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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 2, 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 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 can contribute to more robust HTAs.

Read the research