Making the decision to be a part of a clinical trial is personal. If you are thinking about taking part in a clinical trial but have questions or concerns, it is worthwhile to talk through those thoughts with your medical team. You can also connect with other healthcare professionals, trusted organizations like Tigerlily Foundation, and support networks to get your questions answered and identify trials that may be right for you. It’s important to know that each clinical trial has its own eligibility requirements, and some clinical trials will exclude people who have tried or are currently taking certain medications. Because of this, we encourage people to consider trials early on in their treatment planning process so they don’t miss out on any options that they may be interested in.
Coverage and cost differ by location and by study. Some clinical trials pay for part or all of the associated cost (study medication, travel, etc.), and many insurance companies cover a significant portion of the cost as well. Be sure to ask the research team about what expenses you will be responsible for before agreeing to participate in a clinical trial.
You have the right to stop participation in a clinical trial for any personal or medical reason, including if the treatment is not working or there are serious side effects. You will be working with a team of healthcare and research professionals who can help you make this decision and who will be monitoring changes to your health the entire time you are participating in the trial. Their number one job is to make sure they’re keeping participants as safe as possible during their participation, so it’s important you report any changes or concerns (big and small) to the research team.
Being a participant in a clinical trial study is 100% voluntary. At any point in time, for any reason (including no reason at all), you have the right to withdraw participation from a clinical trial.
All medical decisions come with potential risks and benefits, clinical trials included. While clinical trials are conducted to help scientists learn more about a drug or condition, study medications go through a series of tests before they are ever given to a person. These tests help scientists gather information about safe doses, how often the drug should be administered, and any possible health conditions that might put someone at a higher risk for experiencing a safety issue. Before a clinical trial begins, it also has to be approved by a research ethics committee. They review the plans for the study and all documents that will be given to you, so commonly this is the study protocol, informed consent, and any posters or brochures you might see in your doctor’s office. They also review data gathered while the study is being done, always ensuring that participants rights are being respected and that decisions are being made with the participants best interests in mind.
Other safe guards in place include:
- Participants have the right to withdraw at any time.
- The ethics committee can stop the trial at any time if there are concerns about safety or welfare of the participants.
- Arrangements for compensation should be in place if safety issues do occur.
- Potential benefits and risk must be communicated before you agree to participate along with expectations for visits, cost, and any other responsibilities you might have as a patient participating in a trial.
Fears and myths of being treated like a “guinea pig” are very common. Having an understanding of what’s involved may provide some ease.
- Your personal information remains confidential. In fact, you’re provided a patient ID number that is used in place of your name on study documents to protect your privacy.
- The care received in a trial should meet or exceed the expectations set for standard care for your condition. This means you should get comparable, and in some instances more, care than you would if you opted not to participate.
- Participants are closely monitored and data is carefully recorded, reviewed, and compared to others in the trial so any possible concerns are caught and addressed early.
- There are many safeguards in place for safety. See “Is Participating In A Trial Safe” for some specifics.
- You are free to withdraw at any time you, the researchers, your doctor, etc. feels it is in your best interest with no impact on receiving treatment in the future
Breast cancer trial participants do not receive a placebo in lieu of standard treatment. Some trials may include a chance of receiving placebo or the study medication in addition to a standard treatment, but participants in oncology trials are always provided with some type of active medication to fight their cancer.
In what are called randomized double blind clinical trials, you and your doctor cannot choose which drug/treatment you receive. Randomized means that you are randomly assigned to a dosing group. Double blind means that neither you or your doctor will know what group you were assigned to while you are participating. Often times, once a study ends, you are able to find out what group you were in. Also, it’s important to know that if you have an emergency and need to know what dosing group you were in, the study doctor can easily “unblind” you. This means they can access your dosing group, and this process is quickly and easily done online.
This is a great question to ask the study staff before consenting to participation. They will usually provide you with a direct line to the study coordinator or research nurse, a 24-hour emergency line, and contact information for the ethics board. The emergency number and contact information for the ethics board should also be included in the informed consent document. Of course, the study doctor and research team are a great first place to start if you have a question or concern about the study.
Participation looks different for each trial because they each have their own protocols with information about visits, labs, dosing, and all of the other details to keep participants safe and help the researchers collect important data about whether or not the drug is working. Some common patient responsibilities do exist though, and we’ve listed those here:
- ASK QUESTIONS! Lots of them. Any time you think of a question, it’s a good idea to write it down in a notebook or in the notes app on your phone. Create a running list and bring it to your appointments with you to be sure you get them answered.
- Read the informed consent form. This isn’t fun beach reading by any means, but the informed consent is filled with information about the trial, what you can expect, risks, and other things to consider. They are supposed to be written in patient friendly language, but that doesn’t always happen so be sure to ask your study team to explain the document to you along with any parts that don’t make sense. It is important that you make a decision that you feel good about, and this is the perfect time to gather the information you need to do that!
- Discuss possible benefits, risks, and expectations with the study team to be sure you’ve not missed anything in the consent form.
- Maintain your study appointments to the best of your ability.
- Communicate new health problems, concerns, etc to your study team as soon as they arise.
- There will very likely be additional tests involved like bloodwork or scans. The timing of these is often important, so maintaining these appointments as best you can is key. Sometimes appointments/procedures can be stacked back-to-back to save you a trip and others may be able to be spaced out to give you a chance to rest between. Communicate your needs to the study team so they can work to accommodate you.
- What is the purpose of this study?
- How will this research help?
- What tests or procedures will be done?
- Will I be asked to take investigational drugs or undergo experimental procedures?
- Is it possible that I will receive a placebo and not a drug?
- What are the potential risks and benefits of taking part in the study?
- What other options do I have if I decide not to participate?
- What will happen to the specimens (blood, genetic, etc) that I give?
- What happens if my condition gets worse while I’m in study?
- Who will pay my medical bills if I become sick due to participating in the study?
- Will it cost me anything to be in the study?
- Will you share my test results with my doctor?
With each phase building on the results of the phase before, clinical trials are usually conducted in four phases (phase I-IV.)
Phase 0 – Does This Drug Behave In The Way It Is Supposed To?
- Unusual for trials involving cancer patients
- Small group, low dose (too small to treat cancer)
Phase I Trials – Is It Safe?
- Small studies (~15-20 people)
- Researchers are learning:
- Best way to administer treatment
- Highest dose possible without serious side effects
Phase II Trials – Does it Work?
- Looking at how effective the treatment is
- Slightly larger studies (~25-100 people)
- Start with the dose and method identified in phase I, monitor for benefits
- Benefits sought vary based on research goals
- If a certain percentage benefit and side effects remain acceptable, treatment may move on to phase III
Phase III Trials – Is it Better Than What We Have Now?
- The safety and effectiveness of the study drug is compared to current standard of care. Is the study drug better or different in some way than what we have now?
- Usually large numbers of participants (several hundred to several thousand)
- Participants across US and sometimes around world are included in these trials.
- This is the last step before the U.S. FDA considers approving it for use.
- Participants are still monitored closely for any serious side effects during their participation.
Phase IV Trials – Are There Any Other Uses or Benefits?
- Ongoing review of benefits and any long-term side effects
- Usually done after the drug is approved by U.S. FDA
- Less common than phase I-III
- Large studies (hundreds of thousands of people)