Patients should consider participating in clinical trials and ask their physicians about trials that may be available. Not only do clinical trials advance science and produce important treatments for the future, they may provide the best available treatment to a patient today! In many clinical trials offered today, patients receive not only a standard therapy, but a standard therapy combined with an additional agent that has shown promise. Unfortunately, many clinical trials in the United States close because of lack of participation.
Clinical trials are research studies to determine the safety and efficacy of a new treatment, therapy, or medical device under controlled conditions. Clinical trials are constantly in progress all over the world. A clinical trial is one of the final steps of a long research process. The experimentation does not start on people. Before the clinical trial, there should be extensive review of safety and effectiveness done in the laboratory and on animals. If a treatment is not safe or effective on rats, mice, and other laboratory animals, it does not make it to a clinical trial. If the results show potential benefits that exceed the risks or side effects, human studies may be carried out. Clinical trials often require large numbers of participants to properly evaluate effectiveness and long-term safety of a drug or device.
Clinical trials should follow strict protocols. Protocols may require a treatment to be examined alone or compared with a sugar pill (placebo) or a treatment that already is in use. Alternatively, a trial may be designed to determine whether an existing treatment may have additional uses. Protocols also describe who may participate in the trial, the schedule of tests, procedures, medications and dosages, and the length of the study. Patients participating in a trial are examined regularly by doctors and researchers who monitor their health and follow the safety and effectiveness of the treatment.
Clinical trials of a new drug typically go through four phases. Phase I is primarily designed to determine safe doses and the best modality of the drug or treatment (should it be given by mouth, injection, or inhalation). Phase II continues to test the safety of the drug and how well it works and usually involves a larger number of patients. Phase III involves giving the drug to an even larger number of people over a longer time period, and often compares the drug or treatment being studied against known treatments. Phase IV involves ongoing monitoring of large patient groups after a drug or treatment has been approved and marketed.
Although you may want to participate in a given clinical trial, the trial may not want you. You must satisfy specified criteria to participate. Because each clinical trial looks for answers to different questions, each trial has specific guidelines regarding who is qualified to participate. The guidelines describe characteristics that may include such things as age, type of disease, medical history, and medical condition. For example, it may require that you tried and failed another specified treatment or that you have not undergone other treatments. You must meet these characteristics to qualify for participation in the study. You may not understand the criteria and the criteria do not always make sense from the standpoint of pure treatment of patients. But, first and foremost, clinical trials are studies. We are aware of instances where people who did not satisfy the criteria for a clinical trial have nonetheless been allowed to participate in a clinical trial. Accordingly, it pays to be persistent and to have a physician that is an effective advocate.
Assuming you qualify, the ultimate question is whether you should participate. Before entering a trial, your “informed consent” is required. You will be told such things as: what the trial will consist of (regimen, testing, length of time); why the research is being conducted; what the researchers seek to accomplish; what the risks and benefits are; what other treatments are available; and your right to exit the trial at any time. They will make you sign consent forms.
Before deciding whether to participate in a clinical trial speak with the doctors and researchers and do your own research. Before any trial is carried out with human participants, the protocol is reviewed, approved, and monitored by an institutional review board that follows government guidelines and includes safeguards that purport to protect you and ensure the risks are as low as possible. This board is a fancy name for a group of doctors, statisticians, community members, and clergy that is supposed to ensure the clinical trial is ethical and protects the rights of patient participants. Research centers involved in human research are required by federal law to have such a board in place to approve and review a trial during the time it is active. Board members are not paid.
You should know that other players are involved in these clinical studies. Drug companies, hospitals, insurers, and governmental agencies sponsor clinical trials. The trials generally take place in a university teaching hospital, a local hospital, a clinic, or a doctor’s office. It is always a good idea to “follow the money.” We all have heard about doctors being paid based on containing costs by not ordering tests, not prescribing treatments, or otherwise having a financial incentive that may conflict with the interests of the health of a doctor’s patient. With clinical trials, doctors and researchers may be paid for enrolling each participant in a study. In evaluating whether to participate, you may be interested in whether someone urging you to participate is making a commission.
You are not guaranteed to receive the treatment being tested. You may be a member of the “control group.” Members of this group usually get a placebo or the standard treatment instead of the experimental drug to measure whether there is a mental or emotional element impacting the study or so the drug or treatment being studied can be compared with a baseline. Participants will not know if they are in the control group or the experimental group. Participants in a blinded or masked study are not aware of which treatment they are receiving. When neither the participants nor the research team knows which participants are receiving treatment or placebo, the study is called a double blind study, because everyone is unaware of who is receiving the drug or therapy being tested. This type of study is designed to try to remove both the patients’ and the doctors’ expectations of the results from impacting the study.
Whether or not to participate in a clinical trial depends first and foremost on whether or not there are better treatment options available to you. In some cases, the study may be complementary and not preclude other treatments. Clinical trials offer advantages—the main one is access to new treatments or drugs that are not available to the public. It also may provide a level of care and monitoring the patient may not otherwise be able to afford or have access to. There always is the altruistic motive of benefiting medical science in the hope of helping other patients. Clinical trials are an important part of making new treatment available. Potential risks include the side effects or adverse reactions to medications or treatments. There may be considerable inconveniences associated with trips to the study sites, complex dosage, or monitoring requirements that may not exist with other treatment.
There are several sources available to learn about clinical trials. Leading lymphoma specialists are very knowledgeable about clinical trials and patients should insist that their doctors inform them about trials that may be beneficial. Here are some other resources: National Institutes of Health (www.clinicaltrials.gov); National Cancer Institute (www.cancer.gov/search/clinicaltrials); and Coalition of National Cancer Cooperative Groups (www.cancertrialshelp.org).