By LEE BOWMAN
Scripps Howard News Service
December 13, 2006
Normally, the Food and Drug Administration only allows a small number of patients to take part in the first round of clinical trials for a new drug - often less than 100 for a safety check, followed by several hundred to several thousand, depending on how common the ailment is - to demonstrate that the treatment improves the condition most of the time.
Beyond that, since the 1970s, the FDA has allowed some individuals and certain groups of patients with diseases like AIDS and some cancers to get early access to experimental drugs on a "compassionate use" basis.
But in reality, unless patients were part of a recognized class with no options, access to drugs in the pipeline has mostly been limited to patients at teaching hospitals where drug trials typically are done, or to those with specialists having connections, or maybe to people who were savvy and desperate enough to track down a new medicine before it gets a brand name.
That situation should change under new rules the FDA proposed this week. They would give physicians clearer guidance on when the agency may allow them to give patients unapproved medicines as a last resort.
"FDA hopes this proposal will increase awareness in the health-care community of the range of options available for obtaining experimental drugs for seriously ill patients," said Dr. Janet Woodcock, deputy commissioner for operations.
Under the new plan, access would be open to individual patients, small patient groups and larger populations when there is no satisfactory alternative therapy.
The new guidelines more explicitly lay out how a doctor and patient should weigh the possible benefits of an experimental drug against the seriousness of the disease. For larger groups, the number of patients affected by a disease could also be taken into account.
Additionally, the new rules lay out some guidance for drug companies to charge patients for experimental drugs, at least enough to recoup the direct costs of making and distributing the product, and thus encouraging them to provide early access to more patients.
Depending on the disease and the condition of the patient, the rules could allow people access to drugs even before safety tests are completed. However, this option will probably be reserved for drugs that have shown good potential in lab animals and other tests, while showing little sign of life-threatening side effects.
The FDA still has the option of holding up access to an experimental drug if agency officials feel that wider early use will slow down the approval process. And it's important to remember that only about a quarter of the drugs that even get tested for safety in humans end up being approved by the FDA as "safe and effective" for the pharmaceutical market.
Meanwhile, the FDA will soon begin getting the same sort of adverse-event reports from the makers of dietary supplements and herbal remedies that the government already routinely receives from prescription-drug makers.
Congress approved the new consumer-protection bill, which also encompasses all over-the-counter drugs, shortly before it adjourned. President Bush is expected to sign it into law soon.
Specifically, a manufacturer would have to tell the FDA anytime it becomes aware that someone died, sustained a life-threatening illness, was hospitalized or suffered significant disability, a congenital anomaly or birth defect after consuming its product. Labels on all products will have to include a phone number that patients can call to report a bad reaction.
The adverse-event reports would have to be made to the FDA within 15 days. The manufacturer would have to keep records of any reports of problems with a product, even if it wasn't serious enough to report to the FDA, for six years.
The new rules were strongly supported, even sought, by the dietary-supplement industry, whose members have undertaken voluntary steps to standardize the content and labeling of their products in recent years.
Until now, supplements got little scrutiny from the FDA because they're considered foods, not medicines, so long as the seller makes no specific claims that a product will treat or prevent a disease.
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