Innovative Drugs May Win U.S. Approval After Early Trials

Drugmakers may win approval from U.S. regulators for “breakthrough” therapies after a single round of studies, rather than three, in an effort to speed them to patients, a Food and Drug Administration official said.

The agency assigned three experimental medicines the new status to try to reduce the time needed to get them to market against deadly diseases, Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, said yesterday in an interview before a Bloomberg health conference in New York. Vertex Pharmaceuticals Inc. said last month that two of its drugs for cystic fibrosis had won the designation.

Breakthrough status means the companies will have closer communication with top FDA staff to move drugs for serious diseases to market more quickly, potentially with data from an expanded Phase 1 trial, Woodcock said. Improved understanding of diseases is leading to more candidates for this status.

“We expect many of these would come available very quickly with Phase 1 data,” Woodcock said.

New drugs generally need three phases of clinical testing on safety and effectiveness to win FDA approval. In addition to the three treatments that have won the new designation, 18 other drugs have applied, most for types of cancer, Woodcock said.

The breakthrough designation was created in legislation passed last year reauthorizing the FDA’s user fee programs for drug and device reviews. The agency didn’t receive authorization to hire more full-time employees for the enhanced communication and is attempting to balance its priorities.

“We don’t want to disadvantage ordinary drugs,” Woodcock said.

Biologics Review

In contrast to the breakthrough drugs, Woodcock said the agency is taking its time to consider whether to approval copies of biotechnology medicines, so-called biologics.

While the FDA has been contacted in connection with 12 different biologic therapies, the agency is meeting with companies one by one instead of drafting guidelines on specific classes of drugs, she said.

“It’s probably going to take a while for this program to become established in the United States but there’s robust interest,” she said.

To contact the reporter on this story: Anna Edney in Washington at

To contact the editor responsible for this story: Reg Gale at

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