USP Continues to Refine Its Strategies for Keeping Pace with and Supporting the Rapidly Advancing Biotechnology in the MAb, Vaccine and CGT Arenas
USP is continuing to evolve and refine its strategies for providing standards that can keep pace with and support the rapid advances in biotechnology taking place across the monoclonal antibody (mAb), vaccine, and the cell and gene therapy (CGT) landscape.
Part I: USP Bio Stakeholder Forum Opening Remarks and Mass Spec Standards for Proteins
Part II: USP's CGT Initiatives
Part III: FDA and Industry Experience with CAR T Potency Testing
Part IV: Update on USP Strategies and Initiatives in the MAb, Vaccine, and CGT Arena
Government/Industry/Academia Collaborative Efforts to De-Risk and Accelerate Manufacturing Innovation Draw Strength from Pandemic Learnings
The US government is continuing to strengthen its collaborative efforts with industry and academia to de-risk and accelerate manufacturing innovation, as the learnings from the pandemic are further digested. The five-part IPQ story provides a wealth of insights from key leaders and policy makers on the innovation issues at play and how NIIMBL and other organizations are seeking to address them in the US and internationally.
Part I: NIIMBL and its “Going First Together” Mantra
Part II: US Manufacturing Innovation Leaders Weigh In
Part III: The Importance of Process and Facility Innovation in Global Health
Part IV: Charting the Advanced Therapy CMC Pathways and Other NIIMBL Projects
Part V: CBER’s Marks on Taking CGTs to the Next Level
ICH Q3D Implementation Continues with Workshops, Research, and Guideline and Pharmacopeial Revisions
More sensitive and selective methodologies have facilitated the identification and quantification of elemental impurities (EIs) in pharmaceuticals and the movement away from the old heavy metal analytical tests to the newer risk-based assessment strategy of ICH Q3D.
Part I: Role of PQRI/FDA Workshop in Q3D Implementation Dialogue
Part II: Regulatory Experience and Perspectives in Implementing Q3D
Part III: Pharmacopeial Harmonization with ICH Q3D
Part IV: Outcomes of PQRI Study on Variability in Elemental Impurity Analysis
Strengthened European Regulator Support for Advanced Technologies Includes New EMA Quality Innovation Group
The experience of the pandemic has underscored the criticality of assuring that regulatory processes keep pace with innovation in the manufacturing and control arena and has spurred the efforts that were underway in Europe to assess the issues involved and implement the adaptations needed to address them. The three parts of the IPQ story review the progress of these efforts.
Part I: Europe’s Focus on CMC Innovation and Agile Regulation
Part II: Innovation Issues Explored at CASSS CMC Forum in Europe
Part III: MHRA’s “New Era in Regulation”
EMA Toolbox on CMC Flexibilities has been Evolving to Incorporate Industry Input and Learnings from the Pandemic
EMA’s “toolbox guidance on scientific elements and regulatory tools to support quality data packages” for priority medicines targeting unmet medical needs, released in April 2022, reflects extensive stakeholder input and will evolve further as experience and learnings from the pandemic are evaluated.
Developed as an outcome of the 2018 joint EMA/FDA workshop on quality support to Priority Medicines (PRIME) and Breakthrough Therapy (BT) designated products, the guidance outlines the tools available in the EU to address the challenges in generating robust quality packages under accelerated conditions. It is applicable to small molecules, biological/biotech products, and advanced therapy medicinal products (ATMPs).
The guidance was drafted, consulted on, and revised throughout the COVID pandemic.
Part I: EMA Perspective on its Toolbox Guidance and OPEN Initiative
Part II: Industry View on Efficiency Tools and CMC Flexibility Learnings from COVID
Part III: Panel Discussion on Effective Tools for the Future
CBER’s Advanced Technologies Program Growing Stronger with Increased Funding, Expertise, and Collaboration
mRNA-LNP Vaccines Spur Global Dialogue on Nanomaterial Standards and Regulatory Approaches
The successful development of mRNA-lipid nanoparticle (LNP) vaccines to address the COVID-19 pandemic has highlighted the power of nanoparticle-based formulations in producing safe and efficacious medicinal products.
Part I: Pfizer/BioNTech Lipid Challenges with mRNA-LNP COVID Vaccine
Part II: FDA’s Novel Excipient Review Pilot Program and Nanomaterials Guidance
Part III: USP’s Draft Guideline and Other Efforts on mRNA Vaccine Quality
Part IV: The EDQM Nanomedicines Dialogue and WHO on Regulating mRNA Vaccine Quality
Part V: Potency Assays for mRNA-LNP Vaccines
NASEM-Led Study for FDA is Helping Drive Industry/Regulator Agenda on Innovation Needs
An FDA-commissioned study carried out through the National Academies of Sciences, Engineering, and Medicine (NASEM) is helping drive the industry/regulator agenda on advancing innovation in pharmaceutical manufacturing.
PART I: The NASEM Study and FDA Reflections
PART II: Existing Mechanisms to Enable Innovation
PART III: Challenges and Opportunities
PART IV: The Path Forward