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.
- Paediatric Drugs 10/2012; 14(5):295-7. DOI:10.2165/11640510-000000000-00000 · 1.72 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