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FDA Aims to Expand Access To Experimental Drugs

By JENNIFER CORBETT DOOREN
Wall St Jnl
December 11, 2006 1:43 p.m.

WASHINGTON -- The Food and Drug Administration proposed new regulations Monday that the agency hopes will provide broader access to experimental drugs for seriously ill people who have exhausted all other commercially available treatments.

While the proposals don't establish any new so-called expanded access programs, FDA officials said they believe clarifying how to use the programs and how companies and research institutions can recoup the cost of experimental drugs will make it easier for patients to have access to therapies under development.

Janet Woodcock, the FDA's deputy commissioner for operations, explained that large patient groups with cardiovascular diseases, cancer and HIV have long been able to access drugs that are currently being developed and are in clinical trials. However, outside of clinical trials, FDA regulations have been confusing and potentially a barrier to small groups and individual patients who want to receive access to experimental treatments. The new regulations will explain how patients can receive access to such drugs, even though the patients don't fit the exact criteria for participating in the trial.

"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," Ms. Woodcock said.

One of the biggest changes lays out how companies and researchers can charge patients for the experimental treatment. Ms. Woodcock said that while many large pharmaceutical firms don't charge patients taking experimental drugs, many small or start-up firms simply can't afford to participate in expanded access programs.

The rules allow firms or even research institutions to charge for the cost of manufacturing and providing a drug, but they aren't allowed to make a profit.

The rules also will allow large firms to recoup some costs and could be used in situations where a firm decides a proposed treatment isn't worth developing any further. While a particular drug might have failed to work in most patients, it may have worked in some patients and they may want to keep taking the drug. Only about 10% to 15% of proposed drugs eventually receive FDA approval.

In all cases, the FDA would still decide whether a patient could receive access to the experimental drugs in order to make sure that certain safety rules are met. The proposed rules are open for public comment for 90 days. (Read the proposals.)