Advertise on podcast: Advancing Revolutionary Therapies

Listen as Rachel Smith, Portfolio Director at Veristat, reviews considerations for setting up Natural History studies and the value they bring to rare and ultra-rare clinical research, despite the challenges in their design. 

Cell and gene therapy innovations continue to progress despite their clinical trial complexities. Listen as we replay some of Ewan Campbell’s insights from a recent webinar where Ewan provides a historical recap of the approved cell and gene therapy treatments over the past decade along with insights on where we are headed.

Patient reported outcomes provide an important dimension to assessing the overall patient impact from a novel treatment. Listen as Robin Bliss describes the appropriate execution of PROs using clearly defined endpoints, measurable changes within context to the indication and disease progression, and several other key considerations for deployment in cell and gene therapy studies. 

Administering advanced therapy products during a clinical trial can bring about many unique circumstances. Listen as Rachel Smith discusses Convection Enhanced Delivery (CED) which delivers medicines directly into the brain. Learn how to prepare for its challenges and what it can mean for patients, clinical teams, and sponsors.

Clinical trials involving advanced therapies for the treatment of Central Nervous System (CNS) do not follow a classic road map to market. Listen as Ewan Campbell, Advanced Therapy and Biotech Director for Veristat outlines some of the unique challenges faced when developing advanced therapies and shares some of his experience gained in neurodegenerative disease studies over the past 20 years.

The roadmap to market for a cell or gene therapy is undoubtedly challenging, but also filled with great promise. Listen as we replay some of Rachel Smith’s insights from our recent webinar where Rachel reviews virtual and central site models in cell and gene trials and the logistics involved in planning and execution.

If you are wondering how natural history studies are relevant to cell and gene therapy development, listen as Chris Kenwood, Principal Statistician with Veristat, takes us through an introduction. Learn how natural history studies provide evidence for identifying patient subgroups that may benefit from therapy, help in identifying or developing biomarkers, assist in establishing clinical outcome assessments, and more. 

Developing a successful marketing application involves several strategic steps, as described by Kevin Hennegan, Senior Regulatory Strategist for Veristat. Listen as Kevin identifies some of the key components of a cell & gene therapy marketing application in the US and EU, and outlines several of the most common pitfalls and gaps that he has encountered with sponsor applications. Listen now. 

Our cell and gene experts are back for season two of ART Podcast starting next week! Listen to the trailer to learn more about some of the topics we’ll be covering.

The pros, cons and future of allogeneic versus autologous therapies are explored as Rachel Smith, Portfolio Director for Veristat, shares her observations on the impact of these techniques on advanced cell-based therapies and what the next decade may hold. Factors spanning patient safety, manufacturing, costs and regulations are considered as Rachel responds to the question whether an autologous approach for rare disease therapies is sustainable long-term.

Post-marketing requirements within the European submission process offer conditional approval more flexible than FDA. Rachel Smith, Portfolio Director for Veristat, discusses the three types of post-marketing requirements that can be requested by the EMA and draws upon the recently approved therapies – Tecartus, Zolgensma and Libmeldy, Skysona and Abecma– for real-world context. 

The selection of endpoints for a clinical trial relies on clinical relevance combined with statistical reasoning. Robin Bliss, PhD, and VP of Strategic Consulting for Veristat, takes us through the how-to’s of selecting appropriate endpoints for an early phase study, the importance of considering endpoints in the context of clinical meaning, and the value of ensuring that endpoints are measurable within a reasonable amount of time for continued progression of the clinical development plan.