Claim OwnershipFrom Concept to Medicine - A Comprehensive Drug Development Journey
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© Jim Mitchell 2025
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Welcome to a Complete Drug Development Journey!
Have you ever wondered how groundbreaking medicines go from a scientist’s vision to life-saving treatments in hospitals and pharmacies worldwide? The journey of drug development is an intricate, high-stakes adventure—filled with scientific breakthroughs, regulatory twists, and the relentless pursuit of innovation.
In this podcast, we take you behind the scenes of pharmaceutical research and development, unraveling the complex and fascinating process that transforms a promising compound into a fully approved, market-ready medicine. Each episode is a deep dive into the critical stages of drug development, including:
🔬 Drug Discovery: Where innovation begins—exploring cutting-edge research, target identification, and the quest for the next medical breakthrough.
🧪 Preclinical Testing: Investigating safety, efficacy, and mechanisms of action before a drug ever reaches human trials.
👩⚕️ Clinical Trials: The rigorous, high-stakes world of human testing—what works, what fails, and what it takes to get to the finish line.
📜 Regulatory Approval: Understanding the intricate web of global regulatory agencies, FDA submissions, and the make-or-break moments for new drugs.
🏭 Manufacturing & Scale-Up: From lab to large-scale production—how pharmaceutical companies tackle formulation, stability, and distribution challenges.
💰 Market Access & Commercialization: The economics of drug development—pricing, market dynamics, and the delicate balance between innovation and accessibility.
Whether you're a scientist, industry professional, investor, or simply fascinated by the inner workings of the pharmaceutical world, this podcast brings you expert insights, real-world case studies, and in-depth discussions on the past, present, and future of drug development.
Join us as we demystify the science, strategy, and business behind the medicines that change lives. Subscribe now and be part of the journey!
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***Information in this podcast series is for education only and not meant to construe medical advice. For that, please consult your health care professional.***
270 Episodes
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Embark on a journey through the intricate world of drug development, from the spark of an idea to a life-changing medication available at your local pharmacy. Discover the crucial stages of this process, including discovery, preclinical testing, clinical trials, regulatory hurdles, and post-market surveillance. We'll unravel the complexities of each step, revealing the collaborative efforts of scientists, engineers, and regulatory experts. This episode also introduces Quality by Design (QbD), a groundbreaking approach that emphasizes building quality into every stage, ensuring safer and more effective medicines for all. Join us as we demystify the drug development process, highlighting the challenges, triumphs, and the unwavering commitment to improving human health.From the initial "eureka" moment to the rigorous scrutiny of regulatory bodies, we'll explore how a drug candidate progresses through the pipeline. Understand the importance of preclinical research in evaluating safety and efficacy, and delve into the different phases of clinical trials, from small groups of healthy volunteers to large-scale studies involving diverse patient populations. Learn how QbD principles are revolutionizing the industry, ensuring that every step of the process is driven by science, data, and a patient-centric approach. This episode provides a comprehensive roadmap of drug development, laying the groundwork for a deeper dive into specific aspects in future episodes.
Step back in time and explore the fascinating history of drug discovery, from ancient remedies and traditional healing practices to the cutting-edge scientific breakthroughs of today. Trace the evolution of medicine from trial-and-error methods to the systematic, science-based approaches that drive modern drug development. Discover how ancient civilizations utilized plants and minerals for healing, and how those practices, though often rooted in beliefs, sometimes held genuine therapeutic value. We'll examine key milestones like the discovery of penicillin and the development of aspirin, showcasing the serendipitous moments and painstaking research that have shaped modern medicine.This episode also delves into the rise of rational drug design and the impact of scientific advancements in chemistry and biology. Explore how scientists began to isolate and synthesize active compounds, moving from natural sources to the controlled environment of the laboratory. We'll touch on high-throughput screening and microfluidics, technologies that have revolutionized drug discovery, allowing scientists to test thousands of compounds in a fraction of the time. Join us as we celebrate the ingenuity and perseverance of those who have paved the way for the life-saving medications we rely on today.
Uncover the intricate process of selecting a drug target, the crucial first step in developing new medications. Explore how unmet medical needs and profound biological insights spark drug ideas and guide researchers in their quest to identify the perfect target. This episode delves into the world of models, explaining how mechanistic, empirical, and hybrid models help scientists make sense of the complex landscape of the human body. We'll use clear analogies to explain these concepts, making this complex topic accessible to all.Learn about the challenges of finding a druggable target, one that is not only involved in the disease process but also accessible and selective for drug interaction. Discover how researchers validate their targets, gathering evidence to support their hypotheses. We'll also explore how computational modeling is revolutionizing target selection, allowing scientists to predict druggability and prioritize the most promising candidates. Join us as we unravel the complexities of target selection, revealing the delicate balance between science, intuition, and the unwavering pursuit of effective therapies.
Have you ever swallowed a pill and wondered how that tiny thing actually works inside your body? In this episode, we'll unravel the fascinating world of pharmacology, exploring the intricate ways drugs interact with our biological systems. Discover the key players in this microscopic drama – receptors and enzymes – and learn how drugs can either mimic or block their actions. We'll explain the importance of selectivity in drug design, emphasizing how scientists strive to minimize off-target effects and maximize the desired therapeutic impact.We'll also delve into different mechanisms of drug action, from altering the chemical environment within the body to physically binding to target molecules. Explore how mathematical modeling is revolutionizing drug development, allowing researchers to simulate drug behavior and predict outcomes before even stepping foot in a lab. Finally, we'll touch upon personalized medicine, a groundbreaking approach that tailors treatments to individual patients, taking into account their unique genetic makeup and other factors. Join us for a deep dive into the inner workings of drugs, revealing the intricate science behind these powerful molecules.
This episode maps the intricate journey of drug discovery, from identifying a promising molecule (the "hit") to refining it into a potential drug candidate (the "lead"). We'll explore the key stages of this process, including high-throughput screening, where robots test thousands of compounds simultaneously, and lead optimization, where medicinal chemists fine-tune the structure and properties of promising molecules. We'll also revisit the importance of Quality by Design (QbD) and critical quality attributes (CQAs), highlighting how these principles guide every step of the discovery process.Using the real-world example of L-glutamic acid, we'll illustrate the challenges of controlling crystallization and the importance of selecting the right polymorph for optimal drug performance. Discover how scientists navigate the complex landscape of drug discovery, balancing scientific rigor with creative problem-solving. This episode provides a comprehensive overview of the early stages of drug development, setting the stage for a deeper exploration of preclinical and clinical testing in future episodes.
Before a potential new drug can be tested in humans, it must undergo rigorous preclinical research. This episode explores the crucial role of these studies, both in the lab (in vitro) and in living organisms (in vivo), in assessing a drug candidate's safety and efficacy. Discover how scientists use a variety of tests to evaluate a drug's properties, from its solubility and stability to its interactions with target molecules and potential toxicity. We'll delve into the ethical considerations surrounding animal research, highlighting the strict guidelines and regulations that are in place to ensure animal welfare.Learn how preclinical studies help de-risk a compound, gathering crucial data that informs the design of future clinical trials. We'll also discuss the importance of Quality by Design (QbD) principles in preclinical research, emphasizing how a proactive approach to quality management can help minimize risks and maximize the chances of success. This episode provides a comprehensive overview of the preclinical phase, revealing the meticulous work that goes into preparing a drug candidate for human testing.
Embark on a journey through the crucial phases of clinical trials, from the initial safety assessments in small groups of healthy volunteers to the large-scale pivotal studies that confirm a drug's effectiveness in diverse patient populations. Explore the distinct goals of each phase, from establishing a drug's safety profile and dosage range in Phase 1 to evaluating its efficacy and potential side effects in Phase 2 and confirming its long-term safety and effectiveness in Phase 3. This episode also discusses post-market surveillance, the ongoing monitoring of a drug's performance even after it's been approved and made available to the public.Discover how clinical trials are designed to answer critical questions about a drug's safety and efficacy, and how the results of each phase inform decisions about moving forward in the development process. We'll touch on the ethical considerations involved in conducting clinical trials, highlighting the importance of patient safety and informed consent. Finally, we'll explore emerging trends in clinical trials, such as personalized medicine and the use of technology, and discuss the potential challenges and opportunities these advancements present.
Step into the world of regulatory affairs, a critical but often overlooked aspect of drug development. Discover the essential role of agencies like the FDA in ensuring that the medications we rely on are both safe and effective. This episode explores the rigorous process of drug approval, from the initial Investigational New Drug (IND) application to the final decision on whether a drug can be marketed to the public. Learn how regulatory agencies are involved every step of the way, from monitoring clinical trials to reviewing manufacturing processes and ensuring compliance with strict quality standards.We'll discuss the importance of Quality by Design (QbD) principles in regulatory submissions, emphasizing how a thorough understanding of the drug development process and a robust control strategy are crucial for gaining regulatory approval. We'll also touch on the challenges and complexities of navigating the regulatory landscape, particularly in a global context. This episode provides a valuable overview of the regulatory process, shedding light on the critical role these agencies play in protecting public health.
From the laboratory flask to the large-scale manufacturing plant, this episode explores the intricate world of drug manufacturing. Discover the challenges of scaling up drug production, from synthesizing a few grams of a compound to producing kilograms or even tons while maintaining purity and maximizing yield. We'll discuss the complexities of process engineering, highlighting factors like mixing, heat transfer, and reaction kinetics that can significantly impact the final product. This episode also explores the role of enabling chemistry in optimizing drug synthesis and making it robust and reproducible at scale.We'll delve into the importance of pilot plants in testing and refining the manufacturing process before full-scale production. Learn about the critical role of contract development and manufacturing organizations (CDMOs) in providing expertise and resources to support drug development and manufacturing. Finally, we'll introduce the concept of Current Good Manufacturing Practices (cGMPs), the strict regulations that govern drug manufacturing and ensure the quality and safety of medications.
Discover how a tiny molecule becomes a medicine you can take, from pills and capsules to injections and inhalers. This episode explores the fascinating world of drug formulation and delivery, revealing how scientists transform active drug molecules into marketable products. We'll discuss the importance of bioavailability, the amount of drug that actually reaches the bloodstream in its active form, and explain how different formulation and delivery methods can impact a drug's effectiveness. Using the example of ritonavir, we'll illustrate how researchers optimize drug delivery to maximize bioavailability and ensure the drug reaches its target.We'll also explore the concept of the design space in drug manufacturing, defining the safe operating limits for critical parameters like temperature, mixing speed, and ingredient ratios. We'll touch on the role of continuous manufacturing, a revolutionary approach that could transform drug production, offering increased efficiency, flexibility, and the potential for personalized medicine. Finally, we'll emphasize the crucial importance of clear communication and patient education, highlighting the human element at the heart of drug development.
Protecting innovation is crucial in the world of drug development, and this episode delves into the critical role of patents in safeguarding drug discoveries and enabling companies to recoup their substantial R&D investments. Explore the intricacies of patent law, understanding how these legal safeguards grant exclusive rights to inventors and prevent others from making, using, or selling their inventions for a specified period. We'll discuss the delicate balance between incentivizing innovation and ensuring public access to life-saving medications.We'll examine the strategic decisions involved in patent timing, exploring the long game of protecting an invention early on while waiting for regulatory approval and market entry. Discover how companies navigate the complexities of patent strategy, using innovations like new formulations, delivery methods, and even specific crystal structures (polymorphs) to extend their market exclusivity and stay ahead of the competition. This episode provides a valuable overview of the role of patents in the pharmaceutical industry, highlighting their importance in fostering innovation and driving the development of new treatments.
Explore the complex world of drug development finance, from the multibillion-dollar investments and lengthy timelines to the diverse funding sources that fuel this high-stakes endeavor. Discover how economic considerations influence every stage of the process, from target selection and preclinical research to clinical trials and regulatory approval. We'll discuss the challenges of balancing scientific breakthroughs with commercial viability, highlighting the importance of assessing market trends, unmet medical needs, and the competitive landscape.This episode delves into the various players involved in funding drug development, from pharmaceutical giants with vast R&D budgets to smaller biotech startups relying on venture capital and angel investors. We'll explore the role of government agencies like the NIH in supporting basic research and addressing unmet medical needs in areas where private investment might be hesitant. Finally, we'll discuss the treacherous "Valley of Death," the gap between promising research and commercial investment, and explore strategies for bridging this gap and ensuring that potentially life-saving discoveries reach their full potential.
Drug development is a high-stakes endeavor, and this episode confronts the harsh reality of risk and failure in this complex field. Explore why the vast majority of drug candidates never make it to market, and how those early failures can shape future research strategies and ultimately drive innovation. We'll discuss the common pitfalls that derail drug development, from safety concerns and efficacy issues to technical challenges in manufacturing and formulation. Using real-world examples like the development of Viagra and Taxol, we'll illustrate how valuable lessons can be learned from setbacks and unexpected results.Discover the importance of embracing failure as a learning opportunity, adapting research approaches based on the evidence, and constantly refining our understanding of the drug development process. We'll touch on the concept of Quality by Design (QbD) and its role in mitigating risk and ensuring that every step of the process is driven by science, data, and a patient-centric approach. This episode offers a candid look at the challenges of drug development, highlighting the resilience and perseverance of the scientists and researchers who dedicate their lives to finding new and better treatments.
Drug development isn't a solo act. It's a symphony of scientific expertise, a collaborative effort involving a diverse team of specialists, from chemists and biologists to clinicians and regulatory experts. This episode explores the crucial role of team science in driving innovation and bringing new medicines to market. We'll discuss the challenges of coordinating expertise across different disciplines, highlighting the importance of communication, flexibility, and a shared vision. Using the example of crystallization control, we'll illustrate how different teams work together to overcome challenges and ensure that the final drug product meets strict quality standards.We'll delve into the intricacies of scaling up drug production, from the laboratory bench to the large-scale manufacturing plant, and explore how technology and modeling are being used to address the complexities of this process. We'll also touch on the role of artificial intelligence (AI) in revolutionizing drug discovery and development, highlighting its potential to speed up the process and personalize treatments. This episode celebrates the power of teamwork in science, showcasing the collaborative spirit and dedication that drives the development of life-saving medications.
Join us as we recap the key takeaways from Season 1 of The Deep Dive, revisiting the fascinating world of drug development and looking ahead to the exciting topics we'll explore in Season 2. We'll summarize the core concepts we've covered, from Quality by Design (QbD) and crystallization control to microfluidics, modeling, and the crucial role of teamwork in science. This episode reinforces the roadmap of drug development, highlighting the intricate journey a drug takes from the initial spark of an idea to a life-changing medication.We'll also introduce the concept of spiral learning, where we revisit topics in greater depth and explore new connections as we continue our deep dive. We'll tease the upcoming season, offering a glimpse into the cutting-edge advancements shaping the future of medicine, including personalized therapies, gene editing, and artificial intelligence. Finally, we'll challenge our listeners to actively engage with the material, embrace their curiosity, and continue exploring the fascinating world of drug development.
This episode delves into the crucial process of drug target selection, exploring the criteria for a "good" drug target. We'll discuss the target's role in disease, its druggability, and potential safety implications. We'll examine the importance of biological relevance, market needs, and provide examples like HER2 in cancer. The episode also addresses challenges in target selection, such as historically difficult targets and undruggable proteins. We'll introduce emerging target strategies, including molecular glues and targeted protein degradation.Furthermore, the episode will discuss the concept of "druggability" and how it impacts target selection. We'll explore the analogy of a lock and key, highlighting the importance of finding a drug that can effectively interact with its target. We'll also discuss off-target effects and the need for a deep understanding of the target's interactions throughout the body. Finally, we'll touch upon market needs and the sobering reality that a scientifically promising target might not be developed if there's no market for it. The episode will conclude with a discussion of successful targets like HER2 and the unexpected ways limitations can become advantages in drug development.
This episode explains the experimental methods, both genetic and chemical, used to confirm a target's role in a disease. We'll explore techniques like knockout studies and the use of tool compounds, providing real-world examples from OPR&D. We'll also delve into the world of AI-driven target validation and the role of computational biology in this process. This episode provides a foundational understanding of how scientists validate targets before embarking on the long and costly journey of drug development.Furthermore, we will discuss the limitations of traditional methods, such as off-target effects in CRISPR and false positives in tool compounds. The role of AI in analyzing massive datasets and identifying potential targets will also be explored, highlighting the transformative potential of AI in accelerating drug discovery. We'll also touch on the concept of undruggable proteins and the exciting new strategies scientists are developing to tackle these challenging targets. The episode concludes with a discussion of the importance of target validation for the average person and how it impacts the development of safer and more effective therapies.
In this episode of The Deep Dive, we explore the world of High Throughput Screening (HTS), a crucial process in modern drug discovery. HTS is a highly automated method that allows scientists to rapidly test thousands, even millions, of chemical compounds against biological targets to identify potential drug candidates. Think of it like a speed-dating event for molecules, where researchers seek to find that one special interaction that could lead to a new medicine. We discuss how automation and robotics drive the engine of HTS, enabling the efficient handling of vast amounts of samples and reagents, along with the importance of carefully designed, targeted assays to ensure reliable results.We also delve into the challenges of HTS, particularly distinguishing true hits from false positives, which can be a significant hurdle. We explore the strategies scientists use to filter out "imposters," including reference databases, reference signals, and orthogonal testing. Finally, we examine the factors considered when prioritizing potential drug candidates, such as ADMETox properties, and discuss how the hits identified through HTS are only the starting point of a long journey. Discover how this intricate process underpins much of modern medical progress and consider exploring related topics like assay development, medicinal chemistry, and the drug development pipeline.
This episode introduces the innovative approach of fragment-based drug discovery, where drugs are built piece by piece from small chemical fragments. We'll use the analogy of assembling a puzzle, comparing fragment screening with high-throughput screening (HTS). The discussion will also delve into the intricate details of protein-ligand interactions at the atomic level, providing insights from X-ray crystallography and NMR. Real-world literature examples from OPR&D sources will further illustrate the concepts.We will also discuss the challenges of working with natural products, including their complexity, scarcity, and the difficulties of extraction. The resurgence of interest in natural product screening will be explored, highlighting the innovative ways scientists are combining traditional methods with cutting-edge technology. We'll also examine the ethical and environmental considerations surrounding the use of natural products in drug development. The episode will conclude with a discussion of the future of drug discovery, emphasizing the potential of AI and the importance of a balanced approach that combines the power of both natural products and synthetic libraries.
This episode describes the iterative process of refining "hits" into promising "leads" in drug discovery. We will explore how scientists balance potency, safety, and drug-like properties while making successive chemical modifications. The concept of structure-activity relationships (SAR) will be central to the discussion. We'll also integrate a case study, such as the development of SARS-CoV-2 antivirals, to illustrate the process of lead optimization and the challenges involved.Further, we'll contrast predictive modeling with empirical optimization in lead optimization. We'll discuss the challenges of ensuring a drug can reach its intended target in the body and the complexities of drug resistance. The episode will also delve into the concept of reactive metabolites and how they can complicate drug development. We'll conclude with a discussion of the ethical considerations in drug development and the importance of balancing the benefits of new treatments with potential risks.
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