Pharmaceutical research in oncology is increasingly focused on the development of therapies targeted at newly identified driver mutations. However, the rarity of many of these mutations presents new challenges for trial design and patient recruitment. In particular, it may be difficult in practice to recruit enough patients with the same mutation and tumor histology. One solution has been to evaluate these histology independent therapies (HITs) in basket trials, which recruit patients across multiple histologies that all share a common targetable driver mutation.
Bridging the gap between oncology clinical trials and real-world data: evidence on replicability of efficacy results using German claims data
Randomized control trials (RCTs) are the gold standard for estimating the efficacy of a treatment. They allow us to investigate treatment effects in a controlled setting designed to ensure reproducibility of the results, quality of data, and minimization of selection bias. In contrast, comparative effectiveness from real-world data (RWD) is traditionally regarded as inferior evidence, primarily due to its lack of randomization and limited availability of information on potentially relevant prognostic factors. As such, real-world evidence (RWE) has been mostly used to corroborate RCT results and for post-marketing monitoring.
Recently, the growing body of data from the real world, which accompanied the digitalization of health, has drawn increasing attention to RWD and its potential, leading to questions on whether comparative effectiveness evidence from RWD could support regulatory and HTA decisions.
Career Perspectives: Conversation with Allison Luccock
In this edition of the Career Perspectives series, I interview Allison Luccock, Director of Business Operations for Cytel’s Real World and Advanced Analytics (RWAA) group. Allison is based in Quebec, Canada, and she played a pivotal role in the integration of Purple Squirrel Economics and Cytel. In this interview, we learn about her professional journey, her insights on industry trends including the cutting-edge LiveSLR® platform, and the necessary qualifications to work in Cytel RWAA.
5 Steps to Adjust for Effect Modifiers for Treatment Comparisons
Many thanks to Grammati Sarri and Michael Groff for their comments in developing this blog.
An indirect treatment comparison compares two trials A and B, by first comparing the results of Trial A to Trial C, and then comparing the results of Trial C to Trial B. Unlike head-to-head comparisons where these trials (A and B) would be directly compared with each other, indirect treatment comparisons, or ITCs, utilize at least one other trial to create a network for comparison.
Often, there are fundamental differences between the trials, such as population, or the interaction of the treatment with features of the population (e.g., age, sex, etc.). These are either caused by prognostic variables or by effect modifiers. The methods with which we handle the complexities raised by each differ slightly. Population-Adjusted Indirect Treatment Comparisons (PAICs) use individual patient data from one or more trials to adjust for characteristics of patients and ensure populations across all trials are reasonably comparable.
7 Ways RWD Is Transforming Clinical Research
The ability to draw on electronic medical records is foundational to the use of real-world data (RWD). In China, companies like HappyLife Tech (HLT), an affiliated company of Yidu Tech Inc., can build digital ecosystems with 1.3 billion data records and over 300 million patients, thereby opening new horizons for real world solutions. Such solutions have the potential to improve every stage of the product lifecycle and study journey including feasibility studies, clinical trial design, and market access. They also open new development pathways including those for decentralized trials and AI-driven solutions. According to Dr. Luyan Connie Dai in her recent webinar‚ “Embrace the Era of Digital Clinical Trials in China: Challenges & Opportunities” there are seven key ways in which RWD is transforming the product journey in China.
A Better Way to Track Medication Adherence
Patients’ adherence to the medications or treatment regimens prescribed to them by their clinicians is an important determinant of their healthcare outcomes across a variety of clinical settings. But how do we measure it, and is there a better way?