Veristat Case Studies

1. Supporting A Sponsor From SPA Rejection To BLA Submission 8/22/2022

   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.

2. New Tumor Evaluation Modality To Measure Effectiveness Of Cancer Therapies 5/31/2022

   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.

3. Gaining Regulatory Approval Following A Single-Arm Phase I/II Study 3/23/2022

   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.

4. Writing Multiple Marketing Applications Simultaneously With Accelerated Timelines 4/28/2021

   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.

5. Preparing Data For The Marketing Authorization Application (MAA) Of A Recently Acquired Product 4/28/2021

   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.

6. Groundbreaking Work Leads To First Gene Therapy Ever Approved In Europe 1/14/2021

   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.

7. Supporting Multiple Global Gene Therapy Studies For Rare Diseases 11/17/2020

   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.

8. Cell And Gene Therapy Team Guides Troubled Rare Disease Programs To Success 11/2/2020

   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.

9. Stem Cell Therapy For Stroke Patients 11/2/2020

   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.

10. Full Service Support For A Gene Therapy Trial 11/2/2020

    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.