Photographer: Tomohiro Ohsumi/Bloomberg
FDA Aims to Expand Early-Approval Program for Promising DrugsBy
Commissioner proposes approval based on early, small trials
Scott Gottlieb speaks before House subcommittee on health
The Food and Drug Administration wants to help patients get faster access to promising cancer treatments.
The agency is preparing proposals that would expand an accelerated-approval program for lifesaving medications, FDA Commissioner Scott Gottlieb told House lawmakers on Thursday.
Drugmakers can seek rapid conditional approval for treatments for cancer or other serious diseases based on evidence that a drug is likely to extend patient survival. Later trials once such a drug is on the market are necessary to prove the survival benefit.
The FDA is now looking at ways to conditionally approve an experimental drug if it shows increased survival in early trials in a small number of patients, Gottlieb said.
Typically, early trials are used to determine how best to craft larger studies and aren’t used for approval. But Gottlieb wants early trials to be used for approval if they show an outsized survival benefit. Any drug cleared based on early-trial data would later have to be proven in larger trials to maintain the benefit.
Gottlieb was speaking before the House Energy and Commerce Committee’s health panel on the FDA’s implementation of the 21st Century Cures Act, a bill passed last year that is meant to accelerate development of new pharmaceuticals. The commissioner has also tried to boost competition and ease drug development in an effort to lower prices.
Earlier this year, the FDA said it would update policies to help drugmakers gain approval of treatments targeting specific gene mutations that can spur tumors. Makers of cancer drugs are increasingly embracing such genetic therapies, compared to the traditional manner of identifying cancers by the affected body part, such as the lung, breast or kidney.