Single-trial drug approvals. (Guest Editorial).

To better treat our patients' unmet medical needs, we must have better drugs brought to the market faster. To do this, we need to revamp the way drugs are developed and approved.

The Food and Drug Administration's standard for drug approval has long been that two large, empirical, well-controlled trials are needed to prove that a drug is effective.

Today this two-trial requirement is outdated, inefficient, and too costly Derived 50 years ago in the early days of clinical trial methodology the requirement unnecessarily prolongs the drug development process.

Drug companies routinely come forward armed with much scientific data--from scores of studies--that are neither exhaustively analyzed nor fully appreciated. The FDA and the pharmaceutical companies should fully examine these studies for data that haven't traditionally qualified as evidence of effectiveness. In doing so, they would actually be recognizing and promoting better science--and better drug development.

The Food and Drug Administration Modernization Act of 1997 permits drug approval on the basis of "one adequate and well-controlled clinical trial" plus "confirmatory evidence." The statute allows one-trial approval "at the secretary's discretion," but it doesn't clearly define "confirmatory evidence."

Since the statute was voted into law, the FDA has laid out some instances in which it has allowed one trial. Rep. Richard Burr (R-N.C.), who follows FDA issues in his role as vice chairman of the House Energy and Commerce Committee, says that the agency hasn't acted consistently and that manufacturers are often unclear about how the FDA determines whether evidence is sufficient or not.

The FDA also continues to maintain that, as a general rule, it needs two large trials.