Our practice is at the forefront of cancer research and offers participation in clinical trials for many types of cancer. We believe it is important to provide access to promising new therapies and treatment options to patients in our community. These trials also allow many of our patients to receive newly developed treatments or investigational drugs not yet available outside the study.
What is a clinical trial?
A clinical trial is a study of an investigational medication or treatment to see how well it works and assess its safety. Before a clinical trial can occur, the treatment must have been studied in the laboratory and on animals.
Why do we need clinical trials?
The Food and Drug Administration requires that clinical trials be conducted with investigational medications before it will approve their use with the general public. This is to ensure that the investigational medication is effective and safe.
How do clinical trials work?
Significant achievements have been made in the management of patients with cancer from research involving clinical trials over the last few decades. Clinical trials are designed to achieve specific goals, such as determining the safest and most effective dose of a new drug, uncovering the potential side effects of interventions, and finding new surgical or radiological methods.
Clinical Trial Phases
Phase I
Research, which leads to human testing, often starts at the test tube level. For cancer, this may include treating cancer cells with the drug(s) of interest. If a therapeutic response is observed, the drugs are given to animals. Animal testing provides some insight into possible side effects and generates the starting dose used in the first level of human testing, called a Phase I clinical trial. Patients considered for this treatment have typically exhausted standard of care options, have an advanced stage of cancer, but have few symptoms from their cancer.
Enrollment in a Phase I trial is typically open to cancer patients. The first patients are administered the lowest planned dose and observed for side effects over a period of time. If the low dose is tolerated, another group of patients is enrolled at a higher dose level and monitored for side effects. Escalating drug doses are given to more patients until the maximum tolerated dose is determined. The effectiveness against specific cancers is analyzed once the maximum dose is achieved. It is important to understand that not all cancers will respond to any given agent. Only five to ten percent of Phase I agents are effective enough to proceed to the next level.
Phase II
Phase II trials have more selective enrollment requirements. The inclusion criteria are based on tumor types that appeared to respond to the Phase I trial and often place a restriction on the number of different treatment regimens a patient previously received for their cancer. These patients usually have advanced-stage cancer and are relatively asymptomatic. Many patients are enrolled in a Phase II trial because one of the primary endpoints is to prove effectiveness with acceptable side effects. If a reasonable response rate is seen at the Phase II level, the experimental drug or combination can proceed to a Phase III trial.
Phase III
A Phase III trial usually represents the final destination for a new drug or combination. At this level, the experimental study can be designed for patients with early-stage disease, where a cure is being sought, or more advanced-stage disease, where prolonging life is the goal. Participants are randomly assigned to standard-of-care chemotherapy or the experimental drug(s). The patients and physicians are blinded to the regimen the patient is receiving. Many patients are enrolled, and the study could take years to complete. The experimental drug(s) can become a new standard of care if they are more effective than the current standard of care or equivalent but less toxic.
Cancer research requires a great deal of money and time. However, the most important element is the willingness of cancer patients to be enrolled in clinical trials. Without their participation, there would not be progress in the fight against cancer.
Who is eligible to participate?
The foremost criterion for participation is to have a disease that requires some intervention. The trial will then outline further eligibility requirements. For example, the therapeutic option available through the study could focus on a direct intervention designed to provide primary treatment, such as a new chemotherapy agent to treat cancer. Alternative clinical trials might focus on new interventions to palliate cancer symptoms or the standard treatment, such as nausea or neuropathy.
Who pays for the study?
Many patients asked to participate in a clinical trial believe all their care will be paid for through the study. The reality is that only part of their care might be covered. The trial sponsor would provide additional medications and funding to cover costs outside the standard of care practices. Insurance companies are still required to pay for the components of a patient’s care that are considered the standard of care, i.e., routine provider visits, laboratory tests, radiographic imaging, and prescription medication.
When should you participate?
The National Cancer Care Network guidelines recommend that patients be considered for a clinical trial for most types and stages of cancers. Unfortunately, clinical trials are unavailable for all cancers or may not be readily accessible. Still, Arizona Oncology has a robust research program with various clinical trials open at any time.
What are the benefits of participation?
Commonly prescribed medications such as analgesics, antibiotics, and inhalers have undergone rigorous clinical trial investigations. Participation in clinical trials has become an international phenomenon that improves the health of communities. Choosing to enter a clinical trial may provide the benefit of having another opportunity to treat your disease with a non-standard-of-care option. The altruism of our friends, neighbors, colleagues, family, and strangers provides us with access to medications and diagnostic tests that have improved the quality of life for all. These special individuals take a risk and volunteer to enter a clinical trial when offered the opportunity. As a result, we can celebrate successes and learn from failures.
What are the risks?
The trial is designed to evaluate the safety and effectiveness of the treatment and whether or not there are side effects. There are risks with all clinical trials, which will be described in the informed consent. However, with all investigational medications and treatments, there may be some risks that are currently unknown.
Where to go?
Don’t hesitate to start a conversation with your oncologist; that is where your journey to a clinical trial should begin. Additionally, several resources are available to guide patients through their treatment decisions. Reputable organizations such as the National Cancer Institute or the American Cancer Society have a detailed list of clinical trials available throughout the country.
Today, extensive clinical research is conducted in local community practices like ours. Many studies conducted in private, community-based oncology practices have led to FDA approval of new drugs and treatment regimens.
