• Fulfilling the Promise: Real-World Data and Real-World Evidence in Action

    Real-world data (RWD) – information gathered from the routine delivery of health care rather than from clinical trials – and real-world evidence (RWE) – evidence of a medical product’s effectiveness or safety derived from RWD – have become more prominent in the health care landscape, but questions remain about how to use them effectively in health care decision making.

    By providing insights about outcomes associated with pharmaceuticals and devices in clinical practice, RWD and RWE can complement clinical trial findings, help to fill critical knowledge gaps, and support both product launches by manufacturers and decision making by payers and regulatory bodies.

    By their nature, however, RWD are collected from disparate sources in a variety of settings, often lacking the standards and controls available in clinical trials. This can present unique challenges when it comes to isolating treatment effects and ensuring data validity.

    Analysis Group has deep expertise in handling, analyzing, and generating RWD and RWE to ensure that the data are high quality and fit for purpose, and tell a coherent story about a product’s effectiveness and safety.

    Here are three examples of our work in this area.

    RWE in Regulatory Submissions – Early Engagement, Bias Minimization

    Key question: What can pharmaceutical companies do to improve the chances that the US Food and Drug Administration (FDA) will accept RWE?

    The 21st Century Cures Act called for the use of RWE in regulatory decision making. The FDA created a framework for the potential use of RWD to generate RWE to complement clinical trial data as a way to accelerate the pace of approval of new therapies or new indications. Yet the FDA did not provide clear guidelines for designing and executing adequate RWE studies for successful submissions.

    Working in collaboration with Pfizer Oncology, Analysis Group researchers set out to address this knowledge gap by undertaking a thorough review of successful oncology product submissions to the FDA between 2015 and 2020 that included RWE. They analyzed what RWE was submitted, whether it was accepted, and why. The study revealed a key theme:

    Click here to learn more about the study, or contact Mei Sheng Duh or Maral DerSarkissian.


    Data Integration – Connecting the Dots

    Key question: How can RWD from different data sources be integrated to enhance quality and optimize their value for clinical research and market access?

    Different types of RWD focus on different aspects of patient care in real-world settings, resulting in fragmentary data with compromised research value. Working with organizations researching blood disease in China – where high-quality evidence to support the placement of a treatment on the National Reimbursement Drug List (NRDL) is key to its success – an Analysis Group team established a data-driven disease model to close the gap.

    The model connects different data sources and uses artificial intelligence to obtain a complete picture of the patient journey. It includes a progression algorithm capable of reconstructing lines of therapy, multi-agent treatment regimens, and treatment responses – key elements in understanding real-world treatment patterns and effectiveness.

    Click here to learn more about the study, or contact Eric Wu or Jia Zhong.


    Data Access – Leveraging Patient Support Programs to Generate RWD

    Key question: How can biopharmaceutical companies speed up the process of collecting, compiling, and employing patient-reported RWD?

    Collecting and synthesizing RWD from multiple third-party sources is a complex and time-consuming process, especially when stakeholders (e.g., regulators, payers, clinicians) are interested in patient experiences through patient-reported outcomes about diseases and associated therapies. Analysis Group has designed an innovative methodology, Longitudinal Surveys of Patients with Recruitment Through Patient Support Programs (LEAP), which leverages pharmaceutical companies’ patient support programs (PSPs) for particular therapies. This has proven to be a breakthrough process for generating early and timely RWD directly from a clinical practice setting.

    Click here to learn more about the study, or contact Min Yang. ■