Getting Treatments to Patients Who Need Them
Not everyone is eligible to participate in a clinical trial. Some patients do not fit the exact requirements for studies, some have rare forms of cancer for which only a limited number of studies are underway, and others are too ill to participate. Working with the National Cancer Institute (NCI) and other sponsors, the U.S. Food and Drug Administration (FDA) has established special conditions under which a patient and his or her doctor can apply to receive cancer treatments that have not yet been through the approval process. In the past, these special case applications for new treatments were grouped under the name compassionate uses. More recently, such uses have expanded to include more patients and more categories of experimental or investigational treatments.
Access to Investigational Drugs
The process of new drug development has many parts. In the United States, until a drug has been approved by the FDA, it can generally be obtained only through several mechanisms: enrollment in a clinical trial studying the drug, an expanded access program, or special exemption/compassionate use programs. See Access to Investigational Drugs: Questions and Answers for more information.
Group C Drugs
In the 1970s, researchers from the NCI became concerned about the lag between the date when an investigational drug was found to have anti-tumor activity and the time that drug became available on the market. Working with the FDA, the NCI established the Group C classification system to allow access to certain drugs.
Group C drugs are provided to properly trained physicians who have registered using a special form to assure that their patient qualifies under guidelines - or protocols - for the drug. Each Group C drug protocol specifies patient eligibility, drug use, and how information about the patient's use of the drug should be reported.
Group C designation (now called Group C/Treatment INDs) speeds new drugs to patients who need them most. The process also allows the NCI to gather important information on the safety and activity of the drugs as they are used in the "real world" prior to their final FDA approval. Group C drugs are always provided free of charge, and the Centers for Medicare and Medicaid Services provides coverage for care associated with Group C therapy.
In 1987, the FDA began authorizing the use of new drugs still in the development process to treat certain seriously ill patients. In these cases, the process is referred to as a treatment investigational new drug application (Treatment IND). Clinical trials of the new drug must already be underway and have demonstrated positive results that can be reproduced. The FDA sets guidelines about what constitutes serious and life-threatening illnesses, how much must already be known about a drug's side effects and benefits, and where doctors can obtain the drug for treatment. For many seriously ill patients, the risks associated with taking a not-yet-completely proven drug are outweighed by the possible benefits.
Accelerated approval is the short-hand term for the process by which the FDA quickly conducts its review of new treatment applications while also putting new safeguards in place. Accelerated approval is based on surrogate endpoint judgments: the FDA can grant marketing approval to drugs and treatments that, according to certain indicators, prove they are likely to have beneficial effects on a disease or condition, even before such direct benefits have been shown clinically. Accelerated approval does not mean that additional clinical trials are not needed or that the FDA stops gathering information about the effects of the drug; a follow-up study is required to demonstrate activity by more conventional endpoints.