Cancer Drugs, Part 2

Risks and Benefits
by Food and Drug Administration (FDA)

(Page 2 of 3)

For a drug to treat a common cancer, such as breast cancer, the agency would expect large clinical trials with hundreds or even thousands of participants. But the FDA would consider a drug for rare cancers tested in a small number of people.

The FDA may approve a use for a drug if the drug shows evidence that people live longer or live without the disease getting worse for a certain period of time, even if it's just a few weeks or a few months.

Physical Evidence

In addition to evaluating data from clinical trials, the FDA may look at X-rays, computed tomography (CT) scans, or even photographs that a pharmaceutical company provides to establish whether a drug is truly effective. For the review of Zolinza (vorinostat), a drug to treat skin lesions in cutaneous T-cell lymphoma (CTCL), the agency had asked the drug company to submit before-and-after photographs of skin lesions. CTCL is a cancer of a type of white blood cell, and patients who have it develop a variety of skin lesions.

"In some of the photographs, improvement is easy to see, but in others, it is difficult to evaluate," says Bhupinder Mann, M.B.B.S., an oncologist in the FDA's Office of Oncology Drug Products. The raised surface of a skin lesion may be evident to the touch, but not easily distinguishable in a photograph.

The photographs were considered to be supporting evidence to help assess the benefit of the drug, says Mann. The pharmaceutical company was also required to submit measurements of the lesions and classification of their severity.

Weighing Risks and Benefits as a Patient

The FDA weighs risks and benefits of a drug in its decision-making as a regulatory agency. But every person diagnosed with cancer, in discussions with his or her doctor, must also weigh benefits and risks before making treatment decisions as a cancer patient.

These decisions can be very difficult, especially when a person is first diagnosed, says Patty Delaney, director of the Cancer Liaison Program in the FDA's Office of Special Health Issues, and two-time cancer survivor. "You walk into the doctor's office, and you may feel fine - you walk out, and you're devastated." With some cancers, she says, "you then start the slow degradation into feeling absolutely horrible. You then slowly come to terms with your diagnosis and begin to hope that maybe you will get better - but you also know that the disease may kill you. It's terrifying."

Patients who don't respond to the standard of care treatments also are faced with hard decisions, says Delaney. "Sometimes there's not very strong data in the literature on what to do." The doctor asks the patient what he or she wants to do, and the patient doesn't know what to choose, she says. "People want to be in a partnership with their doctor, but they're often left with making a hard choice with little information."

"You can have your doctor choose for you," adds Delaney. "There is nothing wrong with that."

Philip Rosoff, M.D., agrees, noting an emphasis in society on autonomous decision-making that doctors are often afraid of impinging upon. "We've abandoned our roles as advisors and healers, says Rosoff, an associate professor of pediatric hematology - oncology at Duke University School of Medicine and director of the Duke Hospital Clinical Ethics Program in Durham, N.C. "We give patients this smorgasbord of options: A, B, C, and D - then ask, what do you want to do? Instead of giving A, B, C, and D, and saying, 'this is what I would do.' We do a disservice to patients when we don't use our expertise to give advice and to make recommendations to them."

When a number of treatments have been tried and failed and the person is dying, doctors may discourage further treatment, says Delaney, but patients don't want to run out of options. "Most don't want to hear that they aren't going to get treatments. Some of these drugs are like atom bombs going off in front of you, but a patient is often willing to take many more risks. People have a survival instinct. The mindset of people with cancer is, 'just give me anything and everything.'"

Experimental Cancer Drugs

The agency has put in place a number of regulatory programs and works with manufacturers so that seriously ill patients can get access to promising, but not fully evaluated, products.

Clinical trials. A common method to get an unapproved drug is to enroll in a clinical trial. More than 10,000 cancer clinical trials are ongoing in the United States. People interested in clinical trials should talk with their doctor, check out available trials at clinicaltrials.gov, or call the National Cancer Institute's (NCI's) Cancer Information Service at (800) 4-CANCER (422-6237).

If a person does not meet the criteria to participate in a clinical trial, an investigational new drug sponsor can make an exception to treat the patient. The patient's data would not be analyzed with the primary data from the original trial, but would be evaluated separately. Usually, such exceptions occur in the same institutions that are conducting the original trial, where investigators are familiar with the drug.

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.