PhRMA responds to JAMA article on FDA flagging FDA approval pathway questions

US trade group Pharmaceutical Research and Manufacturers of America (PhRMA) has responded to yesterday’s release of a study published in JAMA, questioning the US Food and Drug Administration’s Accelerated Approval pathway.

Andrew Powaleny, director, public affairs, a PhRMA spokesperson, said: "The US Food and Drug Administration’s (FDA) expedited programs, such as the accelerated approval pathway, have helped to bring safe and effective treatments to patients with serious or life-threatening disease and where there is significant unmet medical need. The use of surrogate endpoints to predict clinical benefit, as agreed upon by both a sponsor organization and the FDA, has allowed many patients with no other treatment options to sooner reap the benefit of innovative new drugs and biologics. Recent studies have demonstrated that such medicines provide substantial health gains.”

PhRMA provided a link to a report from out Tufts which dives into the success of the accelerated approval program: http://content.healthaffairs.org/content/36/8/1408.abstract