News in Depth

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 (ATP) is being strengthened through the expansion of internal and external funding, scientific and regulatory expertise, and collaborative support through the CBER Advanced Technologies Team (CATT). As part of its efforts to promote the adoption of advanced manufacturing, CATT has joined with CDER’s Emerging Technologies Team (ETT) to form the Center for the Advancement of Manufacturing Pharmaceuticals and Biopharmaceuticals (CAMPB). The Office of Regulatory Affairs and other centers are also expected to participate in the center’s activities.

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

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

The potential for faster innovation, and the power of collaboration and workplace and operations modernization are emerging into high relief as the assessment of the pandemic experience continues to deepen. Among recent venues where industry and regulators have come together to explore these themes was the February ISPE Facilities of the Future Conference. Focused on the dialogue that took place there, the IPQ story that follows is divided into three parts:

PART I: The Evolving Landscape of Pharmaceutical Operations

PART II: Government-Industry Collaboration in This and Future Pandemics

PART III: Reducing the Cost of Vaccine Manufacturing for Broader LMIC Access

The progress in understanding and addressing the challenges of impurities was a key focal point of this year’s USP Therapeutic Peptides and Oligonucleotides Workshop, held virtually on February 28, March 2 and March 4, 2022.

PART I: US and European Regulator Perspective on the CMC Challenges of Oligonucleotides

PART II: USP Standards Development Efforts for Peptides and Oligos

PART III: Peptide and Oligo Analytical, Manufacturing and Raw Material Considerations

The pandemic experience and the increasing expectations of regulators regarding supply chain visibility and risk-management are driving forces for incorporating more third-party auditing of excipient suppliers into user quality management systems.

In her new role as FDA’s Principal Deputy Commissioner, Janet Woodcock will be able to continue her strong contribution to the effort in the US and around the world to make drug and biologic regulatory processes as supportive as possible of manufacturing innovation.

FDA’s Knowledge-aided Assessment and Structured Application (KASA) and related Product Quality/CMC (PQ/CMC) initiatives have been laying some important conceptual and information technology (IT) groundwork for the ICH effort now underway to revise its M4Q guideline on the quality component of the Common Technical Document (CTD).

Part I: The Advancing Knowledge-aided Assessment Component of KASA

Part II: Bringing Biologics into the KASA System

Part III: The Progress of FDA’s PQ/CMC Initiative

Part IV: The Goals of Accumulus Synergy in CMC Data IT and Regulatory Communication

Part V: The Drivers for Revising ICH M4Q and Evolving the CMC Regulatory Process

Key focal points in the European GMP arena include revisions to guidance, new veterinary medicine regulations, and adapting assessment/inspection processes to enable manufacturing through the COVID pandemic, foster international collaboration, and address point-of-care manufacturing.

Part I: Update on EMA GMP-related Activities

Part II: MHRA Innovation Pathway and Proposal for Point-of-Care Regulatory Framework

Part III: Insights from Europe and ICMRA Regarding Onsite Inspection Alternatives