ABOUT VERISTAT

Veristat, a global clinical research organization (CRO), enables sponsors to solve the unique and complex challenges associated with accelerating therapies through clinical development to regulatory approval. With more than 26 years’ experience in clinical trial planning and execution, Veristat is equipped to support any development program. Our team has prepared over 100 marketing applications for approval with global regulatory authorities in the last 10 years alone. We supported the trials for the first gene therapy approved in Europe.

Veristat’s focus on novel drug development has led to success when handling the unknowns that arise across complicated therapeutic areas, such as rare/ultra-rare disease, advanced therapies, oncology, and infectious disease trials. We apply this knowledge base every day to solve any clinical program’s challenges, from the simplest to the most complex. Veristat has assembled an extraordinary team of experts worldwide who have mastered therapeutic development intricacies, enabling sponsors to succeed in extending and saving lives.

Whatever the study’s unique considerations – patients, products, process, follow-up, regulatory – Veristat can successfully get you through it. For more information, visit www.veristat.com.

CAPABILITIES

Whether you need a few scientific experts or a fully-staffed function, gain flexibility and results with Veristat’s Strategic Resourcing Solution and confidently meet your study milestones.

Getting a novel therapy through the clinical development process to approval is complicated, full of challenges, and even more complex in the current COVID-19 world. Learn how to advance your novel  medical therapies from Phase I-III clinical development to market with confidence,

Our regulatory affairs experts guide you through the entire drug development and regulatory submission process to help you achieve regulatory success.

Advancing a novel therapy through clinical development is complicated. Not all therapies make it to the patients that need them.

Accelerate your cell or gene therapeutic to market with tailored clinical and regulatory strategies and expert insights.

We understand how challenging it is to adopt a novel approach to drug development. Pivoting to virtual clinical trials is no exception.

Expertise that accelerates therapies for rare and ultra-rare disease through the clinical development process.

Quality medical writing of clinical trial documents, safety updates, and marketing applications for regulatory success

Delivering safety surveillance before and after regulatory approval for pre-approval and marketed health products.

Preparing regulatory marketing applications to their successful conclusions is our focus.

PODCASTS

  • In this 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.

  • Hear members of Veristat’s Regulatory team guide listeners through several publishing best practices to consider when planning your marketing application. 

  • 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.

  • Listen as members of Veristat’s regulatory team shed light on orphan drug designation classifications and the strategic use of subsets in study design.

  • 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.

  • 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.

  • 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.

  • 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.

  • Listen as Kevin, Senior Regulatory Strategist for Veristat, 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. 

  • 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.

  • 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.

  • 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.

  • 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.

  • 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 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.

  • 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.

CONTACT INFORMATION

Veristat, Inc.

134 Turnpike Road, Suite 200

Southborough, MA 01772

UNITED STATES

Phone: 508-306-6281

Contact: Lauren Willis

FEATURED ARTICLES

CASE STUDIES

  • Learn how the clinical trial design and the statistical foundation of the special protocol assessment (SPA), which helped drive to regulatory agreement, was transformed in this case study.

  • In this case study, delve into how the team used intratumor RECIST (itRECIST) criteria as the solution to measuring the effectiveness of a novel cancer therapeutic.

  • A small biotech company engaged Veristat early in clinical development for help with a novel biologic being tested for treatment of an ultra-rare and aggressive hematologic malignancy with no available effective therapies. Discover what happens in this case study.

  • A mid-size pharmaceutical company and its development partner, a small biotechnology company engaged us to complete the medical writing for their product’s Marketing Authorization Application (MAA), New Drug Application (NDA), and New Drug Submission (NDS). Learn how our medical writing team worked closely with the sponsors to write these marketing applications in an accelerated timeline.

  • A mid-sized biopharmaceutical company dedicated to developing innovative therapies for patients with debilitating rare diseases acquired the rights to a new investigational drug from a large pharmaceutical company with the intention of preparing the data for a Marketing Authorization Application (MAA). Learn how our team had to think creatively to enter, clean and standardize the clinical trial data from previous and ongoing trials in order to get the MAA ready for submission in an extremely short timeline.

  • Veristat was brought in to create and manage a full development program for a gene therapy for a very rare inherited disorder. The work involved charting the course in completely uncharted waters – our team had to create industry best practices that didn’t exist before, anywhere. With no classic route to market, Veristat’s experts wrote the map on patient recruitment, regulatory and health agency engagement, natural history studies and the Central Site Model.

WEBINARS

  • The National Institutes of Health (NIH) estimates that 1 in 10 Americans are affected by a rare disease or condition. Find out the challenges and regulatory framework for rare disease in this webinar.

  • Explore the challenges of designing and running an extraordinary complex natural history study - with both retrospective and prospective data – which then had to pivot to virtual patient visits mid-way through the data collection process. 

  • In this webinar experts explore how to develop your marketing application strategy, analyze the benefits and risks associated with your data migration strategy and coordinate with the medical writing team so that all the information needed to write and complete the modules of the submission is provided with adequate time for review and quality control.

  • Submitting an Investigational New Drug (IND) application to the US FDA is a significant step in the development of a new drug or biologic product. But, are you ready? In this webinar, experts explore the minimum required content for an IND submission and the strategic considerations for developing more than the minimum necessary data before filing an IND.

  • Nothing is standard about the study design, study conduct or the regulatory process in cell and gene therapy trials. Every cell and gene therapy is different due to the specific characteristics of each product, with a complicated regulatory approval process and unique therapeutic challenges. In this webinar, participants will learn an end-to-end approach to bringing a cell and gene therapy to market.

WHITE PAPERS