Cancer Drugs, Part 3

Risks and Benefits
by Food and Drug Administration (FDA)

(Page 3 of 3)

Single-patient, or emergency, investigational new drug. If enough is known about an investigational drug's side effects and there is some evidence of effectiveness, the FDA may allow a patient to receive a drug in his or her own specifically designed study. Although the FDA's requirements are relatively simple, setting up this kind of access for an individual patient is not, and involves the following:

• The pharmaceutical company must be willing to provide the new drug to the patient. This provision can be expensive and time-consuming for the company since it must track shipments of the drug, create special instructions for its use, and devise a way to collect information on toxic side effects for each patient.
• The study treatment and an informed consent document must be approved by the local institutional review board, a panel of scientists and non-scientists in hospitals and research institutions who ensure the safety and well-being of human subjects involved in research.
• The patient must give informed consent, understanding that the drug is not approved and may cause known and unknown side effects ranging from mild to fatal.
• The patient's physician must be willing to take responsibility for treating the patient and agree to collect information about the effects of the drug.

Treatment investigational new drug. A promising new drug can be distributed outside of clinical trials under a treatment investigational new drug if

• the drug is intended to treat a serious or immediately life-threatening disease
• there is no comparable or satisfactory alternative drug or other therapy available to treat that stage of the disease in the intended patient population
• there is presumptive evidence that the drug may offer some benefit to certain patients
• proper clinical trials are well under way to see whether the drug really does offer patients any benefit.

The FDA cannot force a pharmaceutical company to give an individual patient an investigational drug outside of its planned clinical trials. The drug manufacturer makes the final decision to provide an experimental treatment to a patient. The company may consider many factors, including the amount of information available about the drug, the amount of drug available, and how best to use its resources to optimize development of the drug.

Cancer Drugs for Children

As with drugs for adults, the FDA weighs the risks and benefits of drugs for children based on clinical trials. "But it's hard to do clinical trials in children for many drugs - not just cancer drugs - because it's difficult to get a large number of patients enrolled," says Karen Weiss, M.D., pediatric oncologist and deputy director of the FDA's Office of Oncology Drug Products.

Even the most common cancer in children, leukemia, strikes only one-tenth as many children - about 3,200 - as adults each year in the United States, according to the NCI. "Acute leukemia has an 85 percent survival rate, so to show that a new drug improves survival, a sponsor would need a large study and it would take many years," says Weiss.

Sometimes, in lieu of conducting randomized trials in children to demonstrate the effectiveness of a drug, the FDA may extrapolate findings from adult trials of a drug that might also be promising for children. This action may be taken when the disease being treated is similar between adults and children.

Because of early diagnosis and successful cancer treatments, about three-fourths of children with cancer are cured and able to live into adulthood, according to the NCI. Although the cure rate is encouraging, the results of a study of survivors of childhood cancer are disturbing.

The Childhood Cancer Survivor Study found that survivors appear to have a high rate of chronic health conditions later in life due to organ damage caused by chemotherapy and radiation during cancer treatments.

The study tracked more than 10,000 children diagnosed with cancer from 1970 to 1986 and compared their health with the health of 3,000 siblings. The researchers reported in the Oct. 12, 2006, issue of the New England Journal of Medicine that 30 years after a diagnosis of cancer, almost three fourths of survivors have a chronic health condition, more than 40 percent have a serious health problem, and more than one-third have multiple conditions."

Doctors and patients need to be aware of the potential for later health problems, says Rosoff, who authored an editorial in the same journal. He emphasizes the importance of monitoring children who survive cancer as they grow up. "We need to try to convince people they need to be vigilant lifelong."

Treatment Decisions

The American Cancer Society (ACS) stresses the importance of having frank, open discussions with your cancer care team and getting answers to all of your questions to help you understand your specific condition and your options so that you can actively participate in your treatment decisions.

The ACS offers free interactive tools on its Web site to help people with cancer make informed decisions about their treatment. The tools provide details specific to a condition, a breakdown of treatment options and side effects, personalized reports with pros and cons of treatment, and questions to ask the doctor.

About the Author

www.fda.gov
FDA is A United States government body that oversees medical devices, including contact lenses, intraocular lenses, excimer lasers and eyedrops. In the US, these products must be approved by the FDA before they can be marketed.