Imiquimod, molluscum, and the need for a better “best pharmaceuticals for children” act.

It was the best research Emily' s doctor had never heard about.* Emily was a delightful 6-year-old with molluscum contagiosum (MC). Characteristic dome-shaped umbilicated papules, some of them itchy, peppered her chest and buttocks. Emily had returned to the clinic, where one of us (KAK) saw her, after she failed treatment with imi-quimod 5% cream. Coincidentally, Emily was born just as 2 rigorously conducted ran-domized controlled trials (RCTs) of imiquimod for treatment of MC in children were concluding. In both studies, imiquimod proved ineffective. Six years later, and 5 years after imiquimod' s package insert (PI) had been updated to include those RCT results, no studies had shown otherwise. So why was Emily prescribed imiquimod? It would be easy to blame Emily' s doctor. But it' s not that simple. The real problem lies in a federal law that allows data from pediatric drug studies requested by the federal government and subsidized by taxpayers to escape meaningful dissemination to physicians. As a result , children like Emily are needlessly exposed to ineffective and/or unsafe medicines. Imiquimod was first approved by the Food and Drug Administration (FDA) for treatment of genital warts in adults in 1997. Physicians soon began using it off-label to treat MC in children. There were good reasons for that. First, other treatments were inconvenient and/or poorly tolerated. Second, imiquimod had proven effective for genital warts, which like MC are caused by a virus. Third, numerous observational studies and review articles, as well as 2 small RCTs, suggested that imiquimod might work. But the evidence was not overwhelming, and imiquimod is not benign. It can cause application site reactions and, rarely, flulike symptoms and other systemic adverse effects. To address questions about imiquimod' s safety and efficacy in treating MC in children, the FDA stepped in, using its authority under the Best Pharmaceuticals in Children Act (BPCA). Signed into law in 2002 and *The name of the child discussed in the manuscript has been changed to protect her privacy. BPCA—Best Pharmaceuticals for Children Act FDA—Food and Drug Administration MC—Molluscum contagiosum PI—package insert RCT—randomized controlled trial Dr Katz conceptualized the manuscript and drafted the initial manuscript; Dr Swetman helped conceptualize the manuscript and reviewed and revised the manuscript; and both authors approved the final manuscript as submitted. The views expressed in this manuscript are those of the authors and do not necessarily represent the views of The Permanente the …