The Pharmaceutical Research and Manufacturers of America (PhRMA) issued a statement on FDA's draft guidance on labelling for human prescription drug and biologics approved under the accelerated approval pathway.
In March 2014, the FDA issued draft guidance, Labeling for Human Prescription Drug and Biological Products Approved Under the Accelerated Approval Regulatory Pathway
to assist applicants in developing the Indications and Usage Section of labeling for human prescription drug and biological products for indications that are approved under the accelerated approval regulatory pathway. The draft guidance focuses on indications and usage statements for drugs approved on the basis of a surrogate endpoint or an effect on a clinical endpoint other than survival or irreversible morbidity. The draft guidance also addresses labeling considerations for indications that were approved under accelerated approval and for which clinical benefit subsequently has been verified and the FDA terminates the conditions of accelerated approval or when the FDA withdraws accelerated approval of an indication while other indications for the drug remain approved.
“PhRMA shares FDA’s goal of ensuring that information on the use of drugs and biological products approved under the Accelerated Approval pathway is consistently presented in the appropriate sections within prescription labeling so that the information is clear and accessible to healthcare providers." said PhRMA Vice President of Scientific and Regulatory Affairs Sascha Haverfield, in a statement. "However, the draft guidance’s proposal of limitations and caveats in the ‘Indications and Usage’ statement could be misinterpreted to mean that products approved under the Accelerated Approval pathway have not met the statutory requirements of safety and efficacy required of all FDA-approved drugs This could result in confusion among healthcare providers and the public, undermining the intent of the Accelerated Approval pathway and recent Congressional efforts to encourage its broader use," she said.
"PhRMA urges FDA to preserve the intent of Accelerated Approval to expedite patient access to treatments for serious conditions, and to preserve the primary purpose of prescription labeling as a mechanism to provide information to health care providers so they may prescribe medicines safely and effectively. Further, we urge FDA to continue to explore ways to enhance its use of existing regulatory flexibility to expedite patient access to treatments for serious or life threatening conditions and to ensure relevant information about both benefits and risks is accurately communicated in labeling. PhRMA suggests that the FDA integrate its efforts regarding communicating benefit, risk, and any uncertainty for the Accelerated Approval pathway with the Agency’s Prescription Drug User Fee Act (PDUFA V) benefit-risk initiatives"
She continued: "We look forward to working with FDA as the Agency continues to implement the Food and Drug Administration Safety and Innovation Act (FDASIA) provisions related to the Accelerated Approval pathway and other programs intended to expedite development and review of new drug and biological products for the treatment of patients with serious or life threatening conditions.”
PhRMA’s complete comments on the draft guidance are available here.