This is a historic occasion. It is remarkable that representatives from Europe (European Society for Developmental Pharmacology), Japan (Society for Pediatric Clinical Pharmacology), and the United States (Pediatric Pharmacology Research Unit [PPRU] Network) have come together with a single purpose: to discuss the solution to the therapeutic orphan problem. I would like to think that the establishment of the PPRU Network in 1993 played a significant role in bringing us all together. Most of you are aware that the PPRU Network was established after a workshop of the Forum on Drug Development of the Institute of Medicine of the National Academy of Sciences on April 23–24, 1990. The workshop proceedings published in 1991 strongly recommended that steps be taken to eliminate the therapeutic orphan situation.
Last year I attended the European Society of Developmental Pharmacology meeting in Hungary. At the meeting I held discussions with Sir David Hull from England, an invited lecturer of the meeting; with Elisabeth Autret, the newly elected President of the European Society of Developmental Pharmacology; and with Gerard Pons, the Executive Secretary of the Society. As a result of our discussions, we agreed to hold a meeting in Washington, DC, at the time of the regularly scheduled meeting of the PPRU Network, just before the Annual Meeting of the American Pediatric Society–Society of Pediatric Research. After many months of planning it is indeed remarkable that we have assembled here to devote an entire day discussing solutions to the therapeutic orphan. It is our hope that by sharing information and strategic approaches to the problem a global assault will be made, and in a few years we will no longer talk of the therapeutic orphan because drugs have been studied in infants and children.
I want to thank Dr Jeffrey Blumer from Cleveland, Ohio, (Rainbow Babies and Childrens Hospital) who has volunteered to serve as the rapporteur of the conference and to put together the manuscripts submitted from each presenter.