standard-of-care treatments that you might otherwise be prescribed. You should also be aware that the new drug might not work as planned.
Moreover, you should make sure you understand what is being studied in the different phases (see above) and decide which one is the most appropriate for you. For example, if you are concerned about side effects, it might be better for you to enroll in a phase 3 trial, as the most common side effects might already have been discovered in the previous phases. On the other hand, since phase 2 and 3 are conducted to investigate if a new experimental drug is better in comparison with an approved drug, you might end up not receiving the new treatment, but rather receive an established standard-of-care treatment instead. Many studies are blinded, meaning you will not know whether you are receiving the experimental treatment or another standard-of-care treatment.
In some trials, a group of patients will receive a placebo (a treatment with no therapeutic value), however, for ethical reasons, this is very rarely the case in breast cancer trials.
While searching for a clinical trial, you will often find bits and pieces of the information mentioned above in the clinical trial listing available online, however, the amount of detail provided varies widely across studies. Make sure you ask the study coordinator or your doctor if anything is unclear to you.
For any clinical trial, researchers are required to submit a study protocol describing in detail how a clinical trial is to be conducted. This protocol is then reviewed by an institutional review board (IRB) that will approve or reject the study of an experimental treatment.
The trial protocol includes the following information:
- Why the study is being conducted and what researchers hope to learn from the study
- What kind of treatment will be administered (e.g. name and dosage of drugs, results of previous studies conducted with the same treatment regimen)
- Which phase of the study is being conducted and how many people will be enrolled
- Who is eligible for the trial (inclusion and exclusion criteria)
- How the treatment will be given
- What tests will be done during the study and how often they’ll be done
- Other information that will be collected on participants
- How long the study will last
4. What does the informed consent document say?
As a patient, once you decide to get involved in a clinical trial, you will need to provide voluntary consent by signing a written consent document. This is a legal document that states that you have received information about your treatment options and that lets your doctor go ahead with the treatment plan for the trial. By signing the informed consent form, you agree that you or your representative fully understand the parameters of a given trial and are willing to participate in the study. Remember, however, that it is your right to leave the study at any time and for any reason.
Informed consent is a process through which you learn details about the trial by asking questions and speaking with the research team, which is made up of doctors and nurses, before deciding whether to take part.
You should make sure you understand the following points:
- The purpose of the research
- How long it is expected to take
- What procedures will be performed on you during the trial
- The risks and benefits that can be expected while participating in the trial
Additionally, the consent form should clarify the following information:
- A statement saying the study involves research
- Your participation as a research subject is made on a voluntary basis
- Any possible discomfort (e.g., injections, frequency of blood tests, etc.)
- Any alternative procedures or treatment (if any) that might benefit the research subject
- How the participant’s information will be kept private during the clinical trial
- Whether any compensation (payment) or medical treatments are available if an injury occurs and where that information may be found
- The research subject’s rights; such as the right to refuse treatment or stop participation in the clinical trial at any time, without losing any treatment benefits
- Contacts for answers to questions related to the clinical trial and to report any injuries that may occur
5. What about cost?
It is important to clarify any costs and insurance questions before enrolling in a clinical trial. In most cases, the trial sponsor will provide the treatment at no cost and will pay for any necessary doctor visits, special tests, and procedures. Some trial sponsors might also offer to pay a fee for transport and travel time. Typically during a trial some of your check-ups and care will still be handled by your normal oncologist and physician team. You should make sure that you and your doctors understand how the trial team plans to collaborate on your care so that things run smoothly and you know what to expect.
There may be extra costs that are not part of the treatment, like transportation and accommodation costs, that you should also take into account before accepting to participate in a trial. It is very likely that you will have to commute to get to your trial site. Sometimes trial sponsors cover these types of costs and for the time you spend traveling. It could be either directly paid by the sponsor, or in the form of vouchers or fixed stipends.
Since costs coverage and reimbursement vary from one study to another, make sure you clarify all these points with the clinical trial coordinator, your insurance provider and your doctor, before you enroll in a study.
6. Will I receive any follow-up care after the trial is over?
You may wonder what happens once the trial has ended and whether you will be able to continue the treatment if it has been beneficial for you. The transfer of care during the trial to after the trial can vary from one study to another. Sponsors are not obligated to offer you access to treatment after the end of the trial, however they are required to inform you about your options before the beginning of the study.
Different scenarios are possible. For example, sometimes the manufacturer of a treatment will offer compassionate use of the drug on humanitarian grounds, in cases where the trial drug provided significant benefit to the trial patient and/or stopping the investigational treatment would likely lead to deterioration of the patient’s overall condition. If the investigational treatment didn’t show enough evidence that it was better and/or safer than established treatments, then patients will be transitioned to other forms of medical care suited to their specific disease.
7. What is the difference between an interventional and an observational trial?
In order to decide which type of trial is best for you, it is also important that you understand the different clinical research terminology used by researchers.
- Interventional studies are clinical trials in which participants with the same conditions or disease are (usually randomly) assigned to one of two or more groups. One or more group(s), called treatment or experimental arms, will be given the intervention, which may be a drug, medical device, or another type of treatment. The other group(s), called the control arm, will receive an intervention that is considered effective (the standard of care treatment), a placebo, or no intervention. Outcomes in the treatment arm will be compared to the control arm in order to determine if there was any significant difference, for example in safety or efficacy.
- Observational studies are studies where researchers observe the effect of a risk factor, diagnostic test, or other intervention on a group of participants with the same disease or condition, over a period of time, without influencing who is or who isn’t exposed to specific interventions/treatment.
8. What are the different types of treatments that can be investigated in a trial?
Clinicaltrials.gov classifies treatments into ten categories: behavioral, biological, combination product, device, diagnostic test, dietary supplement, drug, genetic, procedure, radiation. In order to make things easier for patients, Ancora.ai has created individual icons and filters that are helping users to narrow down the list of trials in your search results according to your preferred type(s) of treatment. For example, if you are interested in receiving a drug rather than with radiation therapy, you can filter to only show the trials which are testing an experimental drug.
9. Why do some drugs have bizarre or multiple names?
If you scroll down a list of trials, you will probably notice that some drugs have names that sound like they are coming straight out from another galaxy, or that multiple names are referring to the same drug. The reason for this is that drugs often go by several different names depending on the context. At the time of its discovery, a drug is often given a chemical name, which describes its atomic and molecular structure. This name is usually too complex and cumbersome for general use, researchers usually create a shorthand version of the chemical name or a code name.
Once a drug is approved by the FDA, it receives a generic and a brand name.
- The generic name is chosen by official agencies, such as the International Non-proprietary Names, British Approved Names or United States Approved Names, following specific guidelines. It is usually more complicated and harder to remember than a brand name, as it’s often a shorthand version of the chemical name.
- The brand name is decided by the company that requested approval for the drug. It is usually catchier and easier to remember than the generic name.
As long as a drug is under patent protection, it is marketed under its brand name. Once the drug is off-patent, other companies that have filed for approval are allowed to market the drug under its generic name or under a brand name that they have created. As a result, the same drug can be sold under either its generic name or one of various brand names.
10. What does IRB approval stand for?
All clinical trials must be approved by an Institutional Review Board (IRB), which reviews research protocols and related materials (e.g. informed consent documents) to ensure the protection of the rights and welfare of human subjects of research. We recommend that you ask the clinical trial staff whether the trial of your interest has been approved and/or check whether the Office for Human Research Protections has registered that specific trial. You should only enroll in trials that have been approved by an IRB.
Enrolling in a clinical trial can seem daunting, however, it can give you access to innovative, cutting-edge treatment options before they are widely available. By joining a trial, you will also help move research forward and contribute to making novel therapies available for other patients in the future.
Remember that it is your right to ask questions to make sure you understand every aspect surrounding a particular trial as they are all unique. In order to make that process easier for you, we have prepared a printable list of questions, with space for notes, that you may want to ask the clinical study team before you decide to take part in a trial.