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


December 2021


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).

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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