If you’ve spent time researching cancer, “clinical trials” is an oft-used phrase. But what is a clinical trial? How is one started? Who can be part of a trial? Well, believe it or not, all of the above are pretty simple questions to answer.
However, not all clinical trials are created equal – and there are certain things to be conscious of when looking at the information.
At its core, a clinical trial is for research on people. These studies are the final step to help doctors find cutting-edge treatments. The work that began in laboratories – under microscopes and in animals – comes to fruition with human clinical trials.
For cancer, clinical trials are available for all stages of the disease. (This is a good thing. Lab work is substantial enough to move on to clinical trials, while animal and cell work continue as additional questions arise regarding the disease. The process has been vetted to the point of documenting the treatment with humans. This is where hope thrives!)
Clinical trials are underway with the end goal of developing better ways to find and diagnose cancer, to treat disease, and – most importantly – to prevent cancer. Other research is probing how cancer patients can manage the symptoms and side effects of treatments.
If you have just been diagnosed with cancer, consider joining a trial before starting treatment. This is a necessary talk to have with your doctor, who can give details on how quickly you need to make a decision about any treatment.
What is a clinical trial?
Generally, a medical doctor – the principal investigator – lays out a plan for the clinical trial. This plan is called a protocol, which details what is done during the process. Also discussed is the who, what, when, where, why, and how – the reason the research is needed, who and how many people can be part of the clinical trial, whether or not drugs or natural treatments are offered (this includes dosing and how often), which type cancer and a particular organ system(s) is being addressed, the institution(s) conducting the study, when the study begins and ends, the timing of treatment and intervention, plus follow-up. This is the nuts and bolts of the process.
The protocol is detailed; it is the foundation on which the research will stand. Successful cancer treatments – including safety and efficacy – are built on clinical trials, and these studies help improve quality of life. (Note: Protocol summaries use medical language to describe and detail the clinical trial. If you do not understand the language or have questions, talk with your healthcare provider.)
After you enter a clinical trial, it is paramount to follow the protocol and the doctor’s instructions. The knowledge gleaned from the research will improve care across the board with today’s and tomorrow’s cancer patients.
Also, not all clinical trials are focused on drugs or natural treatments. Ongoing research is examining new ways to do surgery or to give radiation therapy. There also are clinical trials that use a combination of treatments as well as prevention trials and survival studies. As with all your cancer care, discuss options with your healthcare provider.
Understand that not every clinical trial has the same criteria. A specific type or stage of cancer may disqualify you. Also, you may be disqualified from a clinical trial due to particular kinds of past treatment you may or may not have pursued. Other factors may include your age, medical history, current health, or even specific genetic changes in your tumor.
Phases of a clinical trial
Before treatment is ready for humans, scientific researchers do testing (in vitro and in vivo) with animals to gather information on efficacy and toxicity as well as the absorption, distribution, metabolism, and excretion of the drug.
There are four major phases to a clinical trial. The criteria outlined in each protocol (and each phase) is specifically designed to ensure patient safety and accurate study results. The research will dictate whether a protocol becomes standard:
The purpose of the first phase is to find a safe dosage (often subtherapeutic but with ascending doses), decide how the new treatment is administered, to see how the new treatment fights cancer and its effect on the body, including side effects from the treatment. There are generally 20 to 100 people involved in Phase 1, which typically lasts several months to 1 year.
The second phase determines if the new treatment’s therapeutic dose has an effect on specific cancer and how it affects the body. Participants in Phase 2 ranges from 100-300 people and this lasts for up to 2 years.
This stage is where large amounts of data are collected. Because there could be between 300 and 3,000 people involved, the information from Phase 3 can be substantial (and that’s a game-changer). The purpose of this phase is to compare the use of the new treatment against the current standard and could last several years.
The fourth phase is ongoing after a protocol is approved, tracking safety to determine if there are long-term side effects.
If you are chosen to participate in a clinical trial, you will be randomly assigned to either:
- The control group, which will get the standard treatment for your cancer.
- The investigational group, which will receive the new protocol (the alternative treatment alone or combined with the conventional treatment).
Your doctor will explain how some clinical trials will implement randomization to prevent bias. The bias is a result of individual choices (or other factors not related to the test protocol). Randomization is a way to block unknown factors from affecting the clinical trial results.
“Placebo” – a harmless pill, medicine, or procedure – is another word you will come across while researching clinical trial treatments. A placebo is prescribed more for the psychological benefit to the patient than for any physiological effect. Placebos also help prevent bias in research.
There are federal rules that govern clinical trials. The regulations address patient rights and safety as well as ensure the testing is run and overseen ethically. As a patient, you are protected by an “informed consent” in addition to the review and approval of the clinical trial by a scientific review panel and an institutional review board.
Informed consent lays out the purpose, risks, and benefits of a clinical trial. This information is provided before deciding whether to take part in the clinical trial. (Note: Even after you sign the consent form, you can leave the study at any time.)
The review panels are made up of experts who review the clinical trial protocol from start to finish. If it is government-funded, the trial must go through this review. The government-funded institutional review board generally consists of at least five people. However, clinical trials conducted by drug companies do not have the same evaluation although many do pursue advice from experts on the scientific merit of their results.
Data and safety monitoring boards – most often used in Phase 3 clinical trials – also are part of the safety process. DSMBs are independent experts whose role is to ensure risks are reduced as much as possible, certify that the data are sound, and check that the trial’s objectives have been met (or to stop the trial if risks arise).
How much does a clinical trial cost?
A clinical trial has two types of costs: patient care and research. Patient care costs – doctor visits, hospital stays, conventional cancer treatments, lab testing, X-rays, etc. – often are covered by your health insurance.
Research costs typically are not covered by health insurance but may be paid for by the clinical trial sponsor. The costs would include the protocol treatment, lab tests for research purposes, or X-rays solely for the trial. You also may have additional doctor visits not associated with standard care. (Also, don’t overlook everyday expenses – transportation, child care while you’re at the doctor’s office, meals, etc.)
As you weigh the cost, also consider the pros and cons. Yes, clinical trials offer high-quality care – but you may not receive the new treatment. Still, the standard treatment may be as good (or better) than what’s being researched. You may experience side effects with the protocol – or you could be among the first to benefit from the new cancer treatment.
Key questions before making a decision
Deciding to participate in a clinical trial is not a knee-jerk reaction. Thought and consideration are important. Being involved in a trial takes dedication – making the commitment to follow the protocol exactly as it is laid out is of utmost importance – and it can be time-consuming. It also may be costly.
Be sure you are well-versed with the answers to these discussion points:
- Objective: Does the clinical trial’s purpose align with your treatment goals.
- Eligibility: Does your cancer diagnosis/state of health match the trial’s eligibility criteria?
- Investigator: Who will be overseeing the trial, and will we be in contact through the trial?
- Location: Is the trial near you? How often will you need to receive treatment during the trial?
- Length: How long will the trial last? Will it be a major time commitment? Are you and your family prepared?
Once you have decided on pursuing a clinical trial, seek answers to these questions:
- Has this protocol been tested before?
- What are the possible risks, side effects, and benefits?
- Is long-term follow-up care part of this trial?
- If I benefit, will I continue receiving treatment after the trial ends?
- Will the research results be given to me?
How to find a clinical trial
The discussion for locating a clinical trial that’s right for you should begin with your doctor. Your health care provider knows the ins and outs of your particular case. He can provide insight into a clinical trial that would be best for you. Doctors also can help search for a trial, answer questions you have about the trial, even provide insight on a particular trial.
Then, seek a second opinion. It’s always best to get another viewpoint about treatment options.
If you are searching for a clinical trial on your own, there are details about your cancer diagnosis that are important. (Remember: Not all trials are equal and not every trial is for any cancer patient.) Get the details for the clinical trial before making a decision. The eligibility criteria are non-negotiable with the protocols.
When searching for clinical trials, note whether nonprofit organizations or for-profit groups fund it. Hospitals and cancer center researchers also conduct clinical trials. Regardless of which list you find, know it is not all-encompassing. No site lists all available clinical trials. Also, the amount of information about particular clinical trials may vary. Bottom line: Do your homework before making a decision.
The clinicaltrials.gov database is part of the National Library of Medicine and lists clinical trials for cancer. It features the National Cancer Institute, pharmaceutical, and biotech companies’ trials.