Imiquimod, Molluscum, and the Need for a Better “Best Pharmaceuticals for Children” Act

Abstract

* Abbreviations: BPCA — : Best Pharmaceuticals for Children Act FDA — : Food and Drug Administration MC — : Molluscum contagiosum PI — : package insert RCT — : randomized controlled trial 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 imiquimod 5% cream. Coincidentally, Emily was born just as 2 rigorously conducted randomized 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 … Address correspondence to Kenneth A. Katz, MD, MSc, MSCE, The Permanente Medical Group, Inc., 7601 Stoneridge Dr, Second Floor, Pleasanton, CA 94588. E-mail: kenneth.katz{at}gmail.com