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    Exploring the potential for RWD to reduce uncertainty in HTA decision-making: a case study of long-term survival outcomes

    Pittell H, Kent S, Groves B, Mpofu P, Baxi S, Copeland A, Bargo D, Adamson B, Jonsson P. Exploring the Potential for EHR-Derived Real-World Data to Reduce Uncertainty in HTA Decision-Making: A Case Study of Long-Term Survival Outcomes. Value in Health. Volume 24, Issue 12, S2 (December 2021).

    Lead author Harlan Pittell presents this research and its findings.

    Our summary

    Clinical trials are an important source of evidence for health technology appraisals (HTA). A key concern though is the uncertainty in survival due to the relatively short duration of follow-up in clinical trials, posing an issue for understanding treatment benefits long-term. Researchers from the National Institute for Health and Care Excellence (NICE) and Flatiron Health investigated how real-world data (RWD) may complement findings from clinical trials to reduce uncertainties in HTA decision-making. In this case study, the investigators focused on an immunotherapy drug in advanced lung cancer to see if RWD could have been used to address uncertainty regarding long-term survival.

    Why this matters

    The inherent uncertainty in health technology appraisals (HTAs) can result in suboptimal access to medical interventions by patients in need of therapies. Inconclusive cost-effectiveness appraisals can lead to delays or limited treatment coverage decisions. This study illustrates how RWE can contribute to more robust HTAs.

    Read the research