Hearing The Cry For Help And Information

FDA Consumer, Jan, 2001 by Theresa Toigo

The phone rings.

"My mother has cancer," says the woman on the other end of the line. "Her oncologist says that available cancer treatments probably won't help much. The doctor suggested that she consider enrolling in a clinical trial. I don't know how to do that. Where can I get information about clinical trials?"

We get this kind of call just about every day in the FDA Office of Special Health Issues (OSHI). The patients have serious and life-threatening diseases, particularly HIV/AIDS and cancer. They're afraid. Their families are afraid. They want some options, but they're not sure where to go.

So we take their calls. OSHI tries to provide the information that patients and their families need to make important decisions in such difficult situations. The law limits our discussions about new treatments in the pipeline. We can't share trade secrets or say when new drugs might be approved.

But we can take the time to listen. And we do take the time to answer questions. We can educate consumers about FDA's role in approving drugs, and about what the agency can and cannot do. And we can give them the information that sometimes gives them hope.

The questions have a common thread: clinical trials. The reason is simple. When the known therapies fail, only the unknown remains. There's plenty that's unknown on the frontier of medical research. But there's also hope. Sometimes the new therapies work, miraculously, like the protease inhibitors that helped transform the treatment of AIDS. And the patients who try them first are the ones in the clinical trials.

The common thread means that many of the questions repeat from call to call. Here's a sampling:

What are clinical trials? Clinical trials are research studies designed to test new treatments. No one knows whether the new treatment will work, but there's usually evidence that it may be effective in people. As part of a clinical trial, patients get expert medical care, often in universities. But it's important to remember that the purpose of a clinical trial is to study what a drug's risks and benefits might be. More information can be found in an FDA Consumer article, "Testing Drugs in People," available at www.fda.gov/fdac/special/newdrug/testing.html.> How can I find out about new treatments in development and clinical studies currently underway? It is often difficult for patients to learn about opportunities to participate in clinical trials, but the government has created a new database filled with information about clinical trials. It's on the Internet at http://clinicaltrials.gov/. Developed by the National Institutes of Health's National Library of Medicine and the FDA, it contains information on approximately 5,000 federally funded clinical trials. And it will soon include more drug company-sponsored clinical trials. Information about clinical trials is also available from patient organizations, which can be located through a variety of Internet sources, including MedlinePlus on http://medlineplus.gov.

Patient advocacy organizations can be enormously helpful because they provide advice and support, often from patients who share the same diagnosis. Other information sources include the AIDS Clinical Trials Information Service (1-800-TRIALSA, www.actis.org) for HIV/AIDS trials, and the Cancer Information Service (1-800-4CANCER, http:// cancernet.nci.nih.gov/). Non-government-sponsored Web sites include organizations like the Pharmaceutical Research and Manufacturers of America (www.phrma.org/searchcures/newmeds/).

How can I get an investigational drug if I can't participate in a clinical trial? FDA regulations allow your doctor to obtain investigational treatments outside of a controlled clinical trial, in some circumstances, through a variety of expanded-access programs such as single-patient INDs (or Investigational New Drugs) and treatment IND protocols. More information can be found in an FDA Consumer article, "FDA Finds New Ways to Speed Treatments to Patients," available at www.fda.gov/fdac/special/newdrug/speeding.html.> How does FDA protect patients in a clinical trial? FDA carefully oversees the clinical research process to protect patients from unreasonable risks. In addition, study procedures are reviewed by an Institutional Review Board (IRB), usually a local administrative review group, made up of scientific and non-scientific members. The IRB helps protect the rights and welfare of research participants. The informed consent process provides an opportunity for the investigator and patient to exchange information and ask questions. Patients invited to enter a trial are not obligated to join, but can consent to participate if they find the potential risks and benefits acceptable.

Clinical trials offer no guarantees when standard treatments fail. But they offer an alternative to giving up. The FDA Office of Special Health Issues staff is knowledgeable about clinical trials and ways to find treatment information. We're here to listen to patients and their families and to help them find the information they need to make better-informed decisions with their doctor.