Conversations in Drug Development

In this episode of Conversations in Drug Development, host Harriet Edwards is joined by Kelsey Lennoch and Eamon McGowran to discuss the EU Biotech Act, a major regulatory proposal aimed at revitalizing Europe’s biotech sector. They explore the act’s goals to streamline clinical trial processes, harmonize regulations, and boost funding for late-stage biotech companies. The conversation highlights key changes for medicines and medical devices, the act’s potential impact on innovation and competitiveness, and the importance of ongoing engagement as the EU’s regulatory landscape evolves. For more information, visit www.boydconsultants.com.

In this episode, Eric Hardter and guest Kelsey Lenoch discuss recent US FDA developments, including leadership changes at CBER and CDER, staffing impacts on orphan product reviews, and the new Rare Disease Evidence Principles aimed at accelerating approvals for rare and ultra-rare conditions. They also explore evolving trial endpoints in oncology (including surrogate measures and ctDNA), transparency moves such as the public release of Complete Response Letters and the industry’s reactions, updates on the National Priority Voucher Program, and the FDA Pre-Check initiative to boost domestic drug manufacturing. Tune in for an in-depth discussion offering timely updates and expert perspectives on what may be ahead for drug development stakeholders. 🎧 Chapters Welcome and Introductions (00:00:05) Recap and Recent US Regulatory Affairs Developments (00:01:00) Dr. Vinay Prasad’s Removal and Return to FDA CBER (00:01:20) New Appointments at FDA CDER (00:02:17) FDA Staffing Shortages and Impact on Orphan Products (00:04:22) Rare Disease Evidence Principles Announcement (00:05:29) FDA’s Rare Disease Innovation Hub and Ultra-Rare Disease Focus (00:07:19) Novel Endpoints in Oncology Drug Development (00:09:31) FDA/AACR Joint Meeting on Novel Endpoints (00:10:12) Challenges with Overall Survival as an Endpoint (00:10:36) FDA’s Evolving Guidance on Endpoints (00:11:04) Post-Licensure Requirements and Surrogate Endpoints (00:11:48) Case Study: Myelofibrosis Drug and Patient-Reported Endpoints (00:12:49) Standardization Challenges for New Endpoints (00:13:55) Circulating Tumor DNA as a Biomarker (00:14:51) FDA’s Release of Complete Response Letters – Phase Two (00:16:06) Pros and Cons of Public Complete Response Letters (00:17:19) Industry Pushback and Sponsor Transparency (00:19:22) Case Study: Lycos Therapeutics and Psychedelic Drug Approval (00:21:09) Accessing and Mining Complete Response Letter Data (00:22:34) National Priority Voucher Program Update (00:23:43) FDA Pre-Check Program for Domestic Manufacturing (00:24:26) Closing Remarks and Future Outlook (00:25:12)

In this special episode, host Harriet Edwards sits down with Professor Alan Boyd, founder and CEO of Boyds, to reflect on the company’s remarkable 20-year journey in drug development. Professor Boyd reflects on his pioneering work in gene therapy, the challenges and achievements of leading cutting-edge projects, and the important role of medicines that helped pave the way for future innovation, even if they did not ultimately receive regulatory approval. They also speak about the importance of maintaining a patient-centric vision and the role this has played in Boyds success on a global scale. With stories from Boyds involvement in pivotal projects, like the first gene therapy for brain cancer and contributions to the Covid-19 vaccine, the conversation offers a rare, behind-the-scenes look at how scientific innovation and evolving regulation have shaped modern medicine. Whether you're a seasoned expert or new to the field of drug development, this episode delivers both valuable perspective and fresh inspiration from a figure with decades of experience at the forefront of the industry. 00:00:13 – Introduction to Conversations in Drug Development 00:00:30 – Welcoming Professor Alan Boyd Back 00:01:43 – Reflecting on 20 Years of Boyds 00:02:49 – The Journey to Founding Boyds 00:06:05 – Early Challenges in Gene Therapy 00:07:26 – Manufacturing Gene Therapy Products 00:10:35 – Achievements in Cell and Gene Therapy 00:15:39 – Highlights from Two Decades 00:19:46 – Changes in Drug Development 00:23:32 – Evolution of Clinical Trials 00:28:17 – The Role of the Team at Boyds 00:31:43 – Personal Interests Beyond Medicine 00:38:12 – Future of Drug Development 00:38:42 – Closing Thoughts and Reflections

In the latest episode of Conversations in Drug Development, Dr Katherine Bowen and Dr Eric Hardter discuss the ever-changing US regulatory landscape under the new administration, including new leadership and staff turnover, and their impact on drug development. They examine the FDA's cautious approach to AI, efforts to reduce animal testing, and the new National Priority Voucher Program aimed at expediting drug approvals. Tune in for an in-depth discussion that offers timely updates and expert perspectives on what might be in store for drug development stakeholders.

In this episode, Dr Neil Fish and Dr Ami Patel dive into the real-world challenges of drug development - from early-stage planning to regulatory hurdles and everything in between. Drawing on decades of experience, they share personal stories and expert insights that reveal why a solid strategy and the ability to pivot are essential for success. They explore the value of strategic flexibility, the importance of engaging with regulators early, and how to approach patent protection and manufacturing for advanced therapies. Whether you're preparing an IND or planning scale-up, this episode delivers clear, actionable guidance grounded in industry know-how. A must-listen for biotech teams, clinical leads, and anyone involved in the drug development process.

In this episode, Harriet Edwards from Boyds hosts a discussion on 2025 predictions in drug development and regulatory affairs. Joined by regulatory experts Dr. Katherine Bowen and Dr. Eric Harder, the conversation covers the anticipated trends in the realm of cell and gene therapy, rare diseases, and the increasing role of artificial intelligence (AI). As we look ahead to 2025, Boyds' regulatory experts discuss the evolving regulatory landscape, legislative changes in the US and EU, and the importance of patient-centric approaches. Join us for this insightful journey as we uncover what 2025 has in store for drug development and regulatory strategies.

In this episode, Dr. Nick Meyers and Dr. Eric Hardter, discuss effective strategies for de-risking Investigational New Drug (IND) applications. Discover key insights on how to navigate the complexities of IND submissions, including the importance of pre-IND meetings, optimizing your non-clinical and CMC packages, and understanding FDA expectations. Gain valuable tips on engaging with regulatory agencies, addressing potential issues, and ensuring your IND is well-prepared to avoid clinical holds. Whether you're a seasoned professional or new to the field, this episode offers practical advice to help streamline your drug development process.

In this episode, host Harriet Edwards is joined by Eamonn McGowran, an expert in medical device regulations, to explore the complex and rapidly evolving regulatory landscape for medical devices, particularly within the EU. Eamonn highlights the challenges of developing combination products that integrate devices and pharmaceuticals, the roles of notified bodies and competent authorities, and the importance of early stakeholder engagement. The episode also covers the integration of artificial intelligence in medical devices and the evolving UK regulatory framework post-Brexit.

In this episode, host Harriet Edwards and Senior Clinical Project Manager, Chris Moore, discuss the vital role of patient and public involvement in drug development, particularly within clinical trials. The conversation covers the increasing significance of patient engagement, highlighting how the industry is recognizing the invaluable contributions of patients in shaping clinical trials and exploring the concept of "patients as partners," which is gaining traction among sponsors, ethics committees, and regulatory bodies.

In this episode, host Dr Julie Warner is joined by experts from Decisive Consulting, Esther Nzenza, and Kevin Asher to discuss the upcoming joint clinical assessment (JCA) process set to begin in January 2025. The conversation covers the implications of the JCA for drug developers, particularly smaller companies, and emphasizes the importance of early planning and integrated evidence generation. Join us as we uncover the strategies and considerations essential for success in the ever-evolving world of health technology assessments.

In this episode, host Harriet Edwards is joined by Dr Katherine Bowen to explore the regulatory fundamentals for obtaining marketing authorisation in Europe, focusing on the centralised procedure. Together, they unpack the intricacies of this pathway, comparing it to the FDA application process and emphasising the critical role of thorough dossier preparation and clear communication with regulatory authorities. Key topics include the importance of early engagement with regulators, navigating the challenges of Module 1, developing risk management plans, and addressing current regulatory issues.

In this episode, host Harriet Edwards is joined by Dr Katherine Bowen and Shalini Gupta to explore the transformative changes in the European Union's clinical trial landscape. The focus is on the transition from the Clinical Trials Directive (CTD) to the Clinical Trials Regulation (CTR) and its significant impact on the regulatory environment. Join us for an insightful discussion on how the CTR is shaping the future of clinical research in Europe and the positive impact it aims to have on patients.

In this episode, host Harriet Edwards is joined by two regulatory experts from Boyds, Dr Patrick Ginty and Monica Pianella, to discuss cell-based immunotherapies, focusing on CAR-T cells and TCR-T cells. Patrick and Monica delve into these cutting-edge treatments, discussing their uses, the complexities of their production, and important safety considerations. They also highlight recent advancements and the exciting future prospects for these therapies in cancer treatment and other areas. Tune in for an insightful discussion on the future of cell-based immunotherapies, examining both the hurdles and the potential of these innovative treatments.

In this episode, Dr Katherine Bowen and Dr Julie Warner delve into the world of regulatory designations, providing clarity on what these designations offer, the products that qualify, and the pros and cons of each. They discuss the history of regulatory designations, starting with the Orphan Drug Act of 1983, and explore various designations such as Orphan Drug Designation (ODD), Fast Track Designation (FTD), Breakthrough Therapy Designation (BTD), and Regenerative Medicines Advanced Therapy Designation (RMAT). Tune in to gain valuable insights from their experience and advice and learn how to navigate the regulatory landscape effectively.

In this episode, host Dr. Katherine Bowen, is joined by Harriet Edwards, Associate Director in Regulatory Affairs, to explore the fascinating world of genome editing technologies. Together, they delve into the ethical considerations of editing somatic versus germline cells and trace the historical progression of these technologies from agriculture to healthcare. Join us as we discuss the complexity of genome editing methods, the irreversible nature of genetic changes, and the associated risks.

Welcome to a special edition of Conversations in Drug Development, brought to you by the team at Boyds. In this episode, our host Harriet Edwards hands over the mic to two of her colleagues to discuss a critical topic for early-stage biotech companies: funding challenges and how to overcome them. Dr Nick Myers and Dr Neil Fish, share their extensive experience and insights into navigating the complexities of funding and investment. They delve into what makes an effective pitch deck, the importance of presentation skills, and the role of a well-organized data room. Additionally, they offer valuable advice on matching fundraising activities to the right investors and the impact of market trends on investment strategies.

How can phase I studies with ATMPs address unmet needs in oncology and what are the challenges? In this episode of Conversations in Drug Development, Dr Katherine Bowen is joined by Pharmaceutical Physician, Dr Harriet Gray Stephens, to delve into the complexities of phase I oncology studies involving advanced therapy medicinal products (ATMPs). Join us as we discuss the unique challenges of ATMPs, such as their different benefit-risk profiles compared to traditional therapies, and the need for innovative dose escalation and efficacy evaluation methods as well as the potential of ATMPs to meet unmet needs in cancer treatment.

In this episode of Conversations in Drug Development, our host Harriet Edwards, is joined by Director of Regulatory Affairs, Dr Sabine Ruehle, to discuss the challenges of navigating genetically modified organism (GMO) regulations in clinical trials within Europe. This discussion helps decipher the nuanced environment of GMOs and their associated risks unique within the European framework. Join us as we explore the outdated definition of GMOs, the complexities of their classification, and the impact of genome editing technologies.

In this episode of Conversations in Drug Development, host Dr Harriet Edwards is joined by Katy Rudnick and Dr Julie Warner from Boyds to discuss US regulatory trends and initiatives at the FDA. This episode explores the FDA's recent organizational changes, pilot programs, and focus on innovative clinical trials, including the use of AI in drug discovery. Join us for another Conversation in Drug Development, exploring the dynamic landscape of regulatory trends expected in 2024 and beyond, with a key emphasis on the US scope.

To celebrate Rare Disease Day, we are back with another Conversation in Drug Development, this time discussing the unique challenges of rare disease drug development. Dr Eric Hardter, Associate Director of Regulatory Affairs at Boyds, joins our host Harriet Edwards to explore the need for early stakeholder involvement, the complexities of pediatric clinical trials, including ethical considerations and regulatory requirements and to touch on FDA initiatives supporting rare disease therapeutics and the evolving landscape for cell and gene therapies. To discuss this episode of Conversations in Drug Development with a member of the team, visit www.boydconsultants.com.