Advancing Revolutionary Therapies: Recent Episodes
ART
Cell and gene therapies have made impressive clinical progress but continue to face big hurdles. Nothing is standard about study design, study conduct or regulatory process in this specialized area.
MDR’s Article 117 brings improved patient safety measures with implications for manufacturers, as they must fulfill a significant set of additional requirements. Listen as Andrea Larrañaga, Associate Manager, Regulatory Affairs at Veristat and her colleagues, Laura Ocaña and Gemma Dorrego, discuss the Article and its influence in the marketing of combination products.
Comparability studies are a significant issue for those working in biotherapeutics development, as undetected product changes are considered high risk. Listen as Kevin Hennegan, Lisa Erickson and Sarah Roemer take us through several considerations when Playing the Comparability Game in Biotherapeutics Development.
When developing a new therapy for a rare disease, a thoughtful, strategic approach early in the development process supports a well-designed study and agreement to your approach from regulatory authorities. Listen to our podcast as Veristat experts share important considerations to reduce your regulatory risk along the way.
The development of biosimilar products in the US has been progressing since the regulatory pathway was established as part of the Affordable Care Act. Listen as members of Veristat’s Regulatory team delve into the analytical and clinical science of biosimilars and their regulatory pathway.
Sponsors are familiar with the role of a Regulatory Strategist on the core team but have less familiarity with the responsibilities of a Project Manager. Listen as members of Veristat’s regulatory team bring to light the invaluable contributions and responsibilities of the Regulatory Project Manager, known for their problem-solving mindset.
Listen as members of Veristat’s regulatory team outline the fundamentals— and the pitfalls— as you determine your readiness to file an Investigational New Drug (IND) from a manufacturing perspective.
Hear members of Veristat’s Regulatory team guide listeners through several publishing best practices to consider when planning your marketing application. Get a jump start by learning the key steps that go into making documents “submission ready”.
In this ART podcast, Kevin Hennegan, Veristat’s Director of North American Regulatory Affairs, takes us through the many nuances key to the success of an Emergency Use Authorization application.
FDA’s Orphan Drug Designation provides incentives to encourage the development of treatments for rare diseases. Listen as Mara Holinger, SVP of Regulatory Affairs at Veristat and members of the regulatory team shed light on ODD classification and the strategic use of subsets in study design.
Our Regulatory experts kick off Season 3 of the ART Podcast series in just a few weeks! Listen to the trailer to learn more about some of the topics we’ll be covering.
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.
Scaling up from phase 1 to global phase II/III trials to beyond marketing approval has several implications on a sponsor’s commercial strategy. Hear from Rachel Smith, Portfolio Director for Veristat, as she uses our recent work with a biotech company to illustrate the importance of a comprehensive shipping and traceability strategy, tight management of the patient journey, and adoption of a well-conceived protocol and process documentation to support the commercialization strategy.
Rachel Smith, Portfolio Director for Veristat, takes us through a number of pitfalls to avoid when planning for a gene therapy study. With the stakes high and having conducted trials for the first gene therapy approved in Europe, put Rachel’s lessons learned to work when determining your regulatory pathway, logistics strategies and donor screening requirements.
Mariana Oviedo, Project Manager for Veristat, provides an overview of the key items that must be taken into consideration when designing and running a long-term follow-up study for a gene therapy product, sharing experiences from both a scientific and operational perspective. It is never too early to start thinking about the long-term follow up with so much on the line.
Listen as Kevin Hennegan, Senior Regulatory Strategist for Veristat, shares some of the common hurdles drug developers face when bringing cell therapies to market including international regulatory variability, patient safety assurance, planning for long term follow up and more.