INSIGHTS ON CELL & GENE CLINICAL TRIALS

• Evolution Of Treatment Of Atopic Dermatitis

  The Atopic dermatitis (AD) treatment paradigm has significantly evolved in the last five years. Currently, AD is an area of intense focus for clinical developers. Globally, more than 100 pharmaceutical and biotechnology companies are investigating new therapeutic solutions for AD. This white paper will explore the pathophysiology of atopic dermatitis, as well as the rationale and mechanisms of action of existing and emerging therapies for AD.

• Beware of Underestimating The Value Of A Pre-NDA Meeting

  Drug development is a resource-intensive endeavor. Seeking input from the U.S. Food and Drug Administration throughout the journey can help optimize those resources and maximize the likelihood of regulatory approval. When preparing to submit a new drug application (NDA), a pre-NDA meeting with the FDA can be a critical step in ensuring the submission of a well-organized NDA that fits the expectations of agency reviewers.

• Adaptive Design Methods Offer Rapid, Seamless Transition Between Studies

  The evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy. Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials.

• Tumor-Agnostic Treatments: A Game Changer For Rare Oncology Trials

  As our understanding of the molecular characteristics of tumors has improved, there has been a tremendous leap forward — not only in targeted therapies but also in the development of tumor-or tissue-agnostic treatments. Now, with precision or personalized medicine, treatments can be targeted to a subgroup of patients rather than relying on a one-drug-fits-all model. In this blog post, we explore the changing rare oncology landscape and novel approaches to rare cancer trials.

• Supporting Multiple Global Gene Therapy Studies For Rare Diseases

  A long-term client called upon the Veristat team for help conducting two investigational Phase I/II studies. These global studies focused on proving the safety and efficacy of gene therapy for two rare genetic disorders. Working side-by-side with the client, our cell and gene therapy experts addressed many complicated challenges – including site start-up issues, stringent regulatory requirements, and the logistics of handling highly sensitive genetic material.

• Cell And Gene Therapy Team Guides Troubled Rare Disease Programs To Success

  A clinical-stage biotechnology start-up asked Veristat to run a new European trial of their complex gene therapy. Veristat experts complimented the client’s team taking on their first clinical-stage program. Our global cell and gene therapy team brought knowledge in genetic material handling, relationships with key global regulatory agencies, and expertise in accelerated pathways to approval.

• Stem Cell Therapy For Stroke Patients

  A client engaged the Veristat team in a Phase II study of intracerebral stem cell injection in patients following an ischemic stroke. The goal was to look at limb recovery time and level in patients with stroke. The effort began with identifying the best time for a product to be delivered and then expanded into full-service involvement, including project management, clinical monitoring, medical writing, and regulatory affairs. Learn how we overcame unique challenges in this multi-center trial that led to the client publishing positive Phase IIa data.

• Full Service Support For A Gene Therapy Trial

  A clinical-stage biotechnology start-up asked Veristat to run complex US and EU trials of their unique gene therapy. Veristat brought invaluable global capabilities and expertise to a small client team tackling their first clinical-stage program without the large infrastructure or resources to conduct studies in this space themselves. Our end-to-end support started at Phase I and will continue into post-marketing and regulatory follow-up.

• How a Best-Practice Strategy For Sourcing Ancillary Materials Reduced Risk, Spending and Workload In A Global Diabetes Trial

  It’s impossible to run a global clinical trial of an investigational diabetes drug without ancillary materials. And as one leading multi-national pharmaceutical company recently learned, managing ancillaries can be a challenge every bit as complex as the trial itself.

• 5 Tips To Avoid An IND Clinical Hold: Regulatory Guidance And Insights

  A clinical hold from the U.S. Food and Drug Administration can significantly prolong the time and increase the cost of drug development, which is particularly concerning for emerging/small biotech and specialty pharma companies. In this blog, we discuss common reasons for clinical holds and provide useful tips for both avoiding and addressing them.