Cancer Trials - Are They Ready for Prime Time?
By Keith Hansen, M.D.
Clinical trials are the mechanism for improving survival and quality of life for individuals faced with a cancer diagnosis. Without trials, we would not know that mastectomy for breast cancer is equivalent to lumpectomy and axillary node dissection. We would not have the evidence showing that most patients with Hodgkin's disease, aggressive non-Hodgkin's lymphoma, and advanced testicular cancer can be cured with chemotherapy. In order to achieve these milestones in cancer treatment success, cancer clinical trials are designed in a step-wise fashion.
Phase I Trials: The first step in testing a new approach in humans. Data from previous animal and laboratory studies are used to evaluate drug dose, administration schedule, drug metabolism and side effects. Patients are divided into small groups called "cohorts." Each cohort is treated with increasing doses of the agent or combination until the maximal tolerated dose is reached. The highest (or most effective) dose associated with acceptable side effects is chosen for future studies. Generally, Phase I trials are conducted on patients with a variety of malignancies who have advanced disease.
Phase II Trials: Determining the safety and effectiveness of a new treatment is the primary endpoint of Phase II trials. A new drug, combination or technique is studied on a small and relatively homogeneous group of patients (e.g. 40 - 100 patients with a specific cancer). The type of cancer chosen for a Phase II treatment is based on results of laboratory studies and Phase I trials. The primary purpose of most Phase II cancer trials is to determine the percentage of patients that show a measurable response to treatment. Additional information on side effects and safety is also collected.
Phase III Trials: These large-scale trials compare a new treatment or combination that has shown promise in Phase II trials to the current standard therapy. Patients are randomly assigned to the standard approach or the new treatment. Phase III trials are critical for advancing the quality of cancer treatment and may establish a new standard of care. Hundreds to thousands of participants may be needed for a well-designed Phase III trial.
Experimental or Investigational (NCI definition): An investigational study or clinical trial refers to a drug (including a new drug, dose, combination or route of administration) or procedure that has undergone basic laboratory testing and received approval from the U.S. Food and Drug Administration (FDA) to be tested in human subjects. A drug or procedure may be approved by the FDA for use in one disease or condition, but be considered investigational in other diseases or conditions. Different insurers may have different definitions for these terms.
Medicare and Clinical Trials: Medicare reimburses for routine cancer care as well as for care that is part of a clinical trial. Covered services include routine diagnostic tests, procedures, physician visits, administration costs of any investigational drugs, and treatment/hospitalization if needed for management of side effects. Items that are not reimbursed include any charge for an investigational drug, services or items provided free by a trial sponsor, or any coinsurance or deductible payments. Since most insurance companies follow Medicare guidelines, the same provisions for covering clinical trial costs should be adopted by private health insurers.
By providing coverage of treatment on a nationally-sanctioned clinical trial, public and private insurers are allowing their subscribers access to the best quality, state-of-the-art therapies. Clinical trials help to identify better and safer anti-cancer drugs. Advances in treatment occur as a direct result of clinical trials. These advances lead to new standards of care and improved quality of life for those battling cancer. New and more effective therapies will translate to a reduction in recurrence rates and to the suffering endured by victims of cancer and their families. Moreover, successful clinical trials will contribute to a decrease in healthcare dollars spent to fight the number two killer in the United States. Reducing the burden of cancer requires a dedicated healthcare system that supports research as the only path toward improving outcomes and saving lives.