Medication Development and Testing in Children and Adolescents

Abstract

Progress in pediatric psychopharmacological research has suffered notable delay, especially compared with the achievements in adult psychopharmacology. Although safety and efficacy of the use of many psychotropic agents in children remain largely unproved, their pediatric use has been increasing and their widespread off-label prescribing by practitioners has raised some important concerns. The National Institute of Mental Health, in cooperation with the Food and Drug Administration and leading researchers, has coordinated systematic efforts to identify the major obstacles to research in pediatric psychopharmacology and to propose feasible solutions. In 1995, a conference cosponsored by the national Institute of Mental Health and the Food and Drug Administration gathered more than 100 research experts, family and patient advocates, and representatives of mental health professional associations. Participants met in working groups focused on specific aspects of child research and reached consensus on various recommendations. Each of the various aspects relevant to conducting research in this area (methodological, ethical, legal, regulatory, financial, and family or community context) presents specific challenges, which are herein outlined. Recommendations for possible solutions are presented, some of which are being implemented. Because data about drug safety and efficacy in adults can rarely be extrapolated to children, there is no substitute for pediatric psychopharmacological research. Successful strategies for overcoming the many obstacles with which this research has to contend must enlist the concerted efforts of all the relevant parties (investigators, clinicians, industry, federal agencies, ethicists, families, and community representatives).

Off-label prescribing by psychiatrists

“…The use of off-label prescriptions has particularly exercised child and adolescent psychiatrists (since relatively few psychotropic drugs are licensed for use in children) and psychiatrists dealing with those with a learning disability (British Association for Psychopharmacology, 1997;Vitello & Jensen, 1997). A recently published study (Conroy et al, 2000) looked prospectively at off-label prescribing in five paediatric wards in five countries over a period of 4 weeks.…”

Child and Adolescent Psychopharmacology in the New Millennium: A Workshop for Academia, Industry, and Government

“…A mechanism is needed for deciding and prioritizing the questions. One effective mechanism would be to convene a National Institutes of Health Consensus Development Conference, as was done in 1995, to explore these issues and to help define the research agenda (Vitiello and Jensen, 1997). To be effective globally, however, such a conference needs to have international participation consistent with the spirit and intent of ICH.…”

Children in Need of Medical Innovation

“…In contrast, the increasing off-label use of psychotropics in children, coupled with the lack of adequate data to support their efficacy and safety, was eventually recognized as an important public health issue to be addressed through research in both the U.S. (Vitiello & Jensen, 1997;Jensen et al 1999) and Europe (Fegert 2003). In spite of the orphan drug regulations in Europe since 2001 until now (see below), 51 drugs or application modifications have been licensed.…”