Written by Grace Hsu, Evie Merinopoulou, and Jason Simeone   To establish treatment efficacy and safety, regulatory and reimbursement decision-makers have traditionally preferred evidence from randomized clinical trials, which, by design, have a low risk of bias. However, single-arm trials (SAT) using an external control arm (ECA) are commonly performed for ethical reasons, due to the difficulties in identifying a suitable comparator arm(s) for head-to-head trials in a rapidly evolving therapeutic landscape and in recruiting patients in the case of rare diseases ..read more

In the context of clinical trials, reducing the workload of the clinical team without compromising data quality is imperative for efficiency and cost-effectiveness. One key area that demands attention is the approach to Source Data Verification (SDV) and data review. Typically, the industry relies on 100% SDV, a resource-intensive quality assurance method that often leads to higher costs and staff turnover without necessarily improving data quality. A well-known analysis by TransCelerate1 has revealed that only around 3.7% of clinical trial data changes after its initial entry, and even ..read more

How should health technology developers prepare for future market access activities in Europe? Numerous discussions have already taken place in various forms and on various platforms around the upcoming implementation of the EU Joint Clinical Assessments (JCA); it’s a hot topic and keeps many of us in our industry occupied. Despite the European Commission’s active efforts in developing draft regulation and related materials to support the transition to JCA, to date, some questions remain unanswered. As the EU JCA aims to harmonize and accelerate evaluation processes in Europe, all stakeholder ..read more

Orphan drug designation is a regulatory status granted to pharmaceuticals developed for the treatment of rare diseases. It provides incentives to encourage the research, development, and approval of therapies targeting small patient populations ..read more

In this latest edition of the Career Perspectives series, we are excited to introduce our readers to Joshua Murray, Principal Biostatistician in Functional Service Provision (FSP) at Cytel. He is based in Toronto, Canada, and joined Cytel in August 2022. With a diverse background spanning academia, government, healthcare, and industry, Josh brings a wealth of experience to the forefront. Join us as we delve into his perspectives on mentorship, collaboration, and the dynamic landscape of biostatistics ..read more

Written by Sydney Ringold, Customer Success Manager, and Kevin Trimm, Chief Product Officer   In an ever-changing clinical development environment, sponsors face many challenges when designing clinical trials. These challenges can range from shifting regulations to growing pressure for a rapid time-to-market, and the variety of challenges can complicate trial design requirements. A thorough evaluation of numerous parameters is required in order to choose the best fit or optimal study, and this has led to more complex and innovative clinical trial designs. When designing trials, biostatis ..read more

Thank you to Charlotta Gauffin, Chief Scientific Officer at Dicot, for joining us for our recent webinar, “The Road to First-in-Human Trials: Insights from a Real-World Example.”   Thoughtfully and carefully planned nonclinical studies help pave a smooth path toward first-in-human Phase 1 clinical trials. This involves a collaboration between all areas of nonclinical development, including CMC, toxicology, pharmacology, DPMK, and so on, and keeping in mind early a plan that all stakeholders agree to, but that can be amended as you go along, if needed. Here, we share how Cytel and Dicot w ..read more

Data monitoring committees (DMCs) review ongoing clinical trial data to make recommendations regarding trial conduct based on risk-benefit. DMCs are an essential component to ensuring the integrity and safety of clinical trials. New draft guidance published by the U.S. FDA on the use of DMCs in clinical trials provides six major updates to the FDA’s expectations regarding DMC structure and practice. The draft is open to public comment until April 15, 2024. We highlight the updates provided in this draft guidance and discuss the impact on sponsors ..read more

Written by Lydia Vinals, PhD, and Grammati Sarri, PhD The draft Implementing Act of the EU Health Technology Assessment Regulation (HTAR) for Joint Clinical Assessments (JCA) of medicinal products — the first legal definition of the procedural and methodological details for the new EU HTAR JCA — was published by the European Commission on March 5, 2024, following some delay.1 The draft is currently open for public consultation until April 2, and feedback received will be considered before the European Commission finalizes the Implementation Act. Here, we delve into the key elements of th ..read more

  Group sequential clinical trial designs — a type of adaptive clinical trial design — have emerged as a powerful tool in enhancing the efficiency and ethical conduct of clinical trials, due to the ability to stop the trial early based on accumulating data. Here, I expand on the intricacies of group sequential designs, key design features, applications in clinical trials, their advantages, challenges, and impact on the landscape of clinical trials ..read more