Theme 1: Advances and Innovation in Formulation Development

This theme is dedicated to the recent advancements in formulation development and related regulatory considerations. For biomolecules, this theme covers new approaches in low and high concentration formulations, development and use of novel excipients, high-throughput screening, liquid formulation development, lyophilization, and vaccine formulation development. For chemical entities, topics include development and use of novel excipients, prediction and simulation in formulation development and processing, formulation of molecules with poor solubility and bioavailability, biorelevant dissolution methods, formulation technologies for different routes of administration including particle engineering for pulmonary delivery, and the use of 3D printing in early drug product development.

Theme 2: Formulation Development Strategies for Novel Modalities

Several novel therapeutic modalities are in different stages of development and commercialization to treat a wide range of therapeutic indications. This theme covers the latest advances in formulation technologies and strategies to develop drug products of novel and emerging therapeutic modalities. Scientific approaches and cutting-edge research to fundamentally understand structure function attributes and their connection to formulation development is considered in this theme. Novel modalities require different thinking than traditional drug product development, which is also recognized by regulatory agencies. This theme covers regulatory considerations and related guidance for novel modality development. For biomolecules, topics include formulation aspects of cell therapy, gene therapy, bispecific and trispecific antibodies, BiTE® antibody constructs, diabodies, peptibodies, and oncolytic immunotherapy viruses. For chemical entities, topics include formulation aspects of complex generic drug products, non-biologic complex drugs (NBCDs), synthetic peptides, and oligonucleotides.

Theme 3: Drug Delivery and Device Development

Drug delivery approaches and device development are essential parts of drug product development. Understanding how the end user interacts with the final product is also critical. This theme covers advances in drug delivery approaches, medical device development, connectivity between patient experience and device development, and related regulatory topics. This includes specialty delivery systems such as long-acting delivery, ocular and inhalation products, and implants. This theme also covers aspects of drug product handling and administration, advances in primary packaging development including autoinjectors, pre-filled syringes, dual chamber systems, on-body injectors, connected devices, and low temperature storage for emerging modalities.

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View the entire PharmSci 360 program and speakers

Plenary Speakers

Monday, October 26, 2020

Jennifer Doudna, Ph.D.
Professor of Chemistry and Molecular Biology
U.C. Berkeley

As an internationally renowned professor of Chemistry and Molecular and Cell Biology at U.C. Berkeley, Doudna and her colleagues rocked the research world in 2012 by describing a simple way of editing the DNA of any organism using an RNA-guided protein found in bacteria. This technology, called CRISPR-Cas9, has opened the floodgates of possibility for human and non-human applications of gene editing, including assisting researchers in the fight against HIV, sickle cell disease and muscular dystrophy. Doudna is an investigator with the Howard Hughes Medical Institute and a member of the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors and the American Academy of Arts and Sciences. She is also a Foreign Member of the Royal Society, and has received many other honors including the Breakthrough Prize in Life Sciences, the Heineken Prize, the BBVA Foundation Frontiers of Knowledge Award and the Japan Prize. She is the co-author with Sam Sternberg of A Crack in Creation, a personal account of her research and the societal and ethical implications of gene editing.

Thursday, November 5, 2020

Gregory A. Poland, M.D., MACP, FIDSA, FRCP
Director, Mayo Vaccine Research Group
Mayo Clinic and Foundation

Gregory A. Poland, M.D., is the director of Mayo Clinic’s Vaccine Research Group—a state-of-the-art research group and laboratory that seeks to understand genetic drivers of viral vaccine response and application of systems biology approaches to the generation of immunity, as well as the development of novel vaccines important to public health. The Poland lab has developed the field of viral vaccine immunogenetics, the immune response network theory, and the field of vaccinomics and adversomics.

2020 PharmSci 360 Workshops

AAPS is hosting six workshops and short courses taking place the week of November 9. Each event will consist of four 90-min sessions over a two-day period. Please visit our event website for a detailed agenda and speaker details.

Monday, November 9 – Tuesday, November 10, 2020
10:30 am—12:00 pm & 2:00 pm—3:30 pm ET daily

Workshop: Drug Transporters 2020: Old Challenges and New Opportunities

The goals of this workshop are 1) to bring together experts in the transporter field from academia, industry, and regulatory agencies, 2) to enhance interaction and communication between these three entities, and 3) to inform the audience about existing challenges and new opportunities in the transporter field. Session topics will include: transporter animal models, predicting human PK/IVIVE, transporters as drug targets, and an update on DDI guidances. The workshop will consist of interactive lecture-based sessions, one dialogue and debate session, one podium discussion, and one session focused on regulatory perspectives by FDA representatives.

Workshop: Introduction to Vaccine Clinical Development

The Introduction to Vaccine Clinical Development workshop/short course will provide a general introduction to key concepts in vaccine process development and vaccine clinical development. Case study examples will be used to illustrate key concepts across vaccine process development, clinical development, and regulatory strategy for vaccine licensure. The lectures will set the foundation for a COVID-19 roundtable discussion. Participants will have multiple opportunities to engage the expert panel across hot topic areas to deepen understanding of what is needed to develop and license a novel vaccine and how we may adapt the traditional development process during a pandemic to meet the global need.

Wednesday, November 11 – Thursday, November 12, 2020
10:30 am—12:00 pm & 2:00 pm—3:30 pm ET daily

Workshop: Be Specific – Biomarker Assay Validation in Context

Although the concept of Context of Use (COU) has gained significant traction in the past 2 years, bringing this concept into day-to-day practice for implementation of biomarkers throughout all stages of drug development has been challenging, with ongoing confusion regarding what exactly constitutes a COU. This presentation will provide foundational concepts including clarification on what differentiates a biomarker category from a biomarker endpoint from the biomarker’s COU. Furthermore, since the majority of biomarkers are deployed for exploratory purposes, or to support internal decision making during early clinical development, how to craft specific COU statements to address these scenarios will be discussed. Lastly, the content will highlight how to manage situations where COU is not forthcoming, but an assay is being requested (or demanded). By providing an analytical characterization of the assay along with clear communication of assay performance characteristics and limitations, the analytical biomarker scientist can deliver an assay against which suitability for different COUs can be evaluated. This latter step will then determine whether the assay is valid for a given context or not. Examples of COUs with attendant validation considerations/requirements and/or assay characterizations and the COUs they may support (or not) will be discussed.

Workshop: Gene Therapy: Inserting Old Genes into a New Modality!

This workshop intends to provide a more comprehensive look at the discovery and development of gene therapy. Some of the aspects that will be presented include basic transgene and gene editing target selection, delivery mechanisms, quality testing, immunogenicity, clinical pharmacology, and biomarker responses. The intended target for this workshop is for new scientists and cross-discipline scientists, such as, molecular biologists, drug delivery, CMC manufacturing, regulatory sciences, and clinical teams. Specific topic consideration will be given to dose projections, gene delivery, and manufacturing challenges.

Workshop: Latest Developments In Excipients That Are Relevant To Formulation, Analytical and Manufacturing

With the increasing growth of new and generic drugs entering the market, more and more excipients are being used in specialized drug delivery systems such as biologics, injectables, and modified released dosage forms. As a result, there is a growing need to control critical excipient material properties that contribute to excipient variability in dosage forms. As not all the critical physical and chemical properties may be identified in excipient monographs via compendial tests and specifications, a USP information chapter Excipient Performance was developed to provide an overview of the key functional categories of excipients and tests or procedures that can be used by both the user and maker of the excipient to identify, monitor, and control critical material attributes or properties of the excipient that may impact the dosage form. Further, excipient complexity continues to increase in terms of composition and impurities that create challenges in the selection of excipients that are fit for purpose. Additionally, FDA GMP requirements are limited to finished pharmaceuticals (see 21 CFR 210 & 211). USP is developing a chapter to provide guidance on how best to ensure and maintain product quality through the supply chain over a product’s lifecycle through a toolbox approach used to identify and mitigate risks. This presentation aims to highlight both challenges and opportunities for USP in collaboration with key stakeholders to provide up-to-date USP-NF monograph standards and chapters helpful in qualifying an excipient for its intended pharmaceutical use, providing a better understanding of excipient composition and impurities that may help in the selection of excipients for pharmaceutical, use and creating a toolbox approach to supplier qualification to help mitigate associated risks.

Wednesday, November 11 from 10:30 am—12:00 pm & 2:00 pm—3:30 ET pm

Thursday, November 12, 2020 from 10:30 am—12:00 ET pm

Workshop: Strategic Approaches to Develop Phase-Appropriate Organic Impurity Control Strategies

This program will describe best practices for development of early phase control strategies for organic impurities and extractable/leachables in small molecule drug products. In the current landscape of drug development, the design and implementation of impurity control strategies is a complicated, yet critical process necessary to ensure overall pharmaceutical product quality and patient safety. While there are well-established guidelines for commercial control of organic impurities in drug substances and drug products, there is limited guidance on how to apply these principles throughout the various stages of development, especially in the early phases. Mutagenic impurities are an area of specific interest for which new guidances exist but application of these guidances in an early phase environment represents unique risk-based challenges. One example, nitrosamine impurities, is a current area of fervent interest, with many companies struggling to understand how to set up risk-based approaches to assess, detect, control, and monitor these impurities, especially considering the strict regulatory guidances. This program will also include a discussion of robust and phase-appropriate extractables/leachables strategies to ensure that packaging supports the quality and efficacy of the drug products throughout their shelf life. This program will serve to provide pharmaceutical scientists with case studies and real-world examples of how appropriate control strategies can be successfully applied throughout the drug development for all of these impurity topics. It will also provide a forum to discuss new and innovative technologies that can be applied to the detection, isolation, identification, control, and monitoring of impurities throughout a product lifecycle.