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Module 1
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Therapeutic Areas of Biologics
The biologics therapeutics sector has seen rapid growth driven by advancements in biotechnology and increasing demand for targeted therapies in areas such as oncology, immunology, and rare diseases. As of 2024, biologics accounted for a significant portion of the drug market, with over 400 biologics approved by major regulatory agencies like the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA). This number includes a diverse range of products, such as monoclonal antibodies, vaccines, recombinant proteins, and antibody-drug conjugates (ADCs).
Learning Objectives
Following this lecture, the learner will be able to:
- understand the fundamental characteristics and mechanisms of biologics, including how they differ from traditional small molecule drugs
- list and describe the major therapeutic areas where biologics have made a significant impact
- gain familiarity with key biologic drugs that are on the market and their therapeutic uses, including examples such as monoclonal antibodies and ADCs.
- understand current trends, cutting-edge developments and future directions in biologic therapies
- have an awareness the clinical efficacy, safety profiles, and real-world impact of biologics on patient outcomes and healthcare practices
- understand the regulatory considerations and market factors that influence the development and availability of biologic drugs
Instructor: Stephanie Pasas-Farmer, PhD
President and Principal Consultant, BioData Solutions
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Module 2
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Pharmacokinetics/Pharmacodynamics of Biologics Part 1: Nonclinical to IND/FIH
This presentation is designed to provide an introduction and overview of the pharmacokinetic (PK) and pharmacodynamic (PD) assessment of biologics specifically in the context of regulated studies required by the United States Food and Drug Administration (U.S.F.D.A.) for application for an investigational new drug (IND). Additionally, the presentation will provide an overview of the strategies and PK/PD testing required and/or recommended to support first in human (FIH) dose selection, administration, and PK/PD assessment in FIH clinical safety studies.
Goals of the Presentation:
The objectives of this lecture are to define the classes of biologics as specified by the U.S.F.D.A. center for biologics evaluation and research (CBER). Provide an overview of the United States statutes and international guidelines governing the conduct of regulated the pharmacokinetic (PK) and pharmacodynamic (PD) assessment of biologics in non-clinical and first in human clinical safety assessment studies. Describe the non-clinical studies required for IND submission and review the strategies for first in human dose selection and PK/PD assessment during clinical safety testing.
Learning Objectives
Following this lecture, the learner will be able to:
- Define the classes of biologics and list & demonstrate a broad understanding of the regulations governing PK/PD assessment
- Compare and contrast PK/PD assessment of biologics and small molecule drugs
- Discuss the objectives and studies required for each stage of the drug development path from molecule selection to first in human dosing and list, compare and contrast, the strengths and weaknesses of the FIH dose selection methods
Instructor: John T. Farmer, PhD, Data Scientist, Researcher, Seattle, WA
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Module 3
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Pharmacokinetics & Pharmacodynamics of Biologics Part 2: ADA, PK, PD in Clinical Development
Biologics is a fast-growing class of drug modality generally characterized by large molecular weight and structural complexity . They are also referred to also as large molecule drugs and are different from small molecule drugs in many properties, resulting in special considerations in clinical development. Pharmacokinetics (PK) and Pharmacodynamics (PD) play a fundamental role in drug development. Pharmacokinetics evaluate what happens to a drug after dosing while PD focuses on how a drug works. The focus of this lecture is on PD biomarkers, which measure target binding and target modulation in response to a drug treatment. PD biomarkers are not necessarily associated with clinical efficacy and are commonly used to inform dose selection. The principle and various approaches of FIH dose selection is introduced in this lecture and a case example is presented to describe the key information used and the approach selected to justify the starting dose for FIH study. Also, model informed drug development has become increasingly utilized. Examples are presented to describe various models and their applications.
Learning Objectives
Following this lecture, the learner will be able to:
- describe and define a biologic
- provide a brief description of Pharmacokinetics (PK) and Pharmacodynamics (PD) in terms of development of a biologic-based drug product
- provide and overview and general understanding of a PD biomarker and how PD biomarkers are used in biologics development
Instructor: Meina Liang, PhD, Chief Technology Office (CTO) and President of Bioanalysis & Biomarkets, Amador Bioscience
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Module 4
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Scientific and Regulatory Considerations for the Development and Validation of Ligand Binding Assays (LBAs) in Regulated Bioanalysis
The course outlines critical scientific and regulatory considerations for the development and validation of Ligand Binding Assays (LBAs) in regulated bioanalysis, with emphasis on supporting pharmacokinetic (PK) and toxicokinetic (TK) studies. Here are the key points covered:
Critical Reagents: Emphasizes the importance of reagent quality in LBAs, stating that the assay's accuracy depends on the reagents used. Reagents include antibodies, peptides, and soluble receptors, with lot-to-lot variability posing a significant challenge.
Method Development: This involves defining the design, operating conditions, limitations, and suitability of the bioanalytical method. Method development should cover reference standards, calibration curves, quality control (QC) samples, selectivity, accuracy, precision, recovery, and stability.
Method Validation: Full validation is essential for newly developed bioanalytical methods to ensure the reliability of analytical results. Full validation includes assessing parameters such as selectivity, specificity, matrix effects, calibration range, accuracy, precision, and stability. Partial validation may be conducted when minor changes are made to a fully validated method, while cross-validation is required when multiple methods or laboratories are involved.
Study Sample Analysis: The bioanalytical method must be applied consistently to study samples after validation. The analysis process includes an analytical run consisting of blank samples, calibration standards, QC samples, and the study samples. Acceptance criteria for analytical runs must be predefined, and any reanalysis or reinjection of samples must be justified and documented.
Incurred Sample Reanalysis (ISR): ISR is recommended to confirm the reproducibility of the reported analyte concentrations in study samples. This process is especially important in pivotal studies to ensure the accuracy and reliability of bioanalytical data.
Finally, detailed documentation and record-keeping are emphasized throughout the course to ensure the integrity and traceability of bioanalytical data.
Learning Objectives
Following this lecture, the learner will be able to:
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to explain the importance of critical reagents, including binding proteins and antibodies, in ligand binding assays. They will also understand the procedures for ensuring reagent quality, consistency, and lifecycle management, as well as the impact of reagent variability on assay performance.
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outline the critical components of LBA method development, including the selection and characterization of key reagents, the development of calibration curves, and the establishment of assay specificity, selectivity, accuracy, and precision to ensure reliable quantification of analytes in biological samples
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implement the regulatory guidelines for LBA method validation, including the evaluation of specificity, selectivity, calibration curve accuracy, precision, carryover, dilution linearity, and stability, ensuring compliance with industry standards for bioanalytical method validation in drug development.
- conduct LBA sample analysis by adhering to regulatory requirements, including setting up an analytical run, ensuring proper calibration and quality control, applying predefined acceptance criteria, and handling reanalysis and reinjection of study samples to maintain the integrity of bioanalytical data.
Instructor: Rafiq Islam, Executive Director of CGT and Biologics Bioanalysis, Pharmaron
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Module 5
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Bioanalysis of Biologics Part 2: ADA Analysis
This course will discuss the immunogenicity of biologics, including how it is tested in clinical trials, the regulatory expectations, and the importance of characterization of anti-drug antibody responses and how it informs the clinical understanding of safety and efficacy of biologics.
Learning Objectives
Following this lecture, the learner will be able to:
- Gain an understanding of what immunogenicity is and why it is important to measure during clinical studies for biologic drugs
- Describe the regulatory requirements for immunogenicity assessment and how to develop ADA assays
- Understand the importance of ADA characterization and how it can inform clinician's understanding of drug effects
Instructor: Lynn Kamen, PhD, Scientific Officer and Executive Director, BioAgilytix
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Module 6
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Ensuring Potency and Precision: Cell-Based Considerations for Reliable Biologic Lot Release
This lecture focuses on the design, optimization, and validation of cell-based bioassays used in biologics lot release testing. Key topic include the role of potency testing, the use of cell-based bioassays tailored to drug mechanisms of action, and regulatory requirements for assay qualification and validation. Case studies and practical considerations, such as assay design and mitigation of variability, are highlighted to support reliable biologics lot release potency testing in regulated environments.
Learning Objectives
Following this lecture, the learner will be able to:
- understand the role of potency testing in biologics lot release
- identify critical factors for cell-based bioassay performance
- understand analytical performance characteristics for bioassays
Instructor: Jamison Grailer, PhD, Senior Research and R&D Group Leader, Promega
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Module 7
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Biosimilars: Challenges and Possibilities
With a realization that US health care expenditures were approaching untenable levels for many health care consumers as well as the federal government, the Biologics Price Competition and Innovation Act was signed into law on March 23, 2010. This law allowed for an abbreviated pathway for biological products that were considered similar to biologic products already approved by the US FDA. The goal was to reduce the costs of product development, and by extension to reduce the cost to patients and third party payers, including government programs such as Medicare and Medicaid. Since 2015, there have been 49 biosimilars approved in the US and although biosimilars were slow to be approved and launched, price competition is starting to be evident in the US drug market. This lecture explores the challenges to biosimilar drug development, and discusses the progress that has been achieved over the last decade.
Learning Objectives
Following this lecture, the learner will be able to:
- define biosimilars and the economic factors that lead to the enactment of the Biologics Price Competition and Innovation act
- describe the basic requirements of biosimilar drug development
- discuss challenges to biosimilar adoption globally but particularly in the US
Instructor: Kelly Colletti, PhD, MBA, Director, Preclinical Bioanalytics, Beam Therapeutics
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Module 8
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Biologics Life-Cycle
This presentation covers the comprehensive journey of bioloic products from their initial development to their eventual market release and beyond. This lifecycle includes key stages such as research and development, preclinical and clinical trials, regulatory approval, manufacturing, and post-market surveillance.
Goals of the presentation
This presentation aims to provide insights into the complexities and critical considerations involved at each stage to ensure the safety, efficacy, and quality of biologic products. The primary objectives are to introduce and define biologics, review the history of biotherapeutics, discuss each stage of the life cycle, and provide a high-level view of the future of biologic drug development.
Learning Objectives
Following this lecture, the learner will be able to:
- describe the key phases involved in the development and lifecycle of biologic products.
- identify the main activities and objectives associated with each stage of the biologics lifecycle, from initial research to post-market surveillance.
- discuss the role of regulatory agencies in ensuring the safety and efficacy of biologic products throughout their lifecycle.
- explain the regulatory requirements and guidelines governing the submission of drug approval applications.
Instructor: Rebecca J. Wates, Associate Director, KCAS Bio, Olathe, KS
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