What Will You Gain from Attending this Course?
At the end of these six sessions, the course participant should be able to understand how to incorporate the latest innovative biopharmaceutical development strategies, methodologies, and tools to:
- Explain the entire medical product lifecycle, from molecule or device concept to marketplace
- Apply optimization principles and latest cutting-edge science for improving R&D productivity and increasing success rates
- Analyze a development organization’s structure to understand the integration and contribution of different functional areas to each stage of product development and post-marketing
- Prepare for future developments and changes in the global pharmaceutical, health economics and business environments, as it impacts discovery, development, lifecycle management, regulatory, and business evaluations for medical products
- Design, evaluate, and manage integrated global product development programs for pharmaceutical, biologic, devices, and other innovative products
- Analyze and evaluate the R&D portfolio
- Devise timely Go/No Go decision-making criteria derived from the target product profile, procedures based upon critical analyses of non-clinical and clinical data sets, and literature and competitor information, leading to improved success rates for new drug candidates and devices
- Incorporate present and future regulatory policies, guidances, and opportunities into strategies spanning all stages of discovery, development, and registration
- Participate in decision-making discussions with comprehensive knowledge of medical product development from discovery to market
- Address real world challenges encountered during the development, manufacturing, review, and commercialization of United States Food and Drug Administration (US FDA) and globally regulated medical products
- Formulate integrated product safety management programs
- Incorporate commercial valuations and investment return estimates into milestone decisions, including early in development
- Explain the legal basis of regulations, patents, and recent and new changes in the law, policies, and governmental initiatives affecting drug development and regulatory sciences
- Apply cross-functional product development knowledge to prepare for their next career step
The sessions
The ACDRS Course consists of six sessions over a period of approximately eleven months, including “team” involvement in mentored, case-oriented breakout discussions. Each session is three days of eight hours each in Washington, DC. In addition, approximately eight hours of preparation per session is required to prepare for the case studies and intersession activities.
The syllabus includes the discovery and development of new therapeutics, biopharmaceutical sciences, clinical pharmacology, pharmacometrics, learning-trial methodology, good clinical practice and ethics, pharmacovigilance and risk management, biostatistics, exploratory/confirmatory trial design, regulatory affairs, optimization, health economics, project and portfolio management, marketing, and new therapeutic approaches. Participants will be involved in lectures, panel discussions, team-oriented case studies, and interactive learning.
Session Co-chairpersons: Charlie T. Gombar, PhD, ACDRS Director and Daniela Drago, PhD RAC FRAPS FTOPRA, Expert Consultant, NDA Partners
Learning Outcomes
At the end of Session 1, the course participant should be able to:
- Summarize the history of the pharmaceutical enterprise
- Discuss the principles and organization of global pharmaceutical research and label-driven product development
- Discuss the future directions of global pharmaceutical, health economics and business environments, and their implications for drug selection, drug development, regulatory and business evaluations
- Identify innovation in discovery and development as a response to patient, medical and market needs
- Develop a Target Product Profile to set the strategy for medical product discovery and development programs and projects
- Explain US Food and Drug Administration regulatory pathways for drugs, biologics, biosimilars, cellular and gene therapies and combination products
Topics
- History of the pharmaceutical enterprise
- Development timelines
- Principles of contemporary drug development and regulatory science
- Patient-centered medical product development
- Pharmacoeconomics, health technology assessment, and methods for comparison of economic value for different products
- Intellectual property in drug discovery and development
- Regulatory pathways for drugs and biologics
- Regulatory landscape and development challenges in pediatric, cell and gene therapies, and novel treatment modalities
- Decision points in development of small and large molecules
- Management science: portfolio, projects, and teams
- Target product profile: A key strategic tool in product development
- Special topics based on cutting edge science, changing regulations, controversies, etc.
Session Co-chairpersons: Paolo Vicini, PhD MBA, Chief Development Officer, Confo Therapeutics; and Lois Lehman-McKeeman, PhD ATS, Vice President, Pharmaceutical Candidate Optimization, Bristol Myers Squibb
Learning Outcomes
At the end of Session 2, the course participant should be able to:
- Describe the principles of drug discovery including in vitro and in vivo techniques used to identify and characterize the chemical, biological, and pharmacological properties of drug candidates
- Explain the fundamentals of pharmacokinetics (PK) and describe the use of preclinical data to predict human PK
- Identify the methodology for selecting, optimizing, formulating, assessing the safety, and protecting the intellectual property of small molecule and biologic preclinical drug candidates
- Apply clinical pharmacology principles for the early assessment and development of clinical candidates
- Describe how biomarkers are integrated into and used to accelerate the drug development process
- Discuss the principles of and regulations for conducting nonclinical and first-in-human clinical studies
Topics
- PK, pharmacodynamics (PD) and absorption, distribution, metabolism, and excretion (ADME)
- Discovery chemistry
- Discovery of biologics
- Chemistry, manufacturing, and control (CMC)
- Selection criteria for therapeutic monoclonal antibodies
- Strategies and approaches for preclinical to clinical translation
- Artificial intelligence in drug discovery and development
- Strategies for predicting human PK, exposure-response, and safety
- Determinants of human PK variability
- Guidelines and Beyond for nonclinical toxicology
- Strategies for successful toxicology investigations
- Devices to support early drug development
Session Co-chairpersons: Diane K. Jorkasky, MD FACP, Consultant to Pharma; and Stacey Tannenbaum, PhD FISoP, Consultant and Coach for the MIDD Community, SJTPharm LLC
Learning Outcomes
At the end of Session 3, the course participant should be able to:
- Discuss strategy for taking drugs into humans and through proof of concept in patients
- Discuss the appropriate use of preclinical data for estimating safe and potentially effective dosing in early human clinical studies, especially the limitations of such predictions
- Discuss the pros and cons of early trial designs, the safety management of subjects in these studies, and the decisions required on limited data
- Discuss model-informed drug development (MIDD)
- Address approaches to measuring change in disease, drug efficacy, and safety as generally modulated by dose-exposure-response
- Discuss and plan strategies for determining dose-response prospectively and finding individual dose-exposure-response by exploratory analysis
- Explain the various aspects of the conduct of a clinical trial
Topics
- Choosing doses/exposures for first in human studies, and the role of pharmacokinetics-pharmacodynamics (PKPD) modeling and simulation
- Biomarker strategy and qualification for efficacy and safety
- Developing drugs from first in human dosing to understanding dose-exposure-response in patients – efficacy and safety
- Regulatory perspective: Utilizing pharmacodynamic biomarkers in drug development
- Quantitative principles for drug development decision-making
- Dose-exposure ranging in early drug development
- Exposure response and drug-drug interaction
- Optimization of clinical trial design
- Conundrums of the learning phase of drug development
- Proof of concept
- Data utilization for decision making at the end of the learning phase
Co-chairpersons: Telba Irony, PhD, Senior Scientific Director, Quantitative Sciences, J&J Innovative Medicine; and John Scott, PhD, Director, Division of Biostatistics, Center for Biologics Evaluation and Research (CBER), US FDA
Learning Outcomes
At the end of Session 4, the course participant should be able to:
- Evaluate clinical trial designs and statistical analysis plans to generate credible evidence about a treatment effect
- Interpret clinical trial data to evaluate whether the data constitute substantial evidence of a treatment effect for a regulatory approved label
- Understand the concepts underlying frequentist and Bayesian inference, including hypothesis testing, p-values and Bayesian posterior probabilities
- Recognize the impact of multiple testing in clinical trials and methods to reduce false positive findings
- Construct an appropriate estimand for a clinical trial using the ICH E9(R1) framework
- Compare the pros and cons for various clinical trial designs, including adaptive designs and enrichment designs, and understand how modeling and simulation play an important role in trial optimization
- Discuss considerations for sample size, power, false positive and false negative decisions emanating from interim analyses and final analyses of clinical trials
- Apply statistical considerations for a confirmatory study design and design an analysis plan for a tailored subgroup for phase 3
Topics
- Role of confirmatory phase in drug development
- Statistical principles for clinical trials
- Choice of control groups in clinical trials
- Study objectives, hypothesis testing, sample size, and probability of study success
- Multiplicity in clinical trials
- Missing data and estimands
- Interim analysis
- Bayesian statistics
- The design, analysis, and interpretation of a clinical endpoint trial
- Modeling and simulation
- Adaptive design in clinical trials and optimization
- Design and analysis of adaptive clinical trials
- Enrichment designs
- Perspectives for developing a targeted medicine
- Co-development of a diagnostic and targeted medicine
- Statistical issues related to design, analysis, and reporting of gene therapy clinical trials
- Statistical issues related to design, analysis, and reporting of clinical trials in rare disease and in pediatrics
- Safety assessment and surveillance
- Pharmacovigilance
Session Co-chairpersons: Daniela Drago, PhD RAC FRAPS FTOPRA, Expert Consultant, NDA Partners; and Christine E. Garnett, PharmD, Lead, Interdisciplinary Team for Cardiac Safety Studies and Clinical Analyst, US Food and Drug Administration (US FDA)
Learning Outcomes
At the end of Session 5, the course participant should be able to:
- Explain the requirements for various regulatory applications, including documentation and possible avenues for receiving advice from regulators
- Discuss the procedures for regulatory oversight in the US, the European Union (EU), Japan, and China, including processes for starting a clinical trial and for marketing authorization in these jurisdictions
- Discuss special US regulatory procedures, regulatory strategies, and crisis management
- Recall and apply the benefit-risk framework used by the US FDA for assessment of new drug applications
- Explain the regulatory landscape for artificial intelligence, machine learning, real-world data, and real-world evidence
Topics
- US FDA applicable laws, regulations, and guidance documents
- FDA’s new drug clinical trial and marketing authorization review processes
- US benefit-risk methodology and considerations
- US labeling requirements
- US regulatory actions and outcomes for NMEs, NDAs, and BLAs
- US regulatory approaches to post-marketing
- US regulatory requirements for rare diseases, pediatric therapeutics, and cell & gene therapies
- Challenges with and approaches to multi-country clinical trials
- US FDA advisory committee meetings and CHMP oral explanations
- CMC section of regulatory applications
- FDORA, PDUFA VII, and their impact on US FDA-regulated industry and US FDA
- Starting a clinical trial and obtaining scientific advice in the EU, Japan, China, and the US
- Procedures and approaches to obtain marketing authorizations from the European Medicines Agency (EMA), Pharmaceutical and Medical Devices Agency of Japan, National Medical Products Administration (NMPA) of China, and US FDA
- Enhancing diversity in clinical trial populations
- Real-world data and real-world evidence
- Artificial intelligence and machine learning
Session Co-chairpersons: Charlie T. Gombar, PhD, ACDRS Director; Jean Lee, PhD, Portfolio & Project Management Biopharma Leader, Lee PPM Consulting; and Michael Dyszel, R&D Project Excellence Lead, CSL Behring
Learning Outcomes
At the end of Session 6, the course participant should be able to:
- Discuss the principles of project and portfolio management, including aspects of planning, project evaluation, and decision making
- Explain the limitations of traditional portfolio management and propose innovative methods to improve portfolio management
- Apply the principles of portfolio management to drive decision making in medical product development
- Discuss the principles of business development, partnering, and alliance management throughout the lifecycle of a product
- Apply the principles of regulatory requirements for advertising and promotion of a prescription drug
- Practice leadership strategies for team interactions and other decision making
Topics
- Portfolio management: principles, challenges, applications, and strategies
- Role of licensing in portfolio management
- Project management: principles, challenges, and strategies
- Judgment under uncertainty
- Integrated global strategy
- Turning strategy into plans
- Co-development and alliances
- Strategic marketing (commercial) role in development
- How to launch a new drug successfully into the market
- Risk communication and prescription drug promotion
- Promotional committee review simulation
- Leadership strategies in drug development and regulation
This has truly been the most useful and engaging training that I have ever attended. The content, presenters, and interaction with peers has been outstanding. The opportunity to learn from leaders in the industry is truly unique and provides a perspective that makes the ACDRS course so valuable. Whether you are new to product development, or have some experience in this field, you will undoubtedly find this informative and educational.