FDA/PHARMA/SCOTT GOTTLIEB: “The Food and Drug Administration plans to allow quick approval of some cancer drugs if they show early and ‘outsized’ survival benefits for patients even in small studies, the FDA’s commissioner said Thursday [11-30-17].
Scott Gottlieb told the House Energy and Commerce subcommittee on health that the agency expects to see this situation more, given the advent of drugs that precisely target the genetic underpinnings of diseases…
His concept is essentially an expansion of what the FDA currently does in what the agency calls accelerated approval. It now can approve a drug based on early evidence but require that the manufacturer do a confirmatory study to keep the medicine on the market.
Currently, such approvals are based on endpoints like tumor shrinkage that are believed to be ‘surrogates’ for clinical benefits, such as survival. Dr. Gottlieb’s new proposal would be based directly on overall survival ‘in a rare or deadly cancer,’ he testified. The concept could be used for drugs that don’t have FDA approval or to expand the approval already in existence.”
-Thomas Burton, “FDA to Allow Quicker Approval of Some Promising Cancer Drugs,” The Wall Street Journal online, Nov. 30, 2017 05:09pm