On November 1st, BIO submitted comments in response to the Food & Drug Administration’s (FDA) recent Pharmaceutical Science and Clinical Pharmacology Advisory Committee meeting. The meeting was primarily focused on two topics: the future of the Quality Management Maturity (QMM) program and the next stages of Knowledge-Aided Assessment and Structured Application (KASA). In particular, FDA requested feedback from stakeholders on the development of a CDER QMM program to incentivize investments in mature quality management practices. On this topic, BIO provided three primary recommendations: (1) A QMM program should be voluntary and industry-driven if implemented; (2) Any QMM assessment model should be standardized and well-integrated with related programs; and (3) Details about the proposed implementation of a QMM program should be clarified. During the Advisory Committee meeting, FDA also requested feedback on its vision and plan to expand KASA over the next five years to include drug substances, all generic dosage forms, new drug and biologics applications, and post-approval changes. Regarding KASA, BIO stressed the importance of utilizing the system to build upon international harmonization efforts and to support interoperability of data standards, in contrast to its potential for introducing further integration challenges and regulatory discrepancies. BIO also suggested that a pilot program conducted in partnership with industry would provide valuable insight on the potential benefits, challenges, gaps, and best practices associated with the implementation of KASA.

On October 28th, BIO submitted comments to the World Health Organization on its new draft guideline containing regulatory considerations and requirements for the marketing authorization of monoclonal antibodies (mAbs) and related products based on an immunoglobulin framework. In the comments submitted, BIO stated strong support for WHO’s mission to ensure the equitable and widespread availability of safe, effective, and quality health products. With this objective in mind, BIO also provided several overarching recommendations. First, BIO encouraged further incorporation of the 3Rs principles (replacement, reduction, and refinement of animal use) throughout the guideline. While nonclinical in vivo studies continue to provide critical information to sponsors and regulators on the safety and efficacy of products, BIO urged WHO to reconsider when such studies are truly necessary in the context of this guideline as in vitro technologies and computational models (e.g., physiological based pharmacokinetic models) can be leveraged to inform nonclinical assessments. BIO also suggested that WHO refine the guideline broadly to ensure consistency with existing international guidelines, such as the ICH S6 Guideline. Furthermore, BIO stated that the acknowledgement and incorporation of current regional guidances where possible and applicable, especially those that have been issued since the COVID-19 pandemic, would be beneficial.

On October 28th, BIO submitted comments on the ICH E11A Pediatric Extrapolation Guideline as published by the Food & Drug Administration (FDA). In the comments submitted, BIO voiced appreciation for the guideline’s clear explanation for how pediatric extrapolation can be applied practically to support the safety and efficacy of a product in pediatric populations. BIO also noted agreement with the guideline’s stance that extrapolation decision-making is complex, that knowledge evolves over time, and that gaps in information at the time of the initial pediatric development plan may be addressed during the execution phase of such a plan. In addition to requesting more clarity on the types of products and modalities the recommendations contained within the guideline could apply to, BIO suggested that a discussion section regarding endpoint considerations and data presentations be added. Lastly, BIO noted that a glossary with more precise definitions would be beneficial for sponsors interpreting its recommendations.