As defined by the US Food and Drug Administration (FDA), regulatory science is the “science of developing new tools, standards and approaches to assess the safety, efficacy, quality and performance of all FDA-regulated products” (1). Nowhere is the need greatest for such standardized methods and new approaches than for the regulation of medicines for neonates (2). Recognizing that researchers often generate results that may influence regulatory decision making, yet are not steeped in the rigorous scientific requirements for regulatory review, the Innovative Medicines Initiative recently released a guidance tool for researchers about how to access support from regulators (3,4).

McCune and Mulugeta called for collaboration in the neonatal community to add clarity, rigor, and predictability to the regulatory path during the development of medicines for the market and, more generally, to efforts that optimize the use of existing and new medicines on the market (5). This can be done with: (i) standardized methods, (ii) standards-of-care agreed upon within the neonatal community, (iii) streamlined approaches to working between multiple sites and/or multiple medicines for a given disease/condition, (iv) innovative trial designs, (v) decision criteria for conducting clinical trials of new therapies (e.g., when should studies in neonates be initiated and what information is needed to design neonatal studies?), (vi) drug development tools qualified by the regulatory agencies for a specific context of use, (vii) clinical outcome assessments, and (viii) modeling approaches such as physiologically based pharmacokinetic and disease progression models, as well as clinical trial simulation tools (3,4,5,6). Also important is a shared understanding of formulations for neonates that reflects the practicalities of administration. To date there has been no forum for the neonatal community to develop evidence-based agreements about these approaches to improving the development of drugs for neonates. Because all clinical studies must be informed by the needs and views of people affected by the research, the neonatal collaboration must include a voice for families who have experienced a sick or premature baby. Outcomes need to be meaningful to families and clinicians or be surrogates that predict these meaningful outcomes. Activities could reflect nonclinical disease models and juvenile animal studies when appropriate.

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