Challenges and strategies to facilitate formulation development of pediatric drug products: Safety qualification of excipients

Abstract

A public workshop entitled “Challenges and strategies to facilitate formulation development of pediatric drug products” focused on current status and gaps as well as recommendations for risk-based strategies to support the development of pediatric age-appropriate drug products. Representatives from industry, academia, and regulatory agencies discussed the issues within plenary, panel, and case-study breakout sessions. By enabling practical and meaningful discussion between scientists representing the diversity of involved disciplines (formulators, nonclinical scientists, clinicians, and regulators) and geographies (eg, US, EU), the Excipients Safety workshop session was successful in providing specific and key recommendations for defining paths forward. Leveraging orthogonal sources of data (eg. food industry, agro science), collaborative data sharing, and increased awareness of the existing sources such as the Safety and Toxicity of Excipients for Paediatrics (STEP) database will be important to address the gap in excipients knowledge needed for risk assessment. The importance of defining risk-based approaches to safety assessments for excipients vital to pediatric formulations was emphasized, as was the need for meaningful stakeholder (eg, patient, caregiver) engagement.

Comparison of Safety and Efficacy of Methadone vs Morphine for Treatment of Neonatal Abstinence Syndrome

“…Such expensive and time-consuming processes highlight the need to develop safe, commercially available formulations designed specifically for newborns. 22 Although it is possible that methadone is better than morphine for treatment of NAS, a small retrospective study 7 found lower Bayley III mean composite cognitive and motor scores in methadone-treated infants compared with those receiving morphine. Our ongoing neurodevelopmental follow-up of Abbreviations: IQR, interquartile range; LOS, length of hospital stay; LOT, length of drug treatment; NAS, neonatal abstinence syndrome.…”

Pesquisa E Desenvolvimento De Novas Formas Farmacêuticas Pediátricas No Tratamento De Doençasnegligenciadas

“…A população pediátrica, abrange de recém-nascidos a adolescentes (0-17 anos), é bastante heterogênea em relação a uma multiplicidade de fatores, incluindo a diferença nas habilidades de deglutição. (BALAN et al, 2017;BUCCI-RECHTWEG et al, 2017;BUCKLEY et al, 2017;TRASTULLO et al, 2015).…”

Excipients in Neonatal Medicinal Products: Never Prescribed, Commonly Administered

“…Building this expertise can benefit from cross-talk with non-pharmaceutical disciplines, and from pooling the available expertise and knowledge in a dedicated repository. The concept of cross-talk has been suggested since excipients and decisions on safety to exposure are not unique to drug development, but also occur in the field of the food industry or agro-science [ 44 , 45 ]. Choices about excipient exposure can occur at several stages throughout the lifecycle of a drug, from product development through to clinical use.…”