Ways to Speed Treatments to Patients : Part 2

by Food and Drug Administration (FDA)

(Page 2 of 3)

The regulations reiterate the requirement that, as with all clinical use of investigational drugs, informed patient consent must be obtained, and the product cannot be promoted or otherwise commercialized. FDA also requires that a product administered under a Treatment IND must be under (or have completed) active clinical investigation, and its sponsor must be pursuing marketing approval with "due diligence."

It's critically important to complete definitive clinical trials, because once an investigational product appears in early studies to offer an important therapeutic advance and becomes available for treatment use, "you may never get another crack at it," says Robert Temple, M.D., director of FDA's Office of Drug Evaluation I. "If a study looks favorable — seems to show an effect on survival, for instance — physicians are very reluctant to redo the study. They want the active drug for their patients."

Ethical concerns make it difficult for physicians to withhold a promising investigational drug that might forestall severe disability or death. But if the study that showed promise was not well-designed — if, for example, there was no control group — what looked like favorable results may prove to be an illusion. "So it's very important to do a good study early — right at the beginning before impressions form that might turn out to be wrong," Temple says.

He points out that the early clinical trial showing AZT to be effective in AIDS patients was a placebo-controlled study, the results of which were dramatic and unequivocal. On the other hand, in the case of ganciclovir, an anti-viral drug used to treat an eye infection in AIDS patients, the path to treatment use and ultimate approval was quite different. Early suggestions of ganciclovir's effectiveness led to wide use before controlled clinical trials ever started.

Ganciclovir was approved in 1989 on the basis of a historical comparison with other treatments. But, Temple maintains, approval of ganciclovir was almost certainly delayed for years by the lack of appropriate, controlled clinical investigation.

FDA has indicated, for purposes of Treatment INDs, what constitutes serious or immediately life-threatening illness, what scientific information about the drug's safety and potential usefulness must be in hand, and how physicians can obtain investigational drugs for treatment use.

As of August 1994, 29 agents had been granted Treatment IND status. The conditions for which they have been used include AIDS and its complications, control of infection in kidney transplant patients, severe obsessive-compulsive disorder, Alzheimer's disease, severe Parkinson's disease, various advanced cancers, and respiratory distress syndrome in premature infants. At press time, 24 of these drugs had been approved by FDA and are on the market.

Other Quick Help

An older, more targeted treatment-use initiative is aimed at making investigational cancer drugs available to patients who are not participating in controlled clinical trials. Since the mid-1970s, FDA has reviewed drugs for limited distribution by the National Cancer Institute (one of the National Institutes of Health) to provide promising new anti-cancer drugs and drug combinations to cancer patients for whom established therapy is ineffective.

Another mechanism to permit wider availability of experimental agents is the "parallel track" policy developed by the U.S. Public Health Service in response to the AIDS epidemic. Under this policy, patients with AIDS whose condition prevents them from participating in controlled clinical trials can receive investigational drugs shown in preliminary studies to be potentially useful. At press time, one drug (D4T) had been made available under the parallel track mechanism. D4T was approved for marketing in mid-1994.

Streamlining Review

Less dramatic, perhaps, than rushing investigational drugs to the desperately ill, but almost certainly of more long-range benefit to society, are measures to streamline FDA's review and approval process and expand the agency's resources for this task. Although not the stuff of which gripping movies are made, these efforts can mean earlier arrival of important new drugs in hospital and community pharmacies for the benefit of everyone who needs them.

One change FDA has adopted in recent years to speed drug review is categorizing new drugs as either standard or priority. Standard drugs are those that offer only minor improvement (or none) over drugs already on the market. Priority drugs, on the other hand — which may in fact be a new dosage form of, or new use for, an existing drug — are believed to represent potential major advances in health care. Distinguishing the two categories of drugs permits speedier review even before a new drug application is submitted.

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.