Pediatric Formulations Platform
Inter-Agency Agreement between the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the U.S. Food and Drug Administration (FDA)
The Best Pharmaceuticals for Children Act (BPCA) is a federal legislation designed to improve pediatric therapeutics and labeling. The NIH is mandated to prioritize drugs and therapeutics in need of study, to sponsor the necessary pediatric clinical trials, and to submit the data to the U.S. Food and Drug Administration (FDA) for labeling change. Lack of a pediatric formulation was one consideration for prioritization.
The obvious lack of commercially available oral pediatric formulations is a continuing problem for children, parents, and pediatricians. In addition, there is no coherent approach to the development of pediatric formulations. The ideal pediatric dosage form would be orally dissolvable; be tasteless; have an appropriate dosage increment for the smallest infants; contain minimal amounts of excipients; be stable in light, humidity, and heat; and have the necessary release characteristics, as applicable.
In 2009, the National Institutes of Health (NIH) and the FDA entered into an Inter-Agency Agreement (IAA) entitled Pediatric Formulations Platform using NIH BPCA funds to fund FDA chemists’ assessment of pediatric product formulations and produce an open-source, publicly available approach to pediatric oral formulations manufacturing.
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The work is broken down into six tasks:
- Perform an assessment of all commercially available products to determine which have pediatric formulations
- Determine what technologies are publicly available, how these technologies have been used, and for what types of products
- Use prototypical drug products and employ computational methods to characterize their molecular structure for characteristics such as solubility, permeability, light sensitivity, pH instability, heat instability, hygroscopic properties, and bitterness
- Determine the best formulations technology for specific drug categories, based on information from tasks 1−3
- Produce prototype batches of selected drug products.
The work involved has been quite labor-intensive. The goal is to have these data made publicly available as soon as possible. As a first step, we are posting the Biopharmaceutics Classification System assessment of all the commercially available products. As further stages are completed, they too will be posted on the BPCA website. It is a work in progress, so there may be some blanks which will be filled in as time goes on. Thank you for our interest and stay tuned!
Oral Formulations Platform—Report 1 (PDF 747 kb)
Collaboration with NHLBI, NIAID on Asthma Outcomes Meeting
This Interagency Agreement established a formal cooperative relationship between multiple institutes and agencies including the National Heart, Lung, and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID), and the NICHD. The NICHD provided some funding for the project entitled “Asthma Outcomes Workshop: Developing Standardized Definitions and Methodologies for Outcome Measures for Asthma Clinical Research”. The purpose of the Workshop, held on March 15-16, 2010, was to develop standard definitions and data collection methodologies for establishing and validating outcomes measures in asthma clinical research with the goal of enabling comparisons, including cost/benefit evaluations, across asthma studies and asthma treatment modalities. In addition, the workshop sought to identify promising outcomes for asthma clinical research and record their potential, development status, and further validation needs. Publication of the proceedings is still pending.
Interagency Agreement with the NICHD and the Centers for Disease Control and Prevention National Center for Health Statistics
The purpose of this Interagency Agreement is to participate in the design and implementation of a national survey of physicians about their opinions and use of the National Asthma Education and Prevention Program (NAEPP) Guidelines for the Diagnosis and Management of Asthma (“The Guidelines”). The objectives of this research include the following: 1) to assess familiarity, acceptance, and implementation of key elements of the NAEPP asthma guidelines for the diagnosis and management of asthma; 2) to obtain sufficient sample size to allow comparison between provider specialties and geographic region and; 3) to use the data to improve implementation of guideline-based care and thereby, outcomes for asthma patients. The survey is scheduled to be launched in 2012.
Interagency Agreement with the NICHD and the NHLBI PROP Network
The Prematurity and Respiratory Outcomes Program (PROP) will investigate multiple research hypotheses on the molecular mechanisms that contribute to respiratory disease risk of premature neonates over the first year of life. A standardized bundle of clinical and non-invasive respiratory assessments that is tailored to the respiratory status of the infant at the time of testing will be performed near the estimated due date of very preterm infants to describe the cohort and to predict the severity of respiratory outcomes in the first year of life. It is hoped that these respiratory assessments may be sufficiently predictive to serve as surrogate endpoints in future trials of prevention and therapy.
NICHD/NIGMS T32 Training
Recognizing the need to increase the number of trained pediatric clinical pharmacologists, the NICHD and the National Institute of General Medical Sciences (NIGMS) co-funded six pediatric positions under NIGMS’ T32 Post Doctoral Programs in Clinical Pharmacology. An additional position for post-doctoral training in pediatric pharmaco-epidemiology was transferred from the NICHD’s Division of Epidemiology, Statistics and Prevention Research and is also included in this grouping. In 2011, the NICHD supplemented these sites by adding three new programs through their own Post Doctoral Research Training in Pediatric Clinical and Developmental Pharmacology T32 training grants. Together these eleven programs form the Pediatric Clinical and Developmental Pharmacology Training Network (PCDPTN), a collaborative network that seeks to stimulate interdisciplinary collaboration of clinical, translational, and basic investigators working in complementary areas of research in pediatric therapeutics. Rather than functioning as isolated programs, the goal of the Network is for the principal investigators from each of the sites to function as a coordinating body that would facilitate interactions and collaboration between the programs. Some of the activities the Network is engaged in include harmonization of fellows’ core curriculum, development of strategies to bridge the gap between adult and pediatric therapeutics, and coordination of a monthly lecture series on topics of relevance to pediatric pharmacology.