What are clinical trials?
What are the phases of a clinical trial?
What is a clinical trial protocol?
Where are trials conducted?
Who can participate in a trial?
Is a clinical trial a last resort?
Are clinical trials risky?
Are patients given "sugar pills" on clinical trials?
Will health insurance cover the cost of a clinical trial?
Do you only go on a clinical trial if you have cancer?
A clinical trial is a research study involving patients that is used to find better ways to diagnose, prevent or treat diseases using new drugs or new devices. Trials are designed to answer specific questions about new and established therapies. They help doctors tell if a new treatment works and is safe. Millions of patients have taken part in this kind of research, and nearly all of today’s cancer drugs were tested this way.
Cancer patients must choose to participate in a clinical trial, and they must be carefully screened by their doctors to determine if a trial is appropriate for them. Patients who make an informed decision to enroll in a clinical trial get first access to the latest promising cancer therapies not yet available elsewhere. Participants also contribute to the advancement of cancer research and benefit generations of future patients by providing the information scientists and doctors need to develop new, more effective ways to prevent and treat cancer. Clinical trial participants are closely monitored and can opt out of the trial at any time.
The National Cancer Institute designated and partially funded VCU Massey Cancer Center to conduct research that addresses the nation’s cancer problems. Clinical trials are an essential tool in solving these problems because they evaluate the latest therapies and bring to patients novel laboratory research findings.
VCU Massey has one of the largest offerings of clinical trials in Virginia. Evaluating the latest cancer treatments for adult and pediatric patients, Massey conducts more than 100 trials on more than 20 types of cancers from brain and breast, to leukemia, prostate, lung and more.
Most clinical research progresses in an orderly series of steps or phases.
- Phase 0 trials – A relatively new phase of clinical trials created to speed up the development of promising new treatments. Very small, single doses or applications are used to study how the body processes the treatment and if the treatment works in the body as expected. In this phase, researchers can more quickly weed out the treatments that aren’t producing the desired effects, and can avoid moving those drugs onto further phase trials.
- Phase 1 trials – Evaluate how a new drug should be administered (i.e., orally, intravenously, by injection), how often and in what dosage.
- Phase 2 trials – Provide preliminary information about how well the new drug works and generate more information about the drug’s safety and benefits. A phase 2 study usually focuses on a particular type of cancer.
- Phase 3 trials – Compare a promising new drug, combination of drugs or procedure with a current standard of treatment.
- Phase 4 trials – Include the continuing evaluation that takes place after Food and Drug Administration approval, when the drug is already on the market and available for general use (post-marketing surveillance).
Clinical trial protocols are the action plans or guidelines that must be followed during the course of any study. Guidelines may cover:
- The design of the study – what it will study and how
- Who may participate – criteria for participants who enter the study
- What medications and medical tests will be used with the participants
- What information is being researched
Several research centers may be involved in the study, and each uses the same protocols to ensure that information from all centers can be combined and compared.
Large cancer centers, university hospitals, local medical centers or physician offices may offer clinical trials. There may be only one or two locations involved in a particular study, or hundreds around the country.
Many of the clinical trials at VCU Massey Cancer Center are offered as part of a collaborative effort among multiple cancer centers and research facilities organized into national or regional groups. Working in unison, members of these groups offer the same studies and follow the same protocols for each trial. The protocol is a study’s game plan that each researcher follows to the letter in order to ensure the consistency of the data collected and the integrity of the results drawn from the data.
With strength in numbers, cooperative groups help expand the number of patients on studies and can collect more data to evaluate the effectiveness of the trial. Also, enrolling participants in multiple locations means a trial can reach its enrollment goals sooner, accelerating the research.
Currently, VCU Massey participates in or has access to trials from eleven consortiums:
- American College of Surgeons Oncology Group
- Blood and Marrow Transplant Clinical Trials Network
- Cancer and Leukemia Group B
- Children’s Oncology Group
- Gynecologic Oncology Group
- National Surgical Adjuvant Breast and Bowel Project
- North Central Cancer Treatment Group
- Radiation Therapy Oncology Group
- Southeast Phase 2 Consortium
- Southwest Oncology Group
Each protocol in a trial defines specific characteristics (known as eligibility criteria) that patients should have in order to participate in the study. Characteristics may include type of disease and its stage, as well as the patient’s age and general health.
Eligibility criteria help assure that the study results answer the research question and identify who will benefit in the future from the approach being studied.
Waiting until other treatments are exhausted lessens a patient’s chance of being eligible for a clinical trial. The best time to make a decision about participating in a clinical trial is at the time of first diagnosis and every time a new treatment decision must be made.
At VCU Massey Cancer Center, each new patient is evaluated by a multi-disciplinary team of clinical experts who work together to offer the best treatment plan for that particular patient. When appropriate and based on each individual patient, treatment options may include clinical trials.
Patients on clinical trials are monitored very closely by their doctors and care team. In fact, many patients have said they appreciated the extra attention they received while participating in a clinical trial.
In addition, trials are generally overseen by an independent data and safety monitoring board as well as by any cooperative group study teams through which the trials are offered. Patient data — without individual identifiers — is reported and shared among the cooperative group members.
As part of the monitoring and data-sharing process, clinical researchers can identify any irregularities in symptoms. If a problem arises, the trial can be altered or stopped, and a patient may opt out at any time for any reason. Just like all medical treatments, cancer clinical trials are not risk-free. However, the safety of patients is of utmost importance to the researchers. There are several procedures in place as part of the protocols to protect the safety of the people who participate. Two groups that oversee the safety procedures are the:
- Organization that sponsors the study (for example, the National Cancer Institute).
- The Institutional Review Board (IRB) that oversees clinical research in the health care institution. The IRB includes physicians, other health care providers, consumers and sometimes members of the clergy, who do not have any personal interest in the results of the study. As neutral reviewers, they ensure that the study is conducted fairly and that participants are not likely to be harmed.
Phase 3 trials also have a special group called a Data Safety and Monitoring Committee that looks at the test results, monitors the safety of the participants and decides whether the study should go forward as originally planned.
Sugar pills (placebos) are seldom used in cancer clinical trials and are never used if a known, effective treatment is available. Patients in clinical trials always get the best treatment available. If a new drug is being tested as part of a trial, one group of patients might receive the current best standard treatment plus the new drug, while another group may receive the current best standard treatment plus a placebo. Either way, everyone is getting the best treatment available. Comparing the results of the two groups enables researchers to determine if the new drug is more effective than the current standard treatment.
VCU Massey Cancer Center would never let the cancer go untreated. The only time a placebo would be used alone is if there is currently no standard treatment or if a new cancer prevention measure is being tested for people without cancer.
Many insurers cover the normal costs of treatment for cancer clinical trials and many states, including Virginia, have mandatory coverage for certain types of trials. Research nurses at VCU Massey Cancer Center will gladly contact a patient’s insurance company on his/her behalf.
The cost to VCU Massey is considerable given the infrastructure expenses and labor-intensity of clinical trials, but offering them is a critical component of our research mission. Clinical trials are labor-intensive because we must collect and manage a wealth of data and have more frequent contact with these patients. For example, we have 450 former breast cancer clinical trial participants who are now cancer-free but we must follow up with them at least once a year to continue to report their data and to monitor for any long-term effects or trends.
Several cancer prevention and control studies are also available for people without cancer. Many participants are those who have a strong family health history of cancer and want to explore ways that may reduce their chances of developing cancer.