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
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, then the drugs are given to animals. Animal testing provides some insight into possible side effects and generates the starting dose that will be 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 patients with various cancers. 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 are enrolled at a higher dose level and observed for side effects. Escalating doses of the drug are given to more patients until the maximum tolerated dose is determined. An analysis of the effectiveness against specific cancers is undertaken 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 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 places a restriction on the number of different treatment regimens a patient previously received for their cancer. These patients usually have an 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.
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 prolongation of 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. A larger number of 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 type of intervention. The trial will then outline further eligibility requirements. The therapeutic option available through the study could focus on a direct intervention designed to provide a primary treatment such as new chemotherapy agent to treat cancer. Alternative clinical trials might focus on new interventions to palliate either the symptoms of cancer or from the standard treatment such as nausea or neuropathy.
Who pays for the study?
Many patients who are 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 as well as funding to cover additional costs considered 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. Clinical trials are not available for all cancers or may not be readily accessible, but Arizona Oncology has a robust research program with a variety of clinical trials open at any given time.
What are the benefits of participation?
Commonly prescribed medications such as analgesics, antibiotics, and inhalers have gone through the rigorous process of 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. It is the altruism of our friends, neighbors, colleagues, family and strangers that provide 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. We can celebrate the successes and learn from the 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, a number of 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 of the studies conducted in private, community-based oncology practices have led to FDA approval of new drugs and treatment regimens.
National Cancer Institute:
National Comprehensive Cancer Institute