When laboratory studies indicate that a new drug may show promise in the treatment of disease, researchers develop a plan called a clinical trial protocol. The principal investigator, usually a doctor, prepares this plan and includes the reason for the study, how many people will be enrolled, who will be eligible to participate, what drugs will be used, what medical tests will be necessary, and what information will be gathered. Every doctor or research center participating in the trial uses the same protocol, making certain that patients are treated identically regardless of location, and assuring that all information gathered can be combined and compared.
Clinical trials are sponsored by organizations or individuals seeking better treatments for cancer. The National Cancer Institute (NCI) sponsors numerous clinical trials involving thousands of investigators at over a thousand sites. Participating programs include:
Cancer Centers Program
These research-oriented institutions have been designated by the NCI as Comprehensive or Clinical Cancer Centers for their scientific excellence. Located throughout the country, they play a vital role in cancer research, delivery of high-quality cancer care, and education for the public as well as for professionals.
Cooperative Clinical Trials Programs
This program combines groups of researchers, cancer centers, and community physicians into the NCI network. These cooperative groups enroll nearly 20,000 new patients in treatment trials each year.
The Community Clinical Oncology Program (CCOP)
This program makes clinical trials available in local communities by linking community physicians with researchers in cancer centers. Local hospitals affiliate with larger cancer centers or cooperative groups, enabling physicians to offer their patients easier access to clinical trials without having to travel long distances or leave their usual caregivers.
The earliest (Phase I) trials focus on safety; later trials (Phase II and III) focus on whether or not the drug is effective. The Food and Drug Administration (FDA) will only allow the pharmaceutical manufacturers of a drug sale of their product after it has been proven safe and effective in clinical trials.
After a Phase I or II trial is completed, the researchers carefully examine the collected data and decide whether to move on to the next trial phase, or stop testing of the drug because it has proven unsafe or ineffective. When a Phase III trial ends, the researchers must analyze the data and determine if the results have medical relevance. When the analysis is complete, if the results are positive, the drug is submitted to the FDA for approval. The FDA, must then review studies submitted by the drug’s sponsor (usually its pharmaceutical manufacturer), evaluate any reports of side effects or complications from preclinical and clinical trial studies, and review the drug’s chemistry and manufacturing process.
While the submission process is lengthy, it is meant to ensure that only beneficial drugs with acceptable side effects reach the public. Luckily, recent legislative mandates and streamlined procedures within the FDA have helped to accelerate the approval of effective drugs, especially for cancer. Specific provisions also make some drugs available to patients in special circumstances. One such drug, Alimta, is now open for compassionate use to mesothelioma patients who fit certain eligibility criteria.