PharmaLex Talks

Synopsis: In this podcast, Gerardo Gomez talks about the interactions between validation and technology transfer, considering the views and expectations of the regulators. As Gerardo explains, technology transfer is really a very sophisticated and complex project management activity and therefore needs to be carefully planned to avoid discrepancies or deviations from the regulations. Bio: Dr. Gerardo Gomez has more than 25 years of experience in the Pharmaceutical and Biotechnology industries. He has held technical and leadership roles in the areas of validations, technical transfer, operations, and regulatory compliance in major biopharmaceutical companies. As part of the PharmaLex leadership team, Gerardo is conducting both strategic and tactical (execution) projects, while playing key roles for PharmaLex clients – big and small – and for PharmaLex talent and business development initiatives. Gerardo earned his Ph.D. in Pharmaceutical Sciences from the University of Michigan, after graduating magna cum laude from the Chemical Engineering department at The University of Puerto Rico.

Synopsis: Early access programs are designed to allow patients suffering from a life-threatening unmet medical need or disease to be given promising treatments that have not yet received marketing approval. Examples of these types of programs include named patient programs, which are for a specific patient suffering from a rare, ultra-rare or life-threatening disease, and cohort programs, where patients with the same disease have been identified. Bio: Patrick Larcier is Senior Director at PharmaLex, where he draw on his 30 years of experience in drug development and regulatory affairs at biotechnology companies, CROs and in consulting. Patrick works on Strategic Product Development Solutions for PharmaLex’s global projects. His expertise lies in the design and implementation of regulatory strategies for complex and innovative medicinal products at all stages of clinical development.

Bio Patrick Nieuwenhuizen Quality professional with a Microbiology & Sterile Manufacturing background with over 25 years’ experience in the Pharmaceutical Industry. Worked for several global Pharmaceutical and Biotechnology companies across a variety of platforms including Biologics, Sterile Fill Finish and Solid Oral Dose. Involved with several site and laboratory expansion projects from construction design through to method transfer and operational readiness, and has provided Quality, Sterility Assurance and Microbiology oversight where relevant during these projects. In addition to site responsibilities, involved in several corporate initiatives such as Sterility Assurance Council and the roll-out of corporate standard programs that required collaboration and communication across multiple diverse sites for the improvement and maintenance of organisational quality standards. Next to that acting as a lead auditor and have been involved with audits facing several competent authority inspections including but not limited to the HPRA, FDA, ANVISA, Chinese FDA and Canadian Health Authority inspections. Acted as risk facilitator for Quality Risk Management programs and have gained significant experience with problem-solving and management of complex investigations.

Synopsis: Technology transfer involves two main types: scale-up and transfer to a different site. Scale-up technology transfer involves moving a technology or process from a laboratory or pilot-scale environment to a larger production scale. Transfer to a different site involves relocating a technology or process from one location or organization to another. Listen to the podcast to learn more about technology transfer and important considerations for success. Bio: Olena Chervonenko is Associate Director Quality Management and Compliance at PharmaLex, where she draws on her expertise in quality management and regulatory compliance to support clients and her team. Olena has a Master’s degree in Pharmaceutical Chemistry from Odesa Mechnikov National University.

Bio: Michael Braun-Boghos Michael has been working in medicinal product safety for the last three decades. He spent 13 years at the European PV headquarters of Fujisawa, which later became Astellas, ultimately leading the Safety Data and Quality Management group. Thereafter he joined Relsys, the developer of the Argus software, and finally came to Oracle with the Relsys acquisition in 2009. As a senior director in the safety strategy team, he helps drive the product roadmap of the Oracle Safety One Platform including Argus, Empirica, Safety One Intake, and Oracle Analytics. Bio: Cheryl James Cheryl James has worked at a senior level within the pharmaceutical industry for over 15 years, building extensive business knowledge and implementation experience with the Oracle Life Sciences suite, specifically AERS and now Argus Safety. In her role as Director of Safety Analytics for PharmaLex (formerly pharmasol), Cheryl has been instrumental in developing the Argus components that have culminated in the industry-leading rapidLIVE – PharmaLex’s accelerated, pre-packaged implementation for Oracle Argus. Cheryl was first appointed as Director of Pharmacovigilance for pharmasol in 2008, joining from Oracle, where she served as Senior Life Sciences Consultant specialising in AERS. Prior to Oracle, she held the position of Principal Pharmacovigilance Scientist within the newly created Pharmacovigilance group at PPD, a global CRO. This role provided the opportunity to develop invaluable interdisciplinary experience across numerous Oracle applications and multiple drug safety systems. Previously, Cheryl worked as part of a research group within a cutting-edge medical centre, sparking her career-long interest in drug safety. Cheryl holds a BSc in Biology and Psychology from the University of Bristol.

Market access can be a big hurdle for companies to overcome and might require a different approach to the process of gathering evidence to support reimbursement discussions. Early phase modeling can help to inform feasibility of future adoption, the product development program, support investor communication, and sometimes even to inform decisions on whether to pursue product development or not. The podcast explores early phase modeling, what it is and why it’s becoming a key component in reimbursement and patient access. Bio: Annabelle Fosmark is Senior Manager, Health Economics & Outcomes Research at PharmaLex and has vast experience in managing projects concerning all the Nordic markets, including reimbursement dossiers, supporting payer negotiation processes, health economic modelling, evaluation of clinical and health economic data, and strategic advice on market access.

Combination products offer unique advantages in terms of delivering improved therapeutic outcomes or enhanced patient convenience. Typically, combination products are understood to be a drug-device or biologic-device combination. The regulatory strategy as to which center at the FDA has jurisdiction over product approval depends on their primary mode of action. Listen to the podcast to learn more about combination products and how they are regulated. Guests’ bio: John Lockwood is Senior Director, Head of Medical Devices/IVDs, Americas for PharmaLex, where he leverages his more than 28 years of industry experience across quality, regulatory, operations, development, validation, and purchasing. John has worked at both small and large medical device manufacturers and has spent more than a decade in consulting.

Companies often fall into common traps in early-stage product development. These range from interactions with the regulators to their ability to manufacture their product. Forward planning is crucial, but all too often smaller companies lack the regulatory, business development or marketing expertise needed to support this way of thinking. The podcast explores some of the pitfalls companies can fall into and ways to avoid them. An expert in global drug development, Alex Klein has over 15 years of experience at some of the world’s leading organizations including Abbott, Abbvie, and Hospira, as well as various biotech startups. Her drug development experience includes both biologics and small molecules across multiple therapeutic areas. Possessing an understanding of what it takes to deliver new drugs to market, she has progressed several assets through development from preclinical through commercialization including supporting key regulatory agency meetings and submissions.

The pharmaceutical industry has been globalizing for many years as demand for medicinal products grows and as companies look to expand into the large and increasingly prosperous emerging markets. The impact of rapid globalization is that the industry is dealing with complex and ever-changing regulations and government policies that can vary across jurisdictions. The podcast explores the opportunities and challenges of globalization. Bio: Dr. Jürgen Hönig is Senior Director, Regulatory Business Intelligence, where he participates in the design of regulatory affairs strategy, analyzes trends in the pharmaceutical and regulatory devices industries and supports the interface between regulatory affairs and marketing and sales. Jürgen has been in the pharmaceutical industry for nearly 25 years and previously was a pharmacist. He has a Ph.D. and an undergraduate degree in pharmacy.

For US-based companies seeking to market their product in the EU, it can be a struggle to navigate a different market, and, in particular, to manage requirements from a safety reporting perspective. The podcast explores the challenges faced managing not only the European pharmacovigilance legislation but also dealing with each EU Member State, which can implement these requirements in their own way at the national level. Bios: Monica Buchberger is senior director of pharmacovigilance at PharmaLex, drawing on her many years in industry in pharmacovigilance and regulatory roles, including quality assurance systems and serving as the EU QPPV for Abbott Laboratories. Stephen Sun is a physician by training who heads up the US pharmacovigilance team for global PharmaLex and is also the service area head for benefit-risk management.

Artificial intelligence is playing an increasingly important role in document management in the life sciences industry and in particular in improving management and oversight of the Trial Master File (TMF). The podcast explores why and how AI is a gamechanger for the TMF and what it means for pharmaceutical companies now and in the future. Bio: Jim Horstmann is Product Manager of Clinical Products at Phlexglobal where he draws on his extensive experience in document management solutions that comply with health authority regulations while also being easy to use and interoperable with other systems. For the past 11 years, Jim has focused primarily on electronic Trial Master File solutions. He is passionate about optimizing business processes by leveraging technology to help make organizations more efficient and productive. For more information about how AI is used PhlexTMF, please visit https://www.phlexglobal.com/

Summary: IVD manufacturers must perform two types of performance studies, clinical performance and analytical performance, in order to provide evidence that the device is safe and performs as intended. While both have always been required, IVDR is a lot more prescriptive. Speaker: Keely So is Manager, Medical Devices / IVD at PharmaLex, leveraging her extensive knowledge of the regulatory requirements in Australia, Europe, New Zealand, the USA, Canada and Asia to help clients with new product licensing and launch, regulatory impact and gap assessments, CE marking of devices and IVDs, ongoing product compliance initiatives and audit support. Links: https://www.pharmalex.com/pharmalex-industry-sectors/medtech/

Summary: A lot of statistical work within pharmaceutical companies is repetitive and this costs money and resources, especially if it’s done within GxP environments where there is often a need for double programming, extensive data quality control and meticulous follow-up of procedures. The podcast explores requirements and ways to develop fully automated solutions and processes to address this repetition and improve overall business efficiency. Speaker: Davor Josipovic is senior manager statistics and data science at PharmaLex, focusing on developing innovative solutions for questions the pharmaceutical industry faces, using applied mathematics, probabilistic modelling and machine learning. Links: General page: https://www.pharmalex.com/pharmalex-departments/data-science-in-pharmaceutical-industry/ Custom solutions: https://www.pharmalex.com/software-login/

A strong quality culture in the biopharmaceutical industry is one in which all participants in the organization understand patient safety and how their roles impact patient safety risks. It is one where decisions are prioritized based on patient safety first. From a GMP perspective, that means not only abiding by the formal law but knowing the applicable current best practices, understanding the risks a company’s processes and products pose to patients and implementing effective quality risk management practices. About the speaker: Londa Ritchey is Quality Director with the Quality Management and Compliance group at PharmaLex.

There are unique considerations and nuances to the US Food and Drug Administration’s (FDA) pre-submission process for medical devices that companies need to consider. Known as the Q-submission (Q-sub) program, these pathways are a way of communicating with the regulator to obtain their feedback on particular topics that are germane to both the manufacturer and the regulator. About the speaker: Mohammad Asad is the Regional Sales Manager – Asia Pacific at PharmaLex, where he draws on his consulting and industry experience in Australia, the US, and Singapore and on his engineering analytical background to help start-ups and large companies alike bring new and novel technologies to market.

With pharmaceutical companies increasingly turning to outsourcing partners to support maintenance of marketed products, efficiency and reliability are imperative. Digitalization, in particular automation and AI, plays an integral role in streamlining processes and enhancing that outsourcing relationship. Timm Pauli is Vice President and head of R&D informatics at PharmaLex, where he combines his informatics and systems expertise with his experience in the pharmaceutical industry. Timm has been with PharmaLex for more than 8 years in a series of senior roles. Before joining PharmaLex, Timm worked in various technology-driven R&D roles at leading pharmaceutical and healthcare companies. Timm has a degree in medical informatics. Jim Nichols, Chief Product Officer, brings over 20 years’ experience with compliant software solutions for regulated processes to Phlexglobal. Jim has served in senior management positions at Liquent, Thomson Reuters and DitaExchange. He has a Bachelor of Science in Mathematics from The Pennsylvania State University.

This podcast introduces the concept of strategic product development as part of an integrated strategy to bring new drugs to the patients that need them. This holistic approach helps to save time, reduce overall development costs, and identify and mitigate risk throughout the product lifecycle.

Introduction: The ICH-Q2 and ICH-Q14 together describe the development and validation activities proposed during the life cycle of an analytical method to assess the quality of medicinal products. But what is it that defines the quality of a reportable value? The podcast explores how Bayesian statistics makes it possible to take an observation made during the validation of the analytical procedure and predict the uncertainty around any future result, and in so doing bridge the gaps that exist in the guidelines. About the speaker Bruno Boulanger, Ph.D., is Global Head Statistics and Data Science at PharmaLex, where he draws on his many years of experience in several areas of pharmaceutical research and industry including discovery, toxicology, CMC and early clinical phases. Bruno has authored or co-authored more than 100 publications in applied statistics. He organizes and contributes to Non-Clinical Statistics in Europe and, in 2010, set up the First Applied Bayesian Biostatistics conference.

Brad Carlin is a statistical researcher, methodologist, consultant, and instructor. He currently serves as Senior Advisor for Data Science and Statistics at PharmaLex, an international pharmaceutical consulting firm. Prior to this, he spent 27 years on the faculty of the Division of Biostatistics at the University of Minnesota School of Public Health, serving as division head for 7 of those years. He has also held visiting positions at Carnegie Mellon University, Medical Research Council Biostatistics Unit, Cambridge University (UK), Medtronic Corporation, HealthPartners Research Foundation, the M.D Anderson Cancer Center, and AbbVie Pharmaceuticals. He has published more than 185 papers in refereed books and journals, and has co-authored three popular textbooks: “Bayesian Methods for Data Analysis” with Tom Louis, “Hierarchical Modeling and Analysis for Spatial Data” with Sudipto Banerjee and Alan Gelfand, and "Bayesian Adaptive Methods for Clinical Trials" with Scott Berry, J. Jack Lee, and Peter Muller. From 2006-2009 he served as editor-in-chief of Bayesian Analysis, the official journal of the International Society for Bayesian Analysis (ISBA). During his academic career, he served as primary dissertation adviser for 20 PhD students. Dr. Carlin has extensive experience teaching short courses and tutorials, and won both teaching and mentoring awards from the University of Minnesota. During his spare time, Brad is a health musician and bandleader, providing keyboards, guitar, and vocals in a variety of venues.

Under the Medical Devices Regulation economic operators – that is manufacturers, the authorised representative, importers and distributors – all face new roles and obligations. While some of these responsibilities existed under the Medical Devices Directive, workload will be impacted. From requirements around Eudamed, to having a person responsible for regulatory compliance, to ensuring certain obligations are properly followed through to complaint management, the roles of all economic operators are affected and require careful consideration by companies with devices in the European market. Clare Huntington is Director and Head of Medical Devices / IVD UK at PharmaLex Clare leads the UK and EU teams and consults on industry-wide issues including drug/device combination products, clinical trials, device classification and regulatory submissions, and post-market requirements. Clare has worked in a variety of roles in the medical device space during her career, including the MHRA, a Clinical Research Organisation, as an EU Authorised Representative, as well as industry, leading the regulatory affairs activities. This has given her a broad understanding of the EU regulatory landscape which enables her to provide comprehensive advice to clients that covers the full product life-cycle.