Reconsideration of 2008 decision: Food and Drug Administration approval of etanercept for systemic treatment of moderate to severe pediatric psoriasis.

Although systemic etanercept was approved in 2004 for adults, the Food and Drug Administration (FDA) denied approval for use in children with psoriasis in 2008. Revision of the FDA's risk-benefit assessment in response to understanding of disease burden, unmet medical need, and the effect of off-label use in children with psoriasis led to the 2016 approval as the first systemic biologic product for the treatment of children aged 4-17 with moderate to severe psoriasis. This article delineates the thinking that led to this reconsideration. The underlying thinking paved the way to inform current pediatric drug development as the FDA continues to bring needed medical products to children.

Sources of drug information: FDA-approved labeling and other official FDA sources.

To protect the public health and facilitate the safe and effective use of prescription drugs, the Food and Drug Administration (FDA) disseminates information through drug labeling, communication of safety issues, and the archiving of scientific reviews. The content and format requirements for professional labeling were revised in 2006 to improve the accessibility and usability of the information. New or emerging safety information is communicated using the formats of public health advisories (PHAs), information for heath care professional sheets, and early communications about ongoing safety reviews. The FDA analyses of approved drug marketing applications and Advisory Committee transcripts are posted on the FDA Web site. Prescribers can utilize these resources to inform the care that they provide to patients.

Can we ensure the safe use of known human teratogens?: The iPLEDGE test case.

Minimising the public health burden of isotretinoin-induced teratogenicity has been a challenge for 24 years, the duration of availability of isotretinoin in the US for the treatment of severe, recalcitrant nodular acne. Although the teratogenicity of this drug is well known and risk-management programmes had been implemented, preventable fetal exposures continued to occur, largely as a result of the lack of sufficient controls within the programmes themselves. The manufacturers of isotretinoin implemented a new risk-management programme, iPLEDGE, in March 2006. iPLEDGE is a comprehensive distribution system that includes mandatory registration of patients, healthcare providers, pharmacies, and wholesalers. It allows real-time linkage of pregnancy-test results for verification prior to the dispensing of isotretinoin. Although the challenges of implementing a closed distribution system for a very widely used medication have been extensive, the potential public health benefits from preventing fetal exposure to isotretinoin are substantial.