Measuring the Patient Health, Societal and Economic Benefits of US Pediatric Therapeutics Legislation
ABSTRACT Through at least the mid-1990s, children were often referred to as 'therapeutic orphans' for whom many treatments were administered without the benefit of appropriate studies to guide drug labeling for dosing and other critical therapeutic decisions. At that time, there were no incentives for manufacturers to pursue such work, nor regulatory requirements to compel these studies. Congress addressed this by including an important provision titled the Best Pharmaceuticals for Children Act (BPCA) in the 1997 Food and Drug Administration Modernization and Accountability Act. This was complemented by another key piece of legislation, the Pediatric Research Equity Act (PREA) in 2003. The former Act and its successors created an incentive for firms to study on-patent drugs in pediatric populations by extending the market exclusivity of a medicine by 6 months. The latter was a requirement that provided the US FDA with the authority to require studies of drugs in children if an adult indication also occurs in children. In the current paper, we consider the effects of both pieces of legislation in terms of the health, societal, and economic benefits they have likely imparted and will continue to provide in the future. We conclude that the gains have been substantial - both in terms of safer and more effective use of medicines in children and in terms of new research that has been incentivized by the BPCA exclusivity provision. We estimate the gross economic benefits from the latter alone to be approximately $US360 billion.
SourceAvailable from: Maryann Mazer-Amirshahi[Show abstract] [Hide abstract]
ABSTRACT: Background There is often a lack of safety and efficacy data in the paediatric population at the time of drug approval. Legislative efforts have promoted clinical pharmacology research in this underserved population. We sought to determine the quantity and quality of paediatric clinical pharmacology data in US drug labelling at the time of initial approval and to evaluate trends over time. Materials and methods The labelling data of 213 new molecular entities approved between 2003 and 2012 were systematically reviewed. The type of paediatric pharmacology data present at the time of approval was recorded and stratified by age group. Labelling revisions were analysed for updated paediatric data. The presence of paediatric-specific black-box warnings was noted. Results Of the 213 drugs evaluated, 48 had adult-specific indications. Of the remaining 165 medicines, only 47 (28%) had paediatric study data at the time of initial labelling. The number of approved drugs with paediatric data was the greatest in 2005 (8, 44%) and was at its lowest point in 2012 (3, 11%). Only five medicines had neonatal data, with none of the anti-infective agents presenting neonatal information. Seven medications had a paediatric-specific black-box warning. Additional 16 medicines presented paediatric data during general labelling updates. Conclusions Despite efforts to improve the quality of paediatric clinical pharmacology data, there was not a significant increase in drugs with paediatric data at the time of approval over this 10-year study period. Paediatric drug approvals and labelling revisions continue to lag behind their adult counterparts.Archives of Disease in Childhood 07/2014; 99(9). DOI:10.1136/archdischild-2013-305605 · 2.91 Impact Factor
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ABSTRACT: To provide current information on off-label medication use in pediatric gastroenterology, including a discussion on US legislative efforts to address the issue. Medications used to treat pediatric gastrointestinal illnesses are frequently prescribed off-label. Acid suppressors, antiemetics, laxatives, and antitumor necrosis factor therapies are types of medications frequently used off-label in the pediatric gastroenterology arena. Pediatric studies conducted under US Federal laws are generating much-needed data on the safety and effectiveness of medications used to treat pediatric patients. Moreover, a new US law, the Food and Drug Administration Safety and Innovation Act, may further the development of pediatric medications in part by requiring pediatric-specific study plans earlier in the overall drug development process. As of today, there still are gaps in our knowledge about these medications, including for the treatment of pediatric gastroenterology diseases. Medications are widely used off-label in pediatrics, including medications intended to treat gastrointestinal diseases, such as antitumor necrosis factor and laxatives. Although legislation is helping to generate and make available important information about pediatric medications, most still do not contain pediatric data. Therefore, providers need to understand the potential risks and benefits of prescribing off-label products to pediatric patients.Current opinion in pediatrics 08/2013; 25(5). DOI:10.1097/MOP.0b013e328363ed4e · 2.74 Impact Factor
Article: Clinical Excellence in Pediatrics[Show abstract] [Hide abstract]
ABSTRACT: The 7 core domains of clinical excellence in academic medicine, as defined by the Miller-Coulson Academy of Clinical Excellence at Johns Hopkins, are applicable to the field of pediatrics. The authors use published case reports and teaching models from the pediatric literature to illustrate how thoughtful clinicians have realized distinction in each of the 7 clinical excellence domains, recognizing excellent pediatric patient care serves to strengthen all 3 arms of the tripartite academic mission. Clinicians who feel valued by their institution may be more likely to remain in an academic clinical setting, where they promote the health and well-being of their patients, provide support to families and caregivers, serve as role models for pediatric trainees, and integrate research into their practice with the overall aim of improving patient outcomes.Clinical Pediatrics 05/2014; 53(9). DOI:10.1177/0009922814533408 · 1.26 Impact Factor