- October 22 - 23, 2020
- - ET
- - ET
The U.S. Food and Drug Administration (FDA) in collaboration with the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) will host a two-day virtual public workshop entitled “Pediatric Dose Selection” on October 23, 2020 to discuss the present state of dose selection and how it pertains to pediatric drug development.
Dose selection for new drug products remains a critical part of pediatric drug development. A variety of techniques have been used to predict the pediatric dose, but the prediction is generally based on no or incomplete pharmacokinetic data. The workshop aims to review the current methods used to determine the pediatric dose and address ways to overcome dose selection challenges seen in pediatric drug development.
Dose selection for new drug products for pediatric patients remains a critical part of pediatric drug development. The statement made in 2010 by Drs. Darrell Abernethy and Gilbert Burckart is still true today: “Selection of a drug dose in pediatrics is generally based on no or incomplete pharmacokinetic data. Traditionally, allometric, or scaling, techniques have been used; however, they have inherent limitations and may not make optimal use of the drug-specific clinical pharmacokinetic information that is available. Modeling is a tool that holds promise. The future challenge is to create a structured approach to determining pediatric doses for new therapeutic agents.”1
Pediatric dose selection has been identified as one of the major reasons for failures in pediatric drug development trials.2 This is particularly concerning at a time when the FDA is encouraging the extension of drug development trials into neonates whenever possible. Older children and adolescents also present challenges for dosing in new drug development. The place and method of allometric scaling for dosing for children has been debated, as have other means of filling the gaps of studies that ethically cannot be conducted in children, including bioequivalence, drug-drug interactions3, and renal and hepatic impairment studies.
As stated in 2010, model-informed drug development holds considerable promise for making pediatric dose selection a science. New approaches to MIDD are emerging, such as the use of Bayesian approaches, and would benefit from a robust discussion in a public workshop.
- 1 Abernethy DR, Burckart GJ. Pediatric dose selection. Clinical Pharmacology & Therapeutics 2010; 87:270-1.
- 2 Momper JD, Mulugeta Y, Burckart GJ. Failed Pediatric Drug Development Trials. Clinical Pharmacology & Therapeutics 2015; 98:245-51.
- 3 Salerno SN, Burckart GJ, Huang SM, Gonzalez D. Pediatric Drug-Drug Interaction Studies: Barriers and Opportunities. Clinical Pharmacology & Therapeutics 2019; 105:1067-1070.
- To review the current methods of pediatric dose selection;
- To discuss how to address the gaps in pediatric dose selection that occur because of the inability to perform studies in pediatric patients;
- To evaluate future approaches to pediatric dose selection in all pediatric patient populations.
For registration information, please visit the M-CERSI website. You may register for remote access. This workshop is intended for clinicians, drug developers, and regulators.
This virtual public workshop will be available to view via webcast, but pre-registration is still required. You will receive the link to access the live webinar several weeks prior to the meeting. You must log in with your username and password which you create when you register.
Additional information about this workshop, including an agenda, is available at https://www.pharmacy.umaryland.edu/centers/cersievents/pedsdoseselection/.
If you have any additional questions about this workshop, please contact:
Gilbert Burckart, PharmD.
U.S. Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993