What role does the Court of Justice of the European Union (CJEU) play in holding European Union (EU) regulatory science, ie the science underpinning public regulation, to account for its epistemic quality? Since Pfizer, CJEU case law has strengthened the role of science in both EU risk regulation and litigation, whilst intensifying judicial scrutiny of scientific reasoning based on procedural standards. Scholars often welcome this approach as striking an adequate balance between effective judicial protection and institutional competence. Some praise the Court as an ‘information catalyst’ promoting procedures, in which epistemic quality is ensured through diligent consideration of and deliberation among all relevant voices. These debates, however, overlook the role of economic actors as information providers, in and challengers of EU risk regulation. This paper re-evaluates the modern post-Pfizer approach from a new, socio-legal perspective by studying, for the first time, the interactions between judicial review and the epistemic power of economic actors, ie their relative ability to influence what EU regulators know at the expense of other actors. We combine an epistemologically informed comparative institutional analysis with doctrinal critique of CJEU case law. Our findings show the need to rethink both legal standing and procedural review in EU risk regulation. Instead of catalysing inclusive procedures that open regulatory science to public scrutiny, the modern approach fosters an exclusive bilateral information exchange between the administration and the regulated industry. The Court reduces the function of process values, such as duty of care and reason-giving, to the protection of a small circle of actors, neglecting the public interest dimension of such values. Thus, the modern approach fails to address, and instead further entrenches the epistemic power imbalances inherent in EU risk regulation. We end by sketching out a normative and doctrinally sound vision of how CJEU review could contribute to EU expert accountability in a more publicly oriented way.

Little is known about the number of minors enrolled in clinical research today. IRB administrators at leading pediatric medical centers were surveyed regarding studies with minors. Analyses were descriptive in nature with adaptive Bayesian bootstrap imputation used with missing data. Officials from 17/41 (41.5%) pediatric research centers responded: 74,204 active studies were estimated, 29,078 (39%) included minors, and 6574 (23%) were “more than minimal risk.” Minors accounted for 0.7–2.87M research subjects. Pediatric medicine desperately needs a more accurate and reliable reporting system for tracking the recruitment, retention, and involvement of minors in clinical research.

The last three decades have seen the biotherapeutic drug market evolve from promising concept to market dominance in a range of clinical indications. This growth has been spurred by the success of established drug classes like monoclonal antibodies, but also by the introduction of biosimilars, and more recently, multiple novel cell and gene therapies. Biotherapeutic drug development presents many unique challenges, but unintended immune responses are among the most common reasons for program attrition. Anti-drug antibodies can impact the safety and efficacy of drug products, and related immune responses, like the cytokine release syndrome that occurred in the infamous TGN-1412 clinical trial, can be challenging to predict with nonclinical models. For this reason, it is important that development programs proceed with a scientifically grounded and measured approach to these responses. This process begins at the discovery stage with the application of “quality by design,” continues into the clinic with the development of quality assays and management strategies, and culminates in the effective presentation of this information in regulatory documents. This review provides an overview of some of the key strategic and regulatory considerations for biotherapeutics as they pertain to immunogenicity and related responses.

As the pace of biomedical innovation rapidly evolves, there is a need to train researchers to understand regulatory science challenges associated with clinical translation. We describe a pilot course aimed at addressing this need delivered jointly through the Mayo Clinic Center for Clinical and Translational Science and the Yale-Mayo Center for Excellence in Regulatory Science and Innovation. Course design was informed by the Association for Clinical and Translational Science’s Regulatory Science Working Group’s competencies. The course used didactic, case-, and problem-based learning sessions to expose students to regulatory science concepts. Course evaluation focused on student satisfaction and learning. A total of 25 students enrolled in the first two course deliveries. Students represented several disciplines and career stages, from predoctoral to faculty. Students reported learning “an incredible amount” (7/19, 36.8%) or “a lot” (9/19, 47.4%); this was reflected in individual coursework and their course evaluations. Qualitative feedback indicated that assignments that challenged them to apply the content to their own research were appreciated. The heterogeneity of students enrolled, coupled with assessments and course evaluations, supports the statement that there is a growing need and desire for regulatory science-focused curricula. Future research will determine the long-term impact.

Building on the recent advances in next-generation sequencing, the integration of genomics, proteomics, metabolomics, and other approaches hold tremendous promise for precision medicine. The approval and adoption of these rapidly advancing technologies and methods presents several regulatory science considerations that need to be addressed. To better understand and address these regulatory science issues, a Clinical and Translational Science Award Working Group convened the Regulatory Science to Advance Precision Medicine Forum. The Forum identified an initial set of regulatory science gaps. The final set of key findings and recommendations provided here address issues related to the lack of standardization of complex tests, preclinical issues, establishing clinical validity and utility, pharmacogenomics considerations, and knowledge gaps.

Rapidly advancing technology often pulls the regulatory field along as it evolves to incorporate new concepts, better tools, and more finely honed equipment. When the area impacted by the technological advancement is regulated by the Food and Drug Administration (FDA), a gap develops between the technology and the guidelines that govern its application. Subsequently, there are challenges in determining appropriate regulatory pathways for evolving products at the initial research and developmental stages. Myriad factors necessitate several rounds of iterative review and the involvement of multiple divisions within the FDA. To better understand the regulatory science issues roiling around the area of additive manufacturing of medical products, a group of experts, led by a Clinical and Translational Science Award working group, convened the Regulatory Science to Advance Precision Medicine at the Fall Forum to discuss some of the current regulatory science roadblocks.