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Revised for the Internet January 2006. Originally published in Coping Magazine, September/October 1997
Increasingly, cancer patients are taking a more active role in decisions regarding their treatment, and looking for information about experimental cancer drugs for their type of cancer.
When I was diagnosed with cancer in 1987, I didn't know much about the Food and Drug Administration (FDA). I vaguely understood that is was the federal government agency with responsibility for making certain that the food we ate and the drugs we took were safe. Slowly, as I became more educated about cancer, references to FDA became more frequent. Some patients I talked with thought that the FDA tested, manufactured or distributed drugs. There seemed to be a wide-spread belief, among cancer survivors, that more drugs to cure cancer might be available to cancer patients, if only the FDA would approve them more quickly.
After recovering from my cancer treatment, I went back to work and in 1994 I became an employee of the FDA's Cancer Liaison Program. One of my responsibilities was to answer questions from cancer survivors and their families about cancer drugs that were being researched but not yet approved by the FDA. As a cancer patient and a participant in cancer clinical trial, I knew quite a bit about how clinical trials operated, but I didn't really understand how the FDA fit into the picture. So, my first job responsibility was to educate myself about what the FDA does prior to approving a drug. This is what I learned.
First, I learned what the FDA does not do. FDA does not manufacture drugs or directly research whether a drug is safe and effective. The FDA's role in this process is to oversee the pharmaceutical research conducted by drug companies, university research centers and physicians to make certain the federal regulation governing this research are being followed. A fictional example of how this works follows.
Hypo-Thetical Drugs, Inc. has discovered a new drug that shows some effectiveness in reducing colon cancer tumors in animals. Hypo-Thetical Drugs may not begin to test its drug in human beings until it files an application with the FDA called an Investigation New Drug application or IND. This IND application will contain a plan for how this drug will be studied in people in one or more clinical trials, and a complete description of the drug: its structural formula, animal test results, and manufacturing information. In this hypothetical case, the FDA team, which includes medical oncologists, chemists, pharmacologists and statisticians, reviews the IND application and verifies that Hypo-Thetical's plan 1) protects the safety of cancer patients who may choose to participate in Hypo-Thetical's clinical trial, and 2) clearly states Hypo-Thetical's objective in conducting the clinical trial.
The drug company filing the IND application, often called the "sponsor" of the trial, must specify in which type of cancer it will study the drug, the criteria patients must meet to be enrolled in the clinical trial, and the measures it will use to determine the drug's safety and effectiveness. The sponsor's plan must have an objective such as determining the best dose for a new drug, determining whether the drug will reduce the tumor size or showing that its drug is superior or equivalent to the drug(s) currently used in treating a type of cancer. FDA also requires that the drug company's plan must be reviewed by community research review board, and that patients participating in the clinical trial are informed about the trial and consent to participate.
Once the drug company has completed its clinical trials, the data are tabulated and submitted to FDA in another application known as a New Drug Application (NDA). Complete data from every patient in every clinical trial are included in this application. The clinical trial data and the conclusions reached by the sponsor are carefully reviewed and evaluated, usually by he same team of FDA reviewers who reviewed and evaluated the IND. They prepare formal written reviews and the entire review process for an NDA generally takes between six and twelve months depending on the data submitted in the sponsor's application. However, when a drug shows promise in a type of cancer which has no currently effective treatment, the review is usually completed in less than six months.
Late in the FDA review process, the drug is usually brought before an agency advisory committee. This advisory committee is made up of people who are not employed by FDA, and includes leading scientists, clinical practitioners and consumers and patient representatives who have practical experience with cancer and its treatment. At this meeting, the drug company staff presents its finding from the clinical trials on the safety and efficacy of the drug. This is followed by a presentation of the FDA's review of the drug company's NDA. The advisory committee carefully considers and discusses the presentations and votes on whether to recommend this drug for approval. The committee deliberations assist the FDA in making a final decision regarding marketing approval of the drug.
Until a drug receives final marketing approval, the drug company cannot sell its unapproved drugs and may only provide it to patients who meet the criteria established in the company's clinical trial plan. However at times a cancer patient not eligible for a clinical trial may receive a promising unapproved drug, if the patient's doctor, the drug company and FDA each agree.
The FDA's primary interest is helping to ensure that the drug company's research will not subject cancer patients in the clinical trial to undue risks. FDA is also interested in working with the IND sponsor to provide guidance in developing its clinical trial design so that the data will be scientifically useful. The drug company and FDA are interested in getting the best scientific results as early as possible in an environment that is safe for patients, so that effective new drugs can be approved by the FDA and marketed as soon as possible.
Patients are usually dependent upon their physician to tell them which clinical trial might be appropriate for them. However, this need no longer be the case. Increasingly, cancer patients are taking a more active role in decisions regarding their treatment, and looking for information about experimental cancer drugs for their type cancer.
Cancer patients can directly investigate their own eligibility for a clinical trial by calling the Cancer Information Service (CIS) at 1-800-4-CANCER. A live information specialist works directly with each caller to locate the trials that may be suitable for the patient. Those clinical trials are then sent to the patient or family member within three days of the call. This is a comprehensive information service sponsored by the NCI that, in addition to helping patients search for appropriate clinical trials, contains a wealth of helpful information about the cancer diagnosis and treatment.
The National Library of Medicine's clinical trial database - http://www.clinicaltrials.gov/ - is another valuable resource to help patients locate clinical trials they may be eligible to enter. This database contains cancer clinical trials as well as clinical trials investigating treatments for life-threatening diseases such as heart disease, diabetes, AIDS, Parkinson's disease and many more.
In addition, the Pharmaceutical Research and Manufacturers of America - the trade association for the pharmaceutical industry - also lists some of their trials on line - http://phrma.org/newmedicines/-. Clinical trials for many diseases are listed on this web site along with the name of the company sponsoring the trial and a contact telephone number at the company.
I have learned that the FDA drug review process assures cancer patients that the risks and benefits of cancer drugs have been carefully considered before they are approved and, within that context, the drug review process helps to ensure patients that marketed drugs are safe and effective. Every effort is made by the FDA staff and the drug industry to make that process efficient, so there is no delay in moving cancer therapies through the FDA approval process and making them promptly available to cancer patients. The efficiencies that have been achieved in the past several years are due to a collaborative effort among the pharmaceutical industry, FDA and cancer survivors. We expect this partnership to continue long into the future.
Editor's Note: Patty Delaney is a survivor of Hodgkin's disease and is a member of the U.S. Food and Drug Administration's (FDA) Cancer Liaison Program in the Office of Special Health Issues. This article originally appeared in Coping, September/October 1997.
For Additional Information, Please Contact:
Cancer Liaison Program
Office of Special Health Issues
Food and Drug Administration
5600 Fishers Lane HF-12, Room 9-49
Rockville, MD 20857
301.827.4460
301.443.4555 (FAX)