The American Association of Pharmaceutical Scientists Scientific Programming Committee presents this overview of AAPS’ new meeting programming highlights.
Link on the titles below to jump to your area of interest.
The Five Tracks
End-to-End Hot Topic Sessions
CHEMICAL
PICKING THE WINNING CANDIDATE
HEPATOPAC AND OTHER IN VITRO METHODS TO CHARACTERIZE LOW TURNOVER COMPOUNDS
This presentation will highlight the applicability of improved in vitro hepatocyte models to better characterize the metabolic fate of drug candidates with low turnover. In addition, case examples for improved mechanistic ADME research will also be discussed, emphasizing the potency of combining improved metabolic competencies and longevity of these improved in vitro models to study hepatic drug disposition.
Monday, Nov. 5 9:00 am – 9:30 am
HIGH BINDING TENDENCY AND OTHER DETERRENTS TO CL PREDICTIONS
Prediction of hepatic clearance is critical and has been challenging for drugs that have high plasmatic protein binding (>95% bound). Studies have shown that clearance predictions of highly bound drugs to albumin is not as straightforward as correcting for unbound fraction in plasma as initially thought. This session will describe current knowledge regarding the impact of protein binding on hepatic clearance and the state-of-the-art science on in vitro-in vivo extrapolation prediction of clearance of highly bound drugs.
Monday, Nov. 5 9:30 am – 10:00 am
APPLYING IN SILICO ADME MODELS TOWARD COMPOUND SELECTION: CASE STUDIES
Drug discovery requires multi-parameter optimization to achieve ideal drug efficacy, dose, and safety profiles. In silico ADME modeling is an effective tool in this cycle, but new technologies at the interface of artificial intelligence, PBPK modeling, and PKPD modeling promise to further transform the field. This presentation will describe real-life in silico ADME modeling applications; cover the friends and foes at the interface of science, culture, and technology; and provide a realistic vision of the future of integrated drug discovery.
Monday, Nov. 5 10:00 am – 10:30 am
DEEP LEARNING: A PATH FOR SUCCESSFUL CANDIDATE SELECTION
Decades of pharmaceutical experience, billions of dollars in analytic innovations, and thousands of publications all add up to one thing: abject drug development futility. Information processing challenges have eluded not only human cognition but even several generations of computer algorithms. Finally, that is changing with the flexible blend of deep learning and causality network modeling. This session will present illustrative examples and discuss the novel challenges imposed by emerging target paradigms.
Monday, Nov. 5 10:30 am – 11:00 am
EMERGING RESEARCH TOOLS
IN SILICO TOOLS FOR OPTIMIZING ORAL FORMULATIONS
This presentation will discuss the use of in silico tools to predict the in vivo performance of drug compound formulations, allowing formulations to be ranked by oral bioavailability. Posaconazole will be used as a case study, where different formulations in vivo, in vitro, and in silico were tested.
Tuesday, Nov. 6 9:00 am – 9:30 am
NOVEL PRODUCT PERFORMANCE TOOLS TO OPTIMIZE FORMULATION DEVELOPMENT AND MAXIMIZE THERAPEUTIC OUTCOMES
Combining key product functional characteristics and critical input parameters with appropriate biopharmaceutics studies can elevate product quality with downstream patient benefit. Next generation, integrated, formulation characterization tools not only have the ability to identify attributes of formulation function when establishing BE, they are also capable of providing insights into product mechanism of action or its in vivo effect that can augment overall BE assurance. This session will include details on such tools and their use in drug product development.
Tuesday, Nov. 6 9:30 am – 10:00 am
USE OF IN VIVO AND IN VITRO MODELS TO PREDICT DRUG PENETRATION INTO THE HUMAN BRAIN
This session will cover the makeup of the blood-brain barrier (BBB), how to study and mimic BBB functions, and using in vivo and in vitro models. Current technologies, ranging from static and dynamic platforms to microfluidic systems, and future technologies, including 3D bioprinting and integrated in vivo/in vitro strategies, will also be discussed.
Tuesday, Nov. 6 10:00 am – 10:30 am
USE OF CHIP TECHNOLOGIES: PERSPECTIVES FROM REGULATORY AGENCIES
FDA is very interested in identifying and testing alternative approaches to improve assessment of FDA-regulated product toxicity and efficacy. In December 2017, the presenters published the Predictive Toxicology Roadmap. In September 2018, they held a public meeting to solicit feedback on this effort. This session will explore some of the outreach and research conducted within FDA in the area of alternative methods focusing on microphysiological-type approaches.
Tuesday, Nov. 6 10:30 am – 11:00 am
PRECLINICAL TO CLINICAL TRANSLATION
MECHANISMS UNDERLYING SPECIES DIFFERENCES IN HEPATOTOXICITY
Some nonhuman primate studies have failed to predict exposure-dependent liver toxicity observed in early clinical trials. The mechanisms underlying species differences in hepatoxicity have been a focus of a public-private partnership, the DILI-sim Initiative, which has involved scientists from industry, academia, and FDA. The quantitative systems toxicology approaches employed by the Initiative have helped identify relevant species differences in mechanisms that underlie hepatoxicity, particularly those involving bile acid homeostasis.
Wednesday, Nov. 7 9:00 am – 9:30 am
USE OF ANIMAL MODELS IN TRANSLATIONAL RESEARCH: STRENGTHS, WEAKNESSES, AND POINTS TO CONSIDER
Wednesday, Nov. 7 9:30 am – 10:00 am
MODEL -BASED TRANSLATIONAL RESEARCH: USING SHARED DISEASES IN HUMANS AND ANIMALS TO SUPPORT DRUG PRODUCT DEVELOPMENT
Reverse translational pharmacology is based on the hypothesis that animal models that spontaneously develop analogous diseases to humans will improve the predictability of preclinical models used for biomedical research. In return, PK, efficacy, and safety data gathered from human clinical studies can stimulate veterinary drug development. The success of this approach relies on more communication between scientists, with regulatory incentives to develop parallel (veterinary and human) drug development programs.
Wednesday, Nov. 7 10:00 am – 10:30 am
WHY ANIMAL STUDIES SHOULD BE AVOIDED: A BUSINESS COST/RISK DECISION-BASED PERSPECTIVE
This session will discuss the social and financial costs of developing new drugs relative to the realized benefits and how the industry currently falls short of its potential due to the use of animal models in drug development. Updating legislation to be consistent with modern scientific knowledge will benefit pharmaceutical industry stakeholders, including patients whose health depends on drugs and those who financially rely on drug development.
Wednesday, Nov. 7 10:30 am – 11:00 am
BIOMOLECULAR
PICKING THE WINNING CANDIDATE
LEAD OPTIMIZATION AND SELECTION OF BIOLOGICS: CASE STUDY
This presentation will discuss PK and PK/PD modeling-based strategies that are commonly used in lead optimization and selection of biologics. Using a few case studies, this presentation will demonstrate ways of applying PK/PD-based strategies to efficiently design and plan PK/PD efficacy studies, identify target potency requirements for lead candidates, and establish impact of target expression on PK and human dose.
Monday, Nov. 5 9:00 am – 9:30 am
EMERGING PRECLINICAL MODELS TO IMPROVE ONCOLOGY DRUG DEVELOPMENT
Monday, Nov. 5 9:30 am – 10:00 am
SARS AND IN VITRO STUDIES FOR ADC CANDIDATE SELECTION
Monday, Nov. 5 10:00 am – 10:30 am
MEGALIN: ROLE IN KIDNEY ENDOCYTOSIS OF PROTEINS AND PEPTIDES
An important kidney function is to reabsorb proteins in the proximal tubule by endocytosis. As a result, minimal amounts of protein pass into the urine in healthy kidneys. The tubular protein reabsorption is achieved by the endocytic receptor megalin and the co-localized receptor cubilin. This presentation will highlight emerging findings on the importance of megalin in protein disposition in the kidney and changes with disease.
Monday, Nov. 5 10:30 am – 11:00 am
EMERGING RESEARCH TOOLS
CRISPR/CAS9 GENE EDITING: THE HOW, WHY, AND WHEN
Tuesday, Nov. 6 9:00 am – 9:30 am
CRISPR: IN VITRO AND IN VIVO PRECLINICAL APPLICATIONS
This session will provide an overview of CRISPR/Cas9 technology and its current and future applications in research and preclinical studies, both in vitro and in vivo. In contrast to similar methods, CRISPR/Cas9 is widely accepted as the premier technology for gene engineering. With improvement of gene delivery methodologies and transfection efficiency, CRISPR has overcome many hurdles in its development from early stage cellular engineering to in vivo approaches.
Tuesday, Nov. 6 9:30 am – 10:00 am
ADVANCEMENTS IN THE APPLICATION OF SCAFFOLDS FOR TISSUE REGENERATION
Approaches to regenerating musculoskeletal tissues include the use of programmed stem cells, growth factors, gene therapies, and scaffold matrix materials. These approaches are being developed as standalone therapeutic strategies and in combination. This presentation will show research that suggests nonviral gene-activated scaffolds are effective for bone regeneration and are an attractive therapeutic strategy with significant potential for clinical translation.
Tuesday, Nov. 6 10:00 am – 10:30 am
BEST OF BOTH WORLDS: NANOTHERAPEUTICS FOR CANCER IMMUNOTHERAPY
Passive and active targeting of nanocarriers containing drug molecules to tumor tissues has been explored in preclinical models in which preferential accumulation of nanoparticles has been observed. Since cancer treatment trends are moving from traditional chemotherapy to cancer immunotherapy, this session will look at the nanoparticle opportunity in a different light for cancer immunotherapy and the possibility of leveraging what we already know about nanoparticles.
Tuesday, Nov. 6 10:30 am – 11:00 am
PRECLINICAL TO CLINICAL TRANSLATION
ROAD TO IND: ARX788, A NEXT GENERATION ANTI-HER2 ADC WITH SITE-SPECIFIC CONJUGATION
ARX788 is a HER2-targeting ADC currently being investigated under phase 1 trials in subjects with advanced cancers with HER2 expression. ARX788 uses site-specific technology for chemical conjugation of cytotoxic payload to the unnatural amino acid expressed on the antibody. This presentation will cover a series of preclinical evaluations, including DMPK and toxicology studies that lead to IND application and progression of ARX788 into the clinic.
Wednesday, Nov. 7 9:00 am – 9:30 am
PRECLINICAL TO CLINICAL TRANSLATION OF ADCS USING PK/PD MODELING: A RETROSPECTIVE ANALYSIS OF INOTUZUMAB OZOGAMICIN
Inotuzumab ozogamicin (InO) is an anti-CD22 ADC approved for the treatment of B-cell acute lymphoblastic leukemia in patients who have relapsed or are refractory to standard chemotherapy. This presentation will showcase a mechanistic modeling and simulation approach that integrates InO preclinical PK and PD data along with key in vitro cellular measures to predict InO clinical outcome.
Wednesday, Nov. 7 9:30 am – 10:00 am
TRANSLATION PK/PD APPROACHES FOR DISCOVERY AND DEVELOPMENT OF IMMUNO-CONJUGATES
This presentation will discuss some key modeling strategies for successful preclinical to clinical translation of immunoconjugates (e.g., ADCs and radio-immunoconjugates).
Wednesday, Nov. 7 10:00 am – 10:30 am
THE APPLICATION OF MULTIPLE-ANALYTE INTEGRATED PK/PD-BASED APPROACH TO SUPPORT REGULATORY SUBMISSION OF VC-MMAE-BASED ADCS
In this talk, a multiple-analyte integrated PK/PD approach will be discussed to address key questions to support the regulatory submission of a vc-MMAE-based ADC. An integrated population PK model to characterize the PK of conjugated and unconjugated MMAE was simultaneously developed and applied to address these questions.
Wednesday, Nov. 7 10:30 am – 11:00 am
CHEMICAL
NOVEL THERAPEUTIC MODALITIES
LC-MS/HYBRIDIZATION ELISA APPROACHES FOR BIOANALYSIS OF STEREOPURE OLIGONUCLEOTIDES
Monday, Nov. 5 9:00 am – 9:30 am
BIOANALYTICAL CHALLENGES AND OPPORTUNITIES IN DEVELOPMENT OF RNAI THERAPEUTICS
Monday, Nov. 5 9:30 am – 10:00 am
BIOANALYTICAL STRATEGIES AND CONSIDERATIONS FOR THE BIOANALYSIS OF LIPOSOMAL DRUGS Over the last 10 to 15 years, liposomes have increased in popularity as delivery platforms. From a bioanalytical perspective, developing methods to support studies where liposomes are used as dosing vehicles is challenging. This session will describe general method development considerations for using liposomal dosing vehicles. It will include case studies demonstrating experimental design and mitigating steps that may be taken to overcome various technical challenges.
Monday, Nov. 5 10:00 am – 10:30 am
MEETING BIOANALYTICAL CHALLENGES OF PROTEIN-DRUG CONJUGATES WITH IMPROVED EFFICIENCY USING MULTIPLEXED HYBRID LBA-LC-MS/MS ASSAYS
Because ADCs are generally heterogenous and dynamically undergo changes in vivo, bioanalysis is challenging, and multiple assays are needed to support PK studies. This presentation will focus on bioanalytical strategies to improve efficiency for ADC bioanalysis using hybrid LC-MS/MS technology. Case studies will be presented, concluding with a review of how multiplexed LC-MS/MS assays can address the bioanalytical challenges posed by newer multi-component constructs.
Monday, Nov. 5 10:30 am – 11:00 am
NEW TECHNOLOGY AND NEW APPLICATIONS OF EXISTING TECHNOLOGY
WHAT COULD BE AN ACHIEVABLE LC-MS/MS SENSITIVITY LEVEL: THEORETICAL CONSIDERATIONS
In bioanalysis, the lower limit of quantitation (LLOQ) is considered a principal measurement of assay sensitivity. The desire to achieve a lower level of LLOQ by LC-MS/MS is driven by emerging real-world challenges in drug discovery. This symposium will present theoretical and practical applications of how LC-MS/MS sensitivity is generally determined by multiple interactive parameters and its implications in selecting and optimizing LC-MS systems/methodologies for improving detectability.
Tuesday, Nov. 6 9:00 am – 9:30 am
TOWARD HIGHLY SENSITIVE AND ACCURATE ANALYSIS OF NOVEL BIOTHERAPEUTICS IN TISSUES
Tissue analysis remains challenging because of low tissue levels, high interference, and lack of appropriate sample treatment methods achieving sufficient quantitative accuracy and robustness. This lecture will describe the technical difficulties associated with tissue analysis and analytical techniques developed to improve sensitivity, selectivity, accuracy, throughput, and robustness of LC-MS-based tissue quantification. A number of applications will be given as examples.
Tuesday, Nov. 6 9:30 am – 10:00 am
INTACT LC-MS ANALYSIS OF PEPTIDES AND BIOTHERAPEUTICS FOR PHARMACOKINETICS STUDY
This presentation will discuss some critical questions for bioanalysis and catabolism of protein therapeutics. Workflow of LC-MS analysis of proteins using both bottom-up and top-down will be presented. Protein bioanalysis and biotransformation using LC-high resolution MS for protein half-life extension platforms will be highlighted using real-world case studies.
Tuesday, Nov. 6 10:00 am – 10:30 am
EVALUATION OF VOLUMETRIC ABSORPTIVE MICROSAMPLING AND ITS BIOANALYTICAL CHALLENGES
Blood microsampling continues to gain interest in drug discovery and development. Impact-assisted extraction will be introduced in this presentation as a universal and novel sample preparation workflow for overcoming the recovery bias of blood hematocrit in volumetric absorptive microsampling (VAMS) assays. Efficacy will be illustrated in case studies for both small and large molecule applications. Further, challenges associated with implementation of VAMS in a regulated environment will be discussed.
Tuesday, Nov. 6 10:30 am – 11:00 am
EMERGING REGULATIONS IMPACTING BIOANALYSIS
LC-MS/MS BIOMARKER ASSAY VALIDATION CONSIDERATIONS FROM THE NEW GUIDANCE: DISCUSSION OF THE UPCOMING AAPS WHITE PAPER RECOMMENDATIONS
LC-MS technology and its application to support biomarker analysis in clinical trials is still in an early stage of consideration. This presentation will deliver recommendations from an AAPS group regarding LC-MS-based biomarker validation for both small and large molecules. The recommendations made in the group’s white paper reflect extensive discussions and pertain specifically to practices undertaken during pharmaceutical research and development.
Wednesday, Nov. 7 9:00 am – 9:30 am
SCIENTIFIC AND REGULATORY CONSIDERATIONS FOR THE ANALYTICAL VALIDATION OF ASSAYS USED IN THE QUALIFICATION OF BIOMARKERS IN BIOLOGICAL MATRICES
This presentation will define the latest scientific and regulatory considerations for the analytical validation of assays for fluid-based (any protein, peptide, nucleic acid, lipid, or other chemical entity soluble in plasma, urine, saliva, etc.) biomarkers used in the qualification of drug development tools. The latest industry practices for exploratory biomarkers will be discussed.
Wednesday, Nov. 7 9:30 am – 10:00 am
ICH M10 UPDATE AND THE NEW FDA GUIDANCE
Wednesday, Nov. 7 10:00 am – 10:30 am
PANEL DISCUSSION: EMERGING REGULATIONS IMPACTING BIOANALYSIS
Wednesday, Nov. 7 10:30 am – 11:00 am
BIOMOLECULAR
NOVEL THERAPEUTIC MODALITIES
NOVEL PROTEIN MODALITIES: BIOANALYTICAL CHALLENGES AND SOLUTIONS
Over the past few decades, there has been a surge in the number of protein therapeutics and increasing complexity and diversity of hybrid proteins, probodies, bispecific antibodies, ADCs, etc. This session will discuss novel protein modalities and the complexities of their bioanalysis, demonstrating how a combination of ligand binding, hybrid, and LC-MS/MS assays are used to adequately inform preclinical and clinical development.
Monday, Nov. 5 9:00 am – 9:30 am
BIOANALYTICAL STRATEGIES FOR BISPECIFIC AND MULTI-SPECIFIC BIOLOGICS
Multi-specific biologics, such as bispecific antibodies, are an emerging therapeutic modality. They are one of the fastest growing classes of investigational drugs. The complex molecular designs of this modality present special challenges in bioanalysis, including PK and ADA assay development and validation. This session will discuss these challenges and possible solutions with well-designed bioanalytical strategies in preclinical and clinical development
Monday, Nov. 5 9:30 am – 10:00 am
PROTEIN MISFOLDING AND ITS ROLE
IN PRION DISEASE EXPANSION
The epidemiology of prion diseases continues to expand as additional pathological conditions linked to protein infectious agents are identified. This session will focus on protein misfolding and cover a novel method known as protein misfolding cyclic amplification assay for the detection of prions in blood. This method can be applied to several diseases including Alzheimer disease. Finally, the session will review approaches to treatment.
Monday, Nov. 5 10:00 am – 10:30 am
VACCINE CLINICAL ASSAYS: WHAT CAN WE ADOPT TO SUPPORT EMERGING MODALITIES?
Vaccine clinical assays are used to measure immunogenicity, which is an important clinical efficacy endpoint in vaccine clinical trials. Many practices can be adopted to support immunogenicity studies of new modalities, such as gene therapy, oncolytic viruses, etc. This session will cover vaccine clinical assays and their validations, including FDA expectations. Examples of cell-mediated immunity assays and antibody functional assays will be shared.
Monday, Nov. 5 10:30 am – 11:00 am
NEW TECHNOLOGY AND NEW APPLICATIONS OF EXISTING TECHNOLOGY
OVERVIEW OF THE CONSORTIUM AND MAJOR FINDINGS
The ABIRISK (Anti-Biopharmaceutical Immunization: prediction and analysis of clinical relevance to minimize the RISK) Innovative Medicines Initiative activities spanned February 2012 to April 2018. In this symposium, results from ABIRISK will be presented, including: understanding mechanisms of immunogenicity including the identification of early signature of ADA response, characteristics of ADA and production of ADA assay standards, clinical observations, and genetic associations.
Tuesday, Nov. 6 9:00 am – 9:30 am
UNDERSTANDING MECHANISM OF IMMUNOGENICITY INCLUDING THE IDENTIFICATION OF EARLY SIGNATURE OF ADA RESPONSE
Mechanisms driving antidrug immunogenicity remain uncertain, and reliable biomarkers to predict immunogenicity development are lacking. The research presented in this session has identified novel mechanisms influencing the development of immunogenicity to biopharmaceuticals and a potential biomarker to predict antidrug responses. The findings have clear implications for treatment of patients with multiple sclerosis and potentially other patient groups treated with biopharmaceuticals.
Tuesday, Nov. 6 9:30 am to 10:00 am
CHARACTERISTICS OF ADA AND PRODUCTION OF ADA ASSAY STANDARDS
Tuesday, Nov. 6 10:00 am – 10:30 am
CLINICAL OBSERVATIONS AND GENETIC ASSOCIATIONS
Biopharmaceutical products are a fast-growing class of therapeutics that represent a critical step forward in the treatment of many severe autoimmune diseases. Nevertheless, for some patients these biotherapeutics activate the immune system, leading to the formation of ADAs. This session will present and discuss a cross-diseases strategy for the association and prediction analyses of the first ADA occurrence to identify common immunogenetic pathways.
Tuesday, Nov. 6 10:30 am – 11:00 am
EMERGING REGULATIONS IMPACTING BIOANALYSIS
SYSTEMATIC APPROACHES IN INVESTIGATION OF FAILED BIO-COMPARABILITY STUDY
Wednesday, Nov. 7 9:00 am – 9:30 am
CONSIDERATIONS FOR ESTABLISHING BIOSIMILARITY FOR COMPOUNDS WITH OBSERVED BIOTRANSFORMATION
Establishing interchangeability for a biosimilar and innovator therapeutic is complex. Regulatory submissions for biosimilar therapeutics require evaluation of efficacy, PK profile, and safety. Data will be presented to support the significant impact that assay reagents and assay format may have on the detection of a targeted analyte. In addition, inconsistencies observed when analyzing quality control samples versus patient samples will also be shown.
Wednesday, Nov. 7 9:30 am – 10:00 am
TECHNICAL APPROACHES IN PRESTUDY METHOD VALIDATION AND SAMPLE ANALYSIS TO MINIMIZE BIOANALYTICAL BIAS
Biocomparibility and PK similarity studies follow similar criteria as those used in BE studies to ensure quality, safety, and efficacy of biological products. This session will present the scientific consideration and technical approaches in assessment bias differences during prestudy method validation when comparing a reference product to a testing product.
Wednesday, Nov. 7 10:00 am – 10:30 am
PANEL DISCUSSION: BIOANALYTICAL INVESTIGATION OF FAILED BIO-COMPARABILITY STUDIES
Wednesday, Nov. 7 10:30 am – 11:00 am
CHEMICAL
PBPK MODELING AND CLINICAL PHARMACOLOGY IN REGULATORY APPLICATIONS AND DECISION MAKING
INTEGRATING INTER- AND INTRA-SUBJECTS VARIABILITY IN PBPK MODELING: CONSIDERATIONS AND KNOWLEDGE GAPS
When dosing drugs, understanding drug safety and efficacy in various subgroups rather than an average person is key. Extreme effects, side effects, or lack of therapeutic effects in some subgroups following administration of similar doses require full understanding of variability. Developing population-based PBPK models where such covariates are mechanistically incorporated can assist with predicting between and within subjects variability. Recent advancements and knowledge gaps will be discussed.
Monday, Nov. 5 9:00 am to 9:30 am
GATHERING VARIABILITY DATA IN PHYSIOLOGICAL PARAMETERS FOR USE IN MODELING TO PREDICT BE OUTCOMES
Monday, Nov. 5 9:30 am – 10:00 am
ROLE OF VIRTUAL TRIALS MODELING IN PRODUCT DEVELOPMENT, OPTIMIZATION, AND BEYOND: FDA PERSPECTIVE
An overview of PBPK modeling and simulation in product development, optimization, and regulatory submissions will be provided. Areas with high confidence in model performance and scientific challenges in low confidence areas will be discussed. The session will give an update on the PBPK submissions to FDA. Examples will illustrate how PBPK modeling and simulation have been used for decision making.
Monday, Nov. 5 10:00 am – 10:30 am
USE AND QUALIFICATION OF PBPK STRATEGIES IN REGULATORY SUBMISSIONS AND APPROVAL: EMA’S PERSPECTIVE
This session will describe the use of PBPK modeling in drug development and regulatory submissions. It will cover its biopharmaceutical and drug interaction uses with a special focus on pediatric clinical study design. The methods of assessment of PBPK models will be discussed. An EMA regulatory guidance on the qualification and reporting of the PBPK models will be provided.
Monday, Nov. 5 10:30 am – 11:00 am
NONTRADITIONAL APPROACHES TO BE AND BIOSIMILARS AND APPLICATION OF CLINICAL PHARMACOLOGY TO MINIMIZE BARRIERS TO GENERIC DRUG SUBSTITUTION
SUCCESSES AND CHALLENGES IN ESTABLISHING Q2/Q3 SIMILARITY FOR COMPLEX GENERIC PRODUCTS
This session will clarify strategies on how to approach Q3 characterization of an ANDA ophthalmic suspension. Through a case study, Q3 characterization steps of an ophthalmic suspension will be described.
Tuesday, Nov. 6 9:00 am – 9:30 am
BIOEQUIVALENCE ASSESSMENT FOR INHALATION PRODUCTS
Conventional aerosol performance tests can distinguish between products in the laboratory but fail to predict drug aerosol deposition in different airway regions and to account for variations in lung delivery efficiency. Evolving approaches employing clinically relevant methods capable of comparing the total dose and the aerodynamic particle size distribution of orally inhaled drugs exiting geometrically realistic mouth-throat models under transient, but realistic, inhalation flow conditions will be described.
Tuesday, Nov. 6 9:30 am – 10:00 am
LOCAL AND SYSTEMIC ABSORPTION PREDICTIONS OF NASAL INHALED CORTICOSTEROIDS: A COMBINED APPROACH
Tuesday, Nov. 6 10:00 am – 10:30 am
BIOEQUIVALENCE ASSESSMENT FOR COMPLEX OPHTHALMIC PRODUCTS
Tuesday, Nov. 6 10:30 am – 11:00 am
PHARMACOGENETICS, BIG DATA, AND POPULATION PHARMACOKINETICS IN PERSONALIZED MEDICINE AND SPECIAL POPULATIONS
ROLE OF AI IN CLINICAL BIG DATA ANALYSIS AND DECISION MAKING IN PERSONALIZED MEDICINE
Wednesday, Nov. 7 9:00 am – 9:30 am
EFFECT AND UTILITY OF GENETIC VARIATIONS IN MEMBRANE TRANSPORTERS ON PK IN DRUG DEVELOPMENT
Wednesday, Nov. 7 9:30 am – 10:00 am
TOWARD QUANTITATIVE SYSTEMS PHARMACOLOGY MODELS INFORMED BY INDIVIDUAL GENOMICS DATA
A gap exists between the scope of available genomics data and the scope of well-established quantitative mechanistic models. Systems biology approaches attempt to close this gap with genome-scale mechanistic models of molecular cell biology. Steady state models covering the whole set of metabolic enzymes encoded in the human genome can be constrained by individual genomics data and applied to mechanistic modeling of the effect of genetic polymorphism on the response to pharmacological perturbation. Examples of prototype quantitative systems pharmacology models using genome scale metabolic networks informed by individual genomics data will be discussed.
Wednesday, Nov. 7 10:00 am – 10:30 am
HOW ARTIFICIAL INTELLIGENCE AND MACHINE LEARNING ARE AIDING SCHIZOPHRENIA RESEARCH
This session will describe applying artificial intelligence to the diagnosis and prognosis of psychosis using speech. The results demonstrate that short speech samples suffice to automatically match the expert differential diagnosis of psychosis, achieving accuracies higher than 90%. An interpretation of the speech features that provide for the automated diagnosis, and their universality across different languages, will be provided.
Wednesday, Nov. 7 10:30 am – 11:00 am
BIOMOLECULAR
PBPK MODELING AND CLINICAL PHARMACOLOGY IN REGULATORY APPLICATIONS AND DECISION MAKING
MODELING CLINICALLY RELEVANT IMMUNE RESPONSE: FDA PERSPECTIVE
Monday, Nov. 5 9:00 am – 9:30 am
APPLICATION OF MECHANISTIC PKPD MODELS IN PRECLINICAL TO CLINICAL TRANSLATION OF BIOTHERAPEUTICS Successful implementation of translational PK/PD modeling and simulation early in the drug discovery process can have substantial impact on overall efficiency and quality of decision making in pharmaceutical research. Three case studies will be presented, outlining successful implementation of translational PK/PD modeling in the development of different biotherapeutic modalities, impacting design/selection of lead compounds and prediction of clinical efficacious doses and regimens.
Monday, Nov. 5 9:30 am – 10:00 am
TARGET ENGAGEMENT -BASED TRANSLATIONAL MODELING STRATEGY FOR BIOTHERAPEUTICS DEVELOPMENT
Monday, Nov. 5 10:00 am – 10:30 am
PBPK MODELING OF BIOLOGICS: CURRENT STATUS IN DECISION MAKING
Mathematical modeling and computer simulations using mechanism-based physiologically based PK (PBPK) models are well suited for predicting and assessing complex dose-exposure relationships and may be used to guide the development of therapeutic proteins and antibody-based constructs throughout the development lifecycle. This presentation will describe the basic tenets of PBPK modeling of biologics and present several examples for use in guiding decision making.
Monday, Nov. 5 10:30 am – 11:00 am
NONTRADITIONAL APPROACHES TO BE AND BIOSIMILARS AND APPLICATION OF CLINICAL PHARMACOLOGY TO MINIMIZE BARRIERS TO GENERIC DRUG SUBSTITUTION
FDA’S PERSPECTIVE ON MINIMIZING BARRIERS FOR BIOSIMILAR APPROVALS Biosimilar development programs and applications generally raise unique scientific and regulatory issues. Since Congress passed the Biologics Price Competition and Innovation Act in 2010, FDA has approved 12 biosimilar applications. The presentation will describe the biosimilar regulatory framework through case examples and share steps to minimize barriers to approval and overall success from a regulatory perspective.
Tuesday, Nov. 6 9:00 am – 9:30 am
SWITCHING STUDIES CRUCIAL TO DEMONSTRATE BIOSIMILAR INTERCHANGEABILITY
Tuesday, Nov. 6 9:30 am – 10:00 am
NONTRADITIONAL STATISTICAL STRATEGIES FOR BIOSIMILAR DRUG DEVELOPMENT
This presentation will examine the design and analysis of trials used to determine biosimilarity from a statistical/quantitative perspective. Currently there is no guidance on which quantitative standards should be used. This presentation will discuss some of the challenges of conducting the trials, review methods currently in use, and alternative approaches for the design and analysis of the trials, using case studies.
Tuesday, Nov. 6 10:00 am – 10:30 am
APPLYING PHARMACOMETRIC ANALYSIS IN THE DESIGN OF CLINICAL PHARMACOLOGY STUDIES FOR BIOSIMILAR DEVELOPMENT
The presentation will cover the design of clinical pivotal PKPD similarity studies based on the comprehensive literature review, current EMA and FDA guidelines, and Sandoz’ experiences, together with implementations of various pharmacometric approaches to inform the design of PKPD similarity studies. Dose selection and sensitivity, together with factors affecting the study outcome will be discussed, along with misconceptions associated with the interpretation of bioequivalence results.
Tuesday, Nov. 6 10:30 am – 11:00 am
PHARMACOGENETICS, BIG DATA, AND POPULATION PHARMACOKINETICS IN PERSONALIZED MEDICINE AND SPECIAL POPULATIONS
ROLES OF ARTIFICIAL INTELLIGENCE IN CLINICAL BIG DATA ANALYSES
This talk will explain the relationship between the pharmacometric models and the machine learning models that are commonly used to support the fast growth of artificial intelligence. The roles of these models in drug development and regulatory applications will be discussed.
Wednesday, Nov. 7 9:00 am – 9:30 am
IMPACTING REGULATORY SUBMISSIONS USING PBPK MODELING IN SPECIAL POPULATIONS
Wednesday, Nov. 7 9:30 am – 10:00 am
DOSING DOWN WITH BIOLOGIC THERAPIES: A SYSTEMATIC REVIEW
Wednesday, Nov. 7 10:00 am – 10:30 am
INTEGRATING PMX AND QSP PLATFORM FOR ADVANCEMENT OF BIOLOGICS IN SPECIAL POPULATIONS
Wednesday, Nov. 7 10:30 am to 11:00 am
CHEMICAL
PROCESS CONTROLS, MANUFACTURING, AND ENGINEERING CHALLENGES
NANOPARTICLES THAT ENHANCE CANCER VACCINE EFFICACY
Antigen-loaded or antigen-coated biodegradable particles can be actively taken up by antigen-presenting cells (APCs), and they have shown potential in immunotherapy by initiating a strong immunostimulatory cascade that results in potent antigen-specific immune responses against the target antigen. This symposium will discuss how such particle-based carrier systems offer versatility in that they can simultaneously codeliver adjuvants with the antigens to enhance APC activation and maturation.
Monday, Nov. 5 9:00 am – 9:30 am
CHARACTERIZATION AND EVALUATION OF DRUG-LOADED NANOPARTICLES, REVISITED
Nanoparticles (NPs) are actively investigated as drug carriers with an intention to alter the PK and biodistribution of the drug payloads, enhance their distribution in target tissues, and lower the effective dose and subsequent adverse side effects. This session will discuss common NP characterization and evaluation methods, challenges in proper implementation and interpretation of the methods, and suggestions for future practice in NP development and characterization.
Monday, Nov. 5 9:30 am – 10:00 am
MODULATING THE FUNCTION OF CANCER-DERIVED EXOSOMES THROUGH REPACKAGING
Once secreted from parental cells, exosomes can be captured by neighboring cells and/or released into the systemic circulation for uptake by distant tissues and organs. This session will discuss the role of exosomes in cancer growth and metastasis. RNA-based EXO-codes that are selectively sorted to exosomes have the ability to reprogram pathological, tumorigenic exosomes and repackage them with anticancer small RNAs, representing a novel drug delivery platform.
Monday, Nov. 5 10:00 am – 10:30 am
TARGETING SOLID TUMORS USING NANO-ENGINEERED MESENCHYMAL STEM CELLS
Tumor -targeted drug delivery has the potential to improve therapeutic efficacy and mitigate nonspecific toxicity of anticancer drugs. A unique, active tumor-targeting strategy that relies on engineering mesenchymal stem cells (MSC) with drug-loaded nanoparticles has been developed. The session will focus on the use of nano-engineered MSCs as an efficient carrier for tumor-specific drug delivery and significantly improved anticancer efficacy of conventional chemotherapeutic drugs.
Monday, Nov. 5 10:30 am – 11:00 am
CONTINUOUS MANUFACTURING AND CONNECTED PROCESSES SUPPORTING END-TO-END APPROACHES AND ADAPTING TO THE NONBLOCKBUSTER ERA
NEW DRUG PRODUCT DEVELOPMENT USING CONTINUOUS MANUFACTURING
Tuesday, Nov. 6 9:00 am – 9:30 am
ACADEMIC RESEARCH OPPORTUNITIES TO SUPPORT CONTINUOUS MANUFACTURING
Tuesday, Nov. 6 9:30 am – 10:00 am
CONTINUOUS MANUFACTURING CONTROL STRATEGY FOR ORAL DRUG PRODUCT MANUFACTURING
Tuesday, Nov. 6 10:00 am – 10:30 am
CONTROL STRATEGIES FOR DRUG PRODUCT CONTINUOUS DIRECT COMPRESSION-STATE OF CONTROL, PRODUCT COLLECTION STRATEGIES, AND STARTUP/SHUTDOWN OPERATIONS
Tuesday, Nov. 6 10:30 am – 11:00 am
APPROACHES TO MASTER CMC OF THE FUTURE
CURRENT STATE OF PHARMACEUTICAL MANUFACTURING: TWO SIGMA
FDA estimates that pharmaceutical manufacturing operates at two to three sigma quality. Drug substances, drug products, and their manufacturing processes, in control with respect to common-cause variation at the time of approval, have standards and specifications set from development data that lack mid- and long-term variation. This talk will compare practices in industries that operate at six sigma to those in the pharmaceutical industry and suggest a path for improvement.
Wednesday, Nov. 7 9:00 am – 9:30 am
ADAPTIVE PROCESSES IN PHARMACEUTICAL DEVELOPMENT AND MANUFACTURING
Wednesday, Nov. 7 9:30 am – 10:00 am
KNOWLEDGE INTEGRATION: APPLICATION OF ARTIFICIAL INTELLIGENCE AND MACHINE LEARNING TOOLS IN MANUFACTURING
Wednesday, Nov. 7 10:00 am – 10:30 am
FLEXIBILITY IN FORMULATION
Wednesday, Nov. 7 10:30 am – 11:00 am
BIOMOLECULAR
PROCESS CONTROLS, MANUFACTURING, AND ENGINEERING CHALLENGES
INDUSTRY PERSPECTIVE ON USING PRIOR KNOWLEDGE DURING PRODUCT AND PROCESS DEVELOPMENT
Monday, Nov. 5 9:00 am – 9:30 am
REGULATORY CONSIDERATIONS WITH USING PRIOR KNOWLEDGE FOR NEW SUBMISSIONS
Monday, Nov. 5 9:30 am – 10:00 am
NOVEL APPROACHES TO ROBUST PROCESS CONTROL
Application of ultrahigh-resolution LC-MS/MS-based methods in biotherapeutics upstream and downstream process development has been growing steadily. In the future, online LC/MS in the pilot plant will help provide product attribute control in real time, producing greater batch-to-batch consistency with less off-line, in-process characterization testing. This presentation will share experiences and lessons learned in the application of ultrahigh-resolution MS-based methods for bioprocess development.
Monday, Nov. 5 10:00 am – 10:30 am
CRITICALITY ASSESSMENT OF QUALITY ATTRIBUTES
Characterization of quality attributes and establishing criticality of attributes are key to developing a well-controlled manufacturing process. This talk will discuss a newer paradigm of criticality assessment as well as its role in enabling next generation biologics manufacturing. Additionally, phase-appropriateness of investment in attribute sciences will be discussed. Case studies will be presented for criticality assessment.
Monday, Nov. 5 10:30 am – 11:00 am
CONTINUOUS MANUFACTURING AND CONNECTED PROCESSES SUPPORTING END-TO-END APPROACHES AND ADAPTING TO THE NONBLOCKBUSTER ERA
IMPLEMENTATION OF NEXT-GENERATION TECHNOLOGIES IN BIOMANUFACTURING
Novel approaches to biomanufacturing will help navigate the competitive and emerging business landscape. This presentation will cover recent advances in bioprocessing focusing on drug product manufacturing. Details of fill finish process design for liquid and lyophilized drug products process using fully gloveless isolator-based robotic filling system and ready-to-use container closures will be shared, using case studies to demonstrate technical challenges and strategies to mitigate them.
Tuesday, Nov. 6 9:00 am – 9:30 am
NOVEL APPROACHES FOR ROBOTICS USE IN STERILE PRODUCTS
Tuesday, Nov. 6 9:30 am – 10:00 am
APPROACHES TO REGULATING INNOVATION: CMC CHALLENGES AND OPPORTUNITIES
Tuesday, Nov. 6 10:00 am – 10:30 am
FDA PERSPECTIVE: HOW REGULATORS ADDRESS INNOVATION
Tuesday, Nov. 6 10:30 am – 11:00 am
APPROACHES TO MASTER CMC OF THE FUTURE
CASE STUDIES FOR MABS
Wednesday, Nov. 7 9:00 am – 9:30 am
REGULATORY INPUT ON RISK ASSESSMENTS
Wednesday, Nov. 7 9:30 am – 10:00 am
THE APPLICATION OF RISK ASSESSMENTS FOR THE DESIGN AND DEVELOPMENT OF DEVICES FOR BIOLOGICAL PRODUCTS
Wednesday, Nov. 7 10:00 am – 10:30 am
SPECIFIC CONSIDERATIONS ON RISK ASSESSMENTS AND QBD FOR BIOSIMILARS
Wednesday, Nov. 7, 10:30 am – 11:00 am
CHEMICAL
FORMULATION DEVELOPMENT CHALLENGES AND STRATEGIES
STRATEGIES FOR DELIVERING POORLY SOLUBLE DRUGS
This symposium will give an overview of methods for delivering poorly water-soluble drugs. It will then present a case study, discussing the design and development of CINVANTI injectable emulsion, containing the very poorly water-soluble drug aprepitant. FDA approved CINVANTI in November 2017 for the prevention of chemotherapy-induced nausea and vomiting.
Monday, Nov. 5 9:00 am – 9:30 am
INDUSTRY EXPERIENCE WITH MECHANISTIC UNDERSTANDING OF API PHARMACEUTICAL PROPERTIES TO FACILITATE THE DESIGN OF FORMULATION PROCESSES
Small molecule API can exist in multiple phases with different physicochemical properties, and API phase selection is integral to drug development. Case studies will show industry practice on API phase evaluation to guide formulation and process development for oral and injectable products. The importance of physical characterization to understand the impact of manufacturing and temperature/humidity stress on API phase stability will be discussed.
Monday, Nov. 5 9:30 am – 10:00 am
EXCIPIENTS AND THEIR FUNCTIONALITY FOR ENABLING TECHNOLOGIES IN ORAL DOSAGE FORMS
Excipients modulate solubility, dissolution rate, release kinetics, and spatial control over release and drug targeting. Functionality related characteristics (FRCs) are controllable chemical or physical characteristics of excipients. This talk will focus on excipients in various novel oral drug delivery systems, emphasizing excipient functionality for a given delivery system, their FRCs, and functionality related tests. Impact of excipient variability will be covered.
Monday, Nov. 5 10:00 am – 10:30 am
DEMONSTRATING MICROPRECIPITATION TECHNOLOGY FROM SMALL SCALE TO COMMERCIAL SCALE
Hot melt extrusion and spray drying meet the needs of many poorly soluble compounds, but a class of compounds are not amenable to either due to their high melting point or low solubility in spray drying solvents. Microprecipitation technology yields a high energy form of the drug, microprecipitated bulk powder (MBP). This presentation will cover development of the MBP process from initial screening to scale-up.
Monday, Nov. 5 10:30 am – 11:00 am
QUALITY AND CHARACTERIZATION CONSIDERATIONS FOR FORMULATION DEVELOPMENT
REGULATORY PERSPECTIVE ON CRITICAL QUALITY ATTRIBUTES FOR COMPLEX DRUG PRODUCTS
Tuesday, Nov. 6 9:00 am – 9:30 am
PREDICTIVE DISSOLUTION METHODS Predicting in vivo performance is key. No in vitro or in silico tools can accurately predict PK behavior. By applying in vitro dissolution testing combined with dissolution modeling, we can anticipate differences in PK performance to guide formulation candidate selection and bioperformance risk assessment. Different in vitro approaches to guide formulation candidate selection and to predict in vitro dissolution performance will be presented along with current challenges.
Tuesday, Nov. 6 9:30 am – 10:00 am
HIGH-RESOLUTION CHARACTERIZATION OF STRUCTURE, INTERACTION, AND MISCIBILITY OF DRUG PRODUCTS
For small molecules, drug-polymer interaction ensures that the drug does not crystallize in an amorphous solid dispersion. For proteins, phase separation in lyophilized formulations leads to reduced stability and potential aggregation. This presentation will show the ability to probe these local structures and interactions in small and large molecule systems through case studies demonstrating that structural properties (degrees of interaction, changes in conformation) impact functional properties (crystallization, aggregation).
Tuesday, Nov. 6 10:00 am – 10:30 am
CURRENT TECHNICAL CHALLENGES OF HIGH-RES CHARACTERIZATION OF DRUG PRODUCTS AND INSIGHTS OF IMPACTS OF MOLECULAR INFORMATION ON DRUG DEVELOPMENT
This session will focus on applications of solid-state NMR spectroscopy to gain bulk and molecular-level insights into complex solid dosage forms, including crystalline and amorphous materials, drugs and excipients, and ubiquitous water. NMR is a critical tool in the characterization and understanding of drug product behavior. A variety of applications will be highlighted: quantitation of solid forms, drug-excipient interactions in amorphous formulations, and water distribution throughout a formulation.
Tuesday, Nov. 6 10:30 am – 11:00 am
DEVICE-DRUG COMBINATION
GLOBAL REGULATOR AND INDUSTRY ACTIVITIES ON DRUG/DEVICE COMBINATION PRODUCTS: FOCUS ON EUROPE
The 2017 EU Medical Device Regulation emphasizes review of safety and performance of devices incorporated into drug products. This will involve a new consultation procedure with third party notified bodies from May 2020, but there is no guidance yet. This presentation will explain the regulatory situation and recent developments for combination products in Europe and discuss industry collaboration led by the European Biopharmaceutical Enterprises Biomanufacturing group.
Wednesday, Nov. 7 9:00 am – 9:30 am
HUMAN FACTOR CONSIDERATIONS FOR COMBINATION PHARMACEUTICAL PRODUCTS
Wednesday, Nov. 7 9:30 am – 10:00 am
REGULATORY IMPLICATIONS OF DEVICE CHANGES LATER IN DRUG DEVELOPMENT
Pulmonary diseases are often treated by delivering the drug directly to the lung. This delivery form is complex as it depends on drug, formulation, device, and patient factors. This presentation will focus on regulatory challenges involved in changing drug/formulation/device combinations. The current regulatory landscape and alternatives will be discussed.
Wednesday, Nov. 7 10:00 am – 10:30 am
INHALED PRODUCT ADVANCES FOR AEROSOLIZATION, BREATH COORDINATION, AND PATIENT MONITORING
Orally inhaled drug products rely on patient coordination, breathing, and compliance. After years of poor patient compliance, technological advances make these drug products more intuitive for patients, while providing medical professionals needed data. Key improvement has come from new mechanisms of aerosolization, synchronizing aerosol production with breathing patterns, and new data-capturing devices. This presentation will provide a state-of-the-art review of these new technologies.
Wednesday, Nov. 7 10:30 am – 11:00 am
BIOMOLECULAR
FORMULATION DEVELOPMENT CHALLENGES AND STRATEGIES
EVALUATING PHYSICAL STABILITY OF ANTIBODY DRUG CANDIDATES DURING DEVELOPABILITY ASSESSMENT
This presentation focuses on the physical stability of drug-like property screening. Physical instability (aggregation, solubility, and viscosity) is often observed for biologic drugs and impacts CMC development. The session will show the conformational and colloidal properties and aggregation trends for a series of mAb and bispecific candidates. Examination of aggregation trends through simultaneous evaluation of both conformational and colloidal properties enables classification of candidates as stable or unstable.
Monday, Nov. 5 9:00 am – 9:30 am
GLYCOSYLATED TRIMERIC PROTEIN ANTIGENS: EARLY PHASE FORMULATION CHALLENGES
Monday, Nov. 5 9:30 am – 10:00 am
CHALLENGES FOR INTRAVITREAL FORMULATIONS
Besides clinical challenges, a number of technical challenges (viscosity, dose volume, microbiological safety, particulate matter, etc.) are critical to successful development of an intravitreal product. The symposium will review current understanding of the state of the art in this field, from the biology, formulation, and delivery perspective.
Monday, Nov. 5 10:00 am – 10:30 am
IMMUNOGENICITY RISKS: CONCERN FOR PROTEIN AGGREGATES OR CHEMICAL MODIFICATIONS?
Monday, Nov. 5 10:30 am – 11:00 am
QUALITY AND CHARACTERIZATION CONSIDERATIONS FOR FORMULATION DEVELOPMENT
ESTABLISHING STANDARDS FOR QUALIFICATION AND VALIDATION OF NEW PARTICLE MEASUREMENT TECHNOLOGIES
As particle measurement methods expand beyond light obscuration, membrane microscopy, and visual inspection, how can we establish confidence in these new methods? This session will discuss the application of standards ranging from mixtures of microspheres in the submicrometer range to lithographic particles that have the appearance of large aggregated proteins. Examples will include the application of a NIST reference material for particle size and morphology.
Tuesday, Nov. 6 9:00 am – 9:30 am
BRIDGING THE SUB-MICRON GAP IN PROTEIN AGGREGATE ANALYSIS
Tuesday, Nov. 6 9:30 am – 10:00 am
HANDLE WITH CARE: INDUSTRY EXPERIENCE WITH CSTDS AND BIOLOGICS
Closed system transfer devices (CSTDs) provide a closed mechanism to transfer a drug from its vial to a syringe during patient administration. The symposium will present drug product bio-compatibility data with five commonly used CSTDs: PhaSeal, Codan, ChemoClave, OnGuard, and EquaShield. The effect on a drug’s chemical stability and particulation propensity upon short term CSTD exposure at variable incubation conditions will be reported.
Tuesday, Nov. 6 10:00 am – 10:30 am
PANEL: DISCUSSION AND Q&A ON CSTDS
The session will discuss the compatibility of biologics with closed system transfer devices (CSTDs). Existing data raise concern over the compatibility of the drug product while exposed to CSTDs (e.g., particle formation). Different CSTDs have different hold up volume that may impact the final dosing. This symposium will discuss industry perspective and regulatory expectation around the use of CSTDs and harmonizing their use across industries.
Tuesday, Nov. 6 10:30 am – 11:00 am
DEVICE-DRUG COMBINATION
PATCH PUMP TECHNOLOGY: OPPORTUNITIES AND CHALLENGES
The wearable bolus injector (i.e., patch injector) pipeline and technology and its large injection volumes may present unique challenges relative to the design, clinical suitability, and usability of such devices. Design, use, and manufacturing aspects beyond those associated with more conventional autoinjectors (e.g., fluid path sterility) must be considered. This symposium will review wearable injector technology, and offer insights into the future.
Wednesday, Nov. 7 9:00 am – 9:30 am
APPLICATIONS AND OPTIONS OF PREFILLED SYRINGES WITH LOW-TO-NO SILICONE OIL
Wednesday, Nov. 7 9:30 am – 10:00 am
ELECTRONICALLY POWERED DRUG DELIVERY DEVICES: CONSIDERATIONS AND CHALLENGES
Drug delivery devices are present at the point-of-care, and thus can serve a secondary function as data communications terminals. The miniaturization and commoditization of electronic components and smartphone technology and wireless communication networks and protocols have led to new opportunities in their design. A brief history and a survey of selected state-of-the-art examples will be provided followed by a discussion of the challenges and some predictions.
Wednesday, Nov. 7 10:00 am – 10:30 am
PANEL: STRATEGIC ISSUES IN BIOLOGIC COMBINATION PRODUCT DEVELOPMENT: TO BUILD OR TO LICENSE?
Do you license your drug administration device from a partner, or do you develop and build it yourself? What are the ramifications for product development, approval processes, and your product liability? How does it influence time and costs of development? This session will highlight pros and cons of either approach. Bring your questions and share your experience in this panel discussion with international specialists.
Wednesday, Nov 7 10:30 am – 11:00 am
Keynote Sessions
Monday, November 5
Shyra Gardai, Ph.D. (Seattle Genetics)
Candidate Selection or Preclinical Evaluation of ADC and Immunotherapy Combination
Tuesday, November 6
Ray Deshaies, Ph.D. (Amgen Inc.)
New and Upcoming Drug Modalities
Donald Ingber, M.D., Ph.D. (Harvard University)
Development, Current Applications, and Future Utility of the Organ on a Chip Technology
Wednesday, November 7
Haleh Saber, Ph.D. (U.S. Food and Drug Administration)
Regulatory Considerations into the Translational Aspects and Preclinical Evaluation of Immuno-oncology Drugs
Chand Khanna, D.V.M., Ph.D. (Ethos Vet Health)
Insights on the Homology of Cancers in Dogs and Humans and Similarity Leading to the Parallel Development of Therapeutics
Monday, November 5
Binodh DeSilva, Ph.D. (Bristol-Myers Squibb Company)
The Expanding Frontier of Bioanalysis:
New Modalities
Tuesday, November 6
Nathan A. Yates, Ph.D. (University of Pittsburgh)
Mass Spectrometry Technology
Bernard Maillère, Ph.D. (French CEA)
State of the Art and Future Perspectives on the Predictive Immunogenicity
Wednesday, November 7
Joao Pedras-Vasconcelos (U.S. Food and Drug Administration)
Evolving Regulatory Landscape in Bioanalysis of Biologics; Immunogenicity and Biosimilars
John Kadavil, Ph.D. (U.S. Food and Drug Administration)
The Evolving Regulatory Landscape in Bioanalysis and Bioanalytical Method Validation—PK and Biomarkers
Monday, November 5
Malcolm Rowland, Ph.D. (University of Manchester)
The Evolution of Modeling and Simulation in Drug Development and Regulatory Applications
Tuesday, November 6
Kathleen Uhl, M.D. (U.S. Food and Drug Administration)
Nontraditional Approaches to Bioequivalence and Application of Clinical Pharmacology to Minimize Barriers to Generic Drug Substitution
Speaker TBD
Development of Biologics: Making Affordable High-Quality Medicines by Leveraging Technologies and Data Analytics
Wednesday, November 7
Amita Joshi, Ph.D. (Genentech, Inc.)
Pharmacometrics and Systems Pharmacology to Accelerate Decision Making in Today’s Phase-less Drug Development of Biologics
Jürgen Borlak, Ph.D. (Leipzig University)
Emerging Pharmacogenetics Practices and its Applications in Personalized Medicine
Monday, November 5
Bernard McGarvey, Ph.D. (Eli Lilly and Co.)
Engineering First Principles Applications to Pharmaceutical Manufacturing
Tuesday, November 6
Joseph Shultz (Novartis Pharma AG)
Strategies and Visions for Continuous Biologics Manufacturing from End-to-End
Sharmista Chatterjee, Ph.D. (U.S. Food and Drug Administration)
Continuous Manufacturing and the Role of Models in Implementing Continuous Manufacturing
Wednesday, November 7
Bruce Meiklejohn, Ph.D. (Meiklejohn Consulting)
Global Regulatory Strategies for Biotech Products
Gregory E. Amidon, Ph.D. (University of Michigan)
Advancing the Science of Oral Solid Dosage Form Development and Performance
Monday, November 5
Ashley Boam, MBSE (U.S. Food and Drug Administration)
FDA Perspective on Development of
Drug-Device Combination Products
Tuesday, November 6
Wim Jiskoot, Ph.D. (Leiden University) Lessons Learned from 30+ Years of Attempts to Become a Formulation Scientist
Matthew Burke, Ph.D. (GlaxoSmithKline)
Transforming the Patient Experience through Long-Acting Injectable/Implantable Formulations: New Opportunities and Technologies
Wednesday, November 7
Jamie Moore, Ph.D. (Roche)
Particles in Liquid Drug Products: Causes, Impact, and Strategy
Robert O. Williams III, Ph.D. (University of Texas at Austin)
Formulating Poorly Water Soluble Drugs: Importance of Process Selection
END-TO-END HOT TOPIC SESSIONS
Monday, November 5
IMMUNO-ONCOLOGY: WHAT, WHY, AND WHEN?
9:00 am – 9:30 am
CHIMERIC ANTIGEN RECEPTOR T (CAR-T) CELLS: BUILDING UPON CAR-T19
9:30 am – 10:15 am
FDA approved the first adoptive T cell therapy, the University of Pennsylvania/Novartis CAR-T19 (CTL019), which represents a huge achievement for cancer treatment and paves the way to the use of the CAR-T technology in other cancers and in combination with other agents. This presentation will summarize the key milestones in CAR-T therapy, the technologies that have supported these advancements, and future directions and perspectives for the field.
PK/PD AND SAFETY ASSESSMENT OF BISPECIFIC BIOLOGICS IN PRECLINICAL DEVELOPMENT
10:15 am – 11:00 am
Monday, November 5
FIGHTING CANCERS WITH ONCOLYTIC VIRUSES: BIOANALYTICAL CONSIDERATIONS
3:00 pm – 3:30 pm
The development of an armed or unarmed oncolytic virus program comes with a lot of bioanalytical due diligence required in early clinical development to ensure a thorough assessment of risk can be demonstrated. While the platform to measure the circulating and shed virus is typically qPCR, additional assays are needed to monitor the infectivity of the virus in shedding samples. The immunogenicity risk assessment would also need to consider the risk associated with arming the oncolytic viruses with transgenes. An overview of these topics
will be presented with case studies.
CLINICAL BIOANALYTICAL STRATEGY CONSIDERATION FOR CELL THERAPIES
3:30 pm – 4:00 pm
Unlike conventional small molecules or monoclonal antibody-based therapies, cells are dynamic products. They can migrate, proliferate, differentiate, and respond to their environment in vivo. Monitoring and characterizing cell products for use in clinical study are challenging tasks. These challenges and considerations in the context of clinical bioanalysis including bioanalytical strategy, assay development, and validation will be presented.
BISPECIFIC T-CELL REDIRECTORS PK/AD/NAB STRATEGY
4:00 pm – 4:30 pm
This presentation will focus on challenges and solutions when providing bioanalytical support for CD3 targeting bispecific therapeutic antibodies, specifically focusing on assessment of therapeutic PK and immunogenicity potential.
BISPECIFIC T-CELL REDIRECTORS: RECEPTOR OCCUPANCY STRATEGY
4:30 pm – 5:00 pm
Tuesday, November 6
CAR-T CELL THERAPY IN B-ALL: CLINICAL EVOLUTION AND FUTURE
9:00 am – 9:40 am
IMMUNE CHECKPOINT INHIBITORS: CLINICAL PHARMACOLOGY CONSIDERATIONS
9:40 am – 10:20 am
The patterns of efficacy and safety responses that have been observed for immuno-oncology agents are distinct from those of cytotoxic or targeted agents, and these patterns of response should be considered in dose selection and optimization. The talk will focus on unique clinical pharmacology considerations for immuno-oncology agents.
THERAPEUTIC JOURNEY OF BISPECIFIC T-CELL REDIRECTION: LESSONS LEARNED
10:20 am – 11:00 am
There is evidence that targeting induces cellular and humoral responses that can be transferred to patients after an autologous stem cell transplant for breast cancer in a vaccinate and boost strategy. Clinical trials using anti-CD3 x anti-Her2 BiAb targeting for breast cancer, anti-CD3 anti-EGFR BiAb targeting for pancreatic cancer, and anti-CD3x anti-GD2 bispecific antibody targeting for neuroblastoma will be presented.
Tuesday, November 6
RECENT ADVANCES IN GENE THERAPY PRODUCTS WITH SPECIFIC FOCUS ON ONCOLOGY-RELATED AREAS
3:00 pm – 3:30 pm
Encouraging clinical results placed gene therapy, especially CAR-T therapy, on the top of the list of the most advanced immunotherapy strategies. This presentation will provide a high-level review of the immune-oncology field, the current and potential treatment strategies, and will emphasize gene therapy as the most promising tool.
ADVANCES IN THE MANUFACTURE OF LARGE DNA VIRUSES FOR USE AS ONCOLYTIC THERAPIES
3:30 pm – 4:00 pm
Manufacture of replication competent oncolytic viral therapies is especially challenging. Ongoing clinical data support the special promise of this class of therapies, making the development of large-scale manufacture processes for these vectors critical. Recent progress in this area will be discussed.
CMC CONSIDERATIONS FOR THE DEVELOPMENT OF EARLY PHASE CAR-T CELL PROGRAMS
4:00 pm – 4:30 pm
This session will focus on the various aspects of building a successful early phase CAR-T CMC program. Key elements will include apheresis characterization, selection of the desired starting cell population, introduction of the chimeric antigen receptor, expansion of the target population(s), and final formulation and cryopreservation.
CMC CHALLENGES OF GENE/CELL THERAPY PRODUCTS FROM MANUFACTURING, CHARACTERIZATION AND FORMULATION PERSPECTIVES
4:30 pm – 5:00 pm
Wednesday, November 7
EMERGING MODALITIES FOR CANCER THERAPEUTICS
9:00 am – 9:30 am
Due to new mechanistic aspects for cancer therapeutics, emerging future modalities for cancer immunotherapy will involve drug delivery tools and nanoparticles that can target the immune system. Immune-targeting nanoparticles will be discussed with a view to understanding how to target the nanoparticles to various cell types such as dendritic cells and macrophages. The applicability of various nanoparticles including inorganic and dendrimeric systems will be highlighted. New modalities such as mRNA delivery will also be discussed.
ANALYTICAL CHALLENGES OF FIXED DOSE COMBINATIONS OF MONOCLONAL ANTIBODIES
9:30 am – 10:00 am
To meet all of the new challenges, new concepts and methods must be developed for a control strategy for coformulations that fulfills the expectations of health authorities. This presentation will highlight some of the key challenges with regard to test method development and characterization and setting appropriate specifications.
ANALYTICAL DEVELOPMENT OF CAR-T-CELL THERAPIES
10:00 am – 10:30 am
POTENCY ASSESSMENT OF KYMRIAH CELL THERAPY PRODUCT
10:30 am – 11:00 am
Monday, November 5
USING IN-SILICO MODELS TO INTEGRATE IN-VITRO DATA TO SUPPORT VIRTUAL TRIALS TO SUPPORT COST EFFECTIVE DRUG DEVELOPMENT
9:00 am – 9:30 am
PBPK models provide a unique platform by combining a variety of model information into a single framework for accurate predictions of the complex drug behavior in animals, healthy subjects, and specific patient populations. This presentation will focus on the use of in-vitro data to parameterize these models and describe their utility in designing, or in some cases replacing, clinical studies with virtual trial simulations. We will discuss the current strengths and challenges of this approach and highlight areas for potential improvements.
IN-SILICO, IN-VITRO, AND IN-VIVO ANIMAL MODEL DATA TO PREDICT HEPATIC CLEARANCE OF CANDIDATE MOLECULES
9:30 am – 10:00 am
USE OF BDDCS TO EXPLORE DRUG-INDUCED LIVER TOXICITY
10:00 am – 10:30 am
This presentation will discuss the extent to which Biopharmaceutics Drug Disposition Classification System (BDDCS) defining characteristics, independent of knowing actual drug PK/PD and dose, can be used to evaluate prior published predictive proposals.
UTILIZING ARTIFICIAL INTELLIGENCE AS A PHARMACEUTICAL RESEARCH TOOL: A NEW FRONTIER IN DRUG DEVELOPMENT
10:30 am – 11:00 am
Monday, November 5
TURNING MULTIPLE ASSAYS INTO ONE: STRATEGIC LC-MS DATA ACQUISITION FOR ANTIBODY DRUG CONJUGATES
3:00 pm – 3:30 pm
This presentation will examine how the advances in Immunocapture-LC-MS technologies have enabled monitoring of multiple biotherapeutic drug mass variants from in-life studies with absolute and relative quantitation. Data from an antibody-drug conjugate will be presented as a case study.
MAXIMIZING PK AND PD INFORMATION FROM DISCOVERY SAMPLES
3:30 pm – 4:00 pm
ADVANCING MICROSAMPLING FOR BIOMARKER ANALYSIS WITH LESSONS LEARNED FROM CLINICAL CHEMISTRY
4:00 pm – 4:30 pm
This presentation will describe the latest developments in micro-sampling technologies for both phlebotomy- assisted and self-collection in current patient management. Key considerations during development of micro-sampling technologies regarding minimum specimen, location of micro-sample collection, and sampling technique (lancet versus micro-needles) will be described. Rationalization of technologies to generate plasma and blood fractions from a single collection with internal “volume-normalizing” analysis will be shown.
BUILDING CONFIDENCE IN PATIENT-FOCUSED PRECISION MEDICINE WITH LC-MS-BASED BIOMARKERS AND METABOLOMICS
4:30 pm – 5:00 pm
The metabolic fingerprints in the patient’s phenotypic environment may be used to interpret patient-specific responses to therapeutic intervention. This presentation will explore glycolytic fluxes in preclinical models followed by biomarker and metabolomic applications in clinical models. It will conclude with a forward-looking perspective to patient-centered redox homeostasis during pharmacological intervention and disease.
Tuesday, November 6
CALQUENCE: A CASE STUDY IN THE USE OF BIOPHARMACEUTIC TOOLS TO EXPEDITE THE DRUG DEVELOPMENT PROCESS
9:00 am – 9:30 am
Calquence was recently approved in the U.S. after a rapid development program. This session will describe the role of biopharmaceutical tools that helped build a mechanistic understanding of the drug dissolution and absorption process for the drug which ultimately aided in the rapid development and setting of clinically relevant specifications. The session will explore the use of in vivo smart-pill studies, TIM-1, novel methods for understanding mechanistic dissolution, and the integration of in-vitro data into PBPK modeling and how it culminated in an approved product.
ACCELERATING DEVELOPMENT THROUGH APPLICATION OF QUANTITATIVE MECHANISTIC MODELING TECHNIQUES
9:30 am – 10:00 am
PHARMACOMETRICS TO SUPPORT REGULATORY QUESTIONS AND APPROVAL
10:00 am – 10:30 am
Pharmacometric approaches can help leverage knowledge acquired at each stage of development to inform the next step and optimize the development strategy. Key questions that can be addressed include go/no-go decision, selection of optimal dose and dosing regimen, support of the to-be-marketed formulation, and characterization of clinical pharmacology to support regulatory approval. This session will provide several examples of pharmacometric applications that helped accelerate drug development and support regulatory approval of oncology compounds.
NOVEL CLINICAL TRIAL DESIGNS TO ACCELERATE DRUG DEVELOPMENT
10:30 am – 11:00 am
The capability of conducting exploratory clinical trials is being squeezed from almost every direction. Is it possible to decompress this situation? Yes. This session will explore some new methods, including increasing patient engagement, conducting trials remotely, using platform trial approaches, and real time data analysis, that may help smooth the way to more efficient clinical trial operation.
Tuesday, November 6
COST-EFFICIENCY CONSIDERATIONS: THE VISION
3:00 pm – 3:30 pm
Applying 3D printing techniques will require severe changes from industry. Some 3D printing techniques require feedstock materials that will have to be manufactured industrially. Others, however, could lead to the complete production of medicines in hospitals and pharmacies. Could manufacturing even take place in third world countries without involvement of global pharmaceutical companies? This presentation covers the influence of individualization techniques on the health care sector.
3D PRINTING AS AN APPROACH TO DESIGN AND MANUFACTURING OF PHARMACEUTICAL PRODUCTS
3:30 pm – 4:00 pm
The concept of 3D printing as a novel tool for personalized medicine will be discussed. In particular, the dose flexibility in context with the opportunity for on-demand and decentralized manufacture has led to the idea of using 3D-printed solid oral dosage forms in early clinical trials. This approach will be brought in context with the concept of real-time-release testing and reduced end product quality control testing. In addition, using 3D printing as preparation of marketed drug products as personalized medication in pharmacies on demand and/or upon prescription will be discussed.
3D PRINTING FOR RAPID PROTOTYPING OF PHARMACEUTICAL DEVICES IN DEVELOPMENT
4:00 pm – 4:30 pm
Recently, 3D printing has been used as an alternative for final component production and mass customization. This presentation will look at the evolution of 3D printing technologies in support of pharmaceutical development, as well as opportunities for the future.
3D PRINTING IN PHARMACEUTICAL DEVELOPMENT AND MANUFACTURING: TECHNOLOGICAL REQUIREMENTS, CRITICAL ELEMENTS, AND PROCESS CONTROLS IN THE DEVELOPMENT OF NOVEL DOSAGE FORMS
4:30 pm – 5:00 pm
A novel manufacturing process using 3D printing has been developed to meet the needs of various individual or combined formulation challenges. The dosage form is assembled layer-by-layer without using compression forces or traditional molding techniques. This presentation will explore how novel oral dosage forms can be created and customized; critical elements such as particle size and shape, powder flow, and print head specifications are all significant elements that factor into the process capability.
Wednesday, November 7
STRATEGIC APPROACHES TO DEVELOPMENT OF PHASE-APPROPRIATE SPECIFICATIONS INCLUDING ORGANIC IMPURITIES CONTROL
9:00 am – 9:30 am
There is a lack of regulatory guidance on impurity controls at early stages of clinical development. What are appropriate thresholds for reporting, identification, and qualification of impurities in drug substance and drug products used for phase 1 and phase 2 clinical studies? How do pharmaceutical scientists apply science and risk-based approaches to the development of impurity control strategies prior to phase 3? This presentation will cover best practices and case studies regarding development and implementation of organic impurity control strategies at early stages of small molecule clinical development.
THE USE OF BIOPHARMACEUTICS RISK ASSESSMENT TO GUIDE PRODUCT DEVELOPMENT
9:30 am – 10:00 am
BioRAM is a systems approach, which brings together scientists from across pharmaceutics, manufacturing, clinical, and preclinical disciplines to perform an integrated biopharmaceutics risk assessment. This presentation will describe the application of the BioRAM (Biopharmaceutics Risk Assessment Roadmap) approach, an innovative strategy and framework for patient-centric drug development.
STABILITY MODELING FOR HIGHLY ACCELERATED DETERMINATION OF DRUG PRODUCT SHELF-LIFE
10:00 am – 10:30 am
SAP employs isoconversion with designed temperature/RH conditions to build a model for degradant formation or potency loss for drug products and drug substances. Once the model is built, the shelf-life of inside packaging can be determined based on the calculated RH inside the packaging. This presentation will show how these methodologies provide far better predictions of shelf-life than previously possible in significantly less time.
3D PRINTING FOR DRUG DELIVERY APPLICATIONS
10:30 am – 11:00 am
3D printing has seen rapidly expanding medical applications recently. The complex drug delivery system is an evolving area that benefited from the 3D printing tools, which provide customized and optimized drug delivery solutions. In this session, the attendees will get updates on the latest 3D printing applications in drug delivery to promote product quality and bioequivalence assessment. Discussions will be encouraged to identify potential drug delivery applications by 3D printing technology.
Preconference Workshops and Short Courses
Saturday, November 3
SHORT COURSE: (DAY 1) SURFACTANTS AND BIOPHARMACEUTICALS: A LOVE-HATE RELATIONSHIP
Surfactants play an important role in providing protection to therapeutic proteins against interfacial stresses. However, surfactants, especially if of poor quality, can also result in degradation of the therapeutic protein itself. This course will explore a complete cross-disciplinary picture of the current state of knowledge and related issues. Attendees will be able to apply knowledge gained for formulation development, but also to have a holistic approach to surfactants, starting from raw material storage to final control strategies, and regulatory aspects.
SHORT COURSE: (DAY 1) DEVELOPMENT OF FIXED DOSE COMBINATION (FDC) PRODUCTS: CONSIDERATIONS OF GI PHYSIOLOGY AND OVERALL DEVELOPMENT STRATEGY
The gastrointestinal (GI) tract is both the most popular and most complex route of drug product administration due to the convenience for better patient compliance and the challenge it poses for drug product formulators when developing more complex dosage forms. This short course will offer a comprehensive view of the most influential aspects of GI physiology. Through case studies on FDC product development and regulatory issues, attendees will have an opportunity to explore avenues for successfully developing FDC products and their generic versions.
WORKSHOP: FLIGHT SIMULATOR: LEARNING HOW TO DEVELOP COMPLEX GENERIC DRUG PRODUCTS
The availability of generic drug products can mitigate the risk of drug shortages, help make prescription drugs more affordable, and enhance patient access to medicines. However, there may be substantial uncertainty about how to approach the development of complex generic drug products (CGDPs). This practical, active-participation workshop will help participants learn how to approach a demonstration of API sameness and BE for each type of CGDP and how to engage with FDA about different types of questions during development.
SHORT COURSE: A HARMONIZED STRATEGY FOR THE REGULATORY SUBMISSION OF IMMUNOGENICITY VALIDATION DATA
By harmonizing immunogenicity reporting for regulatory submission, we could streamline regulatory review, drive industry objectives, provide guidance for immunogenicity method assessment, and publish finalized recommendations that will be accessible to the community and adopted by the industry. This short course will provide a detailed update of current expectations for immunogenicity validations and a preview of current recommendations. Presentations and panel discussions will prepare attendees for future regulatory submissions.
SHORT COURSE: IMMUNOGENICITY RISK ASSESSMENT UPDATE FOR BIOLOGIC PROTEINS AND PEPTIDES
Drug developers are devising risk mitigation strategies to assess immune responses to protein therapeutics during both preclinical and clinical phases of development. Many factors contribute to protein immunogenicity; however, T cell-dependent (Td) responses appear to play a critical role in the development of antibody responses to biologic therapeutics. The course will explore a range of methodologies to predict and measure Td immune responses to protein drugs. Benefits and limitations of current in silico and in vitro assay methods and in vivo animal models will be discussed.
Sunday, November 4
SHORT COURSE: (DAY 2) SURFACTANTS AND BIOPHARMACEUTICALS: A LOVE-HATE RELATIONSHIP
A continuation of DAY 1 (above).
SHORT COURSE: (DAY 2) DEVELOPMENT OF FIXED DOSE COMBINATION PRODUCTS: CONSIDERATIONS OF GI PHYSIOLOGY AND OVERALL DEVELOPMENT STRATEGY
A continuation of DAY 1 (above).
WORKSHOP: BIOANALYTICAL METHOD VALIDATION: THE FINALIZED GUIDANCE
On May 18, 2018, FDA issued the final Bioanalytical Method Validation Guidance for Industry. What has been incorporated into the guidance is a new tabular presentation of validation parameters for chromatography and ligand binding assays. The guidance also presents FDA expectations on biomarker assays used during drug development. This event will allow regulators to respond to audience questions using an open forum and panel discussion format. Attendees will also learn about the nonclinical, clinical, generic, and inspection perspectives on bioanalytical method reviews.
SHORT COURSE: SCIENCE AND RISK-BASED STABILITY STRATEGIES: APPLICATIONS OF PREDICTIVE TOOLS
The stability of both drug substances and drug products must be understood at the time of product submission and throughout product life cycle. However, the speed at which drug products are being developed today is unprecedented, and experimental design does not support this accelerated development adequately. This short course will clarify the applicability of science and risk-based predictive stability approaches to optimize the stability protocol to develop an in-depth knowledge of stability performance.
SHORT COURSE: IMMUNOGENICITY DATA EVALUATIONS AND CLINICAL INTERPRETATION
The detection, characterization, and interpretation of antidrug-antibodies (ADAs) formed in response to biotherapeutics are influenced by the data evaluation methods used during prestudy validation and in-study sample analysis. Despite several white-paper publications and guidance documents over the past decade, there are significant gaps in the understanding and implementation of appropriate data analytic methods across immunogenicity method development and clinical impact assessments. This short course will provide an overview of regulatory expectations and experience with data reviews, in-depth statistical guidance, case study illustrations, and a demo of some simple data analysis tools.
SHORT COURSE: QUANTITATIVE-SYSTEM-PHARMACOLOGY (QSP): WHY, HOW AND WHEN IN DRUG DISCOVERY AND DEVELOPMENT
Although complex, QSP provides immense opportunities in terms of drug discovery, lead identification, optimization, and development in the clinic. This short course will offer a holistic approach to understand the vital role of QSP by taking a stepwise approach to its application throughout the drug development process. Using real world case studies and examples, this course will provide a unique opportunity for attendees to better understand the rapidly growing field of QSP and appreciate how it can be applied immediately.
For the complete preliminary program with details and locations, visit the PharmSci 360 app.